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09: Regulatory [clear filter]
Sunday, June 23
 

9:00am

SC21: #21: Basics of European Medical Device Regulation
Component Type: Tutorial
CE: ACPE 3.25 Application UAN: 0286-0000-19-501-L04-P; CME 3.25; IACET 3.25; RN 3.25

This short course will cover what a medical device is (and what it is not) and discuss the legislation surrounding the European medical device regulation and why in Europe there is no combination product legislation. Participants will have the opportunity to classify medical devices which is the first step in the conformity assessment pathway leading to the affixing of a CE Mark to a product. This pathway includes a risk assessment, the basics of which will be explained. Mitigation of risk can be achieved in a variety of ways and the course will address some of them, particularly clinical investigations and labelling. The objective of this short course is to provide a basic understanding of European medical device regulatory requirements and how to ensure compliance with those regulations. Throughout the course there will be short exercises to confirm understanding and opportunities to apply knowledge gained. An additional registration fee is required for all preconference short courses. Back to DIA 2019 Short Courses

Who should attend?

This short course is designed for individuals with no experience of European medical device legislation who require an introduction to the topic and staff working in the pharmaceutical sector who require a basic understanding of medical devices legislation in Europe.

Learning Objectives

At the conclusion of this course, participants should be able to:
  • Define what a medical device is, and is not;
  • Describe the key elements of the European Medical Device Regulation (MDR);
  • Classify a medical device;
  • Recognize the different steps relating to conformity assessment, risk and data required to gain a CE Mark;
  • Interpret the legislation to conduct a clinical investigation and manage a clinical evaluation report.



Speakers
NA

Neil Armstrong

CEO, MeddiQuest Reg Affairs Ltd
avatar for Angela Stokes

Angela Stokes

Vice President, Head Global Regulatory Consulting, Syneos Health
Angela has almost 30 years of experience in medicinal product and medical device development. Her strengths include regulatory and strategic advice, authorship of regulatory submissions, regulatory writing, regulatory research, and compliance with relevant guidelines and standards... Read More →


Sunday June 23, 2019 9:00am - 12:30pm
Room 2 San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

9:00am

SC23: #23: Preparing for a US FDA Advisory Committee Meeting
Component Type: Tutorial
CE: ACPE 3.25 Application UAN: 0286-0000-19-503-L04-P; CME 3.25; IACET 3.25; RN 3.25

What are the critical factors when preparing for an FDA Advisory Committee meeting? Appearing before an FDA Advisory Committee can be one of the most challenging and grueling experiences for any drug, device, or biologic team. In just eight short hours with the FDA Advisory Committee, you not only must thoroughly explain but also defend, in detail, your product in a highly visible, high-stakes public meeting. This short course is structured like an actual FDA Advisory Committee meeting, and presents best practices for preparing for meetings. What You Will Learn:
  • What an advisory committee is
  • How an advisory committee is structured
  • Critical factors for advisory committee preparation
  • How to design the most applicable preparation program for your team
  • Top ten "best practices" and "must avoids"
An additional registration fee is required for all preconference short courses. Back to DIA 2019 Short Courses

Who should attend?

This short course is designed for professionals in regulatory affairs, clinical research leads, and corporate executives.

Learning Objectives

At the conclusion of this course, participants should be able to:
  • Identify the critical success factors in preparing for an advisory committee meeting;
  • Outline those factors that are most applicable to your team;
  • Design the most effective preparation strategy for your team(s).



Speakers
avatar for Kaia Agarwal

Kaia Agarwal

Strategic Lead, PharmApprove, LLC, a member of the NDA Group
Kaia Agarwal is an independent strategic regulatory affairs consultant working with PharmApprove. She helps teams prepare and implement global regulatory strategy for their products as they prepare for critical interactions with regulators, including EMA and FDA. For over 30 years... Read More →
avatar for Lisa Peluso

Lisa Peluso

Director, Coaching and Client Engagement, PharmApprove, a member of the NDA Group
Lisa helps development teams to create and deliver clear, consistent, and convincing messages to critical audiences and decision-makers. She has coached hundreds of individuals and provided presentation, Q&A and facilitation training to teams in pharma, and biotech, and has helped... Read More →


Sunday June 23, 2019 9:00am - 12:30pm
Room 6E San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

9:00am

SC42: #42: Patient Preferences: Using Conjoint Analysis and Stated Preferences in Drug Development and Regulatory Decision Making
Component Type: Tutorial
CE: ACPE 6.50 Application UAN: 0286-0000-19-519-L04-P; CME 6.50; IACET 6.50; RN 6.50

The need to understand patient preferences for health and healthcare has become well established and demand for stated preference studies has grown exponentially in recent years. This short course is designed as an introduction to a range of stated-preference methods and the application of these methods in drug development, regulatory decision-making, and patient advocacy. The short course will also provide an overview of good research practices and principles that are broadly applicable to all stated-preference methods and describe how good research practices can be applied to discrete choice experiments and several other stated-preference methods. Topics to be covered will include designing a survey, developing an experimental design, analyzing data, and presenting results. This short course will include hands-on exercises and detailed case studies of recent empirical examples to illustrate concepts. An additional registration fee is required for all preconference short courses. Back to DIA 2019 Short Courses

Who should attend?

This introductory to mid-level course designed to acquaint attendees with the current state of the art in the use of methods for the development of evidentiary patient preference information. Expected attendees may include: industry, regulators, payers, patients and patient advocacy representatives.

Learning Objectives

At the conclusion of this course, participants should be able to:
  • Identify when & how to successfully develop and present patient preference information for use in a range of applications, including regulatory interactions such as new drug applications;
  • Discuss specific methodologies frequently used in the development of patient preference information.



Speakers
avatar for Rachael DiSantostefano

Rachael DiSantostefano

Senior Director, Benefit Risk, Epidemiology, Janssen Research & Development, LLC
Rachael L. DiSantostefano, MS PhD, is a Senior Director of Benefit-Risk in the Epidemiology Department within Janssen Pharmaceuticals, R&D, LLC. She has 25 years of pharmaceutical research experience across the quantitative disciplines of epidemiology, biostatistics, and health outcomes... Read More →
avatar for Brett Hauber

Brett Hauber

Senior Economist, Vice President of Health Preference Assessment, RTI Health Solutions
Brett Hauber is Senior Economist and Vice President of Health Preference Assessment at RTI Health Solutions and Affiliate Associate Professor in the School of Pharmacy at the University of Washington. His is an expert in stated-preference methods. He was principal investigator for... Read More →
avatar for Carol Mansfield

Carol Mansfield

Senior Economist and Head, Health Preference Assessment, RTI Health Solutions
Carol Mansfield, PhD, is a Senior Economist and Head in the Health Preference Assessment group at RTI-HS, where she conducts stated-preference studies for pharmaceutical applications. She has 25 years of experience conducting research related to health and the environment. She has... Read More →
CP

Christine Poulos

Senior Research Economist and Head, Health Preference Assessment, RTI Health Solutions
avatar for Kristin Bullok

Kristin Bullok

Benefit-Risk Management Scientist, Global Patient Safety, Eli Lilly and Company
Kristin Bullok, PhD, is a research scientist in benefit-risk management at Eli Lilly and Company, Global Patient Safety. Since joining Lilly, she has years of combined experience in conducting structured benefit-risk assessments, consulting on patient preference trade-off studies... Read More →


Sunday June 23, 2019 9:00am - 5:00pm
Room 11B San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

9:00am

SC43: #43: Back to the Future: Combination Products in the 21st Century
Component Type: Tutorial
CE: ACPE 6.50 Application UAN: 0286-0000-19-520-L04-P; CME 6.50; IACET 6.50; RN 6.50

As innovation abounds in science and medicine, there exist endless new opportunities for combining different technologies to solve some of the world’s most pressing medical issues. The lines between engineering, chemistry, and advanced biologic processes are becoming blurred – the age of combination products is here. There are significant differences between the development processes for drugs, devices, and biologics. Theoretically, if the product complies with the regulations specific for each component of the combination product, then your company should be covered, right? Not quite! The requirements and governing bodies for each part of the product are vastly different making it nearly impossible to reconcile all the rules. Factor in new laws and regulations, such as the 21st Century Cures Act (CCA), and the complexity of compliance increases further. This short course provides an overview and comparison of drug, device, and biologics regulation in the United States, and the complex place where combination products fit within these regulations. It follows the product lifecycle, from development through post-market activities, including classification, pre-clinical and clinical requirements, applicability of Good Manufacturing Practices (GMPs) and quality systems, and labeling requirements. It will also introduce the latest statutes, regulations and guidances that impact combination products and discuss how these changes are affecting the existing market. The short course will be interactive. It has been designed to integrate substantive regulatory knowledge and the real-world problems of combination products. You will dive into case studies of unique combination products and get a sense of the gray areas that make the current regulatory scheme so difficult to navigate. It’s an opportunity to ask questions, share your challenges, gain the insights of your instructors, and learn from the experiences of your colleagues. If you are new to the field of combination products, this course will give you the foundation to move forward in your career. An additional registration fee is required for all preconference short courses. Back to DIA 2019 Short Courses

Who should attend?

This short course is designed for regulatory, quality, and clinical professionals and project managers who are new to combination products.


Learning Objectives

At the conclusion of this course, participants should be able to:
  • Discuss how combination products fit into the development processes for drugs/biologics and medical devices;
  • Identify the unique challenges of pre-market and post-market requirements for combination products in the US;
  • Discuss how recent regulatory developments, including the 21st Century Cures Act enacted in 2016 and resulting FDA guidances, affect combination products.



Speakers
CB

Caitlin Bancroft

Associate, Regulatory Affairs Consultant, Pharmatech Associates Inc.
avatar for Lynn Hansen

Lynn Hansen

Director, Regulatory Affairs, Pharmatech Associates Inc.


Sunday June 23, 2019 9:00am - 5:00pm
Room 9 San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

1:30pm

SC30: #30: Machine Learning in Pharmacovigilance
Component Type: Tutorial
CE: ACPE 3.25 Knowledge UAN: 0286-0000-19-509-L04-P; CME 3.25; IACET 3.25; RN 3.25

Since machine learning (ML) requires resources from across the organization, this course is designed for anyone interested in sponsoring or joining a ML project within their organization which focuses on pharmacovigilance (PV). Therefore, we will specifically explore ML and its application within the PV regulatory landscape and provide a high-level introduction to ML, including tools and project tips. The core of the course will also dive deeper into applications within PV, including examples from our own experiences with ICSR identification, and discussion around what the future of ML in PV could look like. There will be time for Q&A but this years course will also be very interactive between the instructors and attendees, with both questions and some relevant tool demonstrations. An additional registration fee is required for all preconference short courses. Back to DIA 2019 Short Courses

Who should attend?

This short course is designed for members of Pharma, Academia, Regulators, and Medicine interested in Machine Learning in PV.


Learning Objectives

At the conclusion of this course, participants should be able to:
  • Identify advances that make ML practical;
  • Describe how ML can be applied to the regulatory and PV landscape;
  • Develop potential future use cases for ML in PV.



Speakers
avatar for Robert Ball

Robert Ball

Deputy Director, Office of Surveillance and Epidemiology, CDER, FDA
Robert Ball MD, MPH, ScM is Deputy Director, Office of Surveillance and Epidemiology (OSE), Center for Drug Evaluation and Research (CDER), FDA. Dr. Ball shares in the responsibilities for leading OSE staff evaluating drug risks and promoting the safe use of drugs by the American... Read More →
avatar for Shaun Comfort

Shaun Comfort

Principal Medical Director, Genentech, A Member of the Roche Group
Dr. Comfort is Principal Medical Director for Roche in the Inflammatory, Infectious Disease, and Ophthalmology Safety Science group and leads innovation work supporting Pharmacovigilance. He is a Board Certified Neurologist with 16 years combined industry/regulatory experience including... Read More →
avatar for Bruce Donzanti

Bruce Donzanti

Senior Group Director, Global Pharmacovigilance Innovation Policy, Genentech, A Member of the Roche Group
Bruce has a PhD in pharmacology/neuroscience with almost 30 years of experience in the pharma/biotech industry. Prior to industry, he performed research on mechanisms of neuronal degeneration and neurotoxicology and lectured in neuropharmacology to graduate and medical students while... Read More →
avatar for Mick Foy

Mick Foy

Head of Pharmacovigilance Strategy, Vigilance Intelligence, and Research Group, Medicines and Healthcare Products Regulatory Agency (MHRA)
Mick Foy is Head of Pharmacovigilance Strategy at the MHRA. Among is responsibilities is the running of the UK ADR reporting system and signal detection activities. Mick is also leads the MHRA's work with WHO and the Bill and Melinda Gates Foundation on capacity building in low and... Read More →


Sunday June 23, 2019 1:30pm - 5:00pm
Room 5AB San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA
  • Level Intermediate
  • Featured Topics Artificial Intelligence
  • Credit Type ACPE, CME, IACET, RN
  • Tags Tutorial

1:30pm

SC33: #33: European Regulatory Meetings: How Best to Prepare and Perform
Component Type: Tutorial
CE: ACPE 3.25 Knowledge UAN: 0286-0000-19-512-L04-P; CME 3.25; IACET 3.25; RN 3.25

High-stakes meetings such as CHMP/PRAC oral explanations are often the ultimate chance for a pharmaceutical sponsor to convince EU regulators that the benefit:risk balance of their product is positive. Being well-prepared and performing professional is key to success. This include demonstrating intimate knowledge of your data to meet regulator’s concern as well as being able to present this in a crisp and clear manner. To get to this stage takes meticulous preparation of key messages, slides and the actual speech and may additionally include training key opinion leaders and/or patient representatives to speak or respond to questions in your support. Finally, preparing a head of questions and being able to respond clear and unequivocally to these are equally important to increase chances of success. This short course focuses on how best to plan, manage, prepare and execute successfully at EU high-stakes meetings. Former CHMP members, pharma executives and communication specialists will walk you through the key steps in preparing as well as the “do’s and don’ts” when presenting and engaging with EU regulators. Focus will be on CHMP oral explanations, but the learnings will be applicable to all other EU regulatory oral interactions. We will discuss how best to present your key massages and supporting slides and manuscript. How to develop a Q&A grid in collaboration with your team and how best to manage back-up slides when responding to questions will be part of the learnings. How best to deal with practicalities and logistics around a CHMP oral explanation in order to minimize the stress on your team will also be touch upon. This short course will include anonymized examples from real or imagined meetings and enable the participants to better understand the process and its challenges as well as how best to cope with these. An additional registration fee is required for all preconference short courses. Back to DIA 2019 Short Courses

Who should attend?

This short course is designed for professionals in Regulatory and Clinical teams.


Learning Objectives

At the conclusion of this course, participants should be able to:
  • Identify the key messages and supporting slides needed to conduct a successful EU regulators meeting;
  • Develop a comprehensive Q&A grid;
  • Develop a strategy and encourage the development of back – up slides when preparing questions.



Speakers
avatar for Steffen Thirstrup

Steffen Thirstrup

Director, NDA Regulatory Advisory Board, NDA Advisory Services Ltd
Steffen Thirstrup is a medical doctor and board certified specialist in clinical pharmacology and therapeutics. He holds a PhD in pharmacology and has a long background in clinical internal medicine. He is an adjunct professor in pharmacotherapy at the Faculty of Health Sciences... Read More →
avatar for Lisa Peluso

Lisa Peluso

Director, Coaching and Client Engagement, PharmApprove, a member of the NDA Group
Lisa helps development teams to create and deliver clear, consistent, and convincing messages to critical audiences and decision-makers. She has coached hundreds of individuals and provided presentation, Q&A and facilitation training to teams in pharma, and biotech, and has helped... Read More →


Sunday June 23, 2019 1:30pm - 5:00pm
Room 6E San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

1:30pm

SC35: #35: Japan Regulatory Environment: Overview of the Organization, Processes, Systems, and Changes Effecting Pharmaceutical Development
Component Type: Tutorial
CE: ACPE 3.25 Knowledge UAN: 0286-0000-19-514-L04-P; CME 3.25; IACET 3.25; RN 3.25

Significant changes in Japanese pharmaceutical regulations and procedures are impacting the development of new drugs in Japan as well as global development programs. This short course will describe the major drivers of the regulatory system, including the Pharmaceuticals and Medical Devices Agency (PMDA) and Ministry of Health, Labor and Welfare (MHLW), regulatory procedures during drug development (consultations with PMDA and clinical trial notifications), the integration of Japanese drug development with East Asian and global drug development, orphan drug regulation and J-NDA preparation and review. Several development strategies available to address Japanese requirements for new drug approval, as well as selected post-approval requirements, will be discussed. An additional registration fee is required for all preconference short courses. Back to DIA 2019 Short Courses

Who should attend?

This short course is designed for professionals involved in regulatory affairs, project management, and clinical development who are involved with global development projects involving Japan.

Learning Objectives

At the conclusion of this course, participants should be able to:
  • Explain the major elements of the Japanese regulatory system;
  • Describe the regulatory procedures during development, registration, and post-approval;
  • Discuss specific attributes of the Japanese regulatory system and their impact on local and global development strategies.



Speakers
avatar for Alberto Grignolo

Alberto Grignolo

Corporate Vice President, Parexel
Alberto Grignolo, PhD is a Corporate Vice President at Parexel, and established the firm's Japan Consulting Services during a two-year assignment in Tokyo. Dr. Grignolo has served as an adviser on human subject protection in clinical trials to the Institute of Medicine of the National... Read More →
avatar for Yoshiaki Uyama

Yoshiaki Uyama

Director, Office of Medical Informatics and Epidemiology, Pharmaceuticals and Medical Devices Agency (PMDA)
Yoshiaki Uyama, PhD, is Director, Office of Medical Informatics and Epidemiology, for the Pharmaceuticals & Medical Devices Agency (PMDA), Japan. His experience in the safety review and assessment of new drugs for PMDA includes serving as the Review Director; as International Conference... Read More →


Sunday June 23, 2019 1:30pm - 5:00pm
Room 4 San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA
  • Credit Type ACPE, CME, IACET, RN
  • Tags Tutorial
 
Monday, June 24
 

11:00am

#106: Moving Forward in EU Pharmacovigilance
Component Type: Forum
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-19-523-L04-P; CME 1.00; IACET 1.00; RN 1.00

This session will introduce delegates to new ways of evaluating evidence of our activities and apply new types of science for our sector based on systems theory. By thinking how patients can be part of the pharmacovigilance team we can out new ways of more effectively managing benefit-risk.

Learning Objectives

Discuss ideas for describing the role and training needed for involving patients; Identify how to constructively criticize pros and cons of involving patients; Describe opportunities to apply systems theory to manage risk; Identify how evaluation of PRAC may help us more effectively manage risk.

Chair

Brian Edwards, DrMed

Speaker

System Analysis of UK Pregnancies Reported During and After Isotretinoin Administration
Brian Edwards, DrMed

Patient Perspective
François Houyez

Removal of Safety Concerns from EU-RMP: Lessons Learned from a Review of PRAC Meeting Minutes
Robert Massouh, MPharm, RPh



Speakers
avatar for Brian Edwards

Brian Edwards

Principal Consultant, Pharmacovigilance and Drug Safety, Vice-President ACRES, NDA Group
After his training in hospital medicine and clinical research for 14 years, Dr. Edwards joined the UK Medicines Control Agency (MHRA) in 1994 where he had various responsibilities as a pharmacovigilance assessor. In 1999 he joined Parexel to become Senior Medical Director before joining... Read More →
avatar for François Houyez

François Houyez

Treatment Information and Access Director, Health Policy Advisor, European Organisation for Rare Diseases (EURODIS)
François Houÿez is working at the European Organisation for Rare Diseases EURORDIS where he is Director of Treatment Information and Access, Policy Advisor. He has always been working as a patient advocate since the early 90s, first in the HIV/AIDS advocacy, and in rare diseases... Read More →
RM

Robert Massouh

Risk Management Product Lead, Pfizer Ltd
Robert is a Risk Management Product Lead at Pfizer, supporting the development and execution of Risk Management strategies for Pfizer products. He was previously at the MHRA working as a Scientific Assessor within the Benefit Risk Management Group. Robert is a registered Pharmacist... Read More →


Monday June 24, 2019 11:00am - 12:00pm
Room 6C San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA
  • Level Intermediate
  • Featured Topics Regulator Thinking,What's Next
  • Credit Type ACPE, CME, IACET, RN
  • Tags Forum

11:00am

#116: Harnessing Power of Advanced Technologies for Digital Transformation in Regulatory Affairs
Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-19-530-L04-P; CME 1.00; IACET 1.00; RN 1.00

This session will provide the insight to future of regulatory affairs in the digital world and how companies need to embrace advanced technology to disrupt the traditional way of handling regulatory information and submission.

Learning Objectives

Describe Regulatory 2.0 and the future of regulatory affairs and impact of advanced technology such as artificial intelligence and machine learning; Discuss how to build a regulatory intelligence system using voice assistant and machine learning; Illustrate how real world evidence ( RWE) gives a new paradigm shift for more predictable, effective, and value-based patient care and the use of RWE in regulatory affairs.

Chair

Susant Mallick, MBA

Speaker

Regulatory 2.0 and Digital Tranformation in Regulatory Affairs Using Advanced Technology
Susant Mallick, MBA

Impact of Artificial Intelligence and Machine Learning in Regulatory Affairs
Hans van Bruggen, MSc

Automated Risk Detection using Machine Learning in Clinical Trials
Francois Torche, MBA



Speakers
avatar for Hans van Bruggen

Hans van Bruggen

Senior Regulatory Affairs Consultant, Qdossier B.V.
Hans van Bruggen is Director of and Senior Regulatory Affairs Scientist at Qdossier and eCTDconsultancy. He has an MSc in Pharmaceutical Medicine from the University of Surrey (UK) and has worked within, or for, the pharmaceutical industry for more than 30 years in Global or European... Read More →
avatar for Susant Mallick

Susant Mallick

Leader and Evangelist, Healthcare and Life Sciences, EMEA, Amazon
Susant Mallick is leading Amazon AWS HCLS professional services for EMEA region. He comes up with 20+yrs of Pharma and IT background on building disruptive solutions/products in Clinical and Regulatory space. He is technology evangelist on cutting edge technology like (Artificial... Read More →
avatar for Francois Torche

Francois Torche

Chief Executive Officer, CluePoints
François holds a Master in Business Administration from the ICHEC School of Management, Brussels. Over the past 20 years in the pharmaceutical industry, he has held positions as statistical programmer, developer and IT project leader for companies such as GSK, UCB and IDDI. During... Read More →


Monday June 24, 2019 11:00am - 12:00pm
Room 6F San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA
  • Level Intermediate
  • Featured Topics Real World Evidence,Artificial Intelligence
  • Credit Type ACPE, CME, IACET, RN
  • Tags Session

11:00am

#117: International Regulatory Convergence
Component Type: Forum
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-19-537-L04-P; CME 1.00; IACET 1.00; RN 1.00

Discuss how regulators approach relative effectiveness in various jurisdictions. The opportunity arises at a time when the EU is discussing joint evaluations and legal framework. Canada is also working on interactions with the bodies responsible for this evaluation. Japan has initiated a pilot on relative effectiveness comparison.
We intend to show how international regulatory authorities could cooperate and share experience to ensure global coordination of data, studies, and evaluation in the context of global development.

Learning Objectives

Discuss how the main regulatory Agencies collaborate and work with those involved in relative effectiveness and added therapeutic value of medicines; Identify the common criteria used and determine when independence should be maintained; Examine specific issues with Advanced Therapies/regenerative medicines and added value; Describe the importance of collaboration at the international level

Chair

Agnès Saint-Raymond, DrMed, MD

Speaker

Panelist
Guido Rasi, MD

Panelist
Pierre Sabourin, MBA

Panelist
Brian O'Rourke, PharmD

Panelist
Giovanni Tafuri, PhD, MS

Panelist
Katsuaki Ura



Speakers
avatar for Brian O'Rourke

Brian O'Rourke

President and Chief Executive Officer, Canadian Agency for Drugs and Technologies in Health
Dr. Brian O’Rourke is the President and Chief Executive Officer of CADTH. He joined CADTH in January of 2009 following a distinguished career as a Pharmacist and Health Service Executive with the Canadian military. With over 30 years of experience in health care, Brian actively... Read More →
avatar for Guido Rasi

Guido Rasi

Executive Director, European Medicines Agency (EMA)
Prof Guido Rasi began his second term as Executive Director of EMA on 16 November 2015. From November 2014 to mid-November 2015, he served as EMA’s Principal Adviser in Charge of Strategy. From November 2011 to November 2014 he was the Executive Director of the EMA and a member... Read More →
avatar for Pierre Sabourin

Pierre Sabourin

Assistant Deputy Minister, Health Products and Food Branch, Health Canada
Pierre Sabourin was appointed Assistant Deputy Minister (ADM) of the Health Products and Food Branch (HPFB) at Health Canada in 2016. The branch plays a vital role in protecting and promoting the health and safety of all Canadians by excelling as a trusted scientific and regulatory... Read More →
avatar for Agnès Saint-Raymond

Agnès Saint-Raymond

Head of International Affairs, Head of Portfolio Board, European Medicines Agency (EMA)
Head of International Affairs, Head of Portfolio Board MD and qualified Paediatrician. Joined EMA in 2000. Head of Special Areas for Human Medicines (Paediatric Medicines, Orphan Medicines, Scientific Advice, SME Office, and Scientific Support & Projects) until 2013. Head of Portfolio... Read More →
avatar for Giovanni Tafuri

Giovanni Tafuri

Senior Scientific Officer, EUnetHTA
Giovanni Tafuri is the Senior Scientific Officer of the European Network for Health Technology Assessment (EUnetHTA) at the EUnetHTA Secretariat in the Netherlands. Prior to his appointment, he worked at both the HTA and the regulatory department of the Italian Medicines Agency (2006-2018... Read More →
avatar for Brian O'Rourke

Brian O'Rourke

President and Chief Executive Officer, Canadian Agency for Drugs and Technologies in Health
Dr. Brian O’Rourke is the President and Chief Executive Officer of CADTH. He joined CADTH in January of 2009 following a distinguished career as a Pharmacist and Health Service Executive with the Canadian military. With over 30 years of experience in health care, Brian actively... Read More →
KU

Katsuaki Ura

Deputy Director, Office of International Regulatory Affairs, Ministry of Health, Labour and Welfare (MHLW)
Mr. Katsuaki Ura is currently Deputy Director, Office of International Regulatory Affairs, Pharmaceuticals Safety and Environmental Health Bureau, Ministry if Health, Labour and Welfare (MHLW), Japan since April 2018. He is engaged in international regulatory harmonisation through... Read More →


Monday June 24, 2019 11:00am - 12:00pm
Room 6B San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

2:15pm

#136: Interpretation of New Pharmacovigilance Regulations: Key Insights
Component Type: Forum
Level: Basic
CE: ACPE 1.00 Knowledge UAN: 0286-0000-19-539-L04-P; CME 1.00; IACET 1.00; RN 1.00

Building on the ongoing collaborative efforts of nineteen sponsor companies, a key workstream has been established on how each is addressing major new pharmacovigilance regulations and guidances. This session will provide feedback on key learnings and best practices. Join the Clinical Safety & Pharmacovigilance Community for a follow up Round Table discussion on Tuesday, June 25, 9:30-10:30AM in the DIA Community Zone 1, Sails Pavilion.

Learning Objectives

Explain some of the challenges associated with major new pharmacovigilance guidelines; Discuss how companies are interpreting and implementing the aforementioned guidelines; Share best practices across the industry and learnings from feedback from regulators.

Chair

Ajay B. Singh

Speaker

Reference Satety Information: Implementing the Recommendations Noted in the CTFG
Ajay B. Singh

Update From MHRA
Mick Foy

Combination Products: Focus on FDA Guidance on Postmarketing Safety Reporting
Jane M. Carroll, BSN, MS, RN



Speakers
avatar for Jane Carroll

Jane Carroll

Head of Global Patient Safety Region - Americas, EMD Serono
Jane Carroll is the interim Head of Global Patient Safety Regions since February 25, 2019 and Head of Global Patient Safety Region Americas for the Biopharma business of Merck KGaA Darmstadt, Germany since 2015. Jane is responsible to ensure a robust system to collect and report adverse... Read More →
avatar for Mick Foy

Mick Foy

Head of Pharmacovigilance Strategy, Vigilance Intelligence, and Research Group, Medicines and Healthcare Products Regulatory Agency (MHRA)
Mick Foy is Head of Pharmacovigilance Strategy at the MHRA. Among is responsibilities is the running of the UK ADR reporting system and signal detection activities. Mick is also leads the MHRA's work with WHO and the Bill and Melinda Gates Foundation on capacity building in low and... Read More →
AS

Ajay Singh

Team Leader, Safety Evaluation and Risk Management, GlaxoSmithKline
Ajay is a nephrologist by training and currently serves as a team leader in the Safety Evaluation and Risk Management group at GSK. The main focus of the group currently is development of oncology/immune-oncology products. Ajay represents GSK on the TransCelerate work stream focused... Read More →


Monday June 24, 2019 2:15pm - 3:15pm
Room 6C San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

2:15pm

#146: Communications with Regulators Beyond Formal Meetings
Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-19-546-L04-P; CME 1.00; IACET 1.00; RN 1.00

FDA and industry experts share best practices when seeking advice via Critical Path Innovation Meetings (CPIM), parallel scientific advice, and the Initial Targeted Engagement for Regulatory Advice on CBER products (INTERACT).

Learning Objectives

Identify when you should seek advice beyond formal meetings with FDA; Discuss best practices in obtaining parallel scientific advice from FDA and EMA; Discuss best practices in obtaining advice via a Critical Path Innovation Meeting (CPIM); and, discuss the impact to regulatory strategies and timelines.

Chair

Khyati Roberts, PharmD, RPh

Speaker

Overview of FDA-EMA Parallel Scientific Advice
Sandra L. Kweder

Seeking Advice from EMA
Tânia Teixeira, PharmD

Overview of Critical Path Innovation Meetings
Chekesha Clingman, PhD, MBA

Industry Perspective on Communications Beyond Formal Meetings
Nicole Mahoney, PhD

Overview of INTERACT Meetings
Christopher Joneckis, PhD



Speakers
avatar for Chekesha Clingman

Chekesha Clingman

Associate Director for Strategic Partnerships, OTS, CDER, FDA
Dr. Chekesha Clingman is a Commander in the US Public Health Service and the Associate Director for Strategic Partnerships in the Office of Translational Sciences (OTS), Center for Drug Evaluation and Research (CDER), US Food and Drug Administration (FDA). Dr. Clingman manages the... Read More →
CJ

Christopher Joneckis

Associate Director for Review Management, CBER, FDA
*
avatar for Sandra Kweder

Sandra Kweder

Deputy Director, Liaison to the EMA, Office of International Programs, OC, FDA
Sandra L. Kweder, MD, is Deputy Director of the Europe Office in the FDA Office of International Programs. Dr. Kweder previously served for more than a decade as Deputy Director, Office of New Drugs, in FDA’s Center for Drug Evaluation & Research, where she actively led numerous... Read More →
avatar for Nicole Mahoney

Nicole Mahoney

Senior Director, Regulatory Policy, Flatiron Health
Nicole Mahoney is the Senior Director of Regulatory Policy at Flatiron Health, helping advance the use of real world evidence for regulatory decision making. Prior to her current role, she was a Director of Global Regulatory Policy at Merck, advocating for incentives to spur antibiotic... Read More →
avatar for Khyati Roberts

Khyati Roberts

Head US/Canada, Regulatory Policy and Intelligence, AbbVie, Inc.
Khyati leads regulatory policy advocacy efforts for the U.S. and Canada and coordiantes international harmonization efforts. She joined AbbVie in 2012 and has over 25 years of regulatory experience. Khyati has also provided regulatory advice to the biopharmaceutical industry on product... Read More →
avatar for Tânia Teixeira

Tânia Teixeira

EMA Liaison Official at FDA, European Medicines Agency (EMA)
Tania Teixeira is the EMA Liaison Official to FDA. She started her career in the pharmaceutical industry and joined EMA in 2004. She held positions as a Head of service for Referrals, dealing with emerging concerns which require a harmonised position across the EU, and as Product... Read More →


Monday June 24, 2019 2:15pm - 3:15pm
Room 6B San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA
  • Level Intermediate
  • Featured Topics Regulator Thinking
  • Credit Type ACPE, CME, IACET, RN
  • Tags Session

2:15pm

#147: Update from Health Canada: The Health Protection Branch
Component Type: Forum
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-19-552-L04-P; CME 1.00; IACET 1.00; RN 1.00

While we generally speak of Health Canada abroad, there is a need to understand that responsibilities for health-related matters are split between the Federal and Provincial and Territorial Governments. This sessions aims to provide a broad and comprehensive picture in order to draw a comprehensive picture of the activities within our mandates.

Learning Objectives

Describe the health system in Canada and the place of the regulator within that system; Discuss the most current regulatory innovations.

Chair

Agnes Victoria Klein, MD

Speaker

Regulatory Reform
Celia Lourenco, PhD

Naming of Biologics and Real World Evidence: Status in Canada
Agnes Victoria Klein, MD



Speakers
avatar for Agnes Klein

Agnes Klein

Senior Medical Advisor, Health Canada
Agnes V. Klein MD is currently the Senior Medical Advisor in the Director General's Office, in the Biologics and Genetic Therapies Directorate. Dr. Klein trained in Endocrinology at UofT and has interests in multiple aspects of drug development and medical bioethics. Dr. Klein is... Read More →
avatar for Celia Lourenco

Celia Lourenco

Director General, Biologics and Genetic Therapies Directorate, HPFB, Health Canada
Celia Lourenco, PhD is the Director General of the Biologics and Genetic Therapies Directorate (BGTD) of the Health Products and Food Branch of Health Canada. She was previously the Senior Executive Director of the Therapeutic Products Directorate (TPD) and held other management positions... Read More →


Monday June 24, 2019 2:15pm - 3:15pm
Room 6F San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

2:15pm

#148: Hot Topics in Digital Health: How is FDA’s Approach Evolving, and What Do Industry and Patients Need to Know?
Component Type: Forum
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-19-553-L04-P; CME 1.00; IACET 1.00; RN 1.00

FDA’s approach to digital health is evolving. The agency has issued a series of guidances and other policies in recent years on digital health technologies <b><b This panel will explore how these policies may impact patients and innovators. As part of this discussion, panelists will also consider how digital health policies are shifting and where additional work could be done to facilitate innovation. We’ll explore FDA’s thoughts on how DH could help to incorporate the patient voice, generate real world evidence, and improve postmarket surveillance. Join us for an interactive discussion among a diverse set of stakeholders.

Learning Objectives

Describe FDA’s current thinking on digital health; Assess the potential impact of digital technologies on FDA priorities such as patient voice, real world evidence generation, post-market safety; Assess future opportunities for digital health to impact medical product development.

Speakers
avatar for Nancy Myers

Nancy Myers

President and Founder, Catalyst Healthcare Consulting, Inc
Nancy Bradish Myers, JD is President/Founder of Catalyst Healthcare Consulting, a boutique regulatory advisory firm that helps innovators navigate FDA and policy circles. She served as a senior advisor in FDA's Office of the Commissioner and held senior positions at PhRMA, BIO and... Read More →
avatar for Jacqueline Corrigan-Curay

Jacqueline Corrigan-Curay

Director, Office of Medical Policy, CDER, FDA
Jacqueline Corrigan-Curay, J.D., M.D., serves as Director of the Office of Medical Policy (OMP) in the Center for Drug Evaluation and Research, FDA. OMP is comprised of the Office of Prescription Drug Promotion (OPDP) and the Office of Medical Policy Initiatives (OMPI). Dr. Corrigan-Curay... Read More →
avatar for Catherine Kopil

Catherine Kopil

Director, Research Partnerships, The Michael J. Fox Foundation for Parkinson's Research
As Director of Research Partnerships at The Michael J. Fox Foundation (MJFF), Katie focuses on building the Foundation’s capacity as an unprecedented stakeholder in Parkinson’s drug development — a nimble, patient-focused problem-solver whose efforts are demonstrably accelerating... Read More →
YP

Yarmela Pavlovic

Partner, Global Regulatory, Hogan Lovells


Monday June 24, 2019 2:15pm - 3:15pm
Room 6E San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA
  • Credit Type ACPE, CME, IACET, RN
  • Tags Forum

2:15pm

#149: Hype Versus Reality: Artificial Intelligence and Drug Development
Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-19-554-L04-P; CME 1.00; IACET 1.00; RN 1.00

This session will discuss maturation of the development of artificial intelligence (AI) over time and how AI has matured more for R&D and signal detection and less so for regulatory support.

Learning Objectives

Discuss how artificial intelligence has matured over time.

Chair

Ranjini Prithviraj

Speaker

Results of DIA (Tufts) Survey of 1000+ Members About Their Ideas About Digital Health
Mary Jo Lamberti, PhD, MA

Artificial Intelligence in Regulatory Affairs
Patrick Brady, PharmD

What is Believed Can Be Done Versus What is Now Being Done
David Meyers



Speakers
avatar for Patrick Brady

Patrick Brady

Vice President, Regulatory Affairs Head, Regulatory Policy and Intelligence, Bayer AG
Dr. Patrick Brady is Vice President, Regulatory Affairs at Bayer. In this role, he leads Bayer’s global regulatory policy and intelligence activities. He has more than 10 years of experience in Regulatory Affairs. Prior to joining Bayer, Patrick led international science and regulatory... Read More →
avatar for Mary Jo Lamberti

Mary Jo Lamberti

Associate Director of Sponsored Research, Research Assistant Professor, Tufts Center for the Study of Drug Development
Mary Jo Lamberti leads multi-company sponsored research studies at the Tufts Center for the Study of Drug Development (CSDD). She has extensive experience conducting research on biopharmaceutical industry practices and trends affecting contract research organizations and investigative... Read More →
avatar for David Meyers

David Meyers

National Director, US Life Sciences, Microsoft
Dave Meyers is National Director - US Life Sciences at Microsoft; focused on strategy, business development, and the solutions portfolio in pharma, genomics and precision medicine. He joined Microsoft in 1997 - held various technical, program management, and product development positions... Read More →
avatar for Ranjini Prithviraj

Ranjini Prithviraj

Global Associate Director, Content Collaboration, DIA
Dr. Ranjini Prithviraj is Global Associate Director, Content Collaboration, at DIA. She provides strategic thought leadership and delivers content that advances DIA’s mission through various media, including traditional journal publications. Prior to DIA, she was a Managing Editor... Read More →


Monday June 24, 2019 2:15pm - 3:15pm
Room 11B San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

2:15pm

#150: TFDA Town Hall: Focus on Regenerative Medicine
Component Type: Forum
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-19-734-L04-P; CME 1.00; IACET 1.00; RN 1.00

Regenerative medicine is a potential treatment that can help repair or replace damaged or diseased human cells or tissues to restore normal function. This forum will discuss the regulatory aspects and experience with regenerative medicine.

Learning Objectives

Discuss regulations governing implementation or use of specific medical technology, examination, laboratory testing and medical devices; Describe the management of regenerative pharmaceutical products; Describe the experience of reviewing regenerative medicine affairs.

Chair

Shou-Mei Wu, PhD

Speaker

New Regulation on Cell Therapy: Challenge and Prospect
Chung-Liang Shih, PhD

Regulation of Cell and Gene Therapy Products
Lien-Cheng Chang, PhD

Regulatory Considerations and Experiences for the Evaluation of Regenerative Medicinal Products
I-Ning Tang, MD, MSc



Speakers
avatar for Chung-Liang Shih

Chung-Liang Shih

Director-General, Department of Medical Affairs, Ministry of Health & Welfare
Dr. Shih, Chung-Liang serves as the Director-General of Department of Medical Affairs, MOHW of Taiwan since 2016. From 2008 to 2012, he was the head of Bureau of Medical Affaires that oversees the health care system and leads the health care reform including the programs addressing... Read More →
avatar for I-Ning Tang

I-Ning Tang

Section Chief, Regulatory Science, Center for Drug Evaluation (CDE)
Dr. Tang is currently the Section chief of Regulatory science, Center of consultation at Taiwan Center for Drug Evaluation. Since joining in the regulatory science field in 2010, Dr. Tang has been involved in various projects. She is an experienced medical reviewer in evaluating market... Read More →
avatar for Shou-Mei Wu

Shou-Mei Wu

Director General, TFDA
Dr. Wu has been the Director-General of the Food and Drug Administration, Taiwan (TFDA) since February 2017. Now she is in charge of the overall policies. Under Dr. Wu’s leadership, TFDA have actively safeguarded food, drug, medical devices and cosmetics, and made best effort overcoming... Read More →
avatar for Lien-Cheng Chang

Lien-Cheng Chang

Section Chief, Division of Medicinal Products, TFDA
Lien-Cheng (Eric) Chang received his PhD degree from the Institute of Biopharmaceutical Sciences at Yang Ming University. Previously, he worked in Taichung Hospital and NTU Hospital in Clinical Pharmacy Services and also served in the former Bureau of Pharmaceutical Affairs for six... Read More →


Monday June 24, 2019 2:15pm - 3:15pm
Room 14B San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA
  • Featured Topics Precision Medicine
  • Credit Type ACPE, CME, IACET, RN
  • Tags Forum

3:30pm

#165: Drug Development Tools in a Digital Era
Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-19-562-L04-P; CME 1.00; IACET 1.00; RN 1.00

Explore opportunities and challenges ahead for digital drug development tools (DDTs) from an EMA, consortium, and industry perspective. Analyze how existing regulatory frameworks apply to the emerging field of digital health technologies including case examples, and discuss issues to be addressed to promote advancement of digital DDTs.

Learning Objectives

Describe the EMA, industry, and consortium views on digital drug development tools (DDTs); Discuss the growing value of digital health tools as part of the drug development process including key learnings from sponsor experience; Evaluate case example(s) in the development of digital DDTs.

Chair

Lauren Oliva, PharmD, RPh

Speaker

Critical Path Update
Stephen Joel Coons, PhD

EMA Update
Zahra Hanaizi, PharmD, MPharm

Industry Update
Josh Cosman, PhD



Speakers
avatar for Josh Cosman

Josh Cosman

Associate Director, Digital and Quantitative Medicine, Biogen
Dr. Cosman has 15 years’ experience exploring novel markers of cognition, motor function, and quality of life in healthy aging and neurological disorders. His work has focused on the development of functional measures using scalable research-grade tools and consumer devices in a... Read More →
avatar for Zahra Hanaizi

Zahra Hanaizi

Scientific officer, PRIME Coordinator, Scientific and Regulatory Management Dept, European Medicines Agency (EMA)
Zahra Hanaizi joined EMA in 2007 as Product Team Leader, managing centralised medicinal products initial marketing authorisation applications, maintenance and life-cycle activities, mostly in the therapeutic area of Oncology. Since 2015, she has been working on the creation, implementation... Read More →
avatar for Lauren Oliva

Lauren Oliva

New Technologies Global Regulatory Policy Lead, Biogen
Lauren Oliva is Global Regulatory Policy Lead for New Technologies at Biogen. She oversees the development and execution of R&D’s policy roadmap for digital health tools and gene therapy to enable Biogen’s neuroscience portfolio. In her time at Biogen she launched a widely used... Read More →
avatar for Stephen Coons

Stephen Coons

Executive Director, PRO Consortium, Critical Path Institute
Stephen Joel Coons is Executive Director of the Patient-Reported Outcome (PRO) Consortium at Critical Path Institute (C-Path). C-Path, an independent nonprofit organization, established the PRO Consortium in cooperation with the U.S. Food and Drug Administration and the pharmaceutical... Read More →


Monday June 24, 2019 3:30pm - 4:30pm
Room 6F San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

3:30pm

#166: Updates on China Regulatory Reform
Component Type: Forum
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-19-563-L04-P; CME 1.00; IACET 1.00; RN 1.00

In this forum, experts from government, academia and industry will present and discuss updates in NMAP's regulatory reform and progress in drug review and approval, and development of Chinese pharmacopeia 2020.

Learning Objectives

Discuss and understand the latest progress in NMPA’s regulatory reform and the impacts on Chinese pharmaceutical regulation and drug development; Recognize China’s evolving regulatory environment and future development.

Chair

Ling Su, PhD

Speaker

Regulatory Reform Enhancing Drug Review and Approval
Xiao Yuan Chen, PhD

Regulatory Reform: Industry EWxoerience and Perspective
Wendy Yan, MD, MBA

Brief Introduction on Chinese Pharmacopoeia 2020
Xiaoxu Hong



Speakers
avatar for Ling Su

Ling Su

Professor, Shenyang Pharmaceutical University
Ling Su, PhD, is Professor and Director, Institute of Drug Regulatory Science in Shenyang Pharmaceutical University, and a Venture Partner with Lilly Asia Ventures. Dr. Su has over 25 years of experience in drug regulatory and development. He had worked in the Chinese regulatory agency... Read More →
avatar for Xiao Yuan Chen

Xiao Yuan Chen

Office Director, Tsinghua University
XH

Xiaoxu Hong

Deputy Division Director, Chinese Pharmacopoeia Commission
avatar for Wendy Yan

Wendy Yan

Senior Vice President, Head of Regulatory Affairs, BeiGene (Beijing) Co., Ltd.
Wendy Yan is Senior Vice President, Global Head of Regulatory Affairs, for BeiGene, a commercial-stage biotechnology company focused innovative molecularly-targeted and immuno-oncology drugs for treatment of cancer. She has more than two decades of regulatory affairs experience, having... Read More →


Monday June 24, 2019 3:30pm - 4:30pm
Room 6E San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

3:30pm

#167: Strategic Priorities of the International Coalition of Medicines Regulatory Authorities in an Increasingly Globalized Industry
Component Type: Forum
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-19-571-L04-P; CME 1.00; IACET 1.00; RN 1.00

The International Coalition of Medicines Regulatory Authorities (ICMRA) will explore its strategic priorities in the context of a globalized world. To overcome these challenges ICMRA can champion greater harmonisation and convergence. The session will be delivered by members of ICMRA and conclude with a panel discussion of questions raised by the audience.

Learning Objectives

Describe strategic priorities of ICMRA in the context of the globalized world.

Chair

Ian Hudson

Speaker

Overview of ICMRA and Strategic Priorities: Including Vigilance, Track, and Trace
Guido Rasi, MD

The 'Innovation' Priority Project
Lorraine Nolan, PhD

Current Topics of Interest to ICMRA Members Including Biosimilars and AMR
John Skerritt, PhD

Panelist
Yasuhiro Fujiwara, MD, PhD

IFPMA View
David B. Jefferys, MD



Speakers
avatar for Yasuhiro Fujiwara

Yasuhiro Fujiwara

Chief Executive, Pharmaceuticals and Medical Devices Agency (PMDA)
Dr. Yasuhiro Fujiwara was previously Director-General, Strategic Planning Bureau of the National Cancer Center, and the Deputy Director of the Hospital (Research), National Cancer Center Hospital. He is a medical oncologist, specializing in breast cancer. Before joining NCCH, he was... Read More →
avatar for Guido Rasi

Guido Rasi

Executive Director, European Medicines Agency (EMA)
Prof Guido Rasi began his second term as Executive Director of EMA on 16 November 2015. From November 2014 to mid-November 2015, he served as EMA’s Principal Adviser in Charge of Strategy. From November 2011 to November 2014 he was the Executive Director of the EMA and a member... Read More →
avatar for Lorraine Nolan

Lorraine Nolan

Chief Executive, Health Products Regulatory Authority (HPRA)
Ms Nolan joined the HPRA in 2001 and since that time has held a number of different roles at senior level within the organisation across a range of areas including inspection and authorisation of human medicines, medical devices, cosmetics and controlled substances. Prior to assuming... Read More →
avatar for Ian Hudson

Ian Hudson

Chief Executive, Medicines and Healthcare products Regulatory Agency (MHRA)
Dr Ian Hudson became Chief Executive of the MHRA in September 2013. He is a physician who practiced as a paediatrician prior to working in the pharmaceutical industry in clinical research and development. In 2001 he joined the former Medicines Control Agency as its Licencing Division... Read More →
avatar for John Skerritt

John Skerritt

Deputy Secretary for Health Products Regulation, Department of Health
Dr John Skerritt joined the Australian Department of Health in 2012 and is currently a Deputy Secretary and member of the executive team of the Department of Health. He was formerly the National Manager of the Therapeutic Goods Administration (TGA), until his role was expanded and... Read More →
avatar for David Jefferys

David Jefferys

Chair of the IFPMA Regulatory Science Committee; Senior Vice President, Eisai


Monday June 24, 2019 3:30pm - 4:30pm
Room 6B San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA
  • Level Intermediate
  • Featured Topics Regulator Thinking
  • Credit Type ACPE, CME, IACET, RN
  • Tags Forum
 
Tuesday, June 25
 

8:00am

#208: Patient Focus as Part of the Regulatory Affairs DNA: Opportunities and Challenges
Component Type: Forum
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-19-576-L04-P; CME 1.25; IACET 1.25; RN 1.25

Patients are at the heart of everything we do. Integrating patient focus into the regulatory mindset can be a challenge. Panelists will discuss their experiences, share best practices, and describe challenges in achieving this goal.

Learning Objectives

Identify best practices for integrating a patient focus mindset into the regulatory affairs organization; Recognize challenges faced by leaders in achieving this goal; Describe experiences with implementing patient focused practices as part of regulatory strategies.

Chair

Max Wegner, PharmD, RPh

Speaker

FDA Perspective
Theresa Mullin, PhD

Patient Representative
Marc M. Boutin, JD

Industry Perspective
Miu Chau, PhD

Patient Representative
Jill Bonjean, MSc



Speakers
avatar for Jill Bonjean

Jill Bonjean

Corporate Relations Advisor, Rare Diseases International
Jill Bonjean is Corporate Relations Advisor for Rare Diseases International - the global alliance of rare disease patient organizations. Jill works with RDI and other patient organizations to build effective relationships with companies and other partners. Previously, she's managed... Read More →
avatar for Marc Boutin

Marc Boutin

Chief Executive Officer, National Health Council (NHC)
Marc M. Boutin, JD, is the Chief Executive Officer of the National Health Council. He has been a leading voice for greater patient involvement at every stage of the health care continuum. Boutin has been actively involved in patient advocacy organization management, health advocacy... Read More →
MC

Miu Chau

Senior Regulatory Program Director, Genentech, A Member of the Roche Group
Miu joined Genentech in 2008 and held different roles. Currently she is a Senior Regulatory Program Director responsible for the global regulatory strategy and execution of projects and leading a matrix team of regulatory professionals in oncology. Miu had been the Global Head of... Read More →
avatar for Theresa Mullin

Theresa Mullin

Associate Director for Strategic Initiatives, OCD, CDER, FDA
Principal advisor on strategy including CDER international cooperation and harmonization, application of decision science, and other initiatives. Leads FDA Patient Focused Drug Development, and heads the FDA delegation to ICH. Previous work includes leading FDA negotiations for 2002... Read More →
avatar for Max Wegner

Max Wegner

Head Regulatory Affairs, Bayer AG
After studying pharmacy at the University of Bonn, Max Wegner commenced his PhD in pharmacology at Bayer and started in Regulatory Affairs as a Dossier Manager in 1996 in Wuppertal. Since then, Max has been employed in various functions within Product Development at Bayer and also... Read More →


Tuesday June 25, 2019 8:00am - 9:15am
Room 5AB San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

8:00am

#213: Facilitating Access: Patient Perspectives on a Streamlined Development Approach for Treatments for Severely-Debilitating or Life-Threatening Diseases
Component Type: Forum
Level: Basic
CE: ACPE 1.25 Knowledge UAN: 0286-0000-19-581-L04-P; CME 1.25; IACET 1.25; RN 1.25

This forum will introduce a streamlined approach to the development of therapies to address severely-debilitating or life threatening diseases and consider the potential value of global regulatory guidance.

Learning Objectives

Discuss the use of streamlined development programs to expeditiously deliver promising new treatments to patients with severely debilitating or life-threatening diseases from the patient perspective.

Chair

Judith Prescott, DVM, PhD

Speaker

Proposal for Development of Pharmaceuticals for Severly-Debilitating or Life Threatening Indications
Judith Prescott, DVM, PhD

Severely Debilitating or Life-Threatening Hematologic Disorders: Nonclinical Considerations
Haleh Saber, PhD

Perspective from The Patients' Academy For Research Advocacy
Susan Schaeffer

Panelist
Catherine Kopil, PhD

Panelist
Isabelle Lousada, MA



Speakers
avatar for Catherine Kopil

Catherine Kopil

Director, Research Partnerships, The Michael J. Fox Foundation for Parkinson's Research
As Director of Research Partnerships at The Michael J. Fox Foundation (MJFF), Katie focuses on building the Foundation’s capacity as an unprecedented stakeholder in Parkinson’s drug development — a nimble, patient-focused problem-solver whose efforts are demonstrably accelerating... Read More →
avatar for Isabelle Lousada

Isabelle Lousada

Chief Executive Officer and President, Amyloidosis Research Consortium
As a former patient, Lousada has spent 20 years dedicated to patient advocacy, accelerating drug development, and promoting awareness of rare diseases. Lousada founded the Amyloidosis Research Consortium (ARC) in 2015 to address the critical needs in clinical trials and related research... Read More →
avatar for Judith Prescott

Judith Prescott

Executive Director, Safety Assessment, Merck & Co., Inc.
Judith Prescott, DVM, PhD, DACVP is an Executive Director in Safety Assessment, Merck & Co, Inc. She has responsibility for all nonclinical drug development programs supporting FIH through post marketing across the Merck portfolio including small molecules, biologics, and vaccines... Read More →
HS

Haleh Saber

Deputy Director, DHOT/ OHOP/ CDER, FDA
Dr. Saber is the Deputy Director in the Division of Hematology Oncology Toxicology (DHOT). In this role, she provides leadership for day-to-day activities, leads and coordinates scientific research, and participates in guidance development. Dr. Saber has extensive industry and regulatory... Read More →
SS

Susan Schaeffer

President and Chief Executive Officer, The Patients' Academy For Research Advocacy
Susan founded The Patients’ Academy for Research Advocacy in 2018 to equip patients and care partners to apply their unique expertise to inform the development of new and better medicines. She spent 15 years at biopharmaceutical industry journal BioCentury informing and educating... Read More →


Tuesday June 25, 2019 8:00am - 9:15am
Room 6B San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA
  • Level Intermediate
  • Featured Topics Patient Focused,Advanced Therapies,Rare Disease
  • Credit Type ACPE, CME, IACET, RN
  • Tags Forum

8:00am

#214: Global Pediatric Policy Update: Are You Ready to Implement FDARA Section 504?
Component Type: Session
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-19-582-L04-P; CME 1.25; IACET 1.25; RN 1.25

The new requirements in FDARA Section 504 represent a significant paradigm shift in pediatric oncology development. This session will review these requirements and their likely global impacts, as well as possible mitigation strategies.

Learning Objectives

Describe and explain the new provisions in FDARA Section 504 that will impact pediatric oncology development in the US within the context of the EU requirements; Analyze the implications and assess the impact of these new provisions on global pediatric oncology development; Describe the use of Master Protocols as a possible mitigation strategy.

Chair

Melodi J McNeil, MS, RPh

Speaker

Industry Perspective: Industry Perspective: Master Protocols as an Option to Design and Conduct Pediatric Oncology Studies under FDARA, Sec 504
Pamela Simpkins, MBA

FDARA 504 Overview/Implementation Planning
Gregory Reaman, DrMed, MD

Lessons Learned from Two Public-Private Partnership Master Protocols
Stacey J. Adam, PhD



Speakers
avatar for Stacey Adam

Stacey Adam

Director, Cancer Reserach Partnerships, Foundation for the National Institutes of Health (FNIH)
Dr. Stacey Adam is the Director of Cancer Research Partnerships at the Foundation for the National Institute of Health (FNIH). She oversees major partnerships, including the Partnership for Accelerating Cancer Therapies (PACT), the Lung Master Protocol (Lung-MAP) clinical trial, and... Read More →
avatar for Melodi McNeil

Melodi McNeil

Director, AbbVie, Inc.
Melodi J. McNeil, R.Ph., M.S. Ms. McNeil is currently a Director in AbbVie’s Rockville, Maryland Regulatory Policy and Intelligence office. She identifies, assesses, and comments on proposed regulatory policies, and ensures appropriate AbbVie personnel are aware of emerging and... Read More →
avatar for Gregory Reaman

Gregory Reaman

Associate Director for Pediatric Oncology , OCE, OC, FDA
Dr. Gregory Reaman is a pediatric oncologist with a long career in clinical investigation and translational research. Prior to joining the FDA in 2011, he was the inaugural Chair of the Children's Oncology Group and was previously the Associate Chair for Scientific Affairs and the... Read More →
avatar for Pamela Simpkins

Pamela Simpkins

Senior Director & Strategy Lead, Child Health Innovation Leadership Dept. (CHILD, Johnson & Johnson
Pamela L. Simpkins, MBA is a biopharmaceutical business leader with 20+ years of experience in leading strategic initiatives and departments across R&D, corporate strategy, regulatory, marketing, business development and finance. She leads internal and external innovation programs... Read More →


Tuesday June 25, 2019 8:00am - 9:15am
Room 6F San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA
  • Level Intermediate
  • Featured Topics Precision Medicine,Advanced Therapies,Rare Disease,Innovative Trial Design
  • Credit Type ACPE, CME, IACET, RN
  • Tags Session

8:00am

#216: Identifying the Reference Listed Drug for ANDA Submission, Overview of FDA’s Orange Book, and Exclusivities for NDAs and ANDAs
Component Type: Session
Level: Basic
CE: ACPE 1.25 Knowledge UAN: 0286-0000-19-590-L04-P; CME 1.25; IACET 1.25; RN 1.25

FDA’s Orange Book and other information resources are essential to potential applicants in developing their generic submissions. This session will provide an overview of these resources and the different information that each contains.

Learning Objectives

Describe the content of FDA’s Orange Book; Identify and understand drug product, patent, exclusivity, and therapeutic equivalence information in the Orange Book; Explain FDA’s process for making exclusivity determinations. Describe the process to qualify for Paragraph IV and Competitive Generic Therapy 180-day exclusivities as well as events which may result in exclusivity forfeiture. Understand which applicants are blocked by these exclusivities and triggering events.

Chair

Kendra Stewart, PharmD, RPh

Speaker

Orange Book Overview
Kendra Stewart, PharmD, RPh

Identifying the Reference Listed Drug for ANDA Submissions
Martha Nguyen, JD

Understanding Exclusivities for ANDAs: 180-Day Exclusivity and Competitive Generic Therapy Exclusivity
Martin Shimer



Speakers
avatar for Martha Nguyen

Martha Nguyen

Director, Division of Policy Development, OGD, CDER, FDA
Martha Nguyen is the Director of the Division of Policy Development in FDA CDER's Office of Generic Drugs, where she provides strategic leadership and direction on broad policy issues affecting generic drugs and oversees the development and clearance of regulations, guidance documents... Read More →
avatar for Martin Shimer

Martin Shimer

Deputy Director, Division of Legal and Regulatory Support, FDA
Captain Martin Shimer is the Deputy Director of the Division of Legal and Regulatory Support within the Office of Generic Drugs Policy (OGDP) at the Food and Drug Administration (FDA). He was commissioned as an officer in the US Public Health Service in July 2000 and joined the Office... Read More →
avatar for Kendra Stewart

Kendra Stewart

Supervisor, Orange Book Staff, Office of Generic Drug Policy, CDER, FDA
Captain Kendra Stewart is the Supervisor of the Orange Book Staff within the Office of Generic Drugs Policy (OGDP) at the Food and Drug Administration (FDA). She began her career in 2001 as a pharmacist for the Veterans Affairs Hospital. In 2003, she was commissioned as a Lieutenant... Read More →


Tuesday June 25, 2019 8:00am - 9:15am
Room 11B San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA
  • Featured Topics Generics,Regulator Thinking
  • Credit Type ACPE, CME, IACET, RN
  • Tags Session

8:00am

#215: Conducting Clinical Trials with GMOs: Strategies to Overcome Regulatory, Operational, and Patient Enrollment Challenges
Component Type: Forum
Level: Intermediate
CE: ACPE 1.25 Application UAN: 0286-0000-19-589-L04-P; CME 1.25; IACET 1.25; RN 1.25

Global clinical trials conducted with GMOs pose unique regulatory, logistical and strategic challenges. This session will presents case studies addressing lessons learned and perspectives from regulatory authorities, investigators and patients.

Learning Objectives

Describe strategies to optimize the conduct of clinical trials with GMOs, in order to facilitate approval across multiple layers and regulatory bodies; Discuss efforts among different stakeholders (Sponsor, CRO, vendors) to enable adequate IP management; Identify how to implement educational plans to overcome cultural barriers (patients, families and site staff) and facilitate enrolment completion.

Chair

Esther Mahillo, PhD

Speaker

Clinical Research with GMOs: Regulatory, Ethical, and Biosafety Challenges
Daniel Kavanagh, PhD

Operational and Logistical Aspects of Working with GMOs
Esther Mahillo, PhD

Patient Perspective
Kathleen Higgins



Speakers
avatar for Daniel Kavanagh

Daniel Kavanagh

Senior Scientific Advisor, Gene Therapy, WIRB-Copernicus Group (WCG)
Dr. Kavanagh is Senior Scientific Advisor, Gene Therapy at WIRB-Copernicus Group (WCG). Prior to joining WCG, he was Assistant Professor of Medicine and Institutional Biosafety Committee Vice Chair at Harvard Medical School, Assistant Immunologist at the Massachusetts General Hospital... Read More →
avatar for Esther Mahillo

Esther Mahillo

Customer Relationship Executive, Oncology and Hematology, Syneos Health
I have been working for 24 years in the clinical research arena. Over the past 20 years, I have conducted 47 phase I-III clinical trials enrolling more than 13,000 patients at over 1,000 sites in 50 countries.I obtained my PhD with a research on the role of multicatalytic proteinase... Read More →
avatar for Kathleen Higgins

Kathleen Higgins

Director of Community Outreach, Li Fraumeni Syndrome Association


Tuesday June 25, 2019 8:00am - 9:15am
Room 6E San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA
  • Level Intermediate
  • Featured Topics What's Next,Patient Focused,Advanced Therapies
  • Credit Type ACPE, CME, IACET, RN
  • Tags Forum

8:00am

#218: Real World Data to Real World Evidence
Component Type: Session
Level: Advanced
CE: ACPE 1.25 Knowledge UAN: 0286-0000-19-583-L04-P; CME 1.25; IACET 1.25; RN 1.25

We discuss the advantages and disadvantages of real world data and real world evidence compared to other sources. Join the Clinical Research Community for a follow up Round Table discussion on Wednesday, June 26, 9:30-10:30AM in the DIA Community Zone 1, Sails Pavilion.

Learning Objectives

Describe the importance of real world data for answering regulatory questions; Discuss the benefits and challenges of evidence derived from real world sources compared to clinical trials.

Chair

Richard Zink, PhD

Speaker

Real World Data to Real World Evidence: A Case Study of Direct-Acting Antivirals for the Treatment of Hepatitis C Infection
Richard Zink, PhD

Machine Learning 2.0: Causal Models and Big Data in the Era of Real World Evidence
Andrew Wilson, PhD, MS

Matching with Replacement: The COLLECTION of ‘Personal’ EFFECTs Should be Handled with Care
Sarah Anselm Short, MPH



Speakers
SS

Sarah Short

Sr. Statistical Mgr, RWE - Biostats & Medical Writing, ICON plc.
Sarah has over ten years’ experience in health related statistical analysis, primarily in disease or product registries and other prospective observational studies. Her experience encompasses a wide array of therapeutic areas and study designs. As a Senior Statistical Manager, she... Read More →
avatar for Andrew Wilson

Andrew Wilson

Director, Pharmacoepidemiology and Statistics RWDS, PAREXEL
I lead the pharmacoepidemiology & statistics initiatives within Real World Data Services at PAREXEL. To be part of the real world evidence movement, it is essential we embrace the data generation process, and keep data context as all-important. Real world data typically comes from... Read More →
avatar for Richard C. Zink

Richard C. Zink

Senior Director, Data Management and Statistics, TARGET Pharmasolutions Inc
Richard C. Zink is Senior Director of Data Management and Statistics at TARGET PharmaSolutions. He is the 2019 Chair of the Biopharmaceutical Section of the American Statistical Association, host of the Biopharmaceutical Section Statistics Podcast, and Associate Editor for the DIA... Read More →


Tuesday June 25, 2019 8:00am - 9:15am
Room 3 San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

8:00am

#219: Personalized Healthcare and Clinical Outcomes: How Real World Endpoints Can Improve Approval and Access to Medicine?
Component Type: Forum
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-19-584-L04-P; CME 1.25; IACET 1.25; RN 1.25

This forum will present examples evaluating real world endpoints using electronic health records, current application, and challenges. A panel of experts will provide interpretation, discuss approaches to increase its reliability and acceptability. Join the Study Endpoints Community for a follow up Round Table discussion on Wednesday, June 26, 9:30-10:30AM in the DIA Community Zone 2, Sails Pavilion.

Learning Objectives

Describe opportunities and challenges utilizing real world endpoints in health care; Discuss examples and approaches to increase objectivity and reliability of real world endpoints; Explain implications for regulatory and payer decision-making.

Chair

Natalia Sadetsky, MD, PhD

Speaker

Panelist
Natalia Sadetsky, MD, PhD

Real World Data in 2019: What's New
Michelle Hoiseth

Real World Data Model with Visualization for Value and Access
Sharon Hensley Alford, PhD, MPH

Panelist
Ronald Cantrell, PhD, MPH

Panelist
Gillis Carrigan, PhD, MS



Speakers
avatar for Michelle Hoiseth

Michelle Hoiseth

Chief Data Officer, Parexel
Michelle has been working in the drug and device development industry for 30 years. She has enjoyed a variety of positions that allowed her to create product development plans, run study operations, and support product commercialization objectives. Michelle presently leads PAREXEL’s... Read More →
avatar for Natalia Sadetsky

Natalia Sadetsky

Senior Scientist, Genentech, A Member of the Roche Group
Natalia Sadetsky, is an medical doctor and epidemiologist by training. She has over 18 years of academic and industry experience in oncology. She joined Genentech, a member of a Roche group in 2013 and currently a Senior Data Scientist in Personalized Health Care Data Science, RWD... Read More →
avatar for Sharon Hensley Alford

Sharon Hensley Alford

Offering Management, IBM Watson Health
Sharon Hensley Alford, PhD is Associated Chief Health Officer for Data & Evidence within the Life Science (LS) pillar of IBM Watson Health. Dr. Alford has been working on data and analytic strategy for life science clients since she joined IBM 2+ years ago. Prior to IBM, she worked... Read More →
RC

Ronald Cantrell

Senior Director and Principal Data Scientist, Verana Health
Ronald Cantrell leads clinical data science at Verana as Senior Director and Principal Data Scientist. He has over a decade of experience working with and analyzing real world data in the life science industry. He previously served as Principal Data Scientist at Genentech, where he... Read More →
GC

Gillis Carrigan

Director, Epidemiology, Covance Market Access
Dr. Carrigan has over 20 years of experience in observational studies with a focus on study design and methodology. He is experienced with the use of electronic medical records (EMR), administrative claims data, and registry data. He has worked with observational data in a number... Read More →


Tuesday June 25, 2019 8:00am - 9:15am
Room 14B San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

10:30am

#229: An Industry Collaboration on Pharmacovigilance Analytics
Component Type: Forum
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-19-592-L04-P; CME 1.00; IACET 1.00; RN 1.00

This session will discuss an initiative and how it is facilitating biopharmaceutical companies to enable earlier and systematic identification of potential safety issues to help minimize harm to patients, improve quality of analytic methods to improve product development, and deliver accelerated method development through collaboration and piloting. Join the Clinical Safety & Pharmacovigilance Community for a follow up Round Table discussion on Wednesday, June 26, 12:45-1:45PM in the DIA Community Zone 1, Sails Pavilion.

Learning Objectives

Discuss methods for collaborating and innovating in analytics to improve patient safety; Identify how to build awareness of opportunities for greater alignment across the industry in signal detection and management; Describe industry best practices of current and future signal detection and management.

Chair

Andres Gomez, PhD, MPH

Speaker

Panelist
John Van Stekelenborg, PhD, MBA, MSc

Panelist
Neal Grabowski, MS



Speakers
AG

Andres Gomez

Vice President, Head of Epidemiology, Safety Science, and Analytics, Bristol-Myers Squibb
Dr. Gomez joined at Bristol Myers Squibb Company as an Associate Director of Pharmacoepidemiology in the Global Pharmacovigilance and Epidemiology department in 2003. He currently heads the epidemiology, safety science and analytics groups which is accountable for the real world research... Read More →
NG

Neal Grabowski

Director, Global Patient Safety and Labeling, Amgen Inc.
Neal Grabowski joined Amgen in 2014 as the Signal Management Officer responsible for signal detection and management processes including the implementation of numerous systems, process oversight and continual improvement and vendor management. He is currently the Head of Safety Signal... Read More →
avatar for John Van Stekelenborg

John Van Stekelenborg

Director, Group Lead, Methods and Analysis, Johnson & Johnson
John van Stekelenborg is the Head of the Methods & Analysis group in the Global Medical Safety department of Janssen, the Pharmaceutical Companies of Johnson & Johnson. John has been with J&J since 2002 and assumed leadership of the M&A group in 2011 where he leads a group of data... Read More →


Tuesday June 25, 2019 10:30am - 11:30am
Room 6C San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

10:30am

#240: Harmonizing Regulatory Science Through the International Council for Harmonization (ICH)
Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-19-599-L04-P; CME 1.00; IACET 1.00; RN 1.00

This session will provide an overview of the ICH Association and offer insight into strategic, long-term views on advancing global convergence of regulatory science through ICH.

Learning Objectives

Identify the value and benefits of global regulatory harmonization and the ICH; Discuss opportunities to advance regulatory science through ICH; Describe the current harmonization initiatives that are currently underway and the impact on global drug development.

Chair

Camille Jackson

Speaker

FDA Update
Theresa Mullin, PhD

EMA Update
Agnès Saint-Raymond, DrMed, MD

MHLW Update
Naoyuki Yasuda, MS



Speakers
CJ

Camille Jackson

Associate Vice President, Science and Regulatory Advocacy, Pharmaceutical Research and Manufacturers of America (PhRMA)
Camille Jackson is currently Associate Vice President of Science and Regulatory Advocacy at PhRMA. In this role, she manages PhRMA’s international regulatory policy initiatives including engagement in the International Council for Harmonization (ICH) and the APEC Regulatory Harmonization... Read More →
avatar for Theresa Mullin

Theresa Mullin

Associate Director for Strategic Initiatives, OCD, CDER, FDA
Principal advisor on strategy including CDER international cooperation and harmonization, application of decision science, and other initiatives. Leads FDA Patient Focused Drug Development, and heads the FDA delegation to ICH. Previous work includes leading FDA negotiations for 2002... Read More →
avatar for Agnès Saint-Raymond

Agnès Saint-Raymond

Head of International Affairs, Head of Portfolio Board, European Medicines Agency (EMA)
Head of International Affairs, Head of Portfolio Board MD and qualified Paediatrician. Joined EMA in 2000. Head of Special Areas for Human Medicines (Paediatric Medicines, Orphan Medicines, Scientific Advice, SME Office, and Scientific Support & Projects) until 2013. Head of Portfolio... Read More →
avatar for Naoyuki Yasuda

Naoyuki Yasuda

Director, Office of International Regulatory Affairs, Ministry of Health, Labour and Welfare (MHLW)
Mr. Naoyuki Yasuda graduated from Osaka University in 1991. He careered industrial policy, industrial chemicals assessment, medical devices revaluation, international cooperation on narcotics and psychotropics, blood/blood product safety and vaccine supply and overall pharmaceuticals... Read More →


Tuesday June 25, 2019 10:30am - 11:30am
Room 6F San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

10:30am

#241: PMDA Town Hall
Component Type: Forum
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-19-600-L04-P; CME 1.00; IACET 1.00; RN 1.00

In this forum, PMDA will share the latest details regarding its policies and initiatives and other related strategic directives.Three senior level representatives from PMDA and MHLW will introduce the latest situations regarding scientific review systems, regulatory capacity building activities, and the current international collaboration.

Learning Objectives

Discuss the latest information regarding PMDA and Japanese regulations; Describe how to foster dialogue between participants and senior PMDA officials concerning drug/device regulatory concerns.

Chair

Nobumasa Nakashima, PhD

Speaker

Regulatory Updates: Facilitating Early Patient Access
Kazuhiko Mori, MSc

The Future of PMDA
Yasuhiro Fujiwara, MD, PhD

Current Initiatives of PMDA Regulatory Science Center
Hiroyuki Arai, PhD



Speakers
avatar for Yasuhiro Fujiwara

Yasuhiro Fujiwara

Chief Executive, Pharmaceuticals and Medical Devices Agency (PMDA)
Dr. Yasuhiro Fujiwara was previously Director-General, Strategic Planning Bureau of the National Cancer Center, and the Deputy Director of the Hospital (Research), National Cancer Center Hospital. He is a medical oncologist, specializing in breast cancer. Before joining NCCH, he was... Read More →
avatar for Hiroyuki Arai

Hiroyuki Arai

Director of Center for Product Evaluation and Center of Regulatory Science, Pharmaceuticals and Medical Devices Agency (PMDA)
Dr. Hiroyuki Arai is currently Director of Center for Product Evaluation & Director of Center for Regulatory Science, Pharmaceuticals and Medical Devices Agency (PMDA). He oversees medical product evaluation and regulatory science initiative in PMDA including collaboration with academic... Read More →
avatar for Kazuhiko Mori

Kazuhiko Mori

Councilor for Pharmaceutical Affairs, Minister’s Secretariat, Ministry of Health, Labour and Welfare (MHLW)
Kazuhiko Mori, MSc., is currently Councilor for Pharmaceutical Affairs, Minister’s Secretariat of the Ministry Health, Lobour and Welfare (MHLW). Mr. Mori has led many of MHLW/PMDA’s drug initiatives. He contributed to introduce new approaches to drug safety regulation including... Read More →
avatar for Nobumasa Nakashima

Nobumasa Nakashima

Senior Director for International Programs, Pharmaceuticals and Medical Devices Agency (PMDA)
Dr. Nakashima joined Pharmaceutical and Food Safety Bureau of Ministry of Health, Labor, and Welfare (MHLW) in 1992. He spent his career in the international field such as at WHO and OECD, not only in the domestic field. He worked as International Planning Director since September... Read More →


Tuesday June 25, 2019 10:30am - 11:30am
Room 6E San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

10:30am

#242: Use of Real World Evidence to Support Regulatory Decision-Making: First-Year Findings From the RCT-DUPLICATE Project
Component Type: Session
Level: Basic
CE: ACPE 1.00 Knowledge UAN: 0286-0000-19-601-L04-P; CME 1.00; IACET 1.00; RN 1.00

The Cures Act and the PDUFA IV mandated that the US FDA develop rules to guide industry on the use of real world evidence (RWE) for drug approvals. The session will bring together researchers and regulators to discuss the role of real world evidence.

Learning Objectives

Describe the DUPLICATE project replicating RCTs with real world data (RWD) analyses and provide an interim update on the findings from the first year; Discuss key considerations in implementing non-randomized studies using real world data to ensure valid and unbiased findings; Facilitate discussion how real world evidence may be used in regulatory decision-making.

Chair

Jeremy Rassen, DrSc, MS

Speaker

Initial Learnings From the RCT DUPLICATE Project
Jessica Franklin, PhD

Industry Perspective
Brande Yaist, MHS

Overview of the DUPLICATE Real World Evidence Demonstration Project
David Martin, MD, MPH



Speakers
avatar for Jessica Franklin

Jessica Franklin

Biostatistician, Div of Pharmacoepidemiology & Pharmacoeconomics;Asst Professor, Brigham and Women's Hospital and Harvard Medical School
Jessica Franklin, PhD, is an Assistant Professor of Medicine at Harvard Medical School and biostatistician in the Division of Pharmacoepidemiology and Pharmacoeconomics at Brigham and Women’s Hospital. Her research focuses on developing and applying statistical methods for the study... Read More →
avatar for David Martin

David Martin

Associate Director for Real World Evidence Analytics, OMP, CDER, FDA
David Martin is the Associate Director for Real World Evidence Analytics, Office of Medical Policy, FDA Center for Drug Evaluation and Research. He oversees demonstration projects intended to support the agency’s evaluation of real world evidence, reviews real world evidence submissions... Read More →
avatar for Jeremy Rassen

Jeremy Rassen

Co-Founder, President and Chief Science Officer, Aetion
Jeremy A. Rassen, MS, ScD is a pharmacoepidemiologist with 25 years of academic and industry experience. He is co-founder, president, and chief science officer at Aetion, a health care technology company that delivers real-world evidence for life sciences companies, payers, and regulatory... Read More →
avatar for Jessica Franklin

Jessica Franklin

Assistant Professor of Medicine, Div of Pharmacoepidemiology and Pharmacoeconomi, Brigham and Women's Hospital and Harvard Medical School
Jessica Franklin, PhD, is an Assistant Professor of Medicine at Harvard Medical School and biostatistician in the Division of Pharmacoepidemiology and Pharmacoeconomics at Brigham and Women’s Hospital. Her research focuses on developing and applying statistical methods for the study... Read More →
avatar for Brande Yaist

Brande Yaist

Senior Director, Global Patient Outcomes & Real World Evidence, Eli Lilly and Company
Mrs. Yaist is the Senior Director of the Center of Expertise in Global Patient Outcomes and Real World Evidence at Eli Lilly and Company. She leads and develops the research talent and capabilities needed to provide scientific services/expertise and support across an array of core... Read More →


Tuesday June 25, 2019 10:30am - 11:30am
Room 6B San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

10:30am

#243: The Future of Combination Products in the EU
Component Type: Forum
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-19-609-L04-P; CME 1.00; IACET 1.00; RN 1.00

This forum will focus on the applicability of the new European Medical Device Regulation (EU-MDR) on 26 March 2020. Implications and challenges of the new legal requirements and technical provisions for drug-device combination products will be discussed from the perspective of regulators, industry and patients.

Learning Objectives

Describe the complexity and consequences of the new EU Medical Device Regulation for drug-device combination products; Identify the challenges of the new requirements for industry, regulators, and Notified Bodies (NB) e.g. new designation process of Notified Bodies and competent staff, accountability, new technical requirements etc; Describe the challenges against patients' expectations and join the discussion.

Chair

Maren von Fritschen, PharmD

Speaker

EMA Perspective
Zahra Hanaizi, PharmD, MPharm

Industry Perspective
Trine B. Moulvad, MSc

Patient Perspective
Nicholas Brooke, MBA



Speakers
avatar for Maren von Fritschen

Maren von Fritschen

Managing Director, Addon Pharma
Maren von Fritschen, PhD has been working for a variety of pharmaceutical/MedTech companies SMEs /Start-ups and global entities for 20+ years. She is Managing Director at AddOn Pharma (Regulatory Affairs, Intelligence, Policy), Professor at the University of Applied Sciences, Berlin... Read More →
avatar for Zahra Hanaizi

Zahra Hanaizi

Scientific officer, PRIME Coordinator, Scientific and Regulatory Management Dept, European Medicines Agency (EMA)
Zahra Hanaizi joined EMA in 2007 as Product Team Leader, managing centralised medicinal products initial marketing authorisation applications, maintenance and life-cycle activities, mostly in the therapeutic area of Oncology. Since 2015, she has been working on the creation, implementation... Read More →
avatar for Trine Moulvad

Trine Moulvad

Vice President, Regulatory, Medical Writing and Pharmacovigilance, Zealand Pharma
Trine Moulvad has an MSc from University of Copenhagen, Denmark. She is an all-round experienced drug developer and leader with more than 25 years of experience within the pharmaceutical industry. She previously worked at Eli Lilly, Ferring and Novo Nordisk in global Regulatory leadership... Read More →
avatar for Nicholas Brooke

Nicholas Brooke

Executive Director, Patient Focused Medicines Development (PFMD), The Synergist.org
Nicholas Brooke is Founder of The Synergist, a collaboration platform incubator that brings key players together with the express aim of solving significant societal problems through collective action. Under Nicholas’ leadership, The Synergist acts as a backbone, providing vision... Read More →


Tuesday June 25, 2019 10:30am - 11:30am
Room 16AB San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA
  • Credit Type ACPE, CME, IACET, RN
  • Tags Forum

11:45am

#250 CH: How to Efficiently Implement a Healthcare Compliance Program in Preparation for Your First Product Launch
Component Type: Workshop
Level: Basic

This interactive workshop will address important government requirements impacting life sciences companies pre- and post-commercialization, as well as discuss approaches to implementing strong healthcare compliance programs with limited resources.

Learning Objectives

Discuss the importance of ethical conduct, and how compliance generates future success (before and after commercialization); Describe the relevant healthcare compliance-related laws, regulations and guidances; Identify methods to implement efficiently implement a healthcare compliance program despite limited resources.

Chair

Marc Adler, JD


Speakers
MA

Marc Adler

President, Global Compliance Strategies


Tuesday June 25, 2019 11:45am - 12:15pm
Content Hub Sails Pav San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

2:00pm

#263: Drug Development for Ocular Disease, New Therapies, Regulations, and Patient Perspectives
Component Type: Session
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-19-614-L04-P; CME 1.25; IACET 1.25; RN 1.25

This session will highlight recent advances and review challenges and opportunities in rare disease ocular therapies. New therapeutic approaches have emerged including promising gene and cell-based therapies in retinal diseases. Relevant case studies and special considerations will be discussed. We will explore the regulatory and scientific issues with experts to provide insights from the FDA and different sponsors.

Learning Objectives

Identify recent advances in in ocular drug development therapies; Discuss the existing regulatory framework; Describe challenges and opportunities in ocular rare disease drug development highlighting what has been done and what might be done in the future.

Chair

Nita Ichhpurani, PMP

Speaker

Gene Therapy for Inherited Retinal Diseases
Melissa Shiao Hui Liew, MD

FDA/CBER Perspective on the Development of Gene Therapy Products for Retinal Disorders
Wei Liang, PhD

Changing What it Means to be Blind: We're All in This Together
Kristin Smedley



Speakers
avatar for Wei Liang

Wei Liang

Pharmacologist, OTAT, CBER, FDA
Dr. Liang is a Pharmacology/Toxicology Reviewer in the Office of Tissues and Advanced Therapies (OTAT) in the Center for Biologics Evaluation and Research (CBER) at the Food and Drug Administration (FDA). She joined OTAT in 2006. Her primary focus is the review of preclinical testing... Read More →
avatar for Nita Ichhpurani

Nita Ichhpurani

Consultant, Phase One Forward
At Pharmacia (now Pfizer), Nita was a discovery Chemist and then PM in Clinical Pharmacology in oncology and CNS research. At MDS PS, she managed Global Central lab teams and later become the Latin American Lab Manager. At MDS PS and Celerion, she managed drug development consulting... Read More →
KS

Kristin Smedley

President, Curing Retinal Blindness Foundation
Kristin Smedley is President and Co-Founder of the Curing Retinal Blindness Foundation, the only patient organization in the world for her two sons’ blindness, CRB1 LCA/RP. Kristin has led the CRBF to raise over $1.3 Million, introduced the first ever legislation in the United States... Read More →
avatar for Melissa Liew

Melissa Liew

Global Head of Translational Medicine-Opthamology, Novartis Institutes of Biomedical Research
Melissa is Vice President and Global Head of Translational Medicine-Ophthalmology, at Novartis Institutes of Biomedical Research, based in Cambridge Massachusetts. In her role, she is working to translate cutting edge basic science discoveries into transformative novel medicines for... Read More →


Tuesday June 25, 2019 2:00pm - 3:15pm
Room 6D San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

2:00pm

#268: Current Status of FDA Framework for the Evaluation of Real World Evidence
Component Type: Session
Level: Basic
CE: ACPE 1.25 Knowledge UAN: 0286-0000-19-619-L04-P; CME 1.25; IACET 1.25; RN 1.25

FDA issued a framework for a program that will evaluate the use of real world evidence (RWE) to help support the approval of new indications for an approved drug or to help support or satisfy drug post approval study requirements. During this session, FDA will share their takeaways from public comments about the RWE framework and provide participants with an update on the current status of the FDA RWE Program.

Learning Objectives

Identify three topic areas included in the FDA real world evidence program; Identify and discuss two FDA takeaways from the analysis of public comments to the real world evidence framework; Identify one area of interest for real world evidence guidance development.

Chair

M. Khair ElZarrad, PhD, MPH

Speaker

FDA Update
Jacqueline A. Corrigan-Curay, JD, MD

Update from CBER
Steven A. Anderson, PhD

FDA Update
David Martin, MD, MPH



Speakers
MK

M. Khair ElZarrad

Deputy Director, Office of Medical Policy, CDER, FDA
Dr. ElZarrad is the Deputy Director of the Office of Medical Policy (OMP) at FDA’s Center for Drug Evaluation and Research (CDER), where he leads the development, coordination, and implementation of medical policy programs and strategic initiatives. Dr. ElZarrad currently leads... Read More →
avatar for Steven Anderson

Steven Anderson

Director, Office of Biostatistics and Epidemiology, CBER, FDA
Steven Anderson, Ph.D., M.P.P., is currently the Director of the Office of Biostatistics and Epidemiology (OBE) at the FDA Center for Biologics Evaluation and Research (CBER). He provides leadership for all CBER statistical, epidemiological and risk assessment programs. He was previously... Read More →
avatar for Jacqueline Corrigan-Curay

Jacqueline Corrigan-Curay

Director, Office of Medical Policy, CDER, FDA
Jacqueline Corrigan-Curay, J.D., M.D., serves as Director of the Office of Medical Policy (OMP) in the Center for Drug Evaluation and Research, FDA. OMP is comprised of the Office of Prescription Drug Promotion (OPDP) and the Office of Medical Policy Initiatives (OMPI). Dr. Corrigan-Curay... Read More →
avatar for David Martin

David Martin

Associate Director for Real World Evidence Analytics, OMP, CDER, FDA
David Martin is the Associate Director for Real World Evidence Analytics, Office of Medical Policy, FDA Center for Drug Evaluation and Research. He oversees demonstration projects intended to support the agency’s evaluation of real world evidence, reviews real world evidence submissions... Read More →


Tuesday June 25, 2019 2:00pm - 3:15pm
Room 6B San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

2:00pm

#269: Driving Complex Generics to Approval: What are the Keys to Success
Component Type: Forum
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-19-620-L04-P; CME 1.25; IACET 1.25; RN 1.25

GDUFA II provides new regulatory processes designed to accelerate access to generic versions of complex products. In thisforum, FDA and industry provide examples on how to effectively use these processes.

Learning Objectives

Explain how the FDA review of ANDAs for complex generics has been improved under GDUFA II; Describe current regulatory and scientific considerations for generic combination products; Develop strategies to communicate more effectively with FDA during development of a complex product intended for submission under an ANDA.

Chair

Robert A. Lionberger, PhD

Speaker

Industry Perspective
Michelle Lee-Bourner, MSc

FDA Update
Martha Nguyen, JD



Speakers
avatar for Martha Nguyen

Martha Nguyen

Director, Division of Policy Development, OGD, CDER, FDA
Martha Nguyen is the Director of the Division of Policy Development in FDA CDER's Office of Generic Drugs, where she provides strategic leadership and direction on broad policy issues affecting generic drugs and oversees the development and clearance of regulations, guidance documents... Read More →
avatar for Michelle Lee-Bourner

Michelle Lee-Bourner

Head Global Respiratory and Biologics Regulatory Sciences, Mylan Pharma UK Ltd
Michelle Lee-Bourner has 30 years’ experience of drug pharmaceutical research and development, focused mainly on NCE’s and more recently generics, biosimilars and biologics. At Pfizer Michelle’s career spanned drug discovery, early clinical research and Regulatory Affairs. Michelle... Read More →
avatar for Robert Lionberger

Robert Lionberger

Director, Office of Research and Standards, Office of Generic Drugs, CDER, FDA
Robert Lionberger, Ph.D. serves as Director of the Office of Research and Standards (ORS) in the Office of Generic Drugs (OGD). Dr. Lionberger leads OGD’s implementation of the GDUFA science and research commitments including internal research activities and external research grants... Read More →


Tuesday June 25, 2019 2:00pm - 3:15pm
Room 6F San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

3:15pm

#275 EE: Access to Investigational Drugs Outside of Clinical Trials: What's Fair?
Component Type: Workshop
Level: Basic

Requests for investigational drugs are increasing, posing ethical, regulatory, and logistical challenges for companies. After discussing a fictional case, participants will understand these challenges and be better prepared to address them.

Learning Objectives

Discuss ethical, legal, and practical concerns surrounding patient requests for access to investigational drugs outside of clinical trials (preapproval access); Apply this knowledge to a fictitious case study; Analyze the pros and cons of different possible responses in real world scenarios.

Chair

Alison Bateman-House, PhD, MA, MPH

Speaker

Facilitator
Beverly L Harrison



Speakers
avatar for Alison Bateman-House

Alison Bateman-House

Assistant Professor, Division of Medical Ethics, NYU Langone Health
Alison Bateman-House, PhD, MPH, MA, is an assistant professor in the Division of Medical Ethics at NYU Langone Health's School of Medicine. She is co-chair of the Working Group on Compassionate Use and Preapproval Access (CUPA), an academic group that studies ethical issues concerning... Read More →
avatar for Beverly Harrison

Beverly Harrison

Head, Patient Support, Janssen Pharmaceutical Companies of Johnson & Johnson
Leading the Patient Support Group @ Janssen R&D (a J&J Co.), reporting directly to the Chief Medical Officer, Beverly focuses on developing strategies for patients’ needs by working with internal/external stakeholders & engaging patient advocacy organizations. She is a R&D & nonprofit... Read More →


Tuesday June 25, 2019 3:15pm - 4:15pm
E and E Area Exh Hall San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

4:15pm

#295: Prescription Drug Labeling: New Guidances from the US FDA
Component Type: Session
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-19-643-L04-P; CME 1.25; IACET 1.25; RN 1.25

This session will provide updates from the US FDA on the latest developments regarding prescription drug labeling (i.e., Prescribing Information). Topics will include overviews of recently published FDA guidances, including Indications and Usage Section of Labeling for Human Prescription Drug and Biological Products -- Content and Format, Pediatric Information Incorporated into Labeling, Product Title and Initial US Approval in the Highlights of Prescribing Information, and Labeling for Biosimilar Products.

Learning Objectives

Discuss labeling updates from the US FDA: Describe recommendations for prescription drug labeling outlined in recently published FDA guidances; Identify labeling information that is relevant for your organization.

Chair

Barbara Chong, PharmD

Speaker

Indications and Usage Section of Labeling for Human Prescription Drug and Biological Products: Content and Format
Iris Masucci, PharmD

Labeling for Biosimilar Products
Stacey Ricci, DrSc

Pediatric Information Incorporated into Labeling; Product Title and Initial US Approval in the Highlights of Prescribing Information
Eric Brodsky, MD



Speakers
avatar for Eric Brodsky

Eric Brodsky

Associate Director, Labeling Development Team, Office of New Drugs, CDER, FDA
Dr. Brodsky [as the Associate Director of the Labeling Development Team in the Office of New Drugs at the Center for Drug Evaluation and Research at the FDA] oversees OND’s implementation of Prescribing Information (PI) regulations, guidances, and policies to help promote consistency... Read More →
BC

Barbara Chong

Special Assistant for Labeling, Office of Medical Policy, CDER, FDA
Dr. Chong, Special Assistant for Labeling, joined the Office of Medical Policy (OMP) in the Center for Drug Evaluation and Research (CDER/FDA) in 2016. Her current position is primarily focused on policy and guidance development for prescription drug labeling. She works with various... Read More →
IM

Iris Masucci

Special Assistant for Labeling, Office of Medical Policy, CDER, FDA
Dr. Masucci joined FDA in 1998 and has been focusing on professional labeling and related policy since 2002. In CDER’s Office of Medical Policy, she serves as the Office lead on professional labeling policies. She leads Office efforts on the development and implementation of labeling... Read More →
SR

Stacey Ricci

Director, Scientific Review Staff, Therapeutic Biologics and Biosimilars, Office, FDA
Dr. Ricci has worked in CDER/FDA for over 11 years and is currently the Director of Scientific Review in the Office of Therapeutic Biologics and Biosimilars OTBB. OTBB works collaboratively with CDER Offices and Review divisions responsible for providing scientific and regulatory... Read More →


Tuesday June 25, 2019 4:15pm - 5:30pm
Room 6B San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA
  • Level Intermediate
  • Featured Topics Regulator Thinking
  • Credit Type ACPE, CME, IACET, RN
  • Tags Session

4:15pm

#296: Informing Development and Authorizations Using Real World Evidence/Artificial Intelligence
Component Type: Session
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-19-645-L04-P; CME 1.25; IACET 1.25; RN 1.25

The evolution of actionable insights on how medicines are used, generated from real world evidence and artificial intelligence will be discussed. This session will focus on knowledge management, regulatory intelligence, and learning healthcare systems.

Learning Objectives

Discuss the potential for artifical intelligence and real world evidence to inform both development and authorization; Identify the opportunities and barriers and how AI and RWE in conjunction with knowledge management can enable better development and regulatory decisions.

Chair

Lawrence Eugene Liberti, PhD, RPh, RAC

Speaker

How Do Companies View Knowledge Management as a Decision-Making Tool?
Magda Bujar, PhD, MSc

Learning Healthcare Systems: Managing Real World Evidence/Knowledge - How Can This Transition Healthcare Data to Actionable Information?
Andrea Julsing Keyter, MS

Potential Uses of AI for Regulatory Intelligence: How Can This be Used Practically and What are the Barriers and Opportunities?
Mukesh Singhal, MBA



Speakers
avatar for Magda Bujar

Magda Bujar

Manager, Strategic Development, Centre For Innovation In Regulatory Science (CIRS)
Dr Magda Bujar is a Manager, Strategic Development at CIRS. In her current role, she is responsible for developing and executing strategies for engaging with CIRS stakeholders - pharmaceutical companies as well as major regulatory authorities. Magda also initiates and coordinates... Read More →
avatar for Lawrence Liberti

Lawrence Liberti

Executive Director, Centre for Innovation in Regulatory Science (CIRS)
Dr Liberti has worked in pharmaceutical regulatory affairs, communications and clinical R&D for the past four decades. Since 2009 he has served as the Executive Director of CIRS (the Centre for Innovation in Regulatory Science, Ltd, forming part of Clarivate Analytics). He has been... Read More →
avatar for Andrea Keyter

Andrea Keyter

Deputy Director, Medical Devices, South African Health Products Regulatory Authority
Andrea is a pharmacist and has 10 years of experience within the field of regulatory affairs, quality assurance, quality risk management, pharmaceutical production, complementary medicines manufacture and risk-based inspection planning. In her current role, Andrea is responsible for... Read More →
avatar for Mukesh Singhal

Mukesh Singhal

Senior Manager, Deloitte
Mukesh is a Senior Manager in Deloitte Consulting's life science R&D practice. He has spent over 15 years of his career between industry and Consulting. He focuses on helping R&D organizations at biotech and pharmaceutical companies address strategic and operational challenges with... Read More →


Tuesday June 25, 2019 4:15pm - 5:30pm
Room 6E San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

4:15pm

#294: Update From the US FDA on Progress and Topics of Current Interest in US Biosimilar Policy, Regulation, and Outreach/Education
Component Type: Session
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-19-636-L04-P; CME 1.25; IACET 1.25; RN 1.25

This session will include an update from members of the US FDA on progress and topics of current interest in US biosimilar policy, regulation, and outreach/education.

Learning Objectives

Discuss topics of current interest in US biosimilar policy.

Chair

Sarah Yim, MD

Speaker

FDA Update
Joseph B. Franklin, JD, PhD

FDA Update
Sarah Crowley-Ikenberry, MA

FDA Update
Christopher Downey, PhD



Speakers
avatar for Sarah Yim

Sarah Yim

Director (Acting), Office of Therapeutic Biologics and Biosimilars, FDA
Sarah Yim, M.D. is acting Director of the Office of Therapeutic Biologics and Biosimilars in CDER's Office of New Drugs (OND). Prior to joining the Biosimilars group in March 2019, she was director of the Division of Clinical Review in the Office of Generic Drugs (OGD) for 2 years... Read More →
avatar for Sarah Crowley-Ikenberry

Sarah Crowley-Ikenberry

Senior Communication Advisor, OTBB, OND, CDER, FDA
Sarah Ikenberry is Senior Communication Advisor for the Office of Therapeutic Biologics and Biosimilars (OTBB), in the Food and Drug Administration’s (FDA), Center for Evaluation and Research (CDER) where she provides communication advice and support to senior leaders and the agency... Read More →
avatar for Christopher Downey

Christopher Downey

Review Chief, OBP, OPQ, CDER, FDA
Christopher (Chris) Downey is a Review Chief in FDA/CDER’s Office of Biotechnology Products (OBP). He was been with FDA since 2012, and currently oversees CMC reviews of therapeutic proteins, including new molecular entities and biosimilars. He received his PhD in Biochemistry from... Read More →
JF

Joseph Franklin

Policy Director for the Principal Deputy Commissioner, FDA
Joe Franklin is the Policy Director for the FDA Principal Deputy Commissioner. Until April 2019, Dr. Franklin was the Director of the Policy Staff in the Office of Therapeutic Biologics and Biosimilars at FDA. Previously, Dr. Franklin was an Associate Chief Counsel in FDA’s Office... Read More →


Tuesday June 25, 2019 4:15pm - 5:30pm
Room 6F San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

4:15pm

#299: Public and Regulatory Response To Drug Pricing Concerns
Component Type: Forum
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-19-638-L04-P; CME 1.25; IACET 1.25; RN 1.25

Stakeholders agree that generics are an effective approach to lowering drug prices, but approvals are often challenging for sponsors to obtain, and the cheap prices that consumers value make it hard for sponsors to stay on the market. This session will describe the strategies that regulators and payers are using to combat those problems. FDA does not regulate the price of drugs, but the regulatory decisions it makes can nevertheless impact them. For generics, that can mean setting a high bar or a low bar for equivalence and deciding which kinds of products are eligible for accelerated review or additional interactions with the agency. Likewise, payers don’t control the application process, but their decisions go a long way to determining which products are developed. This session will analyze the regulatory strategies that can eventually prove successful, explain how companies and policymakers can best handle the development of complex generics, and discuss new steps payers are making to ensure generic sponsors have a market for the products after a long development process.

Learning Objectives

Identify the steps for successfully developing a complex generic product; Describe new reimbursement approaches being used to encourage a stable generic market; Discuss review pathway selection at the US FDA's Office of Generic Drugs; Recognize product categories where FDA is encouraging generic applications.

Chair

Nielsen Hobbs

Speaker

Panelist
Jan Burkett, MBA, RPh

Panelist
Heather Wall, MBA

Panelist
Adam Kroetsch



Speakers
avatar for Jan Burkett

Jan Burkett

Vice President, Drug Sourcing Officer, Express Scripts
Jan is a pharmacist with diverse experience in pharmacy benefit management, contracting and joint venture strategy. She joined Express Scripts in 1993 and is currently VP, Chief Drug Sourcing Officer, responsible for Express Scripts' drug sourcing procurement and strategy. Jan leads... Read More →
avatar for Gregory Daniel

Gregory Daniel

Deputy Director and Clinical Professor, Duke-Margolis Center For Health Policy
Gregory Daniel, PhD, MPH is the Deputy Director of the Duke-Robert J. Margolis, MD Center for Health Policy and a Clinical Professor in Duke's Fuqua School of Business. He directs the DC-based office of the Center leading its pharmaceutical and medical device policy portfolio, and... Read More →
avatar for Nielsen Hobbs

Nielsen Hobbs

Executive Editor, US Policy and Regulatory, The Pink Sheet/Scrip
Nielsen Hobbs has more than 20 years of experience covering the pharmaceutical industry, and writes frequently about the intersection of business, regulation, science and politics. Before joining the “The Pink Sheet” he worked for Congressional Quarterly. Hobbs enjoys discussing... Read More →
avatar for Heather Wall

Heather Wall

Chief Commercial Officer, Civica Rx
Heather’s expertise spans the healthcare continuum focusing on design, implementation and execution of hospital & health system strategies to improve patient care. Heather has a 20+ year career where she has had accountability for strategy, operations, finance & high-value health... Read More →
AK

Adam Kroetsch

Research Director, Duke-Margolis Center For Health Policy


Tuesday June 25, 2019 4:15pm - 5:30pm
Room 14B San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA
 
Wednesday, June 26
 

8:00am

#301: So Much Data, So Little Time: Hot Topics in Benefit-Risk Assessment
Component Type: Session
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-19-646-L04-P; CME 1.25; IACET 1.25; RN 1.25

Both regulators and sponsors are increasingly using structured frameworks to assess the benefit-risk profile of medicinal products. Such frameworks can accommodate a variety of data sources and can be supplemented by an array of graphical presentations. As experience with using structured benefit-risk assessment (SBRA) frameworks in both the pre- and post-market context accrues, a number of important questions remain, including: 1) What has been learned by FDA and EMA in regard to using a structured benefit-risk assessment framework for new drug application assessments? 2) How are these regulatory authorities continuing to train and support their reviewers in conducting such assessments? 3) What are the challenges involved in incorporating real world data into structured benefit-risk assessment analyses? 4) What are good practices in terms of evaluating the impact of different types of uncertainty on the quality of the benefit-risk assessment decision?

Learning Objectives

Describe the FDA’s and EMA’s experiences to date in implementing structured approaches to benefit-risk assessment, including the use of the Effects Tables and other tools; Discuss the key considerations in evaluating the impact of uncertainty on the quality of the benefit-risk assessment decision; Identify the challenges involved in incorporating real world evidence into a structured benefit-risk assessment.

Chair

Meredith Y. Smith, PhD, MPA

Speaker

FDA's Experience to Date Using the Structured Benefit-Risk Assessment Framework to Support Regulatory Decision-Making
Graham Thompson

Use of Real World Evidence in Personalized Benefit-Risk Assessment: Closing the Knowledge Gap
Tarek Hammad, MD, PhD, MS, MSc, FISPE

EMA Perspective
Douwe Postmus, PhD, MSc



Speakers
avatar for Tarek Hammad

Tarek Hammad

Therapeutic Area Strategy Lead, Global Pharmacovigilance, Sanofi Genzyme
Dr. Hammad joined Sanofi-Genzyme, in 2019, as a Therapeutic Area Strategy Lead in the Global Pharmacovigilance group. Prior to this, he served as the Head of Signal Detection and Benefit-Risk Assessment in Merck KGaA/EMD Serono. Previously, he was an Executive Director of Pharmacoepidemiology... Read More →
avatar for Douwe Postmus

Douwe Postmus

Researcher, University Medical Center Groningen
Dr Douwe Postmus is employed as a researcher at the Department of Epidemiology of the University Medical Center Groningen, The Netherlands. His research focuses on applying quantitative modeling techniques to decision making problems in health care, including regulatory, reimbursement... Read More →
avatar for Meredith Smith

Meredith Smith

Global Risk Management Officer, Global Patient Safety, Amgen Inc.
Meredith Smith is Global Risk Management Officer at Amgen, Inc. where she leads a team of 6 scientists responsible for medicinal product benefit-risk assessment and risk management. She is a behavioral scientist and health services researcher by training with over 15 years of experience... Read More →
GT

Graham Thompson

Operations Research Analyst, OSP, CDER, FDA
Graham Thompson joined FDA in 2012 and currently serves as an operations research analyst for the Decision Support and Analysis Team (DSAT) in FDA’s Center for Drug Evaluation and Research (CDER). He works on CDER projects and initiatives related to benefit-risk assessment and supporting... Read More →


Wednesday June 26, 2019 8:00am - 9:15am
Room 6C San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

8:00am

#309: Neoantigen-Based Cancer Therapies: Regulatory Challenges and Opportunities
Component Type: Session
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-19-650-L04-P; CME 1.25; IACET 1.25; RN 1.25

Neoantigen-based cancer therapy is an approach based on the specific genetic information unique to individual tumor(s) of an individual patient. Such information can be utilized in many ways for cancer treatment, e.g., to generate antigens for vaccination or specific T-cells. Results from earlier clinical trials have shown that this approach is feasible, able to elicit the intended immune response, and in some cases generating anti-tumor activities that may mediate the tumor regression. However, this approach has many scientific and regulatory challenges, for example, optimal ways / algorithms in identifying and selecting neoantigens, and regulatory framework for regulating such products given that drug product and indications are not explicitly defined. This session is intended to address these challenges and discuss the opportunities for the neoantigen-based cancer therapies.

Learning Objectives

Describe the concepts of cancer immunoediting and neoantigen;Discuss the recent advances in neoantigen-based novel cancer therapy, e.g., vaccine and cell-based therapies;Discuss scientific and regulatory challenges, focusing on pre-clinical studies and early clinical trials; Identify opportunities in facilitating and accelerating the development.

Chair

Peter F. Bross, MD

Speaker

CMC Considerations: Regulatory Perspective
Elena Gubina, PhD

CMC Considerations: Industry Perspective
Karin Jooss, DrSc

Clinical Consideration for Early-Phase Trials: Academic Perspective
Aaron M. Miller, MD, PhD

Clinical Consideration for Early-Phase Trials: Regulatory Perspective
Peter F. Bross, MD

Panelist
Alyssa K. Galaro, PhD



Speakers
avatar for Alyssa Galaro

Alyssa Galaro

Biomedical Engineer, DCEPT, OTAT, CBER, FDA
Dr. Galaro joined the Office of Tissues and Advanced Therapies in 2018 as a Pharmacology and Toxicology Reviewer. Prior to joining CBER, she completed her PhD in biomedical engineering at the Johns Hopkins School of Medicine where her research focused on engineering nanoparticle-based... Read More →
PB

Peter Bross

Medical Team Leader, OTAT, CBER, FDA
Peter Bross is a clinical oncology team leader in the FDA Center for Biological Evaluation and Research (CBER), Office of Tissue and Advanced Therapies (OTAT) and previously worked as a clinical reviewer in the Division of Oncology Drug Products in the Center for Drug Evaluation and... Read More →
avatar for Elena Gubina

Elena Gubina

Expert Biologist, OTAT, CDER, FDA
KJ

Karin Jooss

Executive Vice President of Oncology and Chief Scientific Officer, Gritstone Oncology
Dr. Karin Jooss serves as executive vice president of research, chief scientific officer at Gritstone and interim head of manufacturing. She joined Gritstone from Pfizer, where she was head of cancer immuno-therapeutics, was a member of the vaccine immuno-therapeutics leadership team... Read More →
AM

Aaron Miller

Medical Oncologist, Assistant Professor of Medicine, University of California San Diego
Dr. Miller is a board-certified medical oncologist who specializes in diagnosing and treating gastrointestinal cancers. He is an assistant professor in the Department of Medicine, where he instructs medical students, residents and fellows at UC San Diego School of Medicine. He holds... Read More →


Wednesday June 26, 2019 8:00am - 9:15am
Room 2 San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA
  • Level Intermediate
  • Featured Topics Regulator Thinking,Advanced Therapies
  • Credit Type ACPE, CME, IACET, RN
  • Tags Session

8:00am

#314: Medical Devices: EU Medical Device Regulation, PMDA Updates, and US MDUFA IV – Where Are We Now?
Component Type: Session
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-19-655-L04-P; CME 1.25; IACET 1.25; RN 1.25

Since 2017 we have analyzed the new EU Medical Devices Regulations, the enabling acts (still to come!) and MDUFA IV. Now one more year on, we examine what has improved, where action is still required and what to do now to keep products on the market and review new initiatives in Japan.

Learning Objectives

Describe how your own organization is managing the change to the new EU regulations; Assess what changes the MDUFA IV regulations have already made and how things might change further; Discuss the changes happening in Japan; Identify areas where your organization still needs to move forward to comply with the new regulations; Interpret the new regulations and guidance to benefit their own working environment.

Chair

Angela Stokes, MSc

Speaker

The Medical Device Regulation: Less Than a Year to Go! Where Are We Now?
Angela Stokes, MSc

Recent Progress of Balancing Between Risk/Benefit Assessment and Patient Access to Medical Devices in Japan
Nobuhiro Handa, DrMed, MD

MDUFA IV: Another Year has Passed! What are the Highlights?
LeeAnn L Chambers, MS



Speakers
LC

LeeAnn Chambers

Principal Research Scientist, Global Regulatory Affairs, CMC - Devices, Eli Lilly and Company
LeeAnn Chambers has been a Regulatory Affairs professional at Eli Lilly and Company for 22 years. She assists teams in developing global registration strategies for medical devices and drug / device combination products. She has guided the preparation of device content in US IND... Read More →
avatar for Nobuhiro Handa

Nobuhiro Handa

Principal Reviewer, Office of Medical Device, Pharmaceuticals and Medical Devices Agency (PMDA)
I graduated the University of Tokyo, Faculty of Medicine in 1985. Since then I had been a cardiovascular surgeon over 28 years. I was trained in the US for 4 years at Mayo clinic as well as Massachusetts General Hospital. I changed my career at age of 52 from clinical physician to... Read More →
avatar for Angela Stokes

Angela Stokes

Vice President, Head Global Regulatory Consulting, Syneos Health
Angela has almost 30 years of experience in medicinal product and medical device development. Her strengths include regulatory and strategic advice, authorship of regulatory submissions, regulatory writing, regulatory research, and compliance with relevant guidelines and standards... Read More →


Wednesday June 26, 2019 8:00am - 9:15am
Room 6F San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

8:00am

#313: Model Integrated Evidence as Pivotal Information for Drug Regulatory Decision Making: When, Where, and Why
Component Type: Session
Level: Basic
CE: ACPE 1.25 Knowledge UAN: 0286-0000-19-654-L04-P; CME 1.25; IACET 1.25; RN 1.25

The use of advanced quantitative methods and computational modeling has become part of modern drug development and assessment. Model Integrated Evidence (MIE) is an approach for generating evidence of bioequivalence via modeling and simulation.

Learning Objectives

Identify the core modeling and simulation capabilities (PBPK and QCP) that a generic drug manufacturer must posses; Evaluate lessons learned from the use of a model informed drug development process for new drugs, and apply common processes to generic drug development programs; Define and prepare for the future use of model informed evidence (MIE) in a generic drug submission and identify the value.

Chair

Liang Zhao, PhD

Speaker

Industry Update
Michael A. Eldon, PhD, RPh

FDA Update From New Drugs Perspective
Yaning Wang, PhD

FDA Update from Generic Drugs Perspective
Liang Zhao, PhD



Speakers
avatar for Yaning Wang

Yaning Wang

Director, Division of Pharmacometrics, OCP, OTS, CDER, FDA
Dr. Yaning Wang is the Director of the Division of Pharmacometrics in the Office of Clinical Pharmacology at FDA. Before joining FDA, Dr. Wang received his Ph.D. in Pharmaceutics and master’s degree in Statistics from the University of Florida from 1999 to 2003. He also obtained... Read More →
LZ

Liang Zhao

Director, Division of Quantitative Methods and Modeling, OGD, CDER, FDA
Dr. Liang Zhao has been serving as Director of the Division of Quantitative Methods and Modeling (DQMM), Office of Research and Standards (ORS) in Office of Generic Drugs, CDER since 2015. He initially joined FDA as a clinical pharmacology reviewer in the Office of Clinical Pharmacology... Read More →
avatar for Michael Eldon

Michael Eldon

Expert Consultant, NDA Partners LLC
Dr. Eldon has more than 37 years of experience in clinical pharmacology, pharmacokinetics/ pharmacodynamics and pharmacometrics in the pharmaceutical industry. He is an Expert Consultant at NDA Partners and as VP of Clinical Pharmacology at Nektar Therapeutics he was responsible for... Read More →


Wednesday June 26, 2019 8:00am - 9:15am
Room 6E San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

10:30am

#340: Digital Technology Advances Labeling Management and Patient Access
Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-19-671-L04-P; CME 1.00; IACET 1.00; RN 1.00

This session will update the labeling management across regions in the EU and the Asia Pacific, in particular, the NMPA and PMDA will elaborate on current labeling management in China and Japan and envisage digital/electronic labeling management in the future. Also, the industry will share some industry initiated eLabeling pilot activities, finally, all will discuss the opportunities and hurdles of introducing digital/electronic labeling

Learning Objectives

Explain digital/electronic labeling policy and its pilot activities across different regions; Discuss the advantages of digital/electronic labeling and benefit to patients and prescribers; Describe the latest eLabeling policy and future trends in EU and the Asia Pacific, particularly in Japan and China

Chair

Vicky (Fengyun) Han

Speaker

Labeling Management Update from the PMDA
Mariko Tsukuda, MSc

NMPA Labeling Management and Envisage of Electronic Labeling Trends in China
Xiao Yuan Chen, PhD

Digital Labeling Innovation in Europe
Shimon Yoshida, PhD

Trends of eLabeling and its Innovation Pilot Activities in Asia Pacific
Vicky (Fengyun) Han



Speakers
avatar for Vicky (Fengyun) Han

Vicky (Fengyun) Han

Senior Director, Asia Pacific Regulatory Policy Group Lead, Asia Pacific, Johnson & Johnson Pte. Ltd.
Vicky Han is Senior Director at Janssen Pharmaceuticals in charge of Asia Pacific Regulatory Policy since 2016. Her experiences spans in Asia & Europe, she dedicated 18 years in GSK, wherein 12 years in GSK China for leading regulatory affairs. In 2008, she moved to Belgium for dealing... Read More →
avatar for Mariko Tsukuda

Mariko Tsukuda

Reviewer, Office of Pharmacovigilance I, Pharmaceuticals and Medical Devices Agency (PMDA)
Mariko Tsukuda is Reviewer at Pharmaceuticals and Medical Devices Agency since 2014. Mrs.Mariko Tsukuda worked for implementation of ICH E2B(R3), which is electronic standard for ICSRs, in Japan as an ICH E2B member since 2014.
avatar for Shimon Yoshida

Shimon Yoshida

Executive Director, International Labeling, Pfizer Inc
Dr Shimon Yoshida is Executive Director at Pfizer, and heads the International Labeling Group in Global Regulatory Affairs. Dr Yoshida obtained a PhD in Chemistry from Reading University, UK, and held postdoctoral positions at Durham University before joining the Royal Society of... Read More →
avatar for Xiao Yuan Chen

Xiao Yuan Chen

Office Director, Tsinghua University


Wednesday June 26, 2019 10:30am - 11:30am
Room 6F San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

10:30am

#341: The Evolving Gene Therapy Regulatory Framework: A Brave New World
Component Type: Forum
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-19-679-L04-P; CME 1.00; IACET 1.00; RN 1.00

This forum will bring together panelists with regulatory expertise in gene therapy to present an update on recent changes to the regulatory framework and discuss its impact on the development of gene therapy products.

Learning Objectives

Outline recent changes to the regulatory framework for gene therapies. Discuss impact on progression of new and existing development programs in gene, genetically-modified cells and genome editing; Identify areas and disciplines where gene therapy sponsors would like to see further change.

Chair

Snehal Naik, PhD, MA

Speaker

Panelist
Peter W. Marks, MD, PhD

Panelist
Anne-Virginie L. Eggimann, MS

Panelist
Sandy Macrae, PhD

Panelist
Robert Pietrusko, PharmD



Speakers
avatar for Peter Marks

Peter Marks

Director, Center for Biologics Evaluation and Research, FDA
Peter Marks received his graduate degree in cell and molecular biology and his medical degree at New York University and completed Internal Medicine residency and Hematology/Medical Oncology training at Brigham and Women’s Hospital in Boston. He has worked in academic settings teaching... Read More →
avatar for Anne-Virginie Eggimann

Anne-Virginie Eggimann

Senior Vice President, Regulatory Science, bluebird bio, Inc.
Anne-Virginie joined bluebird bio in 2011 to lead global regulatory strategy and create innovative pathways to accelerate the development of bluebird bio’s gene therapy products. Previously, AV was an Executive Director at Voisin Consulting, leading rare diseases, oncology, and... Read More →
RP

Robert Pietrusko

Senior Vice President, Regulatory Affairs and Quality Assurance, Voyager Therapeutics
Robert G. Pietrusko, Pharm.D. is Senior Vice President, Regulatory Affairs & QA at Voyager Therapeutics, Inc. He has directed the worldwide approval of more than 30 new products and new indications and is a recognized leader in the pharmaceutical industry. Before joining Voyager... Read More →
avatar for Sandy Macrae

Sandy Macrae

Chief Executive Officer, Sangamo Therapeutics
Sandy Macrae serves as a member of the Company’s Board of Directors. He has twenty years of experience in the pharmaceutical industry most recently serving as the Global Medical Officer of Takeda Pharmaceuticals, where he established and led the Global Medical Office, which encompasses... Read More →
avatar for Snehal Naik

Snehal Naik

Associate Director, Janssen Research & Development, LLC
Snehal brings a confluence of early discovery, innovation and scientific expertise to her current role of North America regulatory liaison for cell & gene therapies and microbiome at Janssen.With a keen interest in influencing policy, she staffs the regulatory committee, gene therapy... Read More →


Wednesday June 26, 2019 10:30am - 11:30am
Room 6E San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA
  • Level Intermediate
  • Featured Topics What's Next,Advanced Therapies
  • Credit Type ACPE, CME, IACET, RN
  • Tags Forum

10:30am

#342: When is Real World Evidence Ready for Prime Time?
Component Type: Forum
Level: Basic
CE: ACPE 1.00 Knowledge UAN: 0286-0000-19-681-L04-P; CME 1.00; IACET 1.00; RN 1.00

We see growing interest in the value of using real world data (RWD) to support label expansions and approvals of drugs for rare conditions and oncology products. The FDA released its Framework for Real World Evidence Program in December 2018, following release of the its MyStudies App designed to incorporate patient-originated data with other RWD for research purposes. In Europe, the Heads of Medicines Agencies and the European Medicines Agency released a joint report in February, 2019 on their big data taskforce summarizing many areas of interest to better understand RWD and its possible uses. We will distill key lessons from demonstration projects and other experience to understand what is being done to evaluate data sets to give confidence in a RW study design, and its findings, including the challenges encountered when comparing data from clinical trials with a medicine’s performance in routine clinical practice, as actually prescribed by physicians and taken by patients.

Learning Objectives

Describe at least one innovative study design that used real world data for label expansion; Characterize the types of diseases and treatments for which real world evidence has been used to expand the labeled use.

Chair

Nancy Dreyer

Speaker

FDA Update
Peter P. Stein, MD

EMA Update
Anthony Humphreys, MPharm

Panelist
Gregory Daniel, PhD, MPH

Panelist
Solomon Iyasu, DrMed, MPH



Speakers
avatar for Gregory Daniel

Gregory Daniel

Deputy Director and Clinical Professor, Duke-Margolis Center For Health Policy
Gregory Daniel, PhD, MPH is the Deputy Director of the Duke-Robert J. Margolis, MD Center for Health Policy and a Clinical Professor in Duke's Fuqua School of Business. He directs the DC-based office of the Center leading its pharmaceutical and medical device policy portfolio, and... Read More →
avatar for Nancy Dreyer

Nancy Dreyer

Chief Scientific Officer & Senior Vice President, IQVIA
Nancy Dreyer is chief scientific officer and SVP at IQVIA. She focuses on generating real-world evidence for regulators, clinicians, patients and payers through pragmatic trials and non-interventional approaches. She is a Fellow of both DIA and the Int’l Society of Pharmacoepidemiology... Read More →
avatar for Anthony Humphreys

Anthony Humphreys

Head of Scientific Committees Regulatory Science Strategy, European Medicines Agency (EMA)
He is the Head of Scientific Committees Regulatory Science Strategy Division (SciRS). He is responsible for providing leadership in the Agency in the areas of Scientific Committees Coordination and Regulatory Science Strategy in support of delivering the EMRN 2020 Strategy. He chairs... Read More →
avatar for Solomon Iyasu

Solomon Iyasu

Vice President and Head of Pharmacoepidemiology, Merck & Co., Inc.
Dr. Iyasu joined Merck and Co. in August of 2015 as head of Pharmacoepidemiology in the Center for Observational and Real-World Evidence (CORE). In his current role, he leads a department that is responsible for designing and conducting postapproval observational safety and effectiveness... Read More →
avatar for Peter Stein

Peter Stein

Director, Office of New Drugs, CDER, FDA
Peter Stein, M.D., is the Director of CDER’s Office of New Drugs (OND). OND is responsible for the regulatory oversight of investigational studies during drug development and decisions regarding marketing approval for new (innovator or non-generic) drugs, including decisions related... Read More →
avatar for Gregory Daniel

Gregory Daniel

Deputy Director and Clinical Professor, Duke-Margolis Center For Health Policy
Gregory Daniel, PhD, MPH is the Deputy Director of the Duke-Robert J. Margolis, MD Center for Health Policy and a Clinical Professor in Duke's Fuqua School of Business. He directs the DC-based office of the Center leading its pharmaceutical and medical device policy portfolio, and... Read More →


Wednesday June 26, 2019 10:30am - 11:30am
Room 6D San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA
  • Credit Type ACPE, CME, IACET, RN
  • Tags Forum

10:30am

#339: Hot Topics in Quality and Regulatory Affairs for Combination Products
Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Application UAN: 0286-0000-19-670-L04-P; CME 1.00; IACET 1.00; RN 1.00

Combination products are a hot topic in healthcare. These products combine two areas of medicine: medical devices and drugs/biologics. While these products open doors to exciting new possibilities in the medical world, they also bring new challenges.

This session will discuss the importance of promoting a culture of compliance to build a compliant lifecycle management infrastructure around combination products to ensure appropriate cGXPs and Quality System regulations are enforced throughout the franchise. Open discussions, case studies and dialogue will be included.

Learning Objectives

Discuss clinical trial to launch including regulatory expectations and differences for devices and drugs, how to manage parallel development programs and minimize risks for regulatory submissions; Outline best practice of handling adverse effects, usability concerns and complaints throughout drug/ device lifecycle; Describe how to establish an on-going and rigorous post-market surveillance strategy to identify product, design, and process improvement.

Chair

Rachel SW Turow, JD, MPH

Speaker

FDA Update
Melissa B. Burns, MS

Industry Perspective
James Wabby, MHS

Industry Perspective
Jim Kershner, MSc



Speakers
MB

Melissa Burns

Senior Program Manager, Office of Combination Products, FDA
Melissa Burns is a Senior Program Manager in FDA’s Office of Combination Products (OCP). In this role, she is responsible for coordinating activities related to combination product review and regulation including development and review of guidance documents, regulations, and procedures... Read More →
avatar for Rachel Turow

Rachel Turow

Executive Counsel, Regulatory Law, Teva Pharmaceutical Industries Ltd.
Rachel Turow is Executive Counsel – Regulatory Law at Teva Pharmaceuticals Ltd. In this role, Rachel provides regulatory legal support to Teva’s specialty and generic pharmaceutical businesses, and supports Teva’s drug-device combination products and digital health projects... Read More →
avatar for James Wabby

James Wabby

Executive Director, Regulatory Affairs, Devices and Combination Products, Allergan
James Wabby is the Executive Director of Regulatory Affairs (Device and Combination Products) at Allergan in Irvine, California. He has over 15 years of experience in increasing quality compliance and regulatory affairs responsibilities within the GxP regulated environment pertaining... Read More →
avatar for Jim Kershner

Jim Kershner

Human Factors Engineering Technical Lead, Eli Lilly and Company
Jim Kershner is a senior technical lead in the Human Factors group at Eli Lilly and Company. In this role, Jim was responsible for establishing the HF group at Lilly, and continues to drive human factors technical input in accordance with regulatory guidance, consensus standards... Read More →


Wednesday June 26, 2019 10:30am - 11:30am
Room 6B San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

10:30am

#343: Efficient Preparation of Global CMC Dossiers
Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-19-676-L04-P; CME 1.00; IACET 1.00; RN 1.00

Preparation and maintenance of dossiers for a global market is exceedingly complex, due to different expectations for regulatory agencies across the globe. Efficiently creating a dossier to effectively meet worldwide requirements requires careful planning. This session will provide some practical and pragmatic approaches for managing the complexity of global CMC dossiers.

Learning Objectives

Describe the key regulatory considerations for preparation of a global CMC dossier; Discuss some of the global harmonization efforts that are underway.

Chair

John Smart, PhD

Speaker

Global CMC Dossiers: Preparation is Key
Angela McGillivary, PhD

Regulatory CMC Roadmaps for Fast-Evolving Global Markets (Brazil, China, India, Russia and Turkey)
Monika Jain, PhD



Speakers
avatar for Monika Jain

Monika Jain

Manager , Regulatory Affairs CMC, Regulatory Development Solutions, PPD
Monika Jain, Ph.D. joined the pharmaceutical industry in 1996. She is an internationally accomplished regulatory Chemistry, Manufacturing and Controls (CMC) scientist, with a long- standing background in pharmaceutical research and development of small molecule drug products.Since... Read More →
avatar for Angela McGillivary

Angela McGillivary

Principal Consultant, PAREXEL
Angela is a Principal Consultant at PAREXEL and is an Emerging Markets Regulatory Affairs Professional with over 20 years’ experience in the Pharmaceutical Industry. Angela McGillivary PAREXEL, Principal Consultant Angela has worked with global cross functional teams to develop... Read More →
avatar for John Smart

John Smart

Senior Director and Group Manager, Regulatory CMC, AstraZeneca
John has a PhD in Chemistry and over 20 years’ experience of CMC development within AstraZeneca including roles in Analytical & Product Development and Project Management. John is currently a Senior Director in Regulatory CMC with responsibility for regulatory submissions from Phase... Read More →


Wednesday June 26, 2019 10:30am - 11:30am
Room 8 San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

12:30pm

#349 EE: Regulatory Affairs Governance: Benchmarking and Sharing of Best Practices
Component Type: Workshop
Level: Intermediate

Gain a deeper understanding of governance in regulatory affairs through this discussion-based workshop where you’ll be teamed with peers to share your experiences and assess your company’s procedures to encourage efficient decision-making processes.

Learning Objectives

Describe how to benchmark internal governance within Regulatory Affairs in other pharmaceutical companies by engaging in peer discussion -Identify the importance and benefits of a well-functioning governance model; Explain how to utilize learnings to identify potential areas of improvement within your own company.

Chair

Benjamin Sugden, PharmD


Speakers
avatar for Benjamin Sugden

Benjamin Sugden

Assistant Director Regulatory Affairs, North America Regional Operations, Bayer U.S. Pharmaceuticals
Benjamin Sugden, Pharm.D., is currently the Assistant Director of Regulatory Affairs, North America Regional Operations for Bayer U.S. Pharmaceuticals. Dr. Sugden has previously served within Regulatory Affairs as a Senior Information Scientist. Before joining Bayer in 2017, he gained... Read More →


Wednesday June 26, 2019 12:30pm - 1:30pm
E and E Area Exh Hall San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

1:15pm

#353 CH: The Current and Future State of RIM
Component Type: Workshop
Level: Basic

An open discussion about the current and future state of RIM within different sized organizations with emphasis on the pros/cons of having a cloud based solution, the benefit of crafting a RIM ecosystem and approaching change management.

Learning Objectives

Describe the current and future state of RIM; Discuss the value added of crafting a complete RIM eco system; Identify and anticipate the impact of these systems on the end users.

Chair

Richard F Fredericks


Speakers
avatar for Richard Fredericks

Richard Fredericks

Director, Regulatory Systems and Information Management, Tesaro
I have been in the life sciences industry for 10+ years operating entirely within the Regulatory Operations space. My career began as a publisher and over the past decade has been gravitating towards the technology and process side of the business. Currently I lead the Regulatory... Read More →


Wednesday June 26, 2019 1:15pm - 1:45pm
Content Hub Sails Pav San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

2:00pm

#367: Model-Informed Drug Development (MIDD) and Complex Innovative Designs (CID) Programs: Where are We and What Have We Learned?
Component Type: Forum
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-19-691-L04-P; CME 1.25; IACET 1.25; RN 1.25

The PDUFA VI ‘goals letter’ identified MIDD and CID as enhancing regulatory decision tools. Starting in FY2018, the FDA committed to conduct pilot program for these. This session presents the experiences and learnings from the two pilot programs.

Learning Objectives

Discuss the status and share experience of the MIDD and CID programs; Summarize high level insights on the scope and process of the programs.

Chair

Rajanikanth Madabushi, PhD

Speaker

Update on PDUFA VI MIDD Pilot Program
Rajanikanth Madabushi, PhD

Update on the PDUFA VI CID Pilot Program
Dionne Price, PhD

Moderator
Issam Zineh, PharmD, MPH

Panelist
Yaning Wang, PhD

Panelist
Cristiana Mayer, DrSc, PhD

Panelist
Brian Corrigan, PhD



Speakers
BC

Brian Corrigan

Global Head, Clinical Pharmacology, Pfizer Inc
Brian Corrigan is Vice President and Global Head of Clinical Pharmacology and Head of Clinical Pharmacology for Global Product Development at Pfizer in Groton, Connecticut. In these roles, Brian directly oversees the Global Clinical Assay Group, the Global Pharmacometrics Group, and... Read More →
avatar for Rajanikanth Madabushi

Rajanikanth Madabushi

Team Leader, Guidance and Policy Team, OCP, OTS, CDER, FDA
Dr. Madabushi has 10 years of regulatory review experience as Pharmacometrics Review and as Team Leader in the Office of Clinical Pharmacology. He was predominantly involved in the application of quantitative clinical pharmacology approaches for regulatory decision making and addressing... Read More →
avatar for Cristiana Mayer

Cristiana Mayer

Director, Statistics and Decision Sciences, Janssen Research & Development, LLC
Cristiana Mayer is Director at Statistics and Decision Sciences, at Janssen R&D, Johnson & Johnson, with 20+ years of experience in the pharmaceutical industry. Cristiana is also the Head of the Adaptive Clinical Trials Center of Excellence at Janssen, and Vice-Chair of the DIA Adaptive... Read More →
avatar for Dionne Price

Dionne Price

Director, Division of Biometrics IV, Office of Biostatistics, OTS, CDER, FDA, FDA
Dr. Price is the Director of the Division of Biometrics IV providing leadership of analytical assessments in a regulatory setting. On the Senior Leadership Team and Statistical Policy Council, Dr. Price promotes collaborative efforts to advance the use of complex innovative trial... Read More →
avatar for Yaning Wang

Yaning Wang

Director, Division of Pharmacometrics, OCP, OTS, CDER, FDA
Dr. Yaning Wang is the Director of the Division of Pharmacometrics in the Office of Clinical Pharmacology at FDA. Before joining FDA, Dr. Wang received his Ph.D. in Pharmaceutics and master’s degree in Statistics from the University of Florida from 1999 to 2003. He also obtained... Read More →
avatar for Issam Zineh

Issam Zineh

Director, Office of Clinical Pharmacology, OTS, CDER, FDA
Dr. Zineh is Director of the Office of Clinical Pharmacology (OCP) at the U.S. FDA. He has held various leadership positions at FDA including Associate Director for Genomics in OCP (2008-2012), Co-Director of the CDER Biomarker Qualification Program (2009-2015), voting member of the... Read More →


Wednesday June 26, 2019 2:00pm - 3:15pm
Room 6B San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

2:00pm

#368: Global Rare Disease Town Hall
Component Type: Forum
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-19-692-L04-P; CME 1.25; IACET 1.25; RN 1.25

FDA and international regulators will address unique regulatory complexities and challenges specific to orphan product development. It will provide key information and updates about programs available to expedite orphan drug development and include audience Q&A.

Learning Objectives

Identify unique regulatory complexities and challenges specific to orphan drug development; Describe FDA and EMA programs available to expedite the development of orphan products; Recognize new regulatory initiatives and policy developments related to orphan drug review.

Chair

James E. Valentine, JD, MHS

Speaker

Panelist
Ilan Irony, MD

Panelist
Peter P. Stein, MD

Panelist
Janet Maynard, MD, MHS

Panelist
Agnès Saint-Raymond, DrMed, MD



Speakers
avatar for Janet Maynard

Janet Maynard

Director, Office of Orphan Products, OC, FDA
Dr. Janet Maynard, as the Director of the Office of Orphan Products Development (OOPD), oversees legislatively mandated designation and grant programs intended to promote the development of products for rare diseases including, orphan drug, rare pediatric disease, and humanitarian... Read More →
avatar for Agnès Saint-Raymond

Agnès Saint-Raymond

Head of International Affairs, Head of Portfolio Board, European Medicines Agency (EMA)
Head of International Affairs, Head of Portfolio Board MD and qualified Paediatrician. Joined EMA in 2000. Head of Special Areas for Human Medicines (Paediatric Medicines, Orphan Medicines, Scientific Advice, SME Office, and Scientific Support & Projects) until 2013. Head of Portfolio... Read More →
avatar for Peter Stein

Peter Stein

Director, Office of New Drugs, CDER, FDA
Peter Stein, M.D., is the Director of CDER’s Office of New Drugs (OND). OND is responsible for the regulatory oversight of investigational studies during drug development and decisions regarding marketing approval for new (innovator or non-generic) drugs, including decisions related... Read More →
avatar for James Valentine

James Valentine

Attorney, Hyman, Phelps & McNamara, PC
James Valentine, JD, MHS is an attorney at Hyman, Phelps & McNamara where he assists medical product industry and patient advocacy organization clients in a wide range of regulatory matters, including new drug and biologic development and approval issues. Mr. Valentine has been central... Read More →
avatar for Ilan Irony

Ilan Irony

Deputy Director, DCEPT, OTAT, CBER, FDA
After training at UCSF and NIH and years of practice in Internal Medicine and Endocrinology, Dr. Irony joined FDA CBER in 2000 as a clinical reviewer. He also worked in the Endocrine Division in CDER as a reviewer and team leader. In 2011, he returned to CBER first as branch chief... Read More →


Wednesday June 26, 2019 2:00pm - 3:15pm
Room 6F San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA
  • Level Intermediate
  • Featured Topics Generics
  • Featured Topics Precision Medicine,Regulator Thinking,Rare Disease
  • Credit Type ACPE, CME, IACET, RN
  • Tags Forum

2:00pm

#369: User-Fee Programs Myth Busting: General Financial Principles Explained
Component Type: Session
Level: Basic
CE: ACPE 1.25 Knowledge UAN: 0286-0000-19-693-L04-P; CME 1.25; IACET 1.25; RN 1.25

With preliminary user-fee reauthorization discussions underway, it is important to refresh on the financial principles behind a user-fee program and hear an update on the PDUFA VI, GDUFA II, and BSUFA II resource management commitments. Don't miss this fantastic educational opportunity to learn more about FDA's financial management.

Learning Objectives

Explain how the FDA financials operate beyond the collection of user-fees; Describe and name the general financial principles behind a user-fee program; Describe how FDA utilizes its resources, and discuss the restrictions FDA is under being part of the federal government; Summarize FDA’s progress in implementing the PDUFA VI, GDUFA II, and BSUFA II resource management related commitments.

Chair

Amy Bertha

Speaker

Panelist
James Tyler

Panelist
Andrew Kish, MS



Speakers
avatar for Amy Bertha

Amy Bertha

Director, North America Regulatory Policy, Bayer
Amy Bertha is currently Director, North America Regulatory Policy at Bayer. Prior to coming to Bayer, Amy worked 14 years at the FDA's Center for Drug Evaluation and Research in various offices, including the Regulatory Affairs Team in the Office of New Drugs and the Special Projects... Read More →
avatar for Andrew Kish

Andrew Kish

Director, Office of Planning and Strategic Analysis, OSP, CDER, FDA
Andrew is the Director of the Office of Strategic Programs and Analysis (OPSA) in the Center for Drug Evaluation and Research (CDER) in the U.S. Food and Drug Administration (FDA). Andrew oversees projects that span the application of economics, program evaluation, decision science... Read More →
avatar for James Tyler

James Tyler

Chief Financial Officer, Office of Operations, OC, FDA
Jay Tyler has 31 years of federal financial management experience serving at multiple federal agencies with diverse missions having broad national and international impact. He joined the U.S. Food and Drug Administration (FDA) in October of 2012 as the Chief Financial Officer (CFO... Read More →


Wednesday June 26, 2019 2:00pm - 3:15pm
Room 6E San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

4:15pm

#388: Convergence of the Regulatory Pathways for Advanced Therapy Medicinal Products
Component Type: Session
Level: Intermediate
CE: ACPE 1.25 Application UAN: 0286-0000-19-707-L04-P; CME 1.25; IACET 1.25; RN 1.25

Advanced Therapy Medicinal Products (ATMPs) include gene therapy, somatic-cell therapy, and tissue-engineered products. Collectively, these products are being developed in an evolving landscape and are associated with unique developmental challenges because of distinct manufacturing, clinical trial, and regulatory requirements. A 75-minute session is proposed facilitating the presentation and discussion of perspectives from several experienced regulatory agencies. An objective would be to provide a global overview of the various frameworks in place for the development of ATMPs, making note of ongoing and planned harmonization and cooperation projects.

This session will include collective short talks by CBER (FDA), European Medicines Agency (EMA), Pharmaceuticals and Medical Devices Agency (PMDA), Health Canada, and possibly a representative from Alliance for Regenerative Medicine (ARM) on the nature and importance of regulatory convergence with ATMPs. This would be followed by a interactive session with the speaker panel and the audience.

Learning Objectives

Describe the regulatory pathway for advanced therapy medicinal products in various global regions.

Chair

Peter W. Marks, MD, PhD

Speaker

EMA Update
Anthony Humphreys, MPharm

PMDA Update
Nobumasa Nakashima, PhD



Speakers
avatar for Peter Marks

Peter Marks

Director, Center for Biologics Evaluation and Research, FDA
Peter Marks received his graduate degree in cell and molecular biology and his medical degree at New York University and completed Internal Medicine residency and Hematology/Medical Oncology training at Brigham and Women’s Hospital in Boston. He has worked in academic settings teaching... Read More →
avatar for Anthony Humphreys

Anthony Humphreys

Head of Scientific Committees Regulatory Science Strategy, European Medicines Agency (EMA)
He is the Head of Scientific Committees Regulatory Science Strategy Division (SciRS). He is responsible for providing leadership in the Agency in the areas of Scientific Committees Coordination and Regulatory Science Strategy in support of delivering the EMRN 2020 Strategy. He chairs... Read More →
avatar for Nobumasa Nakashima

Nobumasa Nakashima

Senior Director for International Programs, Pharmaceuticals and Medical Devices Agency (PMDA)
Dr. Nakashima joined Pharmaceutical and Food Safety Bureau of Ministry of Health, Labor, and Welfare (MHLW) in 1992. He spent his career in the international field such as at WHO and OECD, not only in the domestic field. He worked as International Planning Director since September... Read More →


Wednesday June 26, 2019 4:15pm - 5:30pm
Room 6F San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

4:15pm

#391: Real World Evidence and Artificial Intelligence to Inform Post-Authorization Studies
Component Type: Forum
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-19-717-L04-P; CME 1.25; IACET 1.25; RN 1.25

This forum will discuss SENTINEL and other databases and how they are being mined for important signals. How is AI being used to assess real world effectiveness? Can AI inform a risk-based approach to lifecycle management?

Learning Objectives

Discuss how Sentinel and other databases are being mined for important signals; Identify how real work evidence and artificial intelligence is being used to inform post-authorization activities; Identify if artifical intelligence is a risk-based approach to lifecycle management.

Chair

Sudip Parikh, PhD

Speaker

The New World of Post-Authorization Knowledge Building
Mary Jo Lamberti, PhD, MA

SENTINEL and Other Databases: How Are These Being Mined for Important Safety and Effectiveness Signals?
Gerald J. Dal Pan

The Potential for Real World Data/Real World Evidence to Inform Postmarketing Safety Surveillance
Jose Vega, MD



Speakers
avatar for Mary Jo Lamberti

Mary Jo Lamberti

Associate Director of Sponsored Research, Research Assistant Professor, Tufts Center for the Study of Drug Development
Mary Jo Lamberti leads multi-company sponsored research studies at the Tufts Center for the Study of Drug Development (CSDD). She has extensive experience conducting research on biopharmaceutical industry practices and trends affecting contract research organizations and investigative... Read More →
avatar for Gerald Dal Pan

Gerald Dal Pan

Director, Office of Surveillance and Epidemiology, FDA
Gerald J. Dal Pan, MD, MHS, is Director of the Office of Surveillance & Epidemiology in FDA’s Center for Drug Evaluation and Research, where he oversees adverse event surveillance and analysis, pharmacoepidemiology, risk management, and medication error prevention. A member of the... Read More →
avatar for Sudip Parikh

Sudip Parikh

Senior Vice President and Managing Director, DIA Americas, DIA
Sudip S. Parikh, PhD, serves as Senior Vice President and Managing Director for DIA Americas. He previously served as Vice President and General Manager of Health & Consumer Solutions at Battelle, leading its Healthcare Improvement, Public Health Research, and Consumer, Industrial... Read More →
avatar for Jose Vega

Jose Vega

Vice President, Chief Safety Officer, Merck & Co., Inc.
José M. Vega is Vice President, Global Clinical Safety and Pharmacovigilance (GCS&PV) and Chief Safety Officer at Merck Research Laboratories (MRL). He also serves as Chair of the TransCelerate Pharmacovigilance Steering Committee and as Merck's representative on the Clinical Trials... Read More →


Wednesday June 26, 2019 4:15pm - 5:30pm
Room 16AB San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

4:15pm

#392: Breakthrough Therapy and PRIME Expedited Regulatory Pathways: Experience, Analysis, and Reflections from EMA, FDA, and Industry
Component Type: Session
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-19-735-L04-P; CME 1.25; IACET 1.25; RN 1.25

This session will compare recent hands-on experience with expedited regulatory pathways in EU and US (PRIME and Breakthrough Designation) both from the regulators’ and sponsor’s viewpoint. It will also include analysis of data comparing each program’s utilization and scope. A panel debate will be structured around statements that both panelists and the audience will be able to vote upon with the aim of inspiring honest discussion on the real benefits, drawbacks and future opportunities of these regulatory tools in the EU, US, and globally.

Learning Objectives

Describe Breakthrough Therapy and PRIME program aims, parameters, and regulatory agency experience in implementation; Discuss the sponsor perspectives on utilization of PRIME or Breakthrough Therapy pathways and how decisions about timing of engagement of FDA and EMA are made; Review utilization data of Breakthrough Therapy and PRIME pathways and concurrent and discordant decisions on sponsor applications for these designation.

Chair

Shannon Thor, PharmD, MS

Speaker

Experience with Breakthrough Therapy Applications: Viewpoints on Progress, Potential, and Lessons Learned
Sandra L. Kweder

Experience with PRIME Applications: Viewpoints on Progress, Potential, and Lessons Learned
Zahra Hanaizi, PharmD, MPharm

FDA Update on Breakthrough Therapies
Christopher Joneckis, PhD

Breakthrough Therapy and PRIME Expedited Regulatory Pathways: Industry Perspective
Samuel Rigourd, PharmD



Speakers
avatar for Sandra Kweder

Sandra Kweder

Deputy Director, Liaison to the EMA, Office of International Programs, OC, FDA
Sandra L. Kweder, MD, is Deputy Director of the Europe Office in the FDA Office of International Programs. Dr. Kweder previously served for more than a decade as Deputy Director, Office of New Drugs, in FDA’s Center for Drug Evaluation & Research, where she actively led numerous... Read More →
avatar for Zahra Hanaizi

Zahra Hanaizi

Scientific officer, PRIME Coordinator, Scientific and Regulatory Management Dept, European Medicines Agency (EMA)
Zahra Hanaizi joined EMA in 2007 as Product Team Leader, managing centralised medicinal products initial marketing authorisation applications, maintenance and life-cycle activities, mostly in the therapeutic area of Oncology. Since 2015, she has been working on the creation, implementation... Read More →
CJ

Christopher Joneckis

Associate Director for Review Management, CBER, FDA
*
avatar for Samuel Rigourd

Samuel Rigourd

Senior Director Regulatory Affairs, Alnylam Pharmaceuticals
Samuel Rigourd joined Alnylam Pharmaceuticals in 2016 as Senior Director of Regulatory Affairs, where he leads the global registration strategy of a new class of medicines, RNA interference therapeutics. He has over 15 years of experience in regulatory affairs, in various roles both... Read More →
avatar for Shannon Thor

Shannon Thor

International Policy Analyst, Office of Global Policy and Strategy, European Off, FDA
Lieutenant Commander Shannon Thor is a pharmacist and officer in the United States Public Health Service, serving as an international policy analyst in FDA's Europe Office. LCDR Thor’s FDA career began when she joined the Office of Health and Constituent Affairs in 2014, serving... Read More →


Wednesday June 26, 2019 4:15pm - 5:30pm
Room 6D San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA
  • Featured Topics Regulator Thinking
  • Credit Type ACPE, CME, IACET, RN
  • Tags Session

4:15pm

#390: Clinical Trial Innovation: Pathways for Selecting and Developing Novel, Fit-for-Purpose, Technology-Derived Study Endpoints
Component Type: Session
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-19-709-L04-P; CME 1.25; IACET 1.25; RN 1.25

This session will cover regulatory and research perspectives on challenges and opportunities for selecting, developing and implementing fit-for-purpose digital technology tools for to derive study endpoints to support drug development.

Learning Objectives

Describe the regulatory and practical consideration for successfully selecting, developing, and implementing patient-focused digital technology tools for use to derive study endpoints (e.g., accelerometer or mobile technology derived endpoints) that are fit for purpose for use in clinical trials to support drug development.

Chair

Ebony Dashiell-Aje, PhD

Speaker

What Can Technology-Derived Endpoint Tell us About the Patient’s Experience?
Jean Paty, PhD

Fit-for-Purpose in the Digital Era: Digital Measurement Tools in Clinical Trials
Jennifer Goldsack, MA, MBA, MS

Fit-for-Purpose Digital Health Technology Driven Study Endpoints: Regulatory Considerations for Implementation
Ebony Dashiell-Aje, PhD



Speakers
avatar for Ebony Dashiell-Aje

Ebony Dashiell-Aje

Clinical Outcome Assessments Reviewer, COA Staff, OND, CDER, FDA
Dr. Dashiell-Aje is a reviewer with the Clinical Outcome Assessments (COA) Staff in the Center for Drug Evaluation and Research (CDER) at the FDA. She serves as an expert consultant to CDER and other FDA Centers (e.g., CDRH, CBER), advising on COAs, study endpoint issues and regulatory... Read More →
avatar for Jennifer Goldsack

Jennifer Goldsack

Interim Executive Director, Digital Medicine Society (DiMe)
Jen Goldsack is the Interim Executive Director of the Digital Medicine (DiMe) Society and is Vice President of Digital Measurement at monARC Bionetworks.
avatar for Jean Paty

Jean Paty

Vice President, Patient-Centered Endpoints, IQVIA
Dr. Paty is an acknowledged leader in the effective strategies and practices of capturing patient perspective data for use in the clinical development of new medical products. He has published extensively in the areas of Patient Reported Outcomes (PRO) and electronic PRO (ePRO), as... Read More →


Wednesday June 26, 2019 4:15pm - 5:30pm
Room 6E San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA
  • Level Intermediate
  • Featured Topics Patient Focused,Innovative Trial Design
  • Credit Type ACPE, CME, IACET, RN
  • Tags Session

4:15pm

#389: Transparency, Expanded Access Navigator, Right to Try: Helping Patients Get Access to Investigational Medicines?
Component Type: Forum
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-19-708-L04-P; CME 1.25; IACET 1.25; RN 1.25

Numerous steps have been taken to increase access to investigational medicines for patients with serious and life-threatening diseases. This session will explore whether these initiatives have had the desired impact.

Learning Objectives

Describe changes to expanded access programs, including Right to Try; Assess the impact of these changes to the patient; Discuss ways to improve access to investigational medicines for patients.

Chair

Kim Quaintance-Lunn

Speaker

Patient Perspective on Preapproval Access
Melinda Bachini

Innovating Compassionate Use Request Processes with CompAC
Beverly L Harrison

Assessment of Progress
Alison Bateman-House, PhD, MA, MPH



Speakers
avatar for Alison Bateman-House

Alison Bateman-House

Assistant Professor, Division of Medical Ethics, NYU Langone Health
Alison Bateman-House, PhD, MPH, MA, is an assistant professor in the Division of Medical Ethics at NYU Langone Health's School of Medicine. She is co-chair of the Working Group on Compassionate Use and Preapproval Access (CUPA), an academic group that studies ethical issues concerning... Read More →
avatar for Beverly Harrison

Beverly Harrison

Head, Patient Support, Janssen Pharmaceutical Companies of Johnson & Johnson
Leading the Patient Support Group @ Janssen R&D (a J&J Co.), reporting directly to the Chief Medical Officer, Beverly focuses on developing strategies for patients’ needs by working with internal/external stakeholders & engaging patient advocacy organizations. She is a R&D & nonprofit... Read More →
avatar for Kim Quaintance-Lunn

Kim Quaintance-Lunn

Vice President and Head, Regulatory Policy, North American Regulatory Affairs, Bayer
Kim Quaintance-Lunn serves as Vice President and Head, Regulatory Policy, North American Regulatory Affairs, at Bayer. Kim joined Bayer in February 2014 to establish and lead the US regulatory policy function for the organization. She works with colleagues to analyze regulatory policy... Read More →
MB

Melinda Bachini

Advocacy Coordinator, Cholangiocarcinoma Foundation


Wednesday June 26, 2019 4:15pm - 5:30pm
Room 6B San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

4:15pm

#396: Challenges to Access: Bringing Payers to the Table
Component Type: Session
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-19-711-L04-P; CME 1.25; IACET 1.25; RN 1.25

While the FDA and EMA avail themselves for early engagement with pharmaceutical manufacturers, non-binding joint consultations with regulators and payers have been to-date available to manufacturers primarily in Europe. With the moving target of paradigm shift from volume-based to value-based healthcare delivery in the US, the question remains if viable avenues for engagement with payers exist in the fragmented market place. Traditional engagement opportunities through payer advisory boards and standing panels have proven moderately effective and with the growing focus on health technology assessment in the US that spans comparative effectiveness, short-term affordability and long-term value for money assessments, a dialogue may be warranted to explore more formal ways to engage manufacturers, regulators and payers. A panel composed of the relevant stakeholders will have an open dialogue on the topic offering a forum for generating potential concepts for testing to enable formal early payer engagement in the medicine development process in the US.

Learning Objectives

Identify potential innovative ways of early engagement with payers in the US to enhance medicine development programs and produce a “reimbursable file” at the time of regulatory approval.

Chair

Ruslan Horblyuk, PhD, MA

Speaker

Panelist
Sean Tunis, MD, MSc

EMA Perspective
Tânia Teixeira, PharmD

Industry Perspective
Cristina Masseria



Speakers
RH

Ruslan Horblyuk

Chief Strategic Consulting Officer, AESARA, Inc.
Ruslan Horblyuk started his career in international health policy consulting over 20 years ago working in the countries of former Soviet Union as part of the US Agency for International Development funded Health Policy Reform Initiative. He transferred to the industry in 2003 and... Read More →
avatar for Tânia Teixeira

Tânia Teixeira

EMA Liaison Official at FDA, European Medicines Agency (EMA)
Tania Teixeira is the EMA Liaison Official to FDA. She started her career in the pharmaceutical industry and joined EMA in 2004. She held positions as a Head of service for Referrals, dealing with emerging concerns which require a harmonised position across the EU, and as Product... Read More →
CM

Cristina Masseria

Methods and Capabilities Lead, Pfizer Inc
ST

Sean Tunis

Senior Strategic Advisor, Center For Medical Technology Policy (CMPT)


Wednesday June 26, 2019 4:15pm - 5:30pm
Room 14B San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA
 
Thursday, June 27
 

9:00am

#410: Advancing Benefit-Risk Assessment to Support FDA’s Regulatory Review of Human Drugs and Biologics
Component Type: Session
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-19-724-L04-P; CME 1.25; IACET 1.25; RN 1.25

FDA currently uses a structured, qualitative framework for benefit-risk assessment of human drugs and biologics. This session will cover FDA’s efforts to enhance this qualitative framework and explore more advanced benefit-risk assessment methods.

Learning Objectives

Describe FDA’s approach to benefit-risk assessment under PDUFA V and VI; Discuss efforts to continue enhancing FDA’s methodological approach to benefit-risk assessment; Summarize findings from analysis of BRFs in 2017 novel drug approvals.

Chair

Graham Thompson

Speaker

Implementation of a Structured Benefit-Risk Framework into FDA's Human Drug Review
Leila Grace Lackey, MHS, PhD

CBER’s Experience with Benefit-Risk
Hong Yang, PhD

Planning and Assessing Benefit-Risk Quantitatively in the Next Decade
Susan Duke, MSc

Best Practices for Quantitative Benefit-Risk Assessments
Tommi Tervonen



Speakers
avatar for Susan Duke

Susan Duke

Mathematical Statistician, OB, OTS, CDER, FDA
Susan became involved in safety related industry-wide graphics in 2009 when she joined the FDA/Industry/Academia Safety Graphics WG. She is currently a Mathematical Statistician reviewer in CDER and member of a cross-functional safety monitoring WG co-sponsored by DIA and ASA Biopharm... Read More →
avatar for Leila Lackey

Leila Lackey

Operations Research Analyst, OPSA, OSP, CDER, FDA
Dr. Lackey has a Master of Health Science from Johns Hopkins in Environmental Health Sciences and a doctorate from UCLA in Environmental Science and Engineering. Professionally, she has worked for the Environmental Protection Agency and for the Food and Drug Administration leading... Read More →
avatar for Tommi Tervonen

Tommi Tervonen

Research Scientist, Evidera, PPD
Tommi Tervonen, PhD, is an Associate Director of Patient Preferences and Research Scientist at the Patient-Centered Research team of Evidera in London, UK. Dr. Tervonen joined Evidera in September 2015 after leaving his position of Assistant Professor at the Erasmus School of Economics... Read More →
GT

Graham Thompson

Operations Research Analyst, OSP, CDER, FDA
Graham Thompson joined FDA in 2012 and currently serves as an operations research analyst for the Decision Support and Analysis Team (DSAT) in FDA’s Center for Drug Evaluation and Research (CDER). He works on CDER projects and initiatives related to benefit-risk assessment and supporting... Read More →
avatar for Hong Yang

Hong Yang

Biologist, Office of Biostatistics and Epidemiology, CBER, FDA
Dr. Hong Yang is a senior regulatory scientist in the Office of Biostatistics and Epidemiology, CBER, FDA. She holds Ph.D. degree in Biological Engineering. Dr. Yang has expertise in benefit-risk assessment of biological products. She has been devoted in regulatory review, as well... Read More →


Thursday June 27, 2019 9:00am - 10:15am
Room 6F San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA