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09: Regulatory [clear filter]
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

#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

#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

#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

#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

#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

#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

#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

#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

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

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
 

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

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

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

#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