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Precision Medicine [clear filter]
Monday, June 24
 

11:00am

#118: Statistical Considerations for Trials Using Surrogate Endpoints for Accelerated Approval
Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Application UAN: 0286-0000-19-531-L04-P; CME 1.00; IACET 1.00; RN 1.00

For slowly progressed diseases, it may take years, if not decades, to observe clinical outcomes, such as mortality. Owing to the regulatory agencies’ accelerated approval pathway, drug developers can now consider applying for a drug approval based on surrogate endpoints. In this session, many surrogate endpoints adopted in various diseases and statistical methods for determining the validity of the surrogacy will be discussed, including their implementation in real case examples.

Learning Objectives

Describe cases that utilize the accelerated approval and what good surrogate endpoints are for common and rare diseases; Identify how to apply appropriate statistical methods for assessing the association between surrogate endpoints and clinical outcomes.

Chair

Yeh-Fong Chen, PhD

Speaker

FDA Update
Aloka Chakravarty, PhD

Statistical Assessment of Potential Surrogate Endpoints: Is the Bar too High?
Marc E. Buyse, DrSc

Using the Biomarker Endpoint for Accelerated Approval
Stephanie O. Omokaro, MD



Speakers
avatar for Marc Buyse

Marc Buyse

Chief Scientific Officer, International Drug Development Institute (IDDI)
Marc Buyse holds master’s degrees from Brussels University (Belgium), the Cranfield School of Management (UK) and a doctorate degree from the Harvard School of Public Health (USA). He had worked for 12 years at the EORTC in Brussels and for 2 years at the Dana Farber Cancer Institute... Read More →
avatar for Aloka Chakravarty

Aloka Chakravarty

Deputy Director, Office of Biostatistics, Office of Translational Sciences, CDER, FDA
Dr. Chakravarty joined CDER in 1992. She is an internationally recognized thought leader in the area of safety evaluation, surrogate markers and biomarkers in drug development and has presented and published widely on it. Dr. Chakravarty received the FDA Award of Merit in 2008 and... Read More →
avatar for Yeh-Fong Chen

Yeh-Fong Chen

Mathematical Statistician (Team Lead), Office of Translational Sciences, CDER, FDA
Dr. Chen is the Team Leader of the Division of Biometrics IX within CDER of FDA, supervising reviewers for the Division of Non-Malignant Hematology Products. She joined FDA in 2000 after receiving her Ph.D. from the University of Iowa. She has had years' experience in reviewing drug... Read More →
avatar for Stephanie Omokaro

Stephanie Omokaro

Lead Medical Officer, Office of New Drugs, CDER, FDA
Dr. Omokaro is a Lead Medical Officer in the Division of Gastroenterology and Inborn Errors Products in the Center for Drug Evaluation and Research at FDA. She was educated in Biochemistry at Cornell University, and in medicine at Boston University. She completed a pediatric residency... Read More →


Monday June 24, 2019 11:00am - 12:00pm
Room 3 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
 
Tuesday, June 25
 

2:00pm

#261: Patient Experience Data: How Could this Data Enhance Decision-Making at Different Stages of Medical Product Development?
Component Type: Forum
Level: Basic
CE: ACPE 1.25 Knowledge UAN: 0286-0000-19-613-L04-P; CME 1.25; IACET 1.25; RN 1.25

This forum will provide an opportunity for key stakeholders to discuss examples of various types of patient experience data, and how this data could potentially inform decision-making at different stages of medical product development.

Learning Objectives

Define patient experience data; Describe types of patient experience data that could inform medical product development; Discuss how patient experience data can inform decision making at different stages of medical product development.

Chair

Meghana Chalasani, MHA

Speaker

FDA Perspective
Michelle Campbell, PhD

Panelist
Elizabeth Hart, MD

Panelist
Isabelle Lousada, MA

Panelist
Kristina Bowyer, LPN



Speakers
avatar for Kristina Bowyer

Kristina Bowyer

Executive Director of Patient Advocacy, Ionis Pharmaceuticals
Kristina Bowyer is the Executive Director of Patient Advocacy & Engagement at Ionis Pharmaceuticals. Kristina has worked at Ionis for over 26 years and has spent the last seven years successfully building Ionis’ internal patient engagement program to ensure that the patient perspective... Read More →
avatar for Michelle Campbell

Michelle Campbell

Senior Clinical Analyst for Stakeholder Engagement, DNP, OND, CDER, FDA
Michelle Campbell is the Sr. Clinical Analyst for Stakeholder Engagement and Clinical Outcomes for the Division of Neurology Products, Office of New Drugs (OND), Center for Drug Evaluation and Research (CDER), U.S. Food and Drug Administration (FDA). Previously, Dr. Campbell was a... Read More →
avatar for Meghana Chalasani

Meghana Chalasani

Senior Research Analyst, Office of the Center Director, CDER, FDA
Meghana Chalasani currently serves as a senior research analyst for the Patient-Focused Drug Development (PFDD) Program in FDA’s Center for Drug Evaluation and Research (CDER). She works closely on CDER’s various PFDD initiatives and provides strategic, regulatory, program, and... 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 Elizabeth Hart

Elizabeth Hart

Branch Chief, General Medicine 1, OTAT, CBER, FDA
Dr. Elizabeth Hart is the Branch Chief of General Medicine 1 in the Office of Tissue and Advanced Therapies in the Center for Biologics Evaluation and Research (CBER) at the Food and Drug Administration (FDA). She completed her undergraduate medical training at the University of Pennsylvania... Read More →


Tuesday June 25, 2019 2:00pm - 3:15pm
Room 5AB 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

#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
 
Wednesday, June 26
 

10:30am

#329: Global Clinical Trials: Make Them Really Global and Involve Africa
Component Type: Forum
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-19-674-L04-P; CME 1.00; IACET 1.00; RN 1.00

Changing how we do global clinical trials, with the inclusion of emerging markets in this development process, is imperative. A panel of experts will discuss how to plan and involve pharma/biotech and technology providers.

Learning Objectives

Describe ways in which to integrate emerging markets in global clinical trials using innovative technologies;Discuss approaches to process optimization, mobile health digitalization, blockchain, and change management on how to undertake clinical trials within Africa as the process model: Identify challenges and solutions from actual cases and experience.

Chair

Adama Ibrahim, MBA

Speaker

Next Generation Real World Evidence with Blockchain
Alexis Normand, MPA, MSc

Panelist
Tina Barton, PhD, MBA



Speakers
TB

Tina Barton

Chief Operating Officer, Emerging Markets Quality Trials (EMQT)
Tina Barton is an experienced Board member and Chair, with a BSc, PhD in cancer research and MBA, and has held Senior Management roles in large global through mid-size to small emerging organisations, working in partnership to lead for success, as a customer-focused clinical research... Read More →
avatar for Adama Ibrahim

Adama Ibrahim

Associate Director, Clinical Operations| WIN UK Chapter Co-Lead, Biogen
An innovation award winning operational strategy collaborator, blockchain advocate, eCOA and patient engagement thought leader with over 17 years in the NHS on commission by the DOH and in Industry (Hoffman La-Roche, Amgen, ALMAC, ICON and Biogen). An R&D transformation change champion... Read More →
avatar for Alexis Normand

Alexis Normand

Head of Blockchain for Health Consortium, Embleema
Alexis Normand is head of the Embleema Blockchain Consortium bringing together patients, their advocacy groups, life sciences companies (and regulators, ie FDA to set a new digital standard for sharing Real World Evidence (RWE), to have new treatments approved faster, while ensuring... Read More →


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

10:30am

#344: Meaningful Patient-Focused Drug Development for Rare Disease and Personalized Medicine
Component Type: Forum
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-19-672-L04-P; CME 1.00; IACET 1.00; RN 1.00

This session will provide a platform to discuss measurement challenges and practical considerations for selection of fit-for-purpose clinical outcome assessments (COAs) to support endpoints in rare disease drug development programs.

Learning Objectives

Discuss practical considerations for utilizing clinical outcome assessments (COAs) to support clinical trial endpoint(s) in drug development; Identify the measurement challenges and recommendations for the selection of COAs for rare disease drug development.

Chair

Michelle Campbell, PhD

Speaker

Generalized Pairwise Comparisons for Benefit/Risk Assessment in Personalized Medicine
Marc E. Buyse, DrSc

Endpoint Selection and Use of Clinical Outcome Assessments (COAs) in Rare Disease Drug Development: A Regulatory Perspective
Lili Garrard, PhD

Selection of Fit-for-Purpose COAs to Support Endpoints in Rare Disease
Jean Paty, PhD



Speakers
avatar for Marc Buyse

Marc Buyse

Chief Scientific Officer, International Drug Development Institute (IDDI)
Marc Buyse holds master’s degrees from Brussels University (Belgium), the Cranfield School of Management (UK) and a doctorate degree from the Harvard School of Public Health (USA). He had worked for 12 years at the EORTC in Brussels and for 2 years at the Dana Farber Cancer Institute... Read More →
avatar for Michelle Campbell

Michelle Campbell

Senior Clinical Analyst for Stakeholder Engagement, DNP, OND, CDER, FDA
Michelle Campbell is the Sr. Clinical Analyst for Stakeholder Engagement and Clinical Outcomes for the Division of Neurology Products, Office of New Drugs (OND), Center for Drug Evaluation and Research (CDER), U.S. Food and Drug Administration (FDA). Previously, Dr. Campbell was a... Read More →
LG

Lili Garrard

Mathematical Statistician, Office of Biostatistics, OTS, CDER, FDA
Lili Garrard is a statistical reviewer on the Clinical Outcome Assessment (COA) Statistical Support Team at the Division of Biometrics III, Office of Biostatistics (OB), Office of Translational Sciences (OTS), Center for Drug Evaluation and Research (CDER), U.S. Food and Drug Administration... Read More →
avatar for Jean Paty

Jean Paty

Vice President, Patient-Centered Science, IQVIA
Jean Paty is a leader in effective strategies and practices of capturing patient experience data for use in clinical development and commercial success of new and existing medical products. Jean has been published extensively in the areas of Clinical Outcome Assessments (COA) and... Read More →


Wednesday June 26, 2019 10:30am - 11:30am
Room 3 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
Susanis is a Mathematical Statistician reviewer in CDER, assigned to pulmonary, allergy and critical care indications. She became involved in safety related industry-wide graphics in 2009 when she joined the FDA/Industry/Academia Safety Graphics WG, which lead to subsequent interests... 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, Decision Support and Analysis Team, 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