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Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2016. Advancing the Discipline of Regulatory Science for Medical Product Development: An Update on Progress and a Forward-Looking Agenda: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/23438.
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Appendix B

Workshop Agenda

Advancing the Discipline of Regulatory Science for Medical Product
Development: An Update on Progress and a Forward-Looking Agenda

An IOM Workshop

October 20–21, 2015
Keck Center
500 Fifth Street, NW Room 100
Washington, DC 20001

Background and Workshop Objectives:

The U.S. Food and Drug Administration (FDA) defines regulatory science as the science of developing new tools, standards, and approaches to assess the safety, effectiveness, quality, toxicity, public health impact, or performance of FDA-regulated products. Since its inception, the Institute of Medicine’s Forum on Drug Discovery, Development, and Translation has focused on the need for strengthening the scientific basis of drug regulation. In February 2010, the Forum held a workshop, Building a National Framework for the Establishment of Regulatory Science for Drug Development, that examined the state of the science of drug regulation and considered approaches to enhance regulatory science. In September 2011, the Forum held another workshop, Strengthening a Workforce for Innovative Regulatory Science

Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2016. Advancing the Discipline of Regulatory Science for Medical Product Development: An Update on Progress and a Forward-Looking Agenda: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/23438.
×

in Therapeutics Development, that considered opportunities and needs for advancing innovative regulatory science through workforce and career development. Over the past several years, models to support the discipline have advanced. FDA’s Centers of Excellence in Regulatory Science and Innovation enhance training and educational opportunities for regulatory scientists. Private funders have also established programs: For example, in 2011 the Burroughs Wellcome Fund launched Innovations in Regulatory Science Awards (IRSA), which aim to strengthen regulatory systems capacity by funding regulatory science–based research and collaborations.

This workshop will provide a venue to review progress in building the foundations of regulatory science and to explore a forward-looking agenda for bolstering the field. Participants will examine the current state and scope of the discipline, highlight opportunities to address barriers to success, and explore ways to foster collaboration. The workshop objectives are to:

  • Explore current regulatory science priorities and strategies in federal, academic, and private-sector settings.
  • Consider the current state of regulatory science as a discipline.
    • Discuss professional training successes.
    • Highlight opportunities to further support training, workforce, and career development.
  • Explore the core components of a robust discipline of innovative regulatory science.
    • Consider gaps and key opportunities to address needs to support the discipline of innovative regulatory science.
  • Examine needs and barriers to collaboration among, across, and within the public and private sectors.

DAY ONE (October 20, 2015)

8:00 a.m. Breakfast Available

8:30 a.m.

Opening Remarks

MARTIN PHILBERT, Workshop Co-Chair

Professor and Dean
University of Michigan School of Public Health

ALASTAIR WOOD, Workshop Co-Chair

Partner, Symphony Capital

Professor of Medicine and Pharmacology, Weill Cornell School of Medicine

Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2016. Advancing the Discipline of Regulatory Science for Medical Product Development: An Update on Progress and a Forward-Looking Agenda: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/23438.
×

SESSION I: SETTING THE STAGE FOR INNOVATION IN REGULATORY SCIENCE

Session Objectives:

  • Introduce and discuss workshop theme.
  • Highlight key scientific questions for the field of innovative regulatory science, focusing on the role of information as it is generated across regulatory science domains and ways that it can be better put to use.
  • Discuss how new capabilities and access to new information could advance regulatory science for medical product development.
  • Highlight operational challenges.
8:40 a.m. Background and Session Objectives

Session Chair: Alastair Wood, Partner, Symphony Capital, Professor of Medicine and Pharmacology, Weill Cornell School of Medicine (Workshop Co-Chair)

8:45 a.m.

Workshop Theme and Framework: Innovation in Regulatory Science Through Integration of Information

Transformation of Our Ability to Generate, Analyze, Integrate, and Share Information Across Regulatory Science Applications

RUSS ALTMAN

The Kenneth Fong Professor of Bioengineering, Genetics, Medicine, & (by courtesy) Computer Science

Stanford University

9:00 a.m.

Advancing Regulatory Science Using Information and Information Science: What to Improve and How to Improve It

JIM STEVENS

Distinguished Research Fellow
Eli Lilly
Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2016. Advancing the Discipline of Regulatory Science for Medical Product Development: An Update on Progress and a Forward-Looking Agenda: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/23438.
×

9:15 a.m.

Value of Information to Inform Decision Making Under Uncertainty

KATHERINE VON STACKELBERG

Research Scientist
Harvard Center for Risk Analysis

9:30 a.m.

Fusing RCTs with EHR “Big Data”

DEREK ANGUS

Distinguished Professor and Mitchell P. Fink Endowed Chair, Department of Critical Care Medicine

University of Pittsburgh

9:45 a.m.

Panel Discussion and Audience Q&A (30 mins)

10:15 a.m.

BREAK (15 mins)

SESSION II: LEARNING LESSONS THROUGH CONSIDERATION OF REGULATORY SCIENCE APPLICATIONS

Session Objectives:

  • Discuss how enhanced approaches to obtaining, accessing, and integrating information could advance the science throughout and across development.
  • Through consideration of selected regulatory science applications, discuss current capabilities for regulatory science and strategic priorities in federal, academic, and private sectors.
  • Suggest ways forward to address identified gaps and operational challenges.
10:30 a.m. Background and Session Objectives

Session Chair: Stephen Ostroff, Acting Commissioner, U.S. Food and Drug Administration

10:35 a.m.

Identifying and Developing Meaningful Biomarkers

Panel Moderator: John Wagner, Senior Vice President, Head of Clinical and Translational Sciences, Takeda Pharmaceuticals

Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2016. Advancing the Discipline of Regulatory Science for Medical Product Development: An Update on Progress and a Forward-Looking Agenda: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/23438.
×

10:40 a.m.

Basic Science of Measurement: Metrology Principles for Biomarkers

MARC SALIT

Leader, Genome-Scale Measurements

National Institute of Standards and Technology (NIST)

10:50 a.m.

Opportunities to Develop Meaningful Biomarkers

SHASHI AMUR

Scientific Lead, Center for Drug Evaluation and Research’s (CDER’s) Biomarker Qualification Program

U.S. Food and Drug Administration

11:00 a.m.

Challenges and Opportunities for Qualifying Biomarkers: An Industry Perspective

GABRIELA LAVEZZARI

Assistant Vice President, Science & Regulatory Advocacy

Pharmaceutical Research and Manufacturers of America (PhRMA)

11:10 a.m.

Collaborative Approaches for Developing Kidney Safety Biomarkers

JOHN MICHAEL SAUER

Executive Director, Predictive Safety Testing Consortium (PSTC)

The Critical Path Institute

11:20 a.m.

Panel Discussion and Audience Q&A (30 mins)

11:50 a.m.

BREAK to Lunch (60 mins)

SESSION II, CONTINUED: CONSIDERATION OF REGULATORY SCIENCE APPLICATIONS

12:50 p.m. Clinical Trial Data Integration

Panel Moderator: Rob Califf, Deputy Commissioner for Medical Products and Tobacco, U.S. Food and Drug Administration

Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2016. Advancing the Discipline of Regulatory Science for Medical Product Development: An Update on Progress and a Forward-Looking Agenda: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/23438.
×

12:55 p.m.

Big Data for Randomized Controlled Trials: Opportunities and Challenges for the Reliable Assessment of Treatment Effects

MARTIN LANDRAY

Professor of Medicine and Epidemiology, Deputy Director Big Data Institute

University of Oxford

1:05 p.m.

Approaches to Overcoming Variance Due to Heterogeneity: A Case Study in a Rare Disease

SUSAN WARD

Founder and Executive Director

The TAP Collaboration

1:15 p.m.

Access to Patient-Level Data from Clinical Trials

PERRY NISEN

Chief Executive Officer

Sanford Burnham

1:25 p.m.

The Role of Open APIs (Application Programming Interfaces) and FHIRs (Fast Healthcare Interoperability Resources) Platform for Integrating Patient Care and Clinical Research Data

CHARLES JAFFE

Chief Executive Officer

Health Level Seven International

1:35 p.m.

Data Aggregation Across Diseases and Between Stakeholders

ENRIQUE AVILÉS

Chief Technology Officer

The Critical Path Institute

Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2016. Advancing the Discipline of Regulatory Science for Medical Product Development: An Update on Progress and a Forward-Looking Agenda: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/23438.
×

1:45 p.m.

Panel Discussion and Audience Q&A (30 mins)

Panelists:

  • Clinical Trial Data Integration speakers (above), and
  • Kyle J. Myers, Director, Division of Imaging, Diagnostics, and Software Reliability (DIDSR), U.S. Food and Drug Administration

2:15 p.m.

BREAK (15 mins)

2:30 p.m.

Next-Generation Surveillance

Panel Moderator: Brian Strom, Chancellor of Rutgers Biomedical and Health Sciences, Rutgers, the State University of New Jersey

2:35 p.m.

Next-Generation Surveillance: FDA’s Sentinel Program

RICHARD PLATT

Professor and Chair of the Department of Population Medicine

Harvard Pilgrim Health Care Institute

2:45 p.m.

Harnessing Web Search Data as Complementary Signals for Pharmacovigilance

ERIC HORVITZ

Distinguished Scientist & Managing Director
Microsoft Research

2:55 p.m.

Online Discussion Forums as Potential Sources of Adverse Drug Event Data

JOHN H. HOLMES

Professor of Medical Informatics
University of Pennsylvania

3:05 p.m.

New Frontiers in Safety Surveillance

JOHN BROWNSTEIN

Associate Professor
Harvard Medical School
Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2016. Advancing the Discipline of Regulatory Science for Medical Product Development: An Update on Progress and a Forward-Looking Agenda: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/23438.
×

3:15 p.m.

Panel Discussion and Audience Q&A (30 mins)

Panelists:

  • Next-Generation Surveillance speakers (above), and
  • Danica Marinac-Dabic, Director, Division of Epidemiology, Center for Devices and Radiological Health (CDRH), U.S. Food and Drug Administration

3:45 p.m.

Innovation in Modeling and Integrating Information

Panel Moderator: Darrell Abernethy, Associate Director for Drug Safety, Office of Clinical Pharmacology, U.S. Food and Drug Administration

3:50 p.m.

Statistical Modeling for Efficient and Adaptive Trial Designs Using Composite Endpoints

BRIAN ALEXANDER

Associate Professor of Radiation Oncology
Harvard Medical School

4:00 p.m.

Model Informed Drug Development and Regulatory Decisions Today and Tomorrow

SANDY ALLERHEILIGEN

Vice President, Modeling and Simulation Merck

4:10 p.m.

Innovation in Modeling and Integrating Information: The CAMD Knowledge Model for Alzheimer’s Disease

BRIAN CORRIGAN

Senior Director
Pfizer Inc.

4:20 p.m.

Assessing the Placebo Effect and Drug Efficacy Using Functional MRI

ARIANA ANDERSON

Assistant Research Statistician
University of California, Los Angeles
Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2016. Advancing the Discipline of Regulatory Science for Medical Product Development: An Update on Progress and a Forward-Looking Agenda: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/23438.
×

4:30 p.m.

Panel Discussion and Audience Q&A (30 mins)

Panelists:

  • Innovation in Modeling and Integrating Information speakers (above), and
  • Klaus Romero, Director of Clinical Pharmacology, The Critical Path Institute (C-Path)

5:00 p.m.

Wrap-Up of Day One

5:10 p.m.

ADJOURN

DAY TWO (October 21, 2015)

8:30 a.m. Welcome and Reflections from Day One

MARTIN PHILBERT, Workshop Co-Chair

Professor and Dean

University of Michigan School of Public Health

ALASTAIR WOOD, Workshop Co-Chair

Partner, Symphony Capital

Professor of Medicine and Professor of Pharmacology, Weill Cornell School of Medicine

SESSION III: ENVISIONING THE FUTURE OF REGULATORY SCIENCE: A FORWARD-LOOKING AGENDA

Session Objective:

  • Discuss opportunities and priorities to advance innovative regulatory science through information.
8:35 a.m. Disciplinary Components and Infrastructure Needs

Panel Moderator: Martin Philbert, Professor and Dean, University of Michigan (Workshop Co-Chair)

8:40 a.m.

A Workforce to Bridge the Translational and Regulatory Bottlenecks in Drug Development

Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2016. Advancing the Discipline of Regulatory Science for Medical Product Development: An Update on Progress and a Forward-Looking Agenda: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/23438.
×

GARRET FITZGERALD

Professor of Medicine and Pharmacology

University of Pennsylvania

8:50 a.m.

Core Components of Regulatory Science Curriculum

SCOTT STEELE

Director of Government and Academic Research Alliances

Associate Professor of Public Health Sciences

University of Rochester

9:00 a.m.

Data Science Workforce Challenges and Solutions: Northrop Grumman Perspectives

SAM SHEKAR

Chief Medical Officer

Northrop Grumman

9:10 a.m.

Developing the Regulatory Scientist for Medical Product Development: Successful Examples from Pfizer Worldwide R&D

OWEN FIELDS

Vice President, Regulatory Strategy

Pfizer Inc.

9:20 a.m.

Panel Discussion and Audience Q&A (30 mins)

Panelists:

  • Disciplinary Components and Infrastructure Needs speakers (above), and
  • Peter Honig, Senior Vice President and Head of Worldwide Safety and Regulatory, Pfizer Inc.
  • Frank Weichold, Director, Science and Innovation, Office of the Chief Scientist/Office of the Commissioner, U.S. Food and Drug Administration
Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2016. Advancing the Discipline of Regulatory Science for Medical Product Development: An Update on Progress and a Forward-Looking Agenda: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/23438.
×

9:50 a.m.

Day Two Keynote (15 mins; followed by 5 mins of Q&A)

The Future of Regulatory Science at FDA

STEPHEN OSTROFF

Acting Commissioner

U.S. Food and Drug Administration

10:10 a.m.

BREAK (15 mins)

10:25 a.m.

Presentation of Key Themes/Suggested Paths from Session II Panel Moderators and Session Chair (4 speakers; 10 mins each)

Panel Introduction

STEPHEN OSTROFF

Acting Commissioner
U.S. Food and Drug Administration

10:30 a.m.

Session II Moderators

JOHN WAGNER (moderator of Identifying and Developing Meaningful Biomarkers)

Senior Vice President, Head of Clinical and Translational Sciences

Takeda Pharmaceuticals

BRIAN STROM (moderator of Next-Generation Surveillance)

Chancellor of Rutgers Biomedical and Health Sciences

Rutgers, the State University of New Jersey

DARRELL ABERNETHY (moderator of Innovation in Modeling and Integrating Information)

Associate Director for Drug Safety, Office of Clinical Pharmacology

U.S. Food and Drug Administration

Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2016. Advancing the Discipline of Regulatory Science for Medical Product Development: An Update on Progress and a Forward-Looking Agenda: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/23438.
×

11:10 a.m.

Reflecting and Envisioning the Regulatory Science Discipline of 2020: Panel Discussion with Session Chairs, Panel Moderators, Panelists, and Audience

Panel Moderators: Martin Philbert and Alastair Wood (Workshop Co-Chairs)

Panelists:

  • Session II moderators (above), and
  • Eileen Cannon, President, PhRMA Foundation
  • Mark C. Rogers, Board Chairman, Reagan-Udall Foundation

Discussion Questions:

  • What are the three to five priorities that could advance regulatory science domains?
  • Do we have a cohesive approach to advancing the discipline of regulatory science? Are the strategic priorities that have been articulated and adopted by the key players aligned with, and positioned to advance, innovative regulatory science?
  • What investments and incentives are needed to get us there?
  • How to bridge the gap from regulatory science knowledge to regulation and practice?

12:10 p.m.

ADJOURN

Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2016. Advancing the Discipline of Regulatory Science for Medical Product Development: An Update on Progress and a Forward-Looking Agenda: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/23438.
×
Page 49
Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2016. Advancing the Discipline of Regulatory Science for Medical Product Development: An Update on Progress and a Forward-Looking Agenda: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/23438.
×
Page 50
Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2016. Advancing the Discipline of Regulatory Science for Medical Product Development: An Update on Progress and a Forward-Looking Agenda: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/23438.
×
Page 51
Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2016. Advancing the Discipline of Regulatory Science for Medical Product Development: An Update on Progress and a Forward-Looking Agenda: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/23438.
×
Page 52
Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2016. Advancing the Discipline of Regulatory Science for Medical Product Development: An Update on Progress and a Forward-Looking Agenda: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/23438.
×
Page 53
Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2016. Advancing the Discipline of Regulatory Science for Medical Product Development: An Update on Progress and a Forward-Looking Agenda: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/23438.
×
Page 54
Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2016. Advancing the Discipline of Regulatory Science for Medical Product Development: An Update on Progress and a Forward-Looking Agenda: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/23438.
×
Page 55
Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2016. Advancing the Discipline of Regulatory Science for Medical Product Development: An Update on Progress and a Forward-Looking Agenda: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/23438.
×
Page 56
Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2016. Advancing the Discipline of Regulatory Science for Medical Product Development: An Update on Progress and a Forward-Looking Agenda: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/23438.
×
Page 57
Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2016. Advancing the Discipline of Regulatory Science for Medical Product Development: An Update on Progress and a Forward-Looking Agenda: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/23438.
×
Page 58
Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2016. Advancing the Discipline of Regulatory Science for Medical Product Development: An Update on Progress and a Forward-Looking Agenda: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/23438.
×
Page 59
Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2016. Advancing the Discipline of Regulatory Science for Medical Product Development: An Update on Progress and a Forward-Looking Agenda: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/23438.
×
Page 60
Next: Appendix C: Participant Biographies »
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The field of endeavors known as "regulatory science" has grown out of the need to link and integrate knowledge within and among basic science research, clinical research, clinical medicine, and other specific scientific disciplines whose focus, aggregation, and ultimate implementation could inform biomedical product development and regulatory decision making. Substantial efforts have been devoted to defining regulatory science and communicating its value and role across the scientific and regulatory ecosystems. Investments are also being made in technology infrastructure, regulatory systems, and workforce development to support and advance this burgeoning discipline.

In October 2015, the National Academies of Sciences, Engineering, and Medicine held a public workshop to facilitate dialogue among stakeholders about the current state and scope of regulatory science, opportunities to address barriers to the discipline's success, and avenues for fostering collaboration across sectors. Participants explored key needs for strengthening the discipline of regulatory science, including considering what are the core components of regulatory science infrastructure to foster innovation in medical product development. This report summarizes the presentations and discussions from the workshop.

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