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
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 |
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 |
|
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 |
|
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 |
|
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 |
|
1:45 p.m. |
Panel Discussion and Audience Q&A (30 mins) |
|
Panelists: |
|
|
|
|
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 |
|
3:15 p.m. |
Panel Discussion and Audience Q&A (30 mins) |
|
Panelists: |
|
|
|
|
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 |
|
4:30 p.m. |
Panel Discussion and Audience Q&A (30 mins) |
|
Panelists: |
|
|
|
|
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 |
|
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: |
|
|
|
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 |
|
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: |
|
|
|
|
Discussion Questions: |
|
|
|
|
12:10 p.m. |
ADJOURN |
