Real-World Evidence Generation and Evaluation of Therapeutics: Proceedings of a Workshop (2017)
Chapter: Appendix B: Workshop Agenda
Appendix B
Workshop Agenda
REAL-WORLD EVIDENCE GENERATION AND EVALUATION OF THERAPEUTICS: A WORKSHOP
October 19, 2016
National Academy of Sciences Building, Room 120
2101 Constitution Avenue, NW, Washington, DC 20418
BACKGROUND AND WORKSHOP OBJECTIVES
The traditional process for evaluating new therapeutics does not produce the evidence that patients, clinicians, and payers need for real-world decisions. The volume and complexity of information about individual patients is greatly increasing with use of electronic records and personal devices. Possibilities for medical product development in the context of this wealth of real-world data are great, ranging from the ability to determine both large-scale and patient-specific effects of treatments to assessing how therapeutics affect patients’ lives through measurement of lifestyle changes. However, mechanisms to facilitate efficient use of real-world data to meet the decision-making needs of myriad stakeholders have not been established. An ad hoc committee will plan and conduct a 1-day public workshop that will examine opportunities and challenges for incorporating real-world evidence into evaluation of medical products.
Subject-matter experts will be invited to participate in the workshop through presentations and discussions that will consider
- Quality of data from real-world sources, including
- Relevance and validity of different sources of real-world data (e.g., user collected, practice based) in the context of different clinical/scientific questions; and
- Strengths and limitations of different data sources at different stages of treatment development and the licensing process.
- Methodologies and best practices for high-quality, real-world evidence generation and application, including
- Innovations in clinical trial design to maximize value of information for the full range of stakeholders;
- Considerations of how evidence generation from existing studies could potentially inform the design of future clinical trials and amplify understanding of product efficacy;
- Discussion of how shared goals of payers and regulators can better align evidence generation processes used for regulatory evaluation and decisions on use by payers; and
- Re-evaluation of traditional distinctions between goals and methods of preapproval and postapproval research.
- Other novel methodologies and approaches to improve development and evaluation of products using real-world evidence, including
- Use of Web-based or digital technologies to enhance clinical trial evidence collection and participation; and
- Techniques and case studies for effectively using electronic health record data.
| 8:30 a.m. | SESSION I: BREAKING THE MOLD: STAKEHOLDER PRIORITIES FOR IMPROVING EVIDENCE GENERATION |
Session Objectives:
- Examine different stakeholder evidence needs to support decision making and identify shared goals.
- Discuss priorities for facilitating use of real-world evidence to address stakeholder needs.
- Discuss aligning incentives to maximize generation and sharing of useful evidence.
| 8:30 a.m. | Opening Remarks and Introductions |
|
STEVEN GALSON, Workshop Co-Chair
Senior Vice President for Global Regulatory Affairs and Safety Amgen Inc. |
|
|
GREG SIMON, Workshop Co-Chair
Investigator, Group Health Research Institute Chair, Scientific Advisory Board, Depression and Bipolar Support Alliance |
|
| 8:45 a.m. | Keynote |
|
ROBERT CALIFF Commissioner U.S. Food and Drug Administration |
|
| 9:05 a.m. | Stakeholder Perspectives |
|
JOHN CARROLL Professor of Medicine Co-Medical Director, Cardiac and Vascular Center University of Colorado Hospital |
|
|
JOSEPH CHIN
Deputy Director, Coverage and Analysis Group Centers for Medicare & Medicaid Services |
|
| NAFTALI ZVI FRANKEL Patient and Consumer Advocate |
|
| RHONDA ROBINSON BEALE Chief Medical Officer Blue Cross of Idaho |
|
|
RACHEL SHERMAN
Deputy Commissioner for Medical Products and Tobacco U.S. Food and Drug Administration |
|
|
PATRICK VALLANCE
President, Pharmaceuticals Research and Development GlaxoSmithKline |
| 9:30 a.m. | Moderated Discussion with Session I Speakers and Audience |
| ROBERT CALIFF, Moderator Commissioner U.S. Food and Drug Administration |
|
| 10:45 a.m. | Break |
| 11:00 a.m. | SESSION II: WHAT CAN WE LEARN FROM REAL-WORLD DATA? |
Session Objectives:
- Examine different sources of real-world data (e.g., user collected, practice based) and consider their reliability in the context of different clinical/scientific questions.
- Discuss strengths and limitations of different data sources at different stages of treatment development and licensing process.
| 11:00 a.m. | Background and Session Objectives |
| JOHN HERNANDEZ, Moderator Head of Health Economics, Value and Access Verily Life Sciences |
|
|
NIGAM SHAH, Moderator
Associate Professor of Medicine, Biomedical Information Research Stanford University |
|
| 11:05 a.m. | Sources for and Practical Use of Real-World Data |
| Data Sharing and Linking Records Across Electronic Health Record Vendors | |
|
JON WHITE
Deputy National Coordinator for Health Information Technology Office of the National Coordinator for Health Information Technology |
| PCORnet and Clinical Data Research Networks | |
|
RUSSELL ROTHMAN
Director, Center for Health Services Research Chief, Internal Medicine and Pediatrics Vanderbilt University |
|
| Potential for Data Analytics | |
|
DAVID DORE
Vice President, Epidemiology and Principal Epidemiologist Optum Life Sciences |
|
| Using Data from Activities on Mobile Devices | |
|
LUCA FOSCHINI
Co-Founder and Chief Data Scientist Evidation Health |
|
| 11:45 a.m. | Moderated Discussion with Session II Speakers and Audience |
| 12:30 p.m. | Lunch |
| 1:15 p.m. | SESSION III: THE PROMISE OF REAL-WORLD EVIDENCE: STRATEGIES FOR BUILDING FROM SUCCESSFUL USE CASES |
Session Objectives:
- Discuss examples of successful approaches to generating and incorporating real-world evidence into development and evaluation of medical products.
- Identify opportunities and challenges to scaling up successful practices and adapting them to new purposes.
| 1:15 p.m. | Background and Session Objectives |
|
JESSE BERLIN, Moderator
Vice President and Global Head of Epidemiology Johnson & Johnson |
|
|
CATHY CRITCHLOW, Moderator
Vice President and Head, Center for Observational Research Amgen Inc. |
| 1:20 p.m. | Successful Use Cases of Real-World Evidence |
| Case Study #1: Salford Lung Study | |
|
ANDREW RODDAM
Vice President and Head of Real-World Evidence GlaxoSmithKline |
|
| Case Study #2: Transcatheter Valve Therapy (TVT) Registry | |
|
MICHAEL MACK
Chair, Cardiovascular Service Line Baylor Scott & White Health |
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| Case Study #3: Sentinel Initiative | |
|
LESLEY CURTIS Professor of Medicine Director for Pragmatic Health Services Research Duke Clinical Research Institute |
|
| Case Study #4: Observational Health Data Sciences and Informatics (OHDSI) | |
|
NIGAM SHAH
Associate Professor of Medicine, Biomedical Information Research Stanford University |
|
| 2:20 p.m. | Moderated Discussion with Stakeholder Reaction Panel |
|
MARC BERGER
Vice President, Real-World Data and Analytics Pfizer Inc. |
|
|
LAURA DEMBER
Professor of Medicine Renal, Electrolyte, and Hypertension Division University of Pennsylvania |
|
LOUIS FIORE
Executive Director Massachusetts Veterans Epidemiology Research and Information Center |
|
| RHONDA ROBINSON BEALE Chief Medical Officer Blue Cross of Idaho |
|
|
SEAN TUNIS
Founder, President, and Chief Executive Officer Center for Medical Technology Policy |
|
| 3:30 p.m. | Break |
| 3:45 p.m. | SESSION IV: REAL-WORLD EVIDENCE OF THE FUTURE: POTENTIAL STRATEGIES FOR A WAY FORWARD |
Session Objectives:
- Outline an ideal future state for incorporating real-world evidence into evaluation of medical products.
- Identify short- and long-term next steps at any stage of clinical research to achieve seamless use of real-world evidence.
- Discuss incentives that should be explored.
| 3:45 p.m. | Reflecting on Tactics and Strategies for a Way Forward: Discussion with Workshop Co-Chairs, Session Moderators, Panelists, and Audience |
| MARK MCCLELLAN, Moderator Director Margolis Center for Health Policy Duke University |
|
|
STEVEN GALSON, Workshop Co-Chair
Senior Vice President for Global Regulatory Affairs and Safety Amgen Inc. |
|
GREG SIMON, Workshop Co-Chair
Investigator, Group Health Research Institute Chair, Scientific Advisory Board, Depression and Bipolar Support Alliance |
|
|
JESSE BERLIN
Vice President and Global Head of Epidemiology Johnson & Johnson |
|
|
NAFTALI ZVI FRANKEL
Patient and Consumer Advocate |
|
|
JOHN HERNANDEZ
Head of Health Economics, Value and Access Verily Life Sciences |
|
|
FREDA LEWIS-HALL
Chief Medical Officer and Executive Vice President Pfizer Inc. |
|
|
NIGAM SHAH
Associate Professor of Medicine, Biomedical Information Research Stanford University |
|
|
RACHEL SHERMAN
Deputy Commissioner for Medical Products and Tobacco U.S. Food and Drug Administration |
|
| 4:20 p.m. | Moderated Discussion with Session IV Panel and Audience |
| 5:00 p.m. | Adjourn |
