Appendix A
Workshop Agenda
Innovation in Drug Research and Development for Prevalent Chronic Diseases—A Three-Part Virtual Workshop
February 22, March 1, and March 8, 2021
Part One: February 22, 2021
Opportunities in Discovery and Preclinical Research for
Prevalent Chronic Diseases
11:00 am–3:00 pm ET
11:00 am | Welcome and Opening Remarks |
CARLOS GARNER, Workshop Co-Chair
Vice President, Global Regulatory Affairs Eli Lilly and Company |
|
ANANTHA SHEKHAR, Workshop Co-Chair
Senior Vice Chancellor for Health Sciences and Dean of the School of Medicine University of Pittsburgh |
SESSION I OVERVIEW OF R&D FOR PREVALENT CHRONIC DISEASES
Session Objectives:
- Discuss the unique cross-cutting challenges facing preclinical research for prevalent chronic diseases; and
- Highlight opportunities to overcome those challenges and mobilize the R&D innovation engine.
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1 For more information, see https://www.nationalacademies.org/our-work/innovation-in-drug-research-and-development-for-prevalent-chronic-diseases-a-workshop (accessed October 29, 2021).
11:10 am |
A Patient’s Perspective on Mobilizing the R&D Innovation Engine
RUSS PAULSEN Chief Operating Officer UsAgainstAlzheimer’s |
SESSION II FUNDING AND INVESTMENT DECISION MAKING IN DISCOVERY RESEARCH
Session Objectives:
- Examine common causes of failures in discovery research for prevalent chronic diseases and how failures could be avoided or “go/no-go” decisions could be accelerated in the future;
- Discuss whether investment and cultural incentives are in alignment for spurring the type of R&D that will address unmet need when it comes to prevalent chronic diseases; and
- Consider the factors that determine which research areas key decision makers (e.g., investors, sponsors, researchers) decide to move forward.
11:25 am |
Response and Overview
SUSAN SCHAEFFER, Moderator President and Chief Executive Officer Patients’ Academy for Research Advocacy |
11:35 am |
Funder Perspective
JASON MELLAD Chief Executive Officer and Founder Start Codon |
11:50 am |
Public–Private Partnership Investor Perspective
JOSEPH MENETSKI Associate Vice President of Research Partnerships Foundation for the National Institutes of Health |
12:05 pm | Moderated Panel Discussion and Audience Q&A |
12:30 pm | BREAK (30 minutes) |
SESSION III BIOSPECIMEN COLLECTION AND REGISTRY USE IN DISCOVERY RESEARCH
Session Objectives:
- Consider lessons learned from other disease areas that could have cross-cutting applications for prevalent chronic diseases; and
- Discuss the availability or need for high-quality biospecimen repositories and datasets that represent the patient populations most impacted by prevalent chronic diseases.
1:05 pm |
Introduction and Overview
HOWARD B. ROSEN, Moderator Managing Director, BonVelo Ventures Lecturer, Stanford University |
1:10 pm |
Academic Perspective
ERICA WOODAHL Professor, Department of Biomedical and Pharmaceutical Sciences University of Montana |
SESSION IV NEW TECHNOLOGIES ENABLING DISCOVERY RESEARCH
Session Objectives:
- Discuss the unique cross-cutting challenges in preclinical research for prevalent chronic diseases and consider how new technologies could help researchers overcome these challenges; and
- Consider lessons learned from other disease areas for which new technologies have been a key driver of progress.
1:25 pm |
Academic Discovery Science–Technology Perspective
JOHN NGAI Director BRAIN Initiative, National Institutes of Health |
1:40 pm |
Artificial Intelligence for Discovery Science
ANDREW A. RADIN Chief Executive Officer twoXAR Pharmaceuticals |
1:55 pm |
Regulator Perspective
QI LIU Senior Science Advisor, Office of Clinical Pharmacology & Translational Sciences U.S. Food and Drug Administration |
2:10 pm | Moderated Panel Discussion and Audience Q&A |
2:50 pm |
Closing Remarks
CARLOS GARNER, Workshop Co-Chair Vice President, Global Regulatory Affairs Eli Lilly and Company |
ANANTHA SHEKHAR, Workshop Co-Chair
Senior Vice Chancellor for Health Sciences and Dean of the School of Medicine University of Pittsburgh |
|
3:00 pm | ADJOURN |
Part Two: March 1, 2021
Opportunities in Clinical Research for Prevalent Chronic Diseases
11:00 am–3:00 pm ET
11:00 am |
Welcome and Opening Remarks
CARLOS GARNER, Workshop Co-Chair Vice President, Global Regulatory Affairs Eli Lilly and Company |
ANANTHA SHEKHAR, Workshop Co-Chair
Senior Vice Chancellor for Health Sciences and Dean of the School of Medicine University of Pittsburgh |
SESSION I OVERVIEW OF R&D FOR PREVALENT CHRONIC DISEASES
Session Objectives:
- Discuss the unique cross-cutting challenges facing clinical research for prevalent chronic diseases; and
- Highlight opportunities to overcome those challenges and mobilize the R&D innovation engine.
11:10 am |
A Patient’s Journey
CHRISTIN VEASLEY Co-Founder and Director Chronic Pain Research Alliance |
11:25 am |
Mobilizing the R&D Innovation Engine
CHRONIS MANOLIS Senior Vice President of Pharmacy University of Pittsburgh Medical Center Health Plan |
SESSION II INVESTMENT AND FUNDING DECISIONS IN CLINICAL RESEARCH
Session Objectives:
- Discuss whether investment and cultural incentives are in alignment for spurring the type of R&D that will address unmet need when it comes to prevalent chronic diseases; and
- Consider the factors that determine which clinical programs key decision makers (e.g., investors, sponsors, and researchers) decide to move forward.
11:35 am |
Economics Perspective
KIRSTEN AXELSEN Visiting Fellow American Enterprise Institute |
11:50 am |
A Payer’s Perspective: Pricing and Health Economic Drivers That Incentivize Development Investments
KEN EHLERT Chief Scientific Officer UnitedHealth Group |
12:05 pm | Moderated Panel Discussion and Audience Q&A |
12:25 pm | BREAK (30 minutes) |
SESSION III INNOVATIVE APPROACHES TO EFFICIENT CLINICAL DEVELOPMENT
Session Objectives:
- Discuss the unique cross-cutting challenges in clinical trials for prevalent chronic diseases (e.g., are the regulatory requirements predictable?);
- Brainstorm and prioritize potential strategies to decrease costs and risks for development (i.e., highlight innovative ways to design clinical trials); and
- Discuss ways to meaningfully engage communities and patients in clinical trials.
1:00 pm |
Introduction and Overview
BETTINA DRAKE, Moderator Professor, Washington School of Medicine Associate Director of Community Outreach and Engagement Alvin J. Siteman Cancer Center |
1:05 pm |
Community Health Researcher Perspective
KAREN WINKFIELD Executive Director Meharry-Vanderbilt Alliance |
1:15 pm |
Industry (Regulatory Lead) Perspective
MICHELLE ROHRER Senior Vice President, Global Head of Product Development Regulatory & Policy Roche |
1:30 pm |
Regulatory Perspective
JAMES P. SMITH Deputy Director, Division of Clinical Policy, Office of New Drugs Center for Drug Evaluation and Research U.S. Food and Drug Administration |
SESSION IV NEW TECHNOLOGIES ENABLING INNOVATIVE CLINICAL RESEARCH
Session Objectives:
- Discuss the unique cross-cutting challenges in clinical research for prevalent chronic diseases and consider how new technologies could help researchers overcome these challenges; and
- Consider lessons learned from other disease areas where new technologies have been a key driver of progress.
1:50 pm |
Biotech Perspective
GRACE COLóN Chief Executive Officer InCarda Therapeutics |
2:05 pm |
Regulatory Perspective
ELIZABETH KUNKOSKI Clinical Methodology Team, Office of Medical Policy U.S. Food and Drug Administration |
2:20 pm | Moderated Panel Discussion and Audience Q&A |
2:50 pm |
Closing Remarks
CARLOS GARNER, Workshop Co-Chair Vice President, Global Regulatory Affairs Eli Lilly and Company |
ANANTHA SHEKHAR, Workshop Co-Chair
Senior Vice Chancellor for Health Sciences and Dean of the School of Medicine University of Pittsburgh |
|
3:00 pm | ADJOURN |
Part Three: March 8, 2021
Case Studies in Prevalent Chronic Disease Research
11:00 am–3:00 pm ET
11:00 am |
Welcome and Opening Remarks
CARLOS GARNER, Workshop Co-Chair Vice President, Global Regulatory Affairs Eli Lilly and Company |
ANANTHA SHEKHAR, Workshop Co-Chair
Senior Vice Chancellor for Health Sciences and Dean of the School of Medicine University of Pittsburgh |
SESSION I CASE STUDIES ACROSS THE R&D LIFE CYCLE: MOBILIZING COMMUNITIES AND RESOURCES, ANALYZING PAST SUCCESS
Session Objectives:
- Consider lessons learned in research across the R&D life cycle in several disease areas that could have cross-cutting applications for many prevalent chronic diseases.
- Discuss how research and patient communities have been mobilized to address discovering treatments for some example diseases, and how those approaches led to success.
- Discuss examples of successful development for prevalent chronic disease treatments and what aspects of those approaches led to success.
- Discuss potential strategies to spur drug R&D innovation for prevalent chronic diseases.
11:10 am |
Introduction and Overview
GRACE COLÓN, Moderator Chief Executive Officer InCarda Therapeutics |
11:20 am |
Success Story from Cystic Fibrosis
ROBERT K. COUGHLIN Managing Director, Life Sciences, JLL Former President and Chief Executive Officer, MassBio |
11:35 am |
Digital Innovation for Treating Prevalent Chronic Diseases
RAOLAT ABDULAI Global Clinical Lead, Immunology & Inflammation Sanofi |
11:50 am |
Investing in One Treatment, Applying to Multiple Diseases
ROBERT HEINE Distinguished Eli Lilly Scholar Eli Lilly and Company |
12:05 pm |
Moderated Panel Discussion and Audience Q&A Discussion Questions:
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12:45 pm | BREAK (30 minutes) |
SESSION II RECAP AND POTENTIAL FUTURE STRATEGIES
Session Objectives:
- Reflect on approaches and potential strategies to spur drug R&D innovation for prevalent chronic diseases; and
- Brainstorm potential strategies to spur drug R&D innovation for prevalent chronic diseases (i.e., highlight promising avenues forward that merit additional time, effort, funding, or attention).
1:15 pm |
Summary Presentations by Session Moderators (10 minutes each)
GRACE COLÓN Chief Executive Officer InCarda Therapeutics |
BETTINA DRAKE
Professor, Washington School of Medicine Associate Director of Community Outreach and Engagement Alvin J. Siteman Cancer Center |
|
HOWARD B. ROSEN
Managing Director, BonVelo Ventures Lecturer, Stanford University |
|
SUSAN SCHAEFFER
President and Chief Executive Officer Patients’ Academy for Research Advocacy |
1:55 pm |
Moderated Panel Discussion and Audience Q&A Discussion Questions:
|
2:50 pm |
Closing Remarks
CARLOS GARNER, Workshop Co-Chair Vice President, Global Regulatory Affairs Eli Lilly and Company |
ANANTHA SHEKHAR, Workshop Co-Chair
Senior Vice Chancellor for Health Sciences and Dean of the School of Medicine University of Pittsburgh |
|
3:00 pm | ADJOURN |
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