Page 73 Cite

Suggested Citation: "Appendix A: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2021. Innovation in Drug Research and Development for Prevalent Chronic Diseases: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/26291.

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

Half of all Americans live with at least one chronic disease, such as heart disease, cancer, stroke, or diabetes. These and other chronic diseases are the leading cause of death and disability in the United States and are a leading driver of health care costs. Yet, investment in the leading causes of death and disability, other than cancer, has not kept pace with the public health need. This virtual public workshop will provide a venue for stakeholders to examine bottlenecks to innovation in drug research and development (R&D) for prevalent chronic diseases and highlight opportunities for spurring drug R&D in this space.

The virtual workshop will be conducted in three parts:

Part One (February 22, 2021) will discuss key opportunities and challenges for increasing investment, broadening biospecimen collection and registry use, and supporting innovative discovery and preclinical research in prevalent chronic diseases.

Page 74 Cite

Suggested Citation: "Appendix A: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2021. Innovation in Drug Research and Development for Prevalent Chronic Diseases: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/26291.

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.

___________________

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).

Page 75 Cite

Suggested Citation: "Appendix A: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2021. Innovation in Drug Research and Development for Prevalent Chronic Diseases: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/26291.

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)

Page 76 Cite

Suggested Citation: "Appendix A: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2021. Innovation in Drug Research and Development for Prevalent Chronic Diseases: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/26291.

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

Page 77 Cite

Suggested Citation: "Appendix A: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2021. Innovation in Drug Research and Development for Prevalent Chronic Diseases: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/26291.

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
2:50 pm	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.

Page 78 Cite

Suggested Citation: "Appendix A: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2021. Innovation in Drug Research and Development for Prevalent Chronic Diseases: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/26291.

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?);

Page 79 Cite

Suggested Citation: "Appendix A: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2021. Innovation in Drug Research and Development for Prevalent Chronic Diseases: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/26291.

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

Page 80 Cite

Suggested Citation: "Appendix A: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2021. Innovation in Drug Research and Development for Prevalent Chronic Diseases: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/26291.

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
2:50 pm	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

Page 81 Cite

Suggested Citation: "Appendix A: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2021. Innovation in Drug Research and Development for Prevalent Chronic Diseases: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/26291.

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: How can patient advocacy affect drug research and development? What can we learn from these examples about psychiatric disorders, cardiology, or other prevalent chronic diseases?

Page 82 Cite

Suggested Citation: "Appendix A: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2021. Innovation in Drug Research and Development for Prevalent Chronic Diseases: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/26291.

How might success be replicated, and what might the investment look like for other prevalent chronic disease areas?
What options exist for trials examining multiple indications?
How might these successes be replicated or apply in the future, and what might the investment look like for other prevalent chronic disease areas?
How have digital advancements changed approaches to developing treatments for prevalent chronic diseases, and how might they affect development in the future?

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

Page 83 Cite

Suggested Citation: "Appendix A: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2021. Innovation in Drug Research and Development for Prevalent Chronic Diseases: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/26291.

1:55 pm	Moderated Panel Discussion and Audience Q&A Discussion Questions: Are there common characteristics of disease areas routinely more affected than others by either discovery and preclinical- or clinical-stage research barriers? What cross-cutting strategies could enable investment? How might overall risk for stakeholders innovating in prevalent chronic disease treatments be lowered, with an eye toward integrating policy with stimulus?
2:50 pm	Closing Remarks CARLOS GARNER, Workshop Co-Chair Vice President, Global Regulatory Affairs Eli Lilly and Company
2:50 pm	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

Page 84 Cite

Suggested Citation: "Appendix A: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2021. Innovation in Drug Research and Development for Prevalent Chronic Diseases: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/26291.