The future of medical research, with Donald Berwick, M.D., M.P.P., senior fellow at the Institute for Healthcare Improvement

In the U.S., billions of dollars are spent each year on medical research and drug development, but too often those dollars don't match up with the diseases and patient needs that matter most.

In this episode of Off the Chart: A Business of Medicine Podcast, Donald Berwick, M.D., M.P.P., president emeritus and senior fellow at the Institute for Healthcare Improvement, and former director of the Centers for Medicare & Medicaid Services (CMS) joins Medical Economics Senior Editor Richard Payerchin to discuss a new National Academies of Sciences, Engineering, and Medicine report on aligning therapeutic investments with actual disease burden and unmet needs.

Berwick explains why the U.S. lacks the data needed to track disease burden and research funding, how market forces and policy decisions shape drug development and what role agencies like CMS and the Food and Drug Administration (FDA) can play in closing the gap.

Check out the National Academies article: https://nap.nationalacademies.org/catalog/29157/aligning-investments-in-therapeutic-development-with-therapeutic-need-closing-the

Check out IHI's Turn on the Lights Podcast:
https://www.ihi.org/library/turn-on-the-lights-podcast

And don't miss our recent episodes on building a brand, nutrition, mental health and practice leadership.

Music Credits:
Golden Swing by Fatima Mhedden - stock.adobe.com
Relaxing Lounge by Classy Call me Man - stock.adobe.com
A Textbook Example by Skip Peck - stock.adobe.com

Editor's note: Episode timestamps and transcript produced using AI tools.

Introduction and Podcast Overview (00:00:19) Host introduces the podcast, guests and main topics: medical research alignment and the National Academies report.

Dr. Berwick’s Background (00:01:25) Dr. Berwick shares his education, career and experience in healthcare improvement and policy.

Origins of the National Academies Report (00:02:34) Discussion of how the report on aligning research investments with need was initiated and funded.

Report’s Five Goals and Committee Approach (00:03:29) Overview of the committee’s goals: disease burden, unmet needs, investment mapping, causes of mismatch and recommendations.

Data Gaps and the Need for Transparency (00:05:24) Explains the lack of comprehensive data on disease burden and research investment, and the need for a tracking consortium.

Recommendation: Interagency Consortium (00:06:19) Details on the proposed federal consortium to track unmet needs and investment, and its reporting structure.

Causes of Misalignment: Science and Markets (00:08:41) Explores why research investment doesn’t match disease burden: scientific gaps and market forces.

Role of CMS and Medicare in Drug Development (00:12:32) How CMS coverage, reimbursement and price negotiation can influence research and drug development priorities.

FDA’s Role and Accelerated Approval (00:13:50) Discussion of FDA’s regulatory standards, accelerated approval and the need for confirmatory studies.

Challenges with Confirmatory Studies and FDA Resources (00:17:39) Problems with incomplete confirmatory studies and the need for more FDA resources to ensure drug safety.

Private Sector’s Role and Transparency (00:19:36) How private funders can help realign research, the need for public data and public-private partnerships.

Primary Care and Research Alignment (00:23:39) How research priorities affect primary care, and the importance of physician advocacy for research funding.

Underinvestment in Key Disease Areas (00:26:27) Specific examples of underfunded conditions in public and private sectors, and the need for targeted investment.

Social Determinants of Health and Policy (00:29:44) Emphasis on non-medical factors affecting health, and concern over declining investment in social supports.

Potential of Artificial Intelligence in Research (00:31:06) AI’s promise for identifying research gaps, supporting clinical decisions and accelerating therapeutic development.

Values and Rare Diseases in Research Prioritization (00:32:53) The ethical challenge of balancing investment in common versus rare diseases, and the importance of value judgments.

Message to Primary Care Physicians (00:34:22) Dr. Berwick urges primary care physicians to advocate for science and research funding.

Closing and Podcast Outro (00:35:46) Host wraps up the episode, thanks guests and provides subscription and contact information.