Health Affairs' Jeff Byers welcomes Deputy Editor Leslie Erdelack back to the pod to break down recent turbulence at the FDA following the departure of Vinay Prasad, whose decisions around rare‑disease gene therapies courted controversy. They explore the fast‑growing rare disease therapeutics market, why traditional clinical trials often don’t work for ultra‑rare genetic conditions, and the new FDA draft guidance for rare disease drug development.

On March 24th, join us for our upcoming Insider exclusive event focusing on pharmacy benefit manager reform with Harvard Medical School's Benjamin Rome.

Become an Insider to get access to this event, trend reports, cheat sheets, and exclusive newsletters.

Related Articles:

• FDA vaccines chief who ran afoul of pharma to depart (Politico)
• Rare Disease Therapeutics Market to Surpass US$ 495.27 Billion by 2033 as Gene Therapy, RNA-based Drugs, and Biologics Transform Patient Care (PR Newswire)
• FDA NEWS RELEASE: FDA Launches Framework for Accelerating Development of Individualized Therapies for Ultra-Rare Diseases
• FDA illuminates new approval pathway for bespoke gene editing therapies (Fierce Biotech)
• One Pivotal Trial, the New Default Option for FDA Approval — Ending the Two-Trial Dogma (The New England Journal of Medicine)

Health Affairs' Jeff Byers welcomes Senior Editor Margaret Winchester to the pod to discuss Trump's recent State of the Union Address and unpacking its health care and health policy talking points, including most-favored nation drug pricing, health savings accounts, and if there's a path toward codification for either.

On March 24th, join us for our upcoming Insider exclusive event focusing on pharmacy benefit manager reform with Harvard Medical School's Benjamin Rome.

Related Articles:

• National Health Care Spending Increased 7.2 Percent In 2024 As Utilization Remained Elevated (Health Affairs)
• Trump’s State of the Union trumpets healthcare greatest hits, but no new policies (Healthcare Dive)

Health Affairs’ Jeff Byers welcomes Brown University's Andrew Ryan to the pod to discuss his recent Forefront article that explores whether value-based payment and managed care can lead to addressing the core drivers of spending.

Related Links:

• Value-Based Payment And Managed Care Will Not Solve The Affordability Crisis (Health Affairs Forefront)
• “All I Do Is Win”: Why Beating Benchmarks Doesn’t Mean That ACOs Are Reducing Costs (Health Affairs Forefront)