FDA & Rare Disease Drugs: Why Policy and Politics Are Heating Up

FDA & Rare Disease Drugs: Why Policy and Politics Are Heating Up

From Health Affairs This Week by Health Affairs

March 13, 2026 · 14 min · Episode 239

About this episode

The episode discusses recent developments at the FDA regarding rare disease drugs and the implications of new policies and market trends.

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market, why traditional clinical trials often don’t work for ultra‑rare genetic conditions, and the new FDA draft guidance for rare disease drug development.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…

People in this episode

Host: Jeff Byers

Guest: Leslie Erdelack

Topics covered

  • FDA
  • rare disease drugs
  • gene therapies
  • clinical trials
  • health policy
  • pharmacy benefit manager reform

Keywords

  • FDA
  • rare disease
  • gene therapy
  • clinical trials
  • drug development
  • health policy
  • pharmacy benefit managers

Mentioned in this episode

Organizations: FDA, Harvard Medical School, Politico, PR Newswire, Fierce Biotech, The New England Journal of Medicine

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