The Doctor's Lounge

Hope vs. Power: Gene Therapy, FDA Shake-Up, and Truth in Medicine

Rojas Media Season 1 Episode 9

Send us a text

Original Substack Release: Aug 23, 2025

🎯 Why Listen

When hope collides with billion-dollar incentives, who protects patients? We unpack Vinay Prasad’s FDA resignation, Sarepta’s $3.2M Duchenne gene therapy, and how accelerated approvals can leave families without the long-term data they deserve.


👥 Co-Hosts

  • Dutch Rojas – Founder, Bliksem Health
  • Anthony DiGiorgio, DO, MHA – Neurosurgeon, UCSF; health policy researcher
  • Anish Koka, MD – Cardiologist, Philadelphia; healthcare policy commentator
  • Dan Choi, MD, FAAOS – Orthopedic spine surgeon, Long Island; healthcare advocate and social media voice
  • Sanat Dixit, MD, FACS – Neurosurgeon, Huntsville, AL; Faculty, Vanderbilt University; healthcare entrepreneur

📌 Episode Overview

  • Vinay Prasad’s exit from FDA/CBER and what it signals about evidence, safety, and politics.
  • Duchenne muscular dystrophy, Sarepta’s one-shot gene therapy, and deaths linked to vector toxicity.
  • The promise and peril of accelerated approvals—and why confirmatory trials so often stall.
  • Left vs. right myths about healthcare; markets, incentives, and why “healthcare is a right” can fail in practice.
  • Quick hits: public-good economics in medicine, decentralization vs. central control, and where AI may actually help (RCM, workflow).

💬 Notable Quotes

  • These trials are never gonna get done. It’s a game—with patients as pawns.
  • The FDA is the Midas touch: approval turns hope into revenue—fast.
  • You don’t want a world where healthcare is non-excludable and non-rivalrous.
  • If healthcare is ‘first, do no harm,’ who decides what harm looks like—and who pays the price?


📚 What You’ll Learn

  • How accelerated approval works—and where it breaks.
  • Why safety signals (e.g., vector-related liver failure) matter more than press releases.
  • The difference between hope, evidence, and incentives in rare disease.
  • How political tribes systematically misunderstand healthcare mechanics.
  • Practical places AI can improve care operations today.


The Episode (Timestamps)

  • 00:00–01:10 Welcome back & cadence update (new Sunday drops)
  • 01:10–07:40 Vinay Prasad resigns from FDA/CBER—context and stakes
  • 07:40–13:30 Duchenne 101, dystrophin biology, trial endpoints vs. real outcomes
  • 13:30–17:30 Accelerated approval promises vs. missing confirmatory trials
  • 17:30–22:30 Safety signals: vector immunogenicity, reported deaths, what pulls look like
  • 22:30–28:30 Media/influencer pressure and the politics of “right to try”
  • 28:30–41:30 Is healthcare a public good? Scarcity, incentives, and economic reality
  • 41:30–50:30 Government, morality, and why central planning fails patients
  • 50:30–59:30 AI, agents, and near-term wins in physician workflows
  • 59:30–End What we need next from policymakers—and from clinicians

🔗 Connect with the Hosts:

• Dutch Rojas on X

• Dr. Anthony DiGiorgio on X

• Dr. Anish Koka on X

• Dr. Dan Choi on X

Dr. Sanat Dixit on X