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

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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