Patients, Politics, and the FDA: Who Decides What’s Enough Evidence?

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🎯 Why Listen

Dive into one of the most consequential debates in medicine: how should the FDA balance speed, safety, and patient need in approving new drugs and gene therapies? Dr. Bob Goldberg, joins the Doctors Lounge to unpack regulatory science, RCTs, accelerated approvals, and the controversies around muscular dystrophy treatments.

👥 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

✨ Special Guest

Bob Goldberg, PhD – Co-Founder & Vice President, Center for Medicine in the Public Interest

📌 Episode Overview

This episode explores the tension between innovation and evidence in drug development. Dr. Goldberg shares his decades of work in FDA reform and patient-centered drug development. The discussion spans accelerated approvals, Sarepta’s muscular dystrophy drugs, RCTs vs. real-world evidence, and the risks of leaning too far toward either bureaucratic caution or patient desperation.

💬 Notable Quotes

“Drug development is harder than hitting a fastball Sandy Koufax would throw.” – Bob Goldberg

“Randomization is a very limited tool… there are many more ways to generate meaningful evidence.” – Bob Goldberg

“If we don’t have some dead ends in accelerated approvals, then we’re not doing enough of them.” – Janet Woodcock (quoted by co-hosts)

“Patients don’t care about motor function endpoints—they care about breathing, dignity, and independence.” – Bob Goldberg

📚 What You’ll Learn

• Why RCTs are not always the gold standard in rare diseases
• The backstory of Sarepta’s muscular dystrophy approvals
• How patient-centered drug development reshapes trial endpoints
• The role of politics and policy in FDA decision-making
• Barriers that slow innovation, from trial costs to regulatory red tape
• How AI and adaptive trial design could accelerate safer drug development

⏱ The Episode (Timestamps)

• 00:00 – Regulatory science, biomarkers, and the challenge of precision medicine
• 01:00 – Introduction of guest Dr. Bob Goldberg
• 02:00 – Background: Center for Medicine in the Public Interest & FDA reform
• 06:00 – The limits of randomization and the rise of precision endpoints
• 10:00 – Gene therapy for muscular dystrophy: risks, deaths, and FDA holds
• 14:00 – Critiques of Vinay Prasad and the politics of FDA appointments
• 20:00 – Exondys 51, Sarepta, and the accelerated approval debate
• 30:00 – Historical parallels: AIDS drug approvals and patient advocacy
• 40:00 – Patient-centered drug development and real-world data
• 50:00 – Trial design inefficiencies, AI, and regulatory bottlenecks
• 57:00 – Pharma lobbying, PBMs, and the rebate system
• 01:04:00 – Public perception, patient voice, and FDA trust
• 01:09:00 – Closing though

🔗 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