Former FDA commissioners Robert Califf, Scott Gottlieb and Ned Sharpless criticized current leadership at the Department of Health and Human Services, speaking at the STAT Summit. Their remarks come amid wider debate over how Congress should stabilize the FDA and protect U.S. biotech innovation. The full conversation is available to STAT+ subscribers.
Former FDA commissioners Rob Califf, Scott Gottlieb and Ned Sharpless criticized DOGE at the STAT Summit, calling the Musk-led initiative's cuts of 3,500 workers "one of the biggest disasters in American history" and saying the resulting loss of institutional knowledge will take years to rebuild. Marty Makary resigned as commissioner in May, and White House official Heidi Overton has been nominated to replace him but has not faced a Senate confirmation vote. The ex-officials said acting commissioner Kyle Diamantas and policy lead Grace Graham are restoring rational decision-making, and advised Overton to recruit, support and defend the workforce if confirmed.
The Alliance for Regenerative Medicine (ARM) has published a playbook, building on FDA draft guidance from this summer, showing how gene-editing developers can reuse toxicity, manufacturing and other data across a platform to cut redundant studies and costs. Beam Therapeutics, which helped create the playbook, designs Phase 1 trials as registrational and aims to move from first-in-human studies to FDA approval in four to five years, versus eight to 10 traditionally. ARM also calls for a bifurcated regulatory framework allowing higher-risk, streamlined development for severe rare diseases with no standard of care, while William Blair analysts say more clarity on the plausible mechanism pathway is needed.
A JAMA piece examines how amyloid PET imaging defines amyloid positivity on the Centiloid scale in Alzheimer disease research and clinical care. Amyloid PET has shaped understanding of disease natural history, guided patient selection and served as a trial outcome measure, supporting FDA approval of amyloid-targeting therapies as the first disease-modifying class. Clinically it aids diagnosis in mild cognitive impairment and confirms plaques before treatment.
Karl Deisseroth, Peter Hegemann and Georg Nagel share the 2026 Nobel Prize in Physiology or Medicine for discoveries concerning light-gated ion channels and optogenetics. FDA is due to decide by mid-2027 whether the first optogenetic therapy, a retinal gene therapy, reaches the market. Nanoscope leads direct therapeutic applications of the technology, while MapLight uses it to guide psychiatric drug discovery.
Why it matters: Nobel recognition coincides with the first optogenetic therapy approaching an FDA decision.
The Critical Path Institute's One to Millions Initiative is building a development framework for individualized rare disease therapies, starting with antisense oligonucleotides, and aims to extend it to all genetically defined diseases, including mutation-segmented subtypes of Parkinson's disease. CEO and CSO Klaus Romero said the bottleneck is the absence of a standardized regulatory and development system, and the initiative's first deliverables include a shared safety database meant to reduce repeated preclinical studies. The effort implements FDA's Plausible Mechanism Framework through consortia of companies, academics, patient groups and regulators sharing data.
An analysis of FDA-approved drugs published in Nature Biotechnology on 18 August 2026 finds that the timing and concentration of revenue life cycles vary unevenly across modalities and therapeutic areas. The study draws on drugs approved by the FDA and offers evidence for guiding innovation strategy and policy design in a post-Inflation Reduction Act environment. The authors suggest these distribution patterns can inform decisions by developers and policymakers.