Vinay Prasad

Notable Quotes

"Designing treatments unique to individual patients has always been the promised goal of personalized medicine. After 25 years, the FDA has, for the first time, outlined a framework to facilitate these approvals. The Plausible Mechanism framework is a revolutionary advance in regulatory science." — FDA press announcement, February 2026

from FDA creates new approval pathway for gene therapies tailored to individual rare disease patients · Updated Feb 23

"Dropping the two-trial requirement reflects modern advances that have made drug research increasingly precise and scientific." — NEJM, February 2026

from FDA shifts to single-study standard for drug approvals · Updated Feb 20

Stories

FDA creates new approval pathway for gene therapies tailored to individual rare disease patients

Rule Changes

Returned to CBER director role after brief departure in mid-2025

For decades, the Food and Drug Administration (FDA) required the same basic proof for every drug: show it works in a controlled trial with enough patients to be statistically meaningful. That standard made sense for common diseases but created an impossible barrier for conditions affecting a handful of people worldwide. On February 23, 2026, the FDA issued draft guidance creating a fundamentally different standard—called the "plausible mechanism" framework—that would let developers of individualized gene-editing and ribonucleic acid (RNA) therapies win full approval by demonstrating their treatment targets the root genetic cause, successfully edits or engages the target, and improves outcomes compared to the disease's documented natural course.

Updated Feb 23

FDA shifts to single-study standard for drug approvals

Rule Changes

Active, serving in dual roles

For 63 years, the Food and Drug Administration required drugmakers to prove their products worked in at least two rigorous clinical trials before Americans could take them. On February 18, 2026, Commissioner Marty Makary formally ended that standard, announcing that one trial will now be the "default position" for all new drugs—not just treatments for rare and fatal diseases, but medications for common conditions affecting millions of patients. In accompanying articles published in the New England Journal of Medicine and JAMA, Makary and Deputy Commissioner Vinay Prasad emphasized that the single-trial standard does not eliminate evidence requirements; instead, sponsors must provide "confirmative evidence" through mechanistic data, findings from related indications, animal models, real-world evidence, or data from drugs in the same class.

Updated Feb 20

The six-month cure: gene editing goes bespoke

New Capabilities

Co-developing guidance on personalized therapy approvals

In August 2024, KJ Muldoon was born with a death sentence encoded in his DNA—a single broken letter among three billion that left his body unable to process protein. Six months later, he walked out of Children's Hospital of Philadelphia, cured by a gene-editing therapy that didn't exist when he was diagnosed. The treatment was designed, manufactured, approved, and delivered in 180 days.

Updated Jan 7