
By Michael Erman
NEW YORK (Reuters) -The U.S. Food and Drug Administration can approve new personalized treatments for rare and deadly genetic diseases based on data from a handful of patients, two of the agency's top officials said on Wednesday.
FDA Commissioner Marty Makary and Chief Medical and Scientific Officer Vinay Prasad said in an essay published in the New England Journal of Medicine that for certain conditions, companies could rely on appropriately designed studies with small sample sizes rather than randomized trials. They will rely on biological plausibility and clinical improvements in those early patients.
"Current regulations are onerous and unnecessarily demanding," Makary and Prasad wrote. "For patients and families, there is no time to wait."
The new "plausible-mechanism" pathway would allow the agency to grant marketing authorization after manufacturers demonstrate success with several consecutive patients.
Companies that receive these approvals will be required to collect real-world evidence to confirm efficacy continues and to look for safety issues that might arise.
The new approach will prioritize treatments for rare diseases that are fatal or cause severe childhood disability. Common diseases with unmet medical needs may also qualify.
While makers of cell and gene therapies are likely to be significant beneficiaries of the new approval process, Makary and Prasad said that other types of treatments could also receive licensure this way.
"The FDA will work as a partner and guide in ushering these therapies to market," the officials wrote.
(Reporting by Michael ErmanEditing by Bill Berkrot)
LATEST POSTS
- 1
Transcript: NASA's Jared Isaacman on "Face the Nation with Margaret Brennan," April 5, 2026 - 2
French Senate debates social media ban for under-15s - 3
A 'rampaging lion' nebula roars to life in a stunning deep-space photo - 4
Holden Commodore Turbo BT1 Police Interceptor Offered for Sale in Australia - 5
How we came to be: Scientists get first look at the evolution of early complex animals
Italy Brings In New Measures In 2026 To Tackle Overtourism
Plans for ‘stop anywhere’ night buses recommended by government for women’s safety
The pace of hiring just fell to the lowest since 2011, outside of the pandemic
Excited visitors for NASA's moon launch jockey for prime views
Revvity says it will exceed 2025 profit forecast range
'Stranger Things' star debunks claims of 'unseen footage' from Season 5, Volume 2 as backlash intensifies ahead of the series finale
Toilet rats? Washington health officials warn of possible rodents in sewer systems after floods
A Pompeii site reveals the recipe for Roman concrete. It contradicts a famous architect’s writings
Tech for Learning: Online Courses and Instructive Apparatuses












