
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
Dirty soda started as a Mormon alternative to booze. Now it's everywhere. - 2
Figure out How to Acquire Rewarding Open Record Rewards - 3
Internet Bookkeeping Programming for Consultants - 4
What we know about the 'Stranger Things' spinoff — plus the one cast member who guessed it correctly - 5
Nature carves colossal snowman in Siberia | Space photo of the day for Jan. 2, 2026
Watch interstellar comet 3I/ATLAS make its closest approach to Earth in free livestream on Dec. 18
Improving as a Pioneer: Examples from My Vocation
Figure out How to Forestall Tooth Staining
Figuring out the Business venture Code: The Response to Building an Effective Startup
15 Outrageous Cosplay Outfits That Will Blow You Away
Indian Health Service is digging out of decades-old construction backlog for medical buildings
Heart disease risk greater for women with a common condition they may not be aware they have
Delta flight bound for Atlanta makes emergency landing after engine issue
6 Well known Nissan Vehicles in the U.S.












