
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
Top Music and Dance Celebration: Which One Gets You Going? - 2
7 Countries Where Newcomers Feel Most Welcome, and 3 Where They Often Don’t - 3
This St Nick Truly Can Advise How To Drink And Hack Your Headache - 4
Over 60 local leaders push Netanyahu to halt haredi draft bill, warn of social rift - 5
Andrew McCarthy's awe-inspiring image of a skydiver in front of the sun
Irish defence minister's trip to Lebanon cancelled
Arctic sea ice hits lowest winter level as heat records are shattered worldwide
Euclid space telescope sees gorgeous cosmic cloud | Space photo of the day for Nov. 18, 2025
Woman charged in unprovoked stabbing of tourist changing baby's diaper in Macy’s Herald Square store
Spain breaks jobs record with 22 million Social Security contributors
Freed whale gets stranded again off German coast
Avoid This Common Mistake When Planning Sightseeing Activities For Your Trip To Italy
Most loved Caf\u00e9 Chain: Where Do You Get Your Caffeine Fix
Paraplegic engineer becomes the first wheelchair user to blast into space













