
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
Thousands of small fish defy gravity to climb Congo waterfall - 2
'Stranger Things' character guide: The nerds, the newcomers and the rest of the Season 5 cast - 3
Benihana is 60 years old. Gen Z is lining up. - 4
What you need to know about flu treatments as cases spike across the US - 5
Miss 'Stranger Things' already? Here's how you can get your Upside Down fix in 2026 with spinoffs, games and more
NASA satellite gazes into Medusa Pool | Space photo of the day for Dec. 24, 2025
Doomed SpaceX Starlink satellite photographed from orbit
Satellites capture aftermath of Ethiopian volcano's 1st eruption in recorded history (images)
Mexico says a third of 130,000 missing people might be alive, fueling criticism from families
The Most Astonishing Arising Advances to Watch
6 Pet Sitting Administrations for Your Cherished Pets
The Most Moving TED Talks You Want to Watch
Hidden Island Cameras Capture Rare Tasmanian Species for the First Time Ever
Iran war pushes Germany's deficit to 4.2% as growth outlook is cut by 50%












