
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
German gas price bill signed into law, but consumers not impressed - 2
Divorce filings, feuds and legal trouble: The 'Mormon Wives' drama keeps piling up - 3
NASA begins fueling rocket to launch astronauts on the first lunar trip in half a century - 4
Ageless Tastefulness: An Outline of Valuable Gemstones and Adornments - 5
6 Top of the line Lodgings All over The Planet, Which One Do You Concur With
Influencers are selling a delusional fantasy of being postpartum. Why is it so easy to believe?
Find the Standards of Viable Refereeing: Settling Debates with Strategy
Danish warship sunk by famed British admiral discovered after 225 years
Dinosaur collagen used to create one-of-a-kind handbag
NASA's Artemis 2 moon launch seen from space | Space photo of the day for April 2, 2026
'Women on the floor, riddled with bullets': Ex-hostage Rom Braslavski recounts 'horrors' of Oct. 7
They relied on marijuana to get through the day. But then days felt impossible without it
Overlooked infertility care should be part of national health services, says WHO
5 Language Learning Applications













