
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
Sally Rooney books may be withdrawn from UK sale over Palestine Action ban, court told - 2
Why Tourists May Want To Reconsider Traveling To This Popular Spot In Italy In 2026 - 3
Knesset sets special panel to fast-track Karhi’s communications reform - 4
Share your number one city visit transport that leaves a mark on the world wake up! - 5
Choosing the Ideal Bed for Quality Rest and Solace
What we know about Renee Nicole Good, the woman who was killed by an ICE officer in Minneapolis
Israel approves 19 new West Bank settlements in major annexation push
Famous Network programs in Europe and America
Avoid Slam: Exploring the Pickup Truck Transformation
Elite Execution Gaming PCs for Gamers
Purdue Pharma's deal means money for some victims, end of Purdue company name. Here's what to know
The gay hockey show no one saw coming — and everyone is suddenly obsessed with
Hungary's 'water guardian' farmers fight back against desertification
Best Amusement Park Bite: What Do You Very much want to Crunch On?













