
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
Instructions to Amplify Certifiable Experience While Chasing after an Internet Advertising Degree - 2
Tehran synagogue damaged by missile strike according to Iranian media - 3
Startled Venezuelans express relief but also fear after Maduro arrest - 4
Arctic is again the hottest it's been in 125 years, with record-low sea ice, NOAA report says - 5
Trump administration launches new immigration crackdowns in New Orleans and Minneapolis. Here are all the cities it has targeted so far.
2025 Yachting Editors' Choice Awards: Yachts
This Overlooked Predator Is Running Out of Time—Why Conservationists Are Racing to Save the Striped Hyena
Early diagnosis leads King Charles to scale back cancer treatment in the new year
Merck urges science-led US vaccine schedule after CDC trims childhood vaccine list
AfD faction in western Germany ousts councilman for firebrand speech
Was This Driver Simply Having A great time Or Behaving Like An Ass?
Toyota Reports 2.3% Sales Drop as China Weakness Deepens
Israel faces tough choices over haredi draft exemptions, legal expert warns
The Developing Nearby Food Development and Its Advantages












