
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
Which restaurants and fast food chains will be open on Christmas Eve and Christmas Day? - 2
Vacuum Cleaners That Are Not difficult To Use For Home - 3
Manual for Tracking down Spending plan Agreeable Travel Objections - 4
Bondi Beach survivor criticizes police for inaction during terror attack - 5
Moving Wedding Objections for Paramount Functions
Former GLP-1 users regain lost weight after about 18 months, study says
Geminid meteors streak under green sky | Space photo of the day for Dec. 19, 2025
Venezuelans in Madrid celebrate Maduro's capture
The Best Web-based Courses for Ability Advancement
A Gastronomic Experience in Healthy Enjoyments: A Survey of \Nutritious and Tasty\ Solid Cooking Recipe Book
Nuno Loureiro, MIT physicist, fatally shot at home; police investigate
Ghassan Al-Duhaini to replace Abu Shabab as Popular Forces leader in Gaza
New movies to watch this weekend: See 'The Running Man' in theaters, rent 'One Battle After Another,' stream 'Nobody 2' on Peacock
Judge sets $60K bond for Florida congresswoman accused of stealing $5M in COVID-19 funds












