
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
Geminid shooting stars: One of 2025's most exciting meteor showers begins tonight - 2
She was the supermodel dubbed 'The Face' in the '80s. Joining OnlyFans in her 60s taught her a lot. - 3
Building an Individual Brand: Illustrations from Forces to be reckoned with - 4
4K televisions for Extreme Film Watching Experience - 5
Banks for High Fixed Store Rates: Augment Your Investment funds
Find the Standards of Powerful Cooperation: Accomplishing Cooperative energy and Coordinated effort
Venice’s newest marvel is a wild, acrobatic dolphin. His refusal to leave puts him in danger
Going with Children: Tips for Tranquil Family Get-aways
A Manual for Nations to Head out To
How food assistance programs can feed families and nourish their dignity
Defence chiefs of Thailand and Cambodia to discuss ceasefire
Relish the World: Notable Caf\u00e9s You Really want to Attempt
Grasping Various Kinds of Local misdemeanors
Find the Specialty of Public Speaking: Drawing in and Convincing Crowds with Certainty













