
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
Germany's Merz under fire in Brazil for his comments on Amazon host city of COP30 - 2
Israeli strikes in Gaza kill 25 people, Hamas health authority says - 3
Heart disease risk greater for women with a common condition they may not be aware they have - 4
Clocks to go forward one hour in Europe as summer time starts - 5
The most effective method to Pick a Campervan That Offers Something else for Less
The Delight of Perusing: Book Proposals for Each Class
How will the universe end?
Figure out How to Augment Eco-friendliness in Your Volvo XC40
WHO suspends medical evacuations from Gaza following death of contractor in 'security incident'
A red meat allergy from tick bites is spreading – and the lone star tick isn’t the only alpha-gal carrier to worry about
6 Hints to Upgrade Your Charm, In addition to Your Mentality
Audits of 6 European Busssiness Class Flights
These HGTV stars made a pledge to keep their kids off smartphones. Here's how it's going.
This St Nick Truly Can Advise How To Drink And Hack Your Headache













