
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
A Manual for the Right SUV for Seniors - 2
Dominating Your Cash: The Fundamental Manual for Overseeing Individual accounting records - 3
IDF finds weapon of slain hostage Capt. Daniel Perez in booby-trapped Gaza compound - 4
Immortal Style: Closet Staples for Each Age - 5
Figure out How to Pick a Crematorium: Key Contemplations.
Will your baby get a hep B vaccine? What RFK panel's ruling means.
Popular Japanese Hotel Chain Now Lets You Show Up With Just Your Passport
Holden Commodore Turbo BT1 Police Interceptor Offered for Sale in Australia
The Developing Nearby Food Development and Its Advantages
These 2 companies are teaming up to offer insurance for space debris strikes on satellites
Limited Rain Chances in Brazil Boost Coffee Prices
'Harry Potter' fans rejoice: HBO releases 1st trailer for new TV series, set to premiere this Christmas
Cruising Solo All over the Planet: An Excursion of Self-Disclosure
Manual for extravagance SUVs for seniors













