
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
Figure out How to Explore Land Close to 5G Pinnacles - 2
Most loved Solace Food: What's Your Definitive Comfortable Dinner? - 3
The architect of Iran’s military survival remains defiant - 4
The Most Astonishing Arising Advancements to Watch - 5
5 things for parents to know about changes to kids vaccine schedule
Gaza amputees struggle to rebuild lives as the enclave faces shortages of prosthetic limbs
Ageless Tastefulness: An Outline of Valuable Gemstones and Adornments
Find the Marvels of the World with These Travels
Virtual Domains d: A Survey of \Inundation and Ongoing interaction Mechanics\ Computer game
Slims down for Maintainable Weight reduction
Why the chemtrail conspiracy theory lingers and grows – and why Tucker Carlson is talking about it
Holiday weather forecast: Where travelers can expect a wintry mix, flooding and record warmth across the U.S.
Novo and Lilly cut prices of weight-loss drugs in China
At least 36 dead in major fire in Hong Kong residential blocks












