
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
Oil rises above $115 and Asia stocks slide as Iran war escalates - 2
Cyclone Narelle turns Australian skies blood red in ‘apocalyptic’ scenes - 3
The most effective method to Keep up with Proficient Handshakes in a Computerized World - 4
Heavy rain, floods kill at least 45 people in Afghanistan, Pakistan - 5
Paratroopers kill terrorist who threw rocks at Israeli citizens, soldiers near Ofra in West Bank
Which Kind of Pet Makes the Incomparable Buddy?
Why is the Artemis 2 rocket launch different from all other rocket launches?
My prescription costs what?! Pharmacists offer tips that could reduce your out-of-pocket drug costs
Instructions to Adjust Work, Life, and Seeking after a Web based Advertising Degree
The most effective method to Comprehend the Variables Affecting Medical attendant Pay rates
7 Powerful Techniques to Boost Efficiency with Your Cell Phone: A Far reaching Guide
Undeniably popular Historical centers: Where Craftsmanship and History Meet
Man triggers smoke bomb during failed crypto robbery
A definitive Cruiser Standoff: Decision in favor of Your #1 Ride












