
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
35 million tons of food go to waste yearly in the US. Experts share tips to help stop it - 2
10 Moving Design Frill for Summer 2023 - 3
Yes, NASA's launching Artemis 2 astronauts to the moon on April Fools' Day. It's not a joke. - 4
China and Pakistan issue five-point peace plan for Middle East - 5
Wegmans recalls mixed nuts over salmonella contamination fears
How to identify animal tracks, burrows and other signs of wildlife in your neighborhood
Germany's Deutsche Welle broadcaster declared 'undesirable' in Russia
Extremely Rare Snub-Nosed Monkey Was Just Born for the First Time Outside of Asia
Flu activity rises sharply across US with 7.5 million cases, CDC data shows
'The Drama' plot twist, explained: What did Zendaya's character do, and what happens to her wedding?
What's inside Mexico's Popocatépetl? Scientists obtain first 3D images of the whole volcano
6 Web-based Course Stages for Successful Learning and Educating
Building Tough Connections: Individual Bits of knowledge on Association
Vote in favor of your Favored sort of footwear













