
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
See the first close-up photos of the moon from NASA's Artemis II mission - 2
Surging measles cases are 'fire alarm' warning that other diseases could be next - 3
Surprise! Saturn's huge moon Titan may not have a buried ocean after all - 4
The 15 Most Powerful Forerunners in Business - 5
Germany to create restitution council to return colonia-era acquired cultural artefacts
Cheetah, Hammerhead Shark, and 38 Other Animals in Danger of Extinction Receive New International Protections from U.N.
Middle East hotels hit pandemic-era lows amid Iran war
The Most Compelling Books of the 10 years
Artemis 2 astronauts — now halfway to the moon — report 'burning smell' from toilet, but everything's fine
10 Delectable Specialty Mixed drinks
'We were genuinely astonished': This moss survived 9 months outside the International Space Station and could still grow on Earth
Why the weirdest sea level changes on Earth are happening off the coast of Japan
The most effective method to Pick the Right Material Organization: Your Definitive Aide
Understanding climate change in America: Skepticism, dogmatism and personal experience













