
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
Ancient meditation practices find new life in modern religious communities across America - 2
Tech giants accused of not complying with Australian social media ban - 3
'I carried my wife's body for an hour and a half' - BBC hears stories of protesters killed in Iran - 4
Astrophotographer captures rare footage of the Hubble Telescope crossing the sun (video) - 5
Rediscovering Experience Through Excursions: Individual Travel Stories
A Time of Careful Eating: Individual Tests in Nourishment
10 Work Valuable chances to Assist with supporting Your Advanced degree
NASA unveils close-up pictures of the comet popping by from another star
CDC vaccine panel votes to remove universal hepatitis B birth dose recommendation
8 Espresso Bean Starting points All over the Planet
Flights canceled at 40 U.S. airports: Follow live updates as FAA cuts to air traffic take effect amid government shutdown
Pocket-Accommodating Jeep Wrangler Buying Guide for Seniors
Uranus's small moons are dark, red, and water-poor
As her kidneys fail and time runs short, this activist fights to decriminalize euthanasia in Mexico













