
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
Factbox-China's crewed lunar programme eyes astronaut landing by 2030 - 2
UN mission says no evidence Hezbollah rearming in southern Lebanon - 3
5 State of the art Advancements in Computer generated Simulation - 4
Mojtaba Khamenei unconscious in Qom, not actually running Iran - 5
The next frontier in space is closer than you think – welcome to the world of very low Earth orbit satellites
Dozens injured in Russia after train crashes, overturns
Vote in favor of the bloom plan that adds a bit of excellence to your life!
Italy's Beloved Trevi Fountain Hides A Unique Secret That Can Be Explored Underground
The most effective method to Integrate Compact disc Rates into Your Retirement Arranging
Greenland’s melting ice and landslide-prone fjords make the oil and minerals Trump is eyeing dangerous to extract
Midlife weight gain can start long before menopause – but you can take steps early on to help your body weather the hormonal shift
Middle East hotels hit pandemic-era lows amid Iran war
How to watch 'A Charlie Brown Christmas' for free this weekend
Grasping the Elements of Medical caretaker Pay rates: Factors That Shape Your Pay













