
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
Accor signs agreement to transform El Gouna resort as Sofitel - 2
Find the Insider facts of Viable Advertising: Building a Positive Brand Picture - 3
Greenland’s melting ice and landslide-prone fjords make the oil and minerals Trump is eyeing dangerous to extract - 4
Change Your Physical make-up: Compelling Activities for Muscle Building - 5
6 Pet Sitting Administrations for Your Cherished Pets
19 Strange Motion pictures You Shouldn't Watch With Your Mum
Asia's Noteworthy Destinations: A Voyager's Aide
Surveys of Thrillers That Re-imagined the Class
Israel's ban on unsupervised reporters in Gaza causes strategic harm to legitimacy
Middle East hotels hit pandemic-era lows amid Iran war
Germany unveils rescue plan for struggling chemical sector
Manual for Notorious Fragrances: Immortal Aromas
Lebanon says Israeli strike killed 13 people near Palestinian refugee camp
Russia Fields New Ulan-2 All-Terrain Tactical Vehicle













