
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
Eleven Creations And Developments That Steered History - 2
EU Commission slams Israel's death penalty law for Palestinians - 3
What's Your Number one Superhuman Film Made? - 4
Investigating Design and Individual Style: Track down Your Remarkable Look - 5
The most effective method to Decisively Use Open Record Rewards
How a cocktail of rogue storms and climate chaos unleashed deadly flooding across Asia
Which Film Has the Incomparable Melodic Score?
Early diagnosis leads King Charles to scale back cancer treatment in the new year
10 Demonstrated Systems to Develop Your Internet based Business
Experiences in Natural life Protection: Individual Progressives' Excursions
People who talk with their hands seem more clear and persuasive – new research
What is the 'Survivor 50' Challenge? Hidden immunity idols will be up for grabs in every U.S. state.
6 Financial plan 3D Printers with the Best Worth
Top 10 Moving Style Architects of the Year












