
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
Mystery foot suggests a second early human relative lived alongside Lucy - 2
The cheap health insurance promoted by Trump officials has this catch - 3
Top Frozen yogurt Flavor: Cast Your Vote! - 4
Linda Hamilton, 69, says she doesn't want to 'chase longevity' - 5
Report in relation to renaming Herzog Park set to be withdrawn
The Way to Monetary Health: Individual budget Change
Iran’s Crown Prince Reza Pahlavi backs protests: Join your fellow citizens in the streets
Antivirus Programming for Exhaustive Security
People Are Sharing The One Picture They Can't See Without Laughing, And It's The Comedy Spiral You Need Today
What is colostrum? And should you be taking it?
Independence from the rat race: How to Save and Contribute Shrewdly
Tesla plans to expand production at German car plant
Grasping the Commencement of Criminal Cases: An Extensive Outline
I read 115 books this year. 'Wuthering Heights,' 'Heart the Lover' and 'The House of My Mother' were among my 10 favorites.













