
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)
最近の投稿
10 Asian Countries Perfect for Solo Female Travelers
New hybrid mpox strain discovered in UK after US reports local spread
Voting begins in Uganda’s presidential election during internet shutdown and polling station delays
Japanese H3 rocket fails during launch of navigation satellite (video)
4K televisions for Extreme Film Watching Experience
Catholic influencer shares death of 5-year-old son from 'severe' flu
Aurora chaser catches a fox basking in the glow of Finland's legendary 'fox fires' (photos)
Best Amusement Park in Asia: Which One Is a Must-Visit
I served on the expert committee that advised the government on new dietary guidelines – most of our recommendations were ignored













