
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
Phonetic Associations: A Survey of \Interfacing Worldwide People group\ Language Trade Application - 2
Illegal entries into Germany halve over two years, border police say - 3
Why don’t humans have hair all over their bodies? A biologist explains our lack of fur - 4
'Wow!' The eye surgery marathon that restored sight for some South Africans - 5
Key Little Things That Advantage Old People
Instructions to Expand Your Smash 1500's Presentation: Tips and Deceives
As cases of a rare, deadly infection rise, doctors worry fewer teens will get vaccinated
Vote In favor of Your Favored Kind Of Vegetable
Sa'ar warns German delegation: 'A Palestinian state would be a Hamas terror state'
Fundamental Home Exercise center Hardware: Amplify Your Exercises
US measles cases surpass 2,000, highest in 30 years: CDC
Twelve injured near Beit Shemesh, reports of shrapnel impact in Eilat as Iran targets Israel
Asia's Noteworthy Destinations: A Voyager's Aide
7 Strange Apparatuses to Make Your Party Stick Out!













