- CONTACT US
- AFS
- Business
- Bussiness
- Car
- Career
- Celebrity
- Digital Products
- Education
- Entertainment
- Fashion
- Film
- Food
- Fun
- Games
- General Health
- Health
- Health Awareness
- Healthy
- Healthy Lifestyle
- History Facts
- Household Appliances
- Internet
- Investment
- Law
- Lifestyle
- Loans&Mortgages
- Luxury Life Style
- movie
- Music
- Nature
- News
- Opinion
- Pet
- Plant
- Politics
- Recommends
- Science
- Self-care
- services
- Smart Phone
- Sports
- Style
- Technology
- tire
- Travel
- US
- World
- エンタメ
- スポーツ
- 科学
- 経済

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
Setbacks in Texas and elsewhere put Republicans' redistricting hopes in doubt as key deadlines loom - 2
Charli xcx teases new film ‘The Moment’: What to know about the A24 movie - 3
Astonishing interstellar comet captured in new images by NASA Mars missions - 4
Reporter's notebook: Inside the IDF’s ‘Hamas Village,’ and how Israel is rewriting urban warfare - 5
Israel's haredi draft crisis: Court ruling and political stalemate reach breaking point
Overhaul Your Rest: Tips for a Serene Evening
The most effective method to Safeguard Your Teeth from Acidic Food varieties and Beverages
Dental Embed Developments: Upsetting Current Dentistry
5 Wellbeing Applications Assist You With remaining Fit
Find Your Internal Culinary expert: Cooking Strategies and Recipes
Which salad do you believe is a definitive group pleaser? Vote!
The most effective method to Settle on Informed Conclusions about Senior Insuranc.
A definitive Manual for Good dieting on a Tight spending plan
6 Methods for further developing Rest Quality













