The U.S. Food and Drug Administration (FDA) announced a new process called “Rare Disease Evidence Principles” (RDEP), under which eligible drugs and biologics for ultra-rare diseases caused by known ...

The FDA already authorizes the use of experimental drugs under what’s called “compassionate use,” for people with no other ...

“Designing treatments unique to individual patients has always been the promised goal of personalized medicine. After 25 years the FDA has, for the first time, outlined a framework to facilitate these ...

FDA approval describes when the Food and Drug Administration (FDA) deems a medical product safe and effective for its intended use based on scientific data and testing. Before people use a medical ...

The FDA proposed new guidelines for approving experimental therapies for rare diseases without large group studies.

Scientific, political pressures colliding at FDA, delaying rare disease treatment for patients who have few or no options, experts said at CNBC Cures Summit.

A Senate hearing probed whether the Food and Drug Administration's process for approving rare-disease therapies is ...

The U.S. Food and Drug Administration issued its first formal guidance on how to design and run clinical trials for psychedelic drugs, a move that directly clears a path for psilocybin, the active ...

Montage is still awaiting FDA approval of its Wireless Healthphone, which is meant to send patient data wirelessly to doctors and other caregivers. Healthphone includes blood glucose monitoring ...

Accelerated review was enabled by the Commissioner’s National Priority Voucher Pilot Program, targeting high unmet need and compressing FDA action to 1–2 months with enhanced reviewer communication.