JD Supra

FDA Announces Rare Disease Evidence Principles to Accelerate Treatments for Ultra-Rare Genetic Diseases

The U.S. Food and Drug Administration (FDA) announced Rare Disease Evidence Principles (RDEP) to allow for faster approval of treatments for ultra-rare genetic diseases. A product is eligible if it ...
Pharm Exec

How are the Rare Disease Evidence Principles Reshaping Evidentiary Expectations?

RDEP is a sign that regulators acknowledge that rare disease is a unique space that requires special attention. The rare disease space has always been uniquely difficult for drug development, but ...
JD Supra

FDA’s Rare Disease Evidence Principles Process Provides Guidance for Applications Supported by a Single Pivotal Trial

On September 3, 2025, the US Food and Drug Administration (FDA) announced a new process to support the development of drugs intended to treat rare genetic diseases. The Rare Disease Evidence ...
STAT

The Morris ALS Principles: A model for empowering all disease communities

The FDA’s decision to delay its highly anticipated review of AMX0035, a new drug for treating amyotrophic lateral sclerosis, until September highlights an ongoing rift between the needs of people ...
HealthLeaders Media

Follow 7 Principles for Disease and Risk Factor Screening in Emergency Departments

Emergency department screening is an opportunity to boost individual and population health. Health systems and hospitals should follow seven principles to conduct screening for disease or health risk ...