Better Fda Guidance For Ultra-Rare Diseases Needed

Better FDA Guidance for Ultra-Rare Diseases Needed

FDA Draft Guidance: Rare Disease Clinical Trials

Opportunities to Improve Dose Finding and Optimization for Rare Disease Drug Development Recording

Challenges of the FDA in Regards to Rare Diseases

Janet Woodcock, MD | FDA CDER Re-Organization and Impact on Rare Diseases

EveryLife Foundation for Rare Diseases

Navigating Expanded Access - Rare Disease Drug Development Series Preview

National Organization for Rare Disorders (NORD)

Dr. Janet Maynard Recognizes Successes and Continued Need for Rare Disease Research Grants

U.S. Food and Drug Administration

Natural History Studies for Rare Diseases

U.S. Food and Drug Administration

In the Spotlight: Recent FDA Updates and Guidance for Rare Disease Drug Development

Premier Research

Regulatory Pathway and Applications in Drug Development for Rare Diseases

Critical Path Institute

FDA's Rare Disease Day 2024—Dedicated to Patients and Providers

U.S. Food and Drug Administration

A Look at Rare Disease Listening Sessions with NORD and the FDA

Rare Disease Drug Development

U.S. Government Accountability Office (GAO)

Rare Disease Through a Different Lens

U.S. Food and Drug Administration

FDA Rare Disease Day 2023: “Intersections with Rare Diseases – A patient focused event”

U.S. Food and Drug Administration

Kerry Jo Lee's Work on Rare Diseases

U.S. Food and Drug Administration

How FDA’s Breakthrough Therapy Designation Program Changed the Rare Disease Space

Rare Disease Report

2023 Rare Disease Week - Deep Dive Policy Ask 1: FDA Task Force on Rare Disease Activities

EveryLife Foundation for Rare Diseases

Fireside Chat on Impact on Rare Diseases at FDA

Critical Path Institute

Natural History Studies and Registries in the Development of Rare Disease Treatments - Morning

Reagan-Udall Foundation for the FDA