The resources below are educational recordings about rare disease issues and the FDA. The table below identifies the intended audience for each of the topics; however, these educational resources are meant to serve as a beneficial tool to all rare disease stakeholders.
Additional cross-cutting rare disease topics will continue to be added to this webpage over time.
|Recorded Session Topic||Patients & Advocacy Groups||Research Investigators||Drug Developers||Handout|
|Essentials of Interacting with the FDA|
Larry Bauer, Health Scientist, CDER Rare Disease Program(18 mins)
|Regulatory Applications for Products Intended to Treat Rare Diseases|
Larissa Lapteva, MD, CDER, Rare Disease Program(38 mins)
|Introduction to Patient Focused Drug Development Module 1: Richard Klein, OHCA (13 mins) Module 2: Robert Yetter (10 mins) Module 3: Richard Klein, OHCA (9 mins) Module 4: Theresa Mullin, PhD, CDER (10 mins)||X||X||X|
|Frequently Asked Questions about Expanded Access |
Andrew Mulberg, MD, CDER, DGEIP (18 mins)
|FDA & NIH Science of Small Clinical Trials Course |
|Workshop on Natural History Studies in Rare Diseases: Meeting the Needs of Drug Development and Research |
|Sponsor-Investigator Roles and Responsibilities in Clinical Investigations for Drug and Biological Orphan Products|
Pediatric Medical Device Development
OOPD Grant Program
Gayatri Rao, JD, MD, OOPD; Richard Klein, OHCA; Charles "Tim" Frost, Patient, Alpha-1 Antitrypsin Deficiency; Gayle Greene, MSW, Caregiver, Von Hippel-Lindau Syndrome; Theresa Strong, PhD, Foundation for Prader-Willi Research; Andrew Mulberg, MD, FAAP, CDER/FDA; Jonathan Goldsmith, MD, FACP, CDER-Rare Disease Program/FDA