For Industry

Education and Media Resources

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 TopicPatients & Advocacy GroupsResearch InvestigatorsDrug Developers
Recommended Tips for Creating an Orphan Drug Designation Application, and PowerPoint Slides, Webinar Slides Script
Soumya Patel, PharmD, Health Science Administrator, OOPD (41 mins)


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)


Frequently Asked Questions about Expanded Access disclaimer icon 
Andrew Mulberg, MD, CDER, DGEIP (18 mins)


Orphan Disease Research and Development: Focus on Orphan Diseases at FDAdisclaimer icon
Gayatri R. Rao, JD, MD, OOPD
John J. Whyte, MD,MPH, CDER (11 mins)


FDA & NIH Science of Small Clinical Trials Course disclaimer icon 
Numerous Speakers


Links to education and media resources within program areas:

 Orphan Products Clinical Trials Grants Program: Education and Media Resources

 Orphan Products Natural History Grants Program: Education and Media Resources

 Humanitarian Use Device (HUD) Education and Media Resources

 Pediatric Device Consortia Grant Program:  Education and Media Resources



More in Developing Products for Rare Diseases & Conditions

Page Last Updated: 03/15/2019
Note: If you need help accessing information in different file formats, see Instructions for Downloading Viewers and Players.
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