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  1. Developing Products for Rare Diseases & Conditions

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 Topic Patients & Advocacy Groups Research Investigators Drug 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)
  X

X

Essentials of Interacting with the FDA
Larry Bauer, Health Scientist, CDER Rare Disease Program(18 mins)
X X X
Regulatory Applications for Products Intended to Treat Rare Diseases
Larissa Lapteva, MD, CDER, Rare Disease Program(38 mins)
  X

X

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 disclaimer icon 
Andrew Mulberg, MD, CDER, DGEIP (18 mins)

X X X

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)

X X X

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

X X X

 

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