For Industry

Educational Resources for Patients, Advocacy Groups, Research Investigators and Orphan Product Developers

In recognition of Rare Disease Day 2014, FDA is pleased to launch recorded educational topics about FDA and rare disease issues. Our goal is to make these educational resources accessible to a broad audience. While the table below identifies the intended audience for each of the topics, 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)
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  
FDA & NIH Science of Small Clinical Trials Course disclaimer icon 
Numerous Speakers
X X X  
Workshop on Natural History Studies in Rare Diseases: Meeting the Needs of Drug Development and Research disclaimer icon 
Numerous Speakers
X X X  
Sponsor-Investigator Roles and Responsibilities in Clinical Investigations for Drug and Biological Orphan Products
Numerous Modules
  X X  

Pediatric Medical Device Development
Linda C. Ulrich, MD, OOPD (12 mins)

X X    

OOPD Grant Program disclaimer icon
Katherine Needleman, PhD, RAC (20 mins)

X X X  

 

More in Developing Products for Rare Diseases & Conditions

Page Last Updated: 10/27/2015
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