For Industry

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

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 DevelopersHandout
Essentials of Interacting with the FDA
Larry Bauer, Health Scientist, CDER Rare Disease Program(18 mins)
XXX 
Regulatory Applications for Products Intended to Treat Rare Diseases
Larissa Lapteva, MD, CDER, Rare Disease Program(38 mins)
 XX 
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)XXX 
Frequently Asked Questions about Expanded Access disclaimer icon 
Andrew Mulberg, MD, CDER, DGEIP (18 mins)
XXX 
FDA & NIH Science of Small Clinical Trials Course disclaimer icon 
Numerous Speakers
XXX 
Workshop on Natural History Studies in Rare Diseases: Meeting the Needs of Drug Development and Research disclaimer icon 
Numerous Speakers
XXX 
Sponsor-Investigator Roles and Responsibilities in Clinical Investigations for Drug and Biological Orphan Products
Numerous Modules
 XX 

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

XX  

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

XXX 

Video Discussion on Natural History Studies disclaimer icon(58 mins)

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

XXX 

 

 

More in Developing Products for Rare Diseases & Conditions

Page Last Updated: 07/29/2016
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