Materials

Agenda

Meeting Report

List of Participants

Presentations

About the Rare Diseases Forum 1
 
The Rare Diseases Forum aims to maximize efficiency through collaborations and make use of innovation afforded by the 21st Century Cures Act, including totality of evidence, use of novel/innovative analytics, and efficient/rigorous evaluation of novel biotechnology. 

The Rare Diseases Forum was created attending the request of several patient advocacy and state funded networks, with the purpose to facilitate the development of new therapeutics for rare diseases and advance the regulatory science for the diagnosis and treatment of inborn errors of metabolism and other genetically based rare diseases by providing an independent and neutral venue for ongoing multi-stakeholder dialogue.

 The Rare Diseases Forum will convene in-person on a regular basis, with work streams and working group meetings in between the in-person meetings. The hallmark of the Forum is inclusion of all stakeholder groups, including patient and advocacy organizations, academia, federal agencies, industry, professional societies and other relevant entities. The Forum works closely with the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA), providing a trans-Atlantic regulatory scope and breadth to the deliberations. 
 
The Rare Diseases Forum’s unique approach –continuous, ongoing and focused discussions until a resolution is reached –differentiate this program from other efforts.