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Jul. 7, 2022

TOPIC: Get Involved, RDCA-DAP

A Conversation on Drug Development for Rare Diseases

Posted by Mary Dunkle

Explore the five steps in drug development and discover new opportunities to get involved

More than 95% of rare disease patients lack an FDA-approved treatment for their condition. However, today there are more opportunities than ever before for patients, caregivers and patient organizations to play a key role in advancing R&D on rare diseases. This webinar will explore the basics of research and product development for rare diseases to help patients and patient organizations gain awareness of how they may be able to advance the process.

An upcoming webinar is the latest in a series of resources NORD is offering to help patients, caregivers, and leaders of patient organizations better understand drug development for rare diseases. The goal is to share basic information about research, the drug development process, and how patients and patient organizations can advance development of innovative therapies.

On Wednesday, July 27, from 1 to 2pm ET, NORD will host “Drug Development for Rare Diseases: A Community Conversation.” Expert panelists include:

  • Chandra Clark, Executive Director of The VHL Alliance, an organization advancing research on von Hippel Lindau disease;
  • Kerry Jo Lee, MD, Associate Director of Rare Diseases, Office of New Drugs, FDA CDER, who will provide basic information about drug development and the role of FDA; and
  • Loretta Byrne, RN, National Project Manager for ResearchMatch, an NIH-funded initiative to connect patients with researchers.

Participants will have an opportunity to submit questions before and during the webinar. While the event will be free and open to the entire rare community, the content is designed for patients, parents, caregivers, and patient advocates.

NORD has created resources about the drug development process, including a set of three short, animated videos and a downloadable infographic: