On July 27, 2022, the National Organization for Rare Disorders (NORD) hosted an online webinar titled “Drug Development for Rare Diseases: A Community Conversation.” This program was designed to help introduce patients to the process of drug development in the realm of rare diseases. A number of panelists participated in the webinar to discuss a variety of related topics, such as the role of patient organizations in drug development, the basic steps of the process from start to finish, and ongoing efforts to accelerate the pace of rare disease drug development.
Panelists for this program included:
- Loretta Byrne, RN, National Project Manager, ResearchMatch.org
- Chandra Clark, Executive Director, The VHL Alliance
- Kerry Jo Lee, MD, Associate Director for Rare Diseases, US Food and Drug Administration
The panelists presented their own information and also answered questions from the audience.
Loretta introduced the program and talked in more detail about the clinical trials and research process. She noted that patients should understand that not all participants in a study will necessarily benefit. In fact, when participating in a trial with an experimental drug, there is no way for patients to know how they will respond.
Chandra shared the patient organization perspective, noting the importance of VHL receiving its first-ever FDA approved treatment recently. This can be a major step for a rare patient community, especially when there are no other effective therapies available. She also elaborated on the role of patient organizations in providing education to patients. In addition, patient organizations can help organize patient registries, which can help compile vital data that can propel research and therapy development. They can also play a critical role in fundraising for scientific research.
Kerry Jo’s role is positioned in the Office for New Drugs within the Center for Drug Evaluation and Research (CDER), which is tasked with helping support the acceleration of therapies for rare diseases. This partially through the operation of the Accelerating Rare Disease Cures Program.
The three panelists then offered their own perspectives on questions from the audience, such as:
- How can patients find out if anyone is researching their disease?
- How can patients know whether a study is right for them?
- What are some aspects of study participation that participants and providers may not anticipate?
- What can patients living with a disease that has no approved treatment do to promote development and research?
- How important are patient registries?
Check out the full webinar here.
