On October 14, the U.S. Food and Drug Administration (FDA) launched its Rare Disease Framework Initiative to rethink how drugs and biologics are developed for rare diseases through what the agency describes as a “fit-for-purpose” approach. Read the FDA announcement.
In developing the framework, the FDA will consider the nuances of individual diseases, patient needs, the type of treatment being studied and the evidence that can realistically be gathered in small patient populations. The agency also plans to incorporate patient perspectives early and at defined points in drug development and review.
The FDA’s emphasis on bringing patient perspectives into drug development early aligns with the National Scleroderma Foundation’s Patients as Partners approach. At the Foundation’s inaugural Patient Advocacy Summit in Bethesda, Foundation staff shared insights from the program and early findings from the Patient Needs Assessment. They emphasized that patients are essential partners throughout clinical research and should help shape decisions early, including how trials are designed and what outcomes they measure. Read more about the Patient Advocaty Summit.
Mary Wheatley, IOM, CAE, CEO of the National Scleroderma Foundation, sees the initiative as an encouraging step. The FDA’s emphasis on early patient input echoes conversations at the summit about how to study a disease that can affect two people with the same diagnosis very differently. People living with scleroderma can experience different symptoms, rates of progression and treatment needs.
Advancing treatment for a disease as complex as scleroderma requires listening to the people who live with it. Their perspectives can help researchers decide what outcomes matter to patients before a trial is designed.
Learn how to get involved in scleroderma advocacy through the Foundation’s Advocacy page.