Today, the EveryLife Foundation for Rare Diseases in partnership with Biotechnology Innovation Organization (BIO), the Kith Collective, National Health Council (NHC), and the Pharmaceutical Research and Manufacturers of America (PhRMA), introduced a transformational resource, the “Guide to Patient Involvement in Rare Disease Therapy Development.” The Guide is a go-to navigational resource specifically for rare diseases intended to inform the understanding of how patient-focused medical product development and related FDA guidances can be applied to the development of therapeutics for rare diseases.
“Over the past 10 years, there has been growing recognition that early and continuous integration of patient perspectives is essential to enhance the chances of success for the development of new therapies,” said Annie Kennedy, EveryLife Foundation for Rare Diseases Chief of Policy, Advocacy, and Patient Engagement. “The Guide is intended to embolden all stakeholders involved in developing new therapies for rare diseases to better understand how patient perspectives can be elicited and applied to leverage opportunities and mitigate challenges.”
A series of four virtual “PFDD Guidance Compendium” workshops, held between June and October 2021, convened nearly 100 leaders to consider the application of guidances across four lifecycle stages of medical product development for rare diseases. These discussions informed creation of the Guide. FDA leaders from across the agency provided input and feedback at the midpoint of the Workshop Series and on a late-stage draft of the Guide.
The Guide is available on the Foundation’s website along with other resources, including instructions on how to navigate the Guide and background information on how the Guide was developed.
The Foundation will host a webinar on Thursday, January 27th 1:00 p.m. ET – 2:00 p.m. ET for stakeholders to learn more about this new resource. The webinar is free and open to the public.
To register for the webinar and to view and share the “Guide to Patient Involvement in Rare disease Therapy Development,” click here.
