Patient Perspectives on the Impact of Rare Diseases: Bridging the Commonalities; Public Meeting; Request for Comments

Riepilogo

The Food and Drug Administration (FDA or the Agency) is announcing a public meeting and an opportunity for public comment on "Patient Perspectives on the Impact of Rare Diseases: Bridging the Commonalities." This public meeting is intended to obtain patients' and caregivers' perspectives on impacts of rare diseases on daily life and to assess commonalities that may help the Agency and medical product developers further understand and advance the development of treatments for rare diseases. Developing a treatment for a rare disease can present unique challenges, such as the small number of individuals affected and heterogenous etiologies and manifestations. While the differences between rare diseases are critically important, it is also important to assess commonalities to synergize product development in rare diseases. The goal of this meeting is to identify common issues and symptoms in rare diseases to help advance medical product development, potentially through the creation of novel endpoints or trial designs that focus on commonalities across a variety of rare diseases.