The U.S. Food and Drug Administration (FDA) announced a public meeting and an opportunity for public comment on “Patient Perspectives on the Impact of Rare Diseases: Bridging the Commonalities,” on April 29, 2019.
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 FDA and medical product developers further understand and advance the development of treatments for rare diseases.
With this meeting, the FDA aims 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.
Patient Perspectives on the Impact of Rare Diseases
There are approximately 7,000 rare diseases affecting an estimated 30 million people in the United States, including Alpha-1 Antitrypsin Deficiency (Alpha-1). Each year, the FDA joins the global observance of Rare Disease Day at the end of February, to raise awareness about rare diseases, and this year’s Rare Disease Day theme is Bridging Health and Social Care in recognition that rare diseases deeply impact patients, caregivers and their families, such as Alpha-1.
The Alpha-1 Foundation is committed to finding a cure for Alpha-1 and to improving the lives of those affected by this condition, worldwide, and this open meeting represents a valuable opportunity for Alphas and their caregivers to raise their voice and bring their experiences to the FDA for better outcomes in the development of treatments for rare diseases, including Alpha-1.