Don't Do Different Things…Do Things Differently! Drug Development in Rare Diseases

Abstract

Although definitions of rare disease vary, most acknowledge that there are small numbers of affected patients compared with other conditions. Small numbers of patients, overlapping involvement of investigators as researchers and caregivers, as well as close relationships between researchers and manufacturers require a different pattern of drug development. Regulatory guidances for rare diseases are available, as well as ones for specific rare diseases. Maintaining drug supply for rare diseases also demands innovative approaches.

Challenges and Opportunities in Rare Disease Drug Development

“…Last, Speid emphasizes the need to more thoroughly involve the patient and caregivers of patients with rare diseases. In this set of articles we see paths to the future …”

“…In this set of articles we see paths to the future. [6][7][8][9][10] One challenge a drug developer has with rare diseases is that so little is often known about a particular disease. How do you know what endpoint to examine?…”