Patients highlight commonalities in rare diseases that could streamline drug development

Rare disease patients, advocates and caregivers who spoke at an FDA workshop on Monday highlighted multiple common symptoms across various diseases that could be used to streamline drug development. Attendees also cited the need for patients to access their data as participants in clinical trials and for coordination among patient groups and companies on the development of registries.

The comments came at an FDA workshop designed to identify commonalities among different rare diseases. "Each of you here today has unique challenges with small numbers affected and heterogeneous etiologies and manifestations of those diseases, but it's important for us to assess our commonalities to synergize product development," said Janet Maynard, director of the Office of Orphan Drugs Development at FDA...