FDA officials shared their insights on how drug makers can be more efficient when developing treatments for rare diseases, according to comments at the National Organization for Rare Disorders’ (NORD) Summit in Washington, DC.

Despite the growing number of new therapies in development for rare diseases and approved each year, relatively little is known about many rare conditions.

“Unlike common diseases, there’s a lot of information lacking when you want to develop a drug or other intervention for a rare disorder,” noting a lack of information about the natural history of a disease or its impact on patients’ daily lives.


This is particularly important in orphan territory because there are often few patients in total and there are usually very few near the centers that want to do the studies, Because of the challenges inherent in identifying and enrolling patients with rare disorders in studies, having detailed natural history data “can make a tremendous difference in identifying the manifestations you want to try to treat and identifying the patients you should include” in a study.