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Orphan Drugs: Developing Treatments for Rare Diseases


According to the FDA, millions of Americans are currently affected by more than 7,000 rare diseases, many of which do not have treatment options or are very costly to treat.

Drugs developed specifically to treat rare diseases – called “orphan drugs” – often remain commercially undeveloped because of the limited potential for companies to profit from making them.

In the United States, the median price for an orphan drug is about $100,000 per year – 20 times the price of the median non-orphan drug. Given the staggering cost, you’d think we’d have a solid handle on whether that money is well-spent. But the truth is that we don’t.

In addition to my work at IU School of Medicine, I also write and host a YouTube video series called Healthcare Triage, which aims to explain complex health topics in ways that are entertaining and easier to understand.

In honor of Rare Disease Day, it’s worth revisiting a series of Healthcare Triage videos we produced last year that focuses on orphan drugs, what’s wrong with the system, and how we might fix it. You can check out the full, four-part series embedded below.

Part 1:

Part 2:

Part 3:

Part 4:

The views expressed in this content represent the perspective and opinions of the author and may or may not represent the position of Indiana University School of Medicine.

Aaron Carroll

Professor of Pediatrics

Dr. Carroll is a Professor of Pediatrics and Associate Dean for Research Mentoring at Indiana University School of Medicine, where he is also Director of the Center for Pediatric and Adolescent Comparative Effectiveness Research. His research focuses on ...