The FDA awarded $22 million across 15 clinical trial grants to help increase rare disease drug development. Fantastic! But is it enough?

A third of the new grant award funds went to enrolling patients with rare forms of cancer while the rest went to a broad range of diseases. The Orphan Products Clinical Trials Grants Program is definitely needed and I want to commend it and congratulate the grant recipients. However, just last month, JAMA Network published an article stating that "in this analysis of US Securities and Exchange Commission filings for 10 cancer drugs, the median cost of developing a single cancer drug was $648.0 million."  Though this number is being disputed and some expect it could be much higher, it seems the grants are just a drop in the bucket of what is needed to truly encourage really rare disease product development. 

I want to acknowledge the FDA, the grant winners and the other applicants for pursuing rare disease product development, despite the high financial risks. As someone who has heard of the improvements these products make in the lives of patients and families touched by rare disease, I'm convinced we need to do as much as possible to support further rare disease product development.