Experts say expanding beyond rare disease markets can make ultra-rare therapies financially viable

The piece highlights that investment in ultra‑rare disease treatments remains limited, and experts argue the answer is not to focus solely on the rare condition itself.

They propose that drugmakers improve the economics by looking outside the narrow patient pool, such as linking development to broader indications or using shared platform technologies to spread costs.

If adopted, this approach could make ultra‑rare programs more attractive to investors and increase the pipeline of therapies for patients with the most uncommon disorders.