U.S. policy favors orphans for cancer, well-defined diseases

The U.S. orphan drug landscape is increasingly becoming a tale of two markets. On one side, the government continues to incentivize development of therapies for cancer and other rare diseases with well-characterized biology and clear regulatory pathways. On the other, it is looking away as the space for developing treatments for ultra-rare, heterogeneous, and poorly understood conditions contracts.

Congress recently broadened the orphan drug exemption from Medicare drug price negotiations, a step that will cause biopharma companies to seek multiple orphan indications and to prioritize approvals of orphan indications for drugs that are likely to have a mix of orphan and non-orphan uses. ...