Patients with rare diseases would be directly affected, as this bill could lead to more specialized treatment options becoming available for their conditions, potentially at lower prices due to increased competition. It might also encourage the development of drugs targeting specific symptoms, age groups, or disease stages within a broader rare condition.

Pharmaceutical companies that develop "orphan drugs" (drugs for rare diseases) would also be affected. Companies that currently hold broad market exclusivity for a rare disease might find that exclusivity narrowed to a specific use, potentially facing more competition. Conversely, companies developing new treatments for specific aspects of a rare disease might find it easier to get their drugs approved, even if another drug already exists for the broader condition.