The book surveys a pivotal debate about how to spur drug development for rare diseases while keeping medicines affordable.

The hearing examines H.R. 4160, a bill to modify the Orphan Drug Act. It discusses how the current law grants seven years of market exclusivity and how the proposed changes would set a four-year period with an additional three years for drugs with limited commercial value. The goal is to protect true orphan drugs while avoiding excessive pricing on blockbuster products.

Support comes from key stakeholders, including BIO, NORD, and the administration. The discussion weighs incentives, pricing, and the balance between encouraging research and ensuring access for patients who need these treatments.

• How the Orphan Drug Act has shaped drug designations and approvals.


• Arguments for and against long exclusivity periods and the proposed four-year compromise.


• What influences pricing, competition, and patient access in the orphan drug space.


• Which groups and industries support the changes and why.

Ideal for readers interested in health policy, pharmaceutical regulation, and the politics of rare-disease treatment.

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