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Improved Access to Rare Disease Drugs: New Pricing Rules.

This act aims to improve access to drugs for rare diseases by changing how they are considered in the drug price negotiation program. This could encourage pharmaceutical companies to develop more treatments for conditions affecting a small number of people, potentially leading to more treatment options for patients with rare diseases.
Key points
Drugs for rare diseases (orphan drugs) will have modified rules for price negotiations, potentially affecting their availability.
The act clarifies that drugs for one or more rare diseases will be treated specially to support their development.
The period a drug was designated for a rare disease will not count towards the time after which it becomes subject to price negotiations, giving companies more time to recoup research costs.
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Introduced
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Additional Information
Print number: 119_S_1862
Sponsor: Sen. Barrasso, John [R-WY]
Process start date: 2025-05-22