ARTICLE | Politics, Policy & Law

IRA’s unintended consequences include harm to rare disease patients

Advocates, biopharma companies push Congress to revise the law to reduce barriers to orphan drug development

November 17, 2023 11:11 PM UTC

The orphan drug provisions in the Inflation Reduction Act were intended to help patients with rare diseases by ensuring that drugs marketed for an orphan indication are exempt from Medicare price negotiation. Rare disease patient advocates, along with companies focused on developing the therapies they need, are telling Congress it missed the mark and are urging lawmakers to modify the IRA so that it does not disincentivize orphan drug development.

Because the IRA’s price negotiations will eventually apply to virtually all profitable drugs, providing an exemption for drugs with orphan designation could have helped ensure a steady stream of new therapies for rare diseases. Instead, restricting that exemption to products with only one orphan designation creates an immense barrier to developing a product for multiple indications, including multiple rare indications, which are often needed for the financial viability of a product. ...