Alternative Headline: Orphan Drug Price Talks in Limbo
[MM Curator Summary]: The ORPHAN Cures Act, intended to shield multi-indication orphan drugs from IRA pricing talks, faces likely failure after exclusion from the Senate budget.
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The US Centers for Medicare & Medicaid Services’ power to negotiate certain drug prices under the Inflation Reduction Act (IRA) has been criticised by the pharmaceutical industry for myriad reasons. While drugmakers’ attempts to overturn the programme as a whole have been repeatedly rejected, a legislative fix was in the works to alleviate a specific pain point concerning orphan drugs — but the bill’s chances of making it into law are now looking slim.
Under the IRA, drugs that have been on the market for seven years — or 11 for biologics — are eligible for pricing negotiations. While therapeutics approved by the FDA for a single orphan indication were exempt, if the treatment was later cleared for a second rare disease, or a non-orphan indication, it could then be subject to pricing talks — with its on-market period calculated from its first orphan approval.
The ORPHAN Cures Act, introduced by Congressmen Don Davis and John Joyce into the US House of Representatives in February, aimed to expand the orphan drug exclusion so that therapeutics approved for multiple rare diseases would not be eligible for negotiations. Additionally, if an orphan drug subsequently was greenlit for a non-orphan indication, the timeline to determine its eligibility for pricing talks would be based on that second, non-rare disease approval.
The Biotechnology Innovation Organization (BIO) trade group threw its support behind the ORPHAN Cures Act, saying that it would "ensure the continuation of essential research and development of new medicines for patients with rare diseases." BIO criticised the IRA’s current orphan drug provisions, claiming they "disincentivize companies to test whether existing treatments for rare diseases also benefit other rare diseases."
The ORPHAN Cures Act seemed to be making positive headway after it was included in the House’s 2025 budget, which was approved in May. However, it appears to have been excluded from the Senate’s version of the bill, per reconciliation text released by the finance committee this week.
While the future of the ORPHAN Cures Act is uncertain, another piece of the IRA that has been widely criticised by industry — the so-called "pill penalty" discrepancy between when drugs and biologics are eligible for pricing negotiations — is looking more likely to be fixed. President Donald Trump signed an
in April directing Congress "to align the treatment of small molecule prescription drugs with that of biological products, ending the distortion that undermines relative investment in small molecule prescription drugs."
https://firstwordpharma.com/story/5973650
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