The Orphan Cures Act has been added back into the Senate version of President Donald Trump’s “big, beautiful bill” after being left out in the chamber’s first rendition.
The massive domestic policy bill (PDF) that passed a Senate procedural vote Saturday night now includes provisions that would allow drugs with multiple rare disease indications to be exempt from Medicare price negotiations. Currently, under the Inflation Reduction Act, such exemptions are only available to drugs that treat just one rare disease.
The Senate will launch a “vote-a-rama” process Monday to debate Trump’s bill and potentially vote on its passage as soon as today. If passed, the measure will go back to the House, which endorsed its own version of the tax cut and spending bill in May with the Orphan Cures Act language. Trump has demanded lawmakers in his party finalize the bill by the Fourth of July.
The bipartisan Orphan Cures Act was designed to fix what the biopharma industry views as one of the many unintended consequences of the IRA. By offering exemptions exclusively to drugs with just one rare indication, the original IRA disincentivizes companies from conducting follow-on tests of rare disease therapies, the industry has argued.
An analysis by the National Pharmaceutical Council recently found that after IRA’s passage, the percentage of drugs with an initial orphan designation that went on to receive a second designation decreased by 48%—from 12.1% to 6.3%—despite the industry seeing a steady increase before the law.
In addition to the Medicare negotiation exemption, the orphan drug proposal would also delay the timeline of price cuts for rare disease therapies if they lose their exemptions by adding non-rare-disease indications to their labels.
While other drugs start the price-cut eligibility countdown from their initial FDA approval, for rare disease drugs, the clock would start ticking with the first non-orphan indication.
As it stands, the IRA spares small-molecule drugs from price negotiations for nine years, versus 13 years for biologics, a discrepancy that the biopharma industry is also trying to change.
But critics have argued that drugmakers are looking for ways to evade IRA price cuts. The nonprofit organization Patients for Affordable Drugs Now has described the Orphan Cures Act as a “harmful, pharma-backed proposal.”
“This is a blatant giveaway to the pharmaceutical industry that would keep drug prices high for patients while draining $5 billion in taxpayer dollars,” P4ADNow executive director Merith Basey said in a statement Saturday.
The Congressional Budget Office estimates that the Orphan Cures Act terms would cost taxpayers nearly $5 billion over the next decade, P4ADNOW noted.
The Senate initially left out the orphan drug language in its first draft of the One Big Beautiful Bill Act two weeks ago.
“The ORPHAN Cures Act will enable more options for Americans living with rare disease,” the trade group Biotechnology Innovation Organization wrote on X last week. “Congress must pass the #ORPHANCuresAct to ensure continued investment in lifesaving treatments and to protect U.S. #biotech leadership.”
In seeking to justify the act, the industry association pointed to the lack of treatment options for rare diseases and existing societal costs. Only 5% of rare diseases have an approved treatment, while the economic cost of rare diseases in the U.S. surpassed $997 billion in 2019, BIO noted in its post.