The new version of the bill will still need to go through the entire House and Senate.
Senate Republicans released their version of the Trump administration’s “One Big Beautiful Bill Act” on Monday with a few major departures from the House bill published last month.
The Senate’s package has conspicuously scrapped what’s known as the Orphan Cures Act, which would have excluded orphan drugs with more than one rare disease approval from the Medicare drug price negotiations. The House’s version last month also prolonged the period of exclusivity for rare disease drugs, starting their negotiation countdown only after their approval for non-orphan diseases.
The bill will still have to go through the whole Senate and the House, though Republican Senators aim to hand a final package to Trump by July 4, according to reporting from The Wall Street Journal.
Under the current iteration of the Inflation Reduction Act (IRA), only orphan drugs with one rare disease approval are spared from negotiations. Medicines become open for Medicare negotiations after nine or 13 years, depending on what type of molecule they are. These orphan drug price negotiation provisions were added to prevent companies from evading negotiations by getting a rare disease approval for a popular drug more often used for non-orphan indications, though some observers have expressed concerns that companies would simply not pursue additional approvals.
Experts have raised concerns over the way the IRA deals with rare and orphan diseases. In an online panel discussion hosted by BioCentury in April 2023, Meenakshi Datta, partner at the Chicago-based law firm Sidley Austin, said that the drug negotiations could have a “chilling impact” on drug development in this space.
Drugs for orphan diseases are typically approved later on in their lives for more commonly prescribed indications, at which point they would no longer be exempted from the IRA. In February, Representatives John Joyce and Don Davis recognized this problem. The narrow orphan drug exemptions, they wrote at the time, have “the unintended effect of discouraging and disincentivizing American innovators from engaging in the expensive and time-intensive research necessary to determine if an orphan drug could cure or treat additional rare diseases.”
Their solution is the Orphan Cures Act, a legislative measure that broadened the incentives for orphan drugs under the IRA. These include exemptions for drugs with multiple rare disease indications and stalling the negotiation clock until a non-orphan approval—both of which made it onto the House version of the tax bill, but not onto the Senate’s.
Also included in the Senate bill on Monday are the steep cuts to Medicaid—which would require certain able-bodied beneficiaries to put in at least 80 hours of work per month to qualify for coverage—as well as certain restrictions on providers.
