The pharmaceutical industry’s lobbying muscle scored a high-stakes victory on Monday when the Senate parliamentarian reversed a previous decision and ruled that the Optimizing Research Progress Hope and New (ORPHAN) Cures Act can ride inside the Republican budget reconciliation package now racing toward a floor vote. The quiet procedural shift immediately reshaped the sprawling measure—already under fire for deep Medicaid and nutrition cuts—by restoring a provision that would spare certain high-revenue “orphan drugs” from Medicare price negotiations for years to come.
Under current law, medications that treat a single rare disease may escape Medicare bargaining, but drugs earning approval for multiple rare conditions must eventually face negotiated prices once they have been on the market long enough. The ORPHAN Cures Act rewrites that rule so any drug with an orphan designation—even one treating several rare diseases—would remain exempt. The change pleases brand-name manufacturers that have steadily turned once-niche therapies into blockbuster revenue streams, yet it alarms patient advocates who say seniors and taxpayers will shoulder the bill. The nonpartisan Congressional Budget Office (CBO) estimates the carve-out would cost the federal government about $5 billion over the next decade, money that otherwise would have been saved through the negotiation program created in 2022.
If the proposal were already law, Medicare “would have been barred from negotiating lower prices for important treatments like cancer drugs Imbruvica, Calquence, and Pomalyst,” the consumer-rights group Public Citizen noted after Monday’s announcement. Those three medicines together generated more than $7 billion in U.S. sales last year, according to public filings, underscoring how “rare-disease” therapies can also become fixtures of everyday oncology practice.
Much of the spotlight now falls on Sen. Martin Heinrich of New Mexico, the only Democratic cosponsor of the stand-alone ORPHAN Cures bill and the senator who confirmed the parliamentarian’s course correction. He joins lead sponsor Sen. John Barrasso of Wyoming, a member of Republican leadership, in championing the exemption on the Senate floor. Their alliance has bewildered progressive healthcare advocates who otherwise expected Democrats to close loopholes in the fledgling negotiation framework, not open new ones.
“Sen. Heinrich should be ashamed of prioritizing drug corporation profits over lower medicine prices for seniors and people with disabilities,” Steve Knievel, access-to-medicines advocate at Public Citizen, said Monday. “Patients and consumers breathed a sigh of relief when the Senate parliamentarian stripped the proposal from Republicans’ Big Ugly Betrayal, so it comes as a gut punch to hear that Sen. Heinrich welcomed the reversal and continued to champion a proposal that will transfer billions from taxpayers to Big Pharma.” Knievel added that “People across the country are demanding lower drug prices and for Medicare drug price negotiations to be expanded, not restricted. Sen. Heinrich should apologize to his constituents and start listening to them instead of drug corporation lobbyists.”
Those lobbyists were quick to hail the ruling. The Biotechnology Innovation Organization (BIO), whose members include many of the world’s largest drugmakers, urged lawmakers to pass the revived language “as soon as possible,” praising it as a bulwark for rare-disease innovation. Yet independent analysts note that the Food and Drug Administration has approved more than 100 orphan indications since 2018, many for drugs already earning billions. Critics argue the generous market incentives embedded in the 1983 Orphan Drug Act—seven years of exclusivity, tax credits and waiver of regulatory fees—were never intended to protect monopolies once a company discovered multiple profitable uses for the same compound.
In New Mexico, where more than 420,000 people rely on Medicare, Heinrich’s position has sparked a rare rupture with progressive activists who typically count him among reliable allies. The senator’s office defended his support by framing the measure as a boost for biotech firms pursuing therapies in small patient populations, though it has yet to publicly address the $5 billion CBO score. Republicans, for their part, hope the bipartisan imprimatur helps inoculate the broader reconciliation bill—which also extends Trump-era tax cuts—from charges that it only benefits the wealthy.
Whether the strategy succeeds could hinge on timing. GOP leaders want the 940-page package on the president’s desk before the July 4 recess but still face resistance inside their own caucus over deficit concerns. Adding a high-profile pharmaceutical giveaway may complicate talks with fiscally conservative senators already uneasy about the bill’s $3.3 trillion addition to the national debt. House progressives, meanwhile, have signaled they will try to strip the orphan-drug language if the Senate sends the bill back for final approval.
Merith Basey, executive director of Patients For Affordable Drugs Now, warned lawmakers not to underestimate public anger at prescription costs. “Patients are infuriated to see the Senate cave to Big Pharma by reviving the ORPHAN Cures Act at the eleventh hour,” she said. “This is a blatant giveaway to the pharmaceutical industry that would keep drug prices high for patients while draining $5 billion in taxpayer dollars. We call on lawmakers to remove this unnecessary provision immediately and stand with an overwhelming majority of Americans who want the Medicare Negotiation program to go further. Medicare negotiation will deliver huge savings for seniors and taxpayers; this bill would undermine that progress.”
For now, the revived carve-out sits in the package’s legislative text, poised to become law unless opponents muster the votes—or procedural ingenuity—to excise it once more. The coming days will reveal whether the Senate’s eleventh-hour embrace of the ORPHAN Cures Act is an immovable concession to pharmaceutical power or just another fragile piece of a budget bill still teetering on the brink.
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