WASHINGTON, D.C. — The ORPHAN Cures Act is proving even more destructive than initially reported. Over the weekend, The Wall Street Journal revealed that the Congressional Budget Office (CBO) will re-score the bill to account for its impact on even more blockbuster drugs, meaning its already massive $5 billion price tag is set to grow. 

When first scored, the ORPHAN Cures Act was projected to cost taxpayers nearly $5 billion over the next decade, allowing drugmakers to reap the rewards of a weakened Medicare Negotiation Program. But that initial estimate understated the damage: major high-cost drugs, including blockbusters like Keytruda, weren’t counted in the original analysis. With a new CBO score underway, the full scale of this pharma-backed carveout will soon be clear — and it’s worse than anyone thought.

This carveout is a blatant loophole designed for Big Pharma to protect its monopolies and keep prices artificially high. Pharma’s claim that ORPHAN is necessary to protect rare disease innovation doesn’t stand up to scrutiny either, since Medicare negotiation already preserves all the incentives for rare disease research under the 42-year-old Orphan Drug Act.

This should serve as a warning to Congress: the ORPHAN Cures Act is already funneling billions more to Big Pharma than lawmakers originally understood, doing even greater damage to Medicare negotiation. The last thing patients and taxpayers can afford is another pharma-backed giveaway like the EPIC Act that would hand the industry even more money and further undermine the program’s ability to lower drug price

The Wall Street Journal: Why Drug Prices for Some Big Medicines Will Remain High for a Longer Time
Joseph Walker | August 3, 2025

###

Patients For Affordable Drugs Now is the only national, patient advocacy organization focused exclusively on policies to lower drug prices. We empower and mobilize patients and allies, hold accountable those in power, and fight to shape and achieve system-changing policies that make prescription drugs affordable for all people in the United States. P4ADNow is bipartisan and does not accept funding from organizations that profit from the development or distribution of prescription drugs. To learn more, visit: PatientsForAffordableDrugsNOW.org