News and Reports | March 19, 2026
Analysis is part of a new series on Pharma-driven price hikes of essential medicines
WASHINGTON, D.C. — Patients For Affordable Drugs is releasing a new analysis finding that pharmaceutical companies raised prices on rare disease drugs just a few months after the passage of the ORPHAN Cures Act in the 2025 Reconciliation Package, which weakened Medicare’s ability to negotiate lower prices for those treatments.
In the first week of January alone, pharmaceutical companies raised prices on 208 drugs used to treat rare diseases, with more than 80% of the hikes exceeding the rate of inflation. These increases include high-cost drugs like Firdapse, Tibsovo, Brukinsa, Juxtapid, and Tyvaso — medications that already cost patients tens to hundreds of thousands of dollars each year. While each individual condition treated by this class of drugs may be rare, an estimated 1 in 10 Americans lives with a rare disease.
These price hikes follow closely behind the passage of the ORPHAN Cures Act, which weakened Medicare’s ability to negotiate prices for some blockbuster rare disease drugs. The new law allows drugs that treat multiple rare conditions to avoid Medicare drug price negotiation entirely, even if they generate billions in revenue.
“The ORPHAN Cures Act weakened the government’s ability to negotiate with drugmakers, and now the industry is cashing in,” said Merith Basey, CEO of Patients For Affordable Drugs. “Despite nine in ten Americans demanding greater congressional action to lower drug prices, lawmakers instead handed a $8.8 billion giveaway to Big Pharma, and patients and taxpayers are paying the price.”
The harm to patients is already clear. Some of the most expensive drugs, including blockbuster cancer drugs Keytruda ($12,031/dose) and Opdivo ($7,787/dose), were widely expected to be selected in the third round of negotiations, but were excluded because of the passage of ORPHAN.
Steven from North Carolina: “There isn’t a generic option for Brukinsa, so I have no choice but to buy this medication. So many people need the drug to stay alive. At times, I’ve had to forgo or ration some of my drugs because of their high costs.”
Cheryl Ann from Kentucky: “I take Brukinsa for my chronic lymphocytic leukemia (CLL). A grant pays for the drug, but if it weren’t paid for by someone else, I would not be getting this chemotherapy treatment — the drug would cost me $14,000 a month after insurance.”
Ginny from Pennsylvania: “In 2018, a new drug company obtained FDA approval to produce 3,4 DAP under the brand name Firdapse. With the new company came new pricing, and the list price for my 80 mg dosage soared to $500,000 annually.”
Big Pharma Hikes Prices on Rare Disease Drugs ReportRead the full analysis here.
This analysis is part of a series examining how pharmaceutical companies are hiking prices on lifesaving medications across disease areas — including cancer and HIV — while lobbying Congress to delay the reforms designed to lower them. On March 17th, we released an analysis exposing how pharmaceutical companies are raising prices on HIV medications, while lobbying Congress to pass the EPIC Act to delay Medicare negotiation for many of those same drugs.
Methodology: P4AD analyzed the wholesale acquisition cost (WAC) data for 487 unique National Drug Codes (NDCs) with price hikes greater than 1% between January 1 and January 5, 2026.
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Patients For Affordable Drugs is the only national patient advocacy organization focused exclusively on policies that lower prescription drug prices. We empower and mobilize patients by amplifying their experiences with high drug prices to hold those in power to account and fight to shape and achieve system-changing policies that make prescription drugs affordable for all people in the United States. P4AD does not accept funding from organizations that profit from the development and distribution of drugs. To learn more, visit PatientsForAffordableDrugs.org