ORPHAN Cures Act Removed from Senate Budget Reconciliation Bill

Rare Daily Staff

The Senate Finance Committee has released its sweeping budget reconciliation bill and has removed language in the House version that was designed to restore a key incentive for drug companies to invest in the development of therapies to treat rare diseases.

Although the Optimizing Research Progress Hope And New (ORPHAN) Cures Act had been incorporated into the House version of the bill, the Senate has removed this provision, which industry has sought to end a disincentive created by the Inflation Reduction Act. Under the IRA, orphan drugs that treat a single condition are exempted from price negotiations with Medicare. Industry has argued that the provision’s unintended consequence is that it discourages companies from pursuing research to explore additional indications for rare disease drugs since it would make those products subject to price negotiations.

One in five orphan drugs are approved by the U.S. Food and Drug Administration for more than one use, and 60 percent of those second indications are for another rare disease, according to the Rare Disease Company Coalition, an industry group.

While industry groups and rare disease patient advocacy organizations, such as the Biotechnology Innovation Organization and National Organization for Rare Disorders, support passage of the ORPHAN Cures Act, other groups, such as Public Citizen, AARP, and Patients for Affordable Drugs Now, have opposed it.

“This decision helps protect the popular and effective Medicare Drug Price Negotiation Program and its promise of lower prescription drug prices for millions of Americans on Medicare,” said Merith Basey, executive director of Patients for Affordable Drugs Now. “We remain vigilant against any effort to revive this harmful proposal, or anything like it, and will continue fighting to ensure every patient can get the medications they need at prices they can afford.”