Rare Disease Makers Push Back on Medicare Price‑Cut Pilots

Last week a handful of biotech firms that focus on ultra‑rare disorders gathered at the White House’s Office of Management and Budget. Their purpose? To plead for a carve‑out that would keep their specialty medicines out of two new Medicare pilots designed to lower drug prices across the board.

The pilots are part of President Trump’s broader “most‑favored‑nation” push, which essentially says: if a drug sells for less abroad, Medicare should pay no more than that foreign price. In practice, that means brand‑name drugs could see steep cuts that would bring their U.S. price tags in line with what European or Canadian governments pay.

For companies that make orphan drugs—treatments for conditions that affect fewer than 200,000 Americans—the stakes feel especially high. Their products often cost well over $100,000 a year, but the market is tiny, and the revenue from a handful of patients is what keeps the entire development pipeline alive.

“We’re not asking for a free pass,” said a spokesperson for the Rare Disease Company Coalition, the informal group that brought the request. “We’re asking that the government recognize that the economics of orphan drugs are fundamentally different from blockbuster medicines.”

The coalition’s appeal comes on the heels of an earlier decision by the Centers for Medicare & Medicaid Services (CMS) to exclude a few of the biggest pharmaceutical players from the pilots. Those exclusions were largely symbolic, covering companies that already have robust patient‑assistance programs and whose prices are already under pressure.

What the rare‑disease firms want is a broader exemption—one that would let them continue to set prices that reflect the high cost of research, small patient pools, and the need to fund future innovations. They argue that applying the same pricing formula to a drug that treats 10 patients as to a drug that treats millions would be, at best, unfair and, at worst, a death sentence for the next generation of therapies.

Critics, however, warn that such carve‑outs could create loopholes that undermine the whole point of the pilots. “If you start carving out more and more products, you end up with a patchwork system that defeats the purpose of price transparency and control,” noted a health‑policy analyst at a Washington think‑tank.

The meeting with the OMB is just the latest chapter in a long‑running debate over how to balance drug affordability with the need to incentivize innovation. As the pilots move toward a possible rollout later this year, both sides are bracing for a showdown that could shape the future of rare‑disease treatment in the United States.