Gov. Kathy Hochul talks a big game about making New York affordable. Pressers. Slick websites. Big speeches about “fighting for your family.” Sounds great—until you look at what Albany is doing to families with boys battling Duchenne muscular dystrophy (DMD).
Behind closed doors, her Medicaid Drug Utilization Review Board, which is supposed to evaluate and recommend how to handle high-cost breakthrough medicines, just voted to pause coverage of Elevidys, the first gene therapy for Duchenne. If the Health Commissioner signs off, most Medicaid kids are effectively locked out. Other states are watching—and could copy New York’s playbook. That’s not affordability. That’s a death panel custom made for kids with DMD
Here’s what that means in real life. In June 2024, the FDA expanded Elevidys for kids 4 and older who can still walk—the group most likely to benefit. In a major study, kids who got Elevidys moved faster, stood up from the floor quicker, and held on to strength longer over a year. Earlier patients followed for several years have shown slower decline with manageable side effects. Translation: This treatment helps kids keep abilities longer while their disease tries to take them away.
Why does that matter? In Duchenne, staying on your feet longer usually means better heart and lung health later—and that’s tied to living longer. This isn’t a cosmetic win; it’s about buying time kids can use.
New York has tools to deal with expensive drugs. The state can negotiate extra rebates on top of the federal rebates it already gets. It can cut value-based deals. It can demand data. What it can’t do—while cashing those federal rebate checks—is turn a pricing board into a shadow coverage cop and slam the door on an FDA-approved treatment.
Medicaid isn’t a private HMO. If a drug is part of the federal rebate program and used the way doctors and the FDA say is acceptable, states must cover it—with only narrow exceptions. Washington has said this time and again. When Massachusetts tried to run a closed Medicaid formulary and keep the federal rebate money, the feds said pick one. New York wants it both ways—enjoy the rebates, block the access.
DURB also loves executive sessions—“confidential” pricing talks behind closed doors. Legal? Sure. Transparent? Not even close. New Yorkers can’t see the models or the thresholds that end up creating a de facto ban on a therapy that can change a kid’s life. It’s a black box with a human cost.
Gene therapy is expensive. But the right answer is negotiate the price—don’t nuke the access. New York built a “high-cost drug” process to do exactly that: haggle hard, protect the budget, keep patients covered. Instead, the DURB veered into coverage decisions it wasn’t designed to make. Wrong forum. Wrong authority. Wrong outcome.
Meanwhile, families are stuck. Doctors know who qualifies. Parents are watching the clock. Duchenne doesn’t wait for Albany’s paperwork. Every month without treatment means lost muscle, lost function, lost time they’ll never get back.
Gov. Hochul doesn’t have to let this stand. Order coverage of Elevidys for FDA-accepted uses while talks continue. Shine sunlight on the process. And if New York wants to lead, strike a smart value-based deal that protects taxpayers and gives Duchenne kids a fair shot.
Hochul promised to fight for New York families. The Duchenne community is a New York family. Don’t price them out with a pause that plays like a ban. Reverse the DURB move. Keep Elevidys on the table. And show the country that affordability doesn’t mean shutting the door on kids who need hope now.
