If a boy in New York has been convinced to think he’s a girl, the state Medicaid program has got you covered. But if a boy with muscular dystrophy just wants to walk again, he’s in trouble.
That’s the outcome of the state’s high-cost drug initiative, which empowers the state’s Department of Health to arbitrarily decide which medicines it will pay for and which it will refuse. It can designate drugs as too expensive and thereby deny care, which it has done with a miracle gene therapy called Elevidys that can help children regain mobility.
A one-time gene therapy treatment, Elevidys, was developed to counter Duchenne muscular dystrophy. A nasty degenerative disease, DMD starts with muscle weakness around the thighs and pelvis, typically around age 4. The weakness gets worse and worse until age 12, when most DMD find themselves unable to walk — it can even be fatal. DMD primarily afflicts boys.
RFK JR. MAHA FOOD REGULATIONS HAVE BIPARTISAN APPEAL, NEW POLL FINDS
Although the Food and Drug Administration has approved Elevidys, New York’s Drug Utilization Review Board unanimously voted to pause coverage. DUR wants the FDA to update safety labeling and evaluate an enhanced immunosuppression regimen, which certainly isn’t a terrible idea, but federal law requires state Medicaid programs to cover FDA-approved drugs with a Medicaid Drug Rebate Agreement.
At a recent meeting of the DUR Board, parents reported that Elevidys provided fast, meaningful improvements to their children. One parent testified that her son, Anderson, improved so much after therapy that he could ride his bike again and even go mountain climbing! In justifying its decision, however, the DUR Board downplayed evidence of benefit and overemphasized potential side effects again, rejecting the FDA’s findings.
Alas, New York’s stance reflects a pattern of insurers using cost to deny gene therapy treatments, underscoring the broader need for policy reforms. Yes, patient families under New York law can seek supplemental rebates or appeals, but this process is long and difficult. Children can lose eligibility if they turn 6 before an approval is granted, creating a needless race against time between a disease’s unforgiving clock and senseless bureaucratic dilly-dallying.
This is despite the fact that New York Medicaid has a federal coverage floor, meaning once a manufacturer has a Drug Rebate Agreement, state Medicaid programs must cover FDA-approved drugs under Section 1927 of the Social Security Act. Elevidys has such an agreement and is FDA-approved for DMD. The decision to slam the brakes on Elevidys is a direct rejection of federal law, a terrible trend of state governments ignoring the FDA’s expertise: About two-thirds of state Medicaid gene therapy policies impose clinical requirements more restrictive than the already rigorous FDA label.
In one terrible example, an insurer denied coverage to 15-year-old Sarah Jenssen, a wheelchair-bound victim of the disease, claiming Elevidys would only cover patients who could still walk — even after the FDA expanded its approval to include non-ambulatory patients aged 4 and older.
Again, the harmfully ideological New York Department of Health will throw away billions of Medicaid dollars on so-called “gender affirming care” and grizzly abortion procedures. So, they will mutilate or destroy young bodies, but won’t pay to heal them. Democrats in Albany will also gleefully steal money from the overly burdened New York taxpayer to pay the medical bills of illegal aliens, but not American children with muscular dystrophy.
This is insane.
TRUMP IVF ACTION GETS RESTRAINED PUSHBACK FROM ANTI-ABORTION GROUPS
There’s nothing as devastating as telling a child that their life might have ended before it even starts, and a diagnosis of DMD does exactly that. Thanks to the miracle research of the American medical industry, there is hope for those who once had none, if state bureaucracies don’t interfere. New York’s decision to pause Medicaid coverage of Elevidys is a blow to the children whose young bodies are being eaten away by DMD.
The state’s high-cost drug policy fails to prioritize patients who have the opportunity to receive an exciting new treatment. Contradicting FDA approval makes no sense. The Empire State must align coverage with federal labeling to ensure every child with DMD gets treatment before it’s too late. Doing so doesn’t just relieve human suffering, but probably saves money in the long term as expenses for continuing care become unnecessary.
Jared Whitley is a longtime politico who has worked in the U.S. Senate, White House, and defense industry. He has an MBA from Hult business school in Dubai. In 2024, he won the Top of the Rockies best columnist award.
