The maxim that the government which governs closest governs best is usually sound. Local leaders understand their constituency better than someone, say, 2,000 miles away federal policymakers. However, with some recent, bizarre healthcare decisions, some state leaders have decided to buck that trend – with a vengeance.

Last year, New York’s Drug Utilization Review Board (DURB) voted unanimously that state Medicaid program should halt coverage of a treatment for Duchenne muscular dystrophy (DMD). A horrible but thankfully rare disease, DMD eats away at a child’s leg and arm muscles, eventually making it impossible to even walk. It is thankfully very rare – about one in 3,000 to 5,000 births – affecting boys as young as 2. Many families had found relief thanks to a gene-therapy treatment called Elevidys, which delivered relief for affected boys.

Despite DMD’s rarity, the affliction is gaining considerable attention. In March, advocates staged a mock funeral at the offices of the Food and Drug Administration (FDA), complete with caskets!

A few years ago, the Food and Drug Administration granted Elevidys traditional approval for patients ages 4+ who could still walk and accelerated approval for such patients who could no longer walk. However, in response to hyped-up safety controversies, New York’s Medicaid program put a “pause” on providing access to Elevidys, thereby condemning affected children from finding the treatment they need to life ordinary lives.

Under Section 1927 of the Social Security Act, once a manufacturer signs a Medicaid Drug Rebate Agreement, states are required to cover FDA-approved drugs for their medically accepted indications.

Arizona kind of splits the difference. On the one hand, Arizona has mandated that every newborn must be screened for Duchenne, because as with most diseases early detection can save lives. On the other hand, the Arizona Health Care Cost Containment System (AHCCCS – oof, there’s a mouthful of a government acronym for you) is now enforcing an Elevidys policy that only pays for children who can still walk.

So the Grand Canyon State is funding the diagnostic side of a rare, rapidly acting, potentially fatal disease while gutting the treatment side – quietly rationing the treatment in a race against time. One of the problems with this is that DMD is progressive by nature: boys who can walk at age 4 can quickly lose that capability within a few years, so an “ambulatory-only” coverage rule isn’t a stable clinical line. It’s a ticking clock that forces sick kids to wait while insurers slow-walk prior authorization.

Moreover, Arizona’s own newborn screening statute won’t even take effect until October 2027, which means Arizona will soon be identifying DMD in infants who then face roughly four years of watching the window for gene therapy narrow before they’re even old enough to qualify for care.

AHCCCS pulled Elevidys as a covered service in July 2025 with no evidence of a public hearing or vote, meaning the restriction was an administrative reaction to the manufacturer’s own temporary pause that was never revisited once ambulatory shipments resumed.

Dealing with rare diseases is no picnic, especially when the treatment is both expensive and new. Of course, families can appeal rejections – but then that adds drawn-out bureaucratic headaches to the health nightmares while racing against a ticking clock. Payers routinely delay and deny gene therapies, making parents fight through multiple appeals while children age out of criteria or lose function.

But it seems better to allow access to the victims of rare diseases regardless of even rarer side-effects. The FDA made the right decision, state governments who disagree need to (ahem) trust the science rather than abandon dying children.

Jared Whitley is a longtime DC politico, having worked in the US Senate, White House, and defense industry. This year, the Society of Professional Journalists named him among the best columnists the Intermountain West.