One of the nation’s largest health insurers has decided not to cover a controversial Duchenne muscular dystrophy drug after raising doubts about clinical trial data that regulators relied on to approve the medicine last month.
In a bulletin issued to clients on Friday, Anthem reviewed the results from various studies and concluded that Exondys 51, which is sold by Sarepta Therapeutics, is “not medically necessary” and that “the clinical benefit … has not been demonstrated.” Duchenne is a rare disease that confines boys to wheelchairs and condemns them to an early death.
The Anthem decision is significant because the insurer is one of the largest in the country, covering nearly 38 million lives. Not only does Anthem have a significant presence serving individuals and small employers, the insurer is also a major player serving large employers that operate in different states.
Not surprisingly, Sarepta stock fell on the news. At one point, shares were down 8 percent on Friday morning.
A Sarepta spokesman sent us this note: “We cannot comment on ongoing reimbursement discussions but we do have a robust team that is actively educating payers on the safety and efficacy of Exondys 51.”
The investor reaction underscores concerns about insurance coverage.
Sarepta is charging about $300,000, on a net annual basis, for its drug, although the cost could rise, depending upon the weight of the patient. A recent survey by RBC Capital Markets found that nearly 60 percent of the neurologists expect insurance reimbursement could limit access, despite the lack of available options. Only 2 percent of those surveyed did not expect any issues with reimbursement.
The decision is not only a setback for Sarepta, but also amounts to a rebuke of the US Food and Drug Administration, which approved the drug last month after unusually protracted bickering among high-ranking agency staff.
For months, FDA officials quarreled behind the scenes over study results. A key issue had been whether the drug can sufficiently produce higher levels of a protein called dystrophin. Without this protein, muscle fibers degenerate and voluntary movement becomes impossible. A study published in 2013 concluded the Sarepta drug produced enough of the protein to help boys walk.
However, some agency staff questioned the results and argued the study should be retracted, a sentiment openly endorsed by FDA Commissioner Dr. Robert Califf. FDA reviewers also raised doubts about the viability of a six-minute walking test that trial participants underwent. Consequently, Anthem pointed to the uncertainty over these findings to justify its decision.
“Overall, both studies failed to meet their primary endpoints of a significant improvement … and methodological study limitations hinder the ability to interpret the efficacy of [Exondys 51] as a disease-modifying therapy for Duchenne muscular dystrophy,” Anthem wrote to its clients.
To some, the Anthem decision is not a surprise.
“The FDA is not doing patients any favors when they approve a drug or device that is not proven to work,” said Diana Zuckerman, who heads the National Center for Health Research, a nonprofit think tank. “In order to keep health insurance affordable, companies need to ensure that they are paying for safe and effective treatments. When FDA fails to ensure those standards, then FDA approval is no longer a gold standard that insurance companies can rely on.”
Of course, other large insurers may choose to cover the drug.
UnitedHealthcare and Cigna have agreed to do so. “We will continue to follow the clinical trials of Exondys 51 to ensure that this new drug is delivering value to Cigna’s customers and clients for the money they are spending,” a Cigna spokeswoman wrote us last month.
In any event, one patient advocate expressed concern.
“Exondys 51 is the only approved drug for Duchenne. We hope the insurers will consider the professional opinion of the physicians who treat, prescribe, and monitor Duchenne patients,” said Debra Miller of CureDuchenne, an advocacy group that also donates to, and invests in, drug makers.