Why Health Insurers Won't Cover this $300,000-a-year Rare Disease Drug

(Source) — Alison Willis Hoke’s twin sons were among the 12 boys in a key clinical trial that helped get Sarepta Therapeutics Inc.’s Exondys 51 approved, as the first treatment for the rare degenerative disease Duchenne muscular dystrophy.
But her health insurer will only cover the more than $300,000-a-year drug for DMD patients who can walk, a blow for her wheelchair-bound sons.
“The thought of this drug being taken away from my boys after everything they’ve been through over the last five years is astounding,” she said. “It’s really alarming.”
Willis Hoke’s story exemplifies the new bind for patients with DMD and their families. Until this fall, the often-deadly disease had no treatment. But highly variable insurance coverage could keep this new treatment out of reach for many patients.
It also signals a bumpy path forward for drugmaker Sarepta SRPT, +2.99% Health insurers have cited the controversial approval history of the drug, also known as etiplirsen, as rationale for constraints on coverage, or for not covering the drug at all.
“The thought of this drug being taken away from my boys after everything they’ve been through over the last five years is astounding. It’s really alarming.”
Alison Willis Hoke, mother of twin boys who suffer from Duchenne muscular dystrophy
That has led to a reversal in Sarepta’s once-soaring shares and raised questions about its recently-cemented status as a biotech darling.
What happens next could test patients as well as the company. Willis Hoke does not expect Sarepta to continue providing Exondys 51 to her boys for free, forever. But her family can’t afford to pay for the pricey drug out-of-pocket, and she says she “can’t fathom” taking her boys off the medication.
Five of the top 13 commercial health insurers in the U.S. will cover the drug, according to a report last week by J.P. Morgan analyst Anupam Rama.
Of that number, some have more stringent requirements than simply meeting the genetic profile for the drug, which studies show about 13% of DMD patients do.
Aetna Inc. AET, -0.27% and Humana Inc. HUM, -1.14% both have an “ambulatory requirement,” so patients must be able to walk. Aetna has also imposed an under-14 age requirement to start the medication, according to Rama.
Many other insurers simply won’t cover it. Anthem made the earliest and most prominent such decision, with Kaiser Foundation Group also not covering the drug.
More patients are taking five or more prescription medications at once, putting them at risk for side effects and drug interactions. Amid concern about the potential harm of taking too many drugs, more doctors are de-prescribing, and getting patients off prescriptions that are no longer necessary. WSJ’s Laura Landro explains on Lunch Break with Tanya Rivero. Photo: iStock
Exondys 51 was approved under an accelerated pathway, which is intended to speed up treatments for diseases like DMD. The FDA looked at what’s called a “surrogate endpoint” in making its decision, or an increase in the protein dystrophin in patients treated with the drug.
Critics, including an advisory committee that recommended against approving the drug, zeroed in on the small patient population involved in the research — just 12 boys — and certain aspects of how the clinical trial was designed.
See: More bad news for those that have this disease with no treatment
But members of the patient community advocated strongly and loudly for the drug’s approval, with many telling stories about how Exondys 51 had made a difference in their children’s lives.
Other insurers’ policies on the drug appear to still be in the works, Rama said.
There are a couple of reasons health insurers could be making these decisions, Rama said, including its high cost, the “contentious/flawed nature” of the FDA’s approval, the FDA label on Exondys 51 that says its benefit hasn’t yet been established, a lack of protest from medical providers and current development of potentially cheaper treatment alternatives.
Read more: New Anthem policy will cost DMD patients $300,000 or more
Sarepta relied upon improvements in walking for patients on its drug in applying for FDA approval. Many DMD patients can no longer walk when they reach their teenage years. Indeed, nearly all of the company’s clinical trials enrolled ambulatory patients, according to Rama’s report.
The Exondys 51 application was based on “a large significant advantage” of over 100 meters on a six minute walk test for boys treated with Exondys 51, according to the FDA advisory committee’s report. The effect was consistent and held across several analyses and a FDA follow up, the report said.
Read: Sarepta’s controversial Duchenne muscular dystrophy drug was contested right up to approval
DMD, which by and large affects boys, first manifests in early childhood as muscle weakness and degeneration and gets worse over a patient’s life. Those symptoms are particularly dangerous once they extend to the heart and respiratory systems.
Patient advocates say Sarepta’s drug can improve those symptoms.
Christine McSherry, co-founder and executive director of the DMD nonprofit the Jett Foundation, says she’s seen “subtle but significant changes” in her son with DMD since he started on Exondys 51 in 2014.
She calls the drug — which is covered by her insurer, Blue Cross Blue Shield — a “miracle in my son’s life.”
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