One of the nation’s major health insurers has placed limitations on access to the first-ever approved drug for the rare spinal muscular atrophy, validating patient fears that the high price tag of the drug, nusinersen (Spinraza), might make it hard for some with the disease to get treatment.
Indianapolis-based Anthem (NYSE: ANTM) has disclosed its coverage policy for nusinersen, the SMA drug from Biogen (NASDAQ: BIIB) and Ionis Pharmaceuticals (NASDAQ: IONS). Anthem said the drug is only “medically necessary” for patients with Type 1 SMA. Those are patients diagnosed with the disease within six months of birth who might never walk or even sit up, and who often die within a few years.
Anthem is also instituting a pay-for-performance model for nusinseren. The insurer will only cover treatment for Type 1 SMA patients past six months if there is “documentation of clinically significant improvement” in symptoms, like a slowing decline in motor function, compared to the “natural trajectory of the disease.” Anthem says it will require evidence of the drug’s benefit every subsequent six months, as well. The insurer covers almost 40 million Americans total.
Khrystal Davis, whose five-year-old son Hunter has Type 1 SMA, took a defiant stance when she heard about Anthem’s policy, which she said contradicts the FDA-approved label—that is, who is allowed to receive treatment and how often they should receive it.
“Unfortunately, we are accustomed to insurance denials in the SMA community. We regularly appeal such denials for lifesaving equipment for our children,” she says. “One thing is for sure, those who receive denials will not face them alone. The SMA community will stand with them to ensure all SMA patients obtain access to [nusinersen]. The FDA approved [nusinersen] for all SMA patients, and that is who will obtain access to the treatments.”
For patients who don’t meet its coverage criteria, Anthem said nusinersen would be considered “investigational and not medically necessary.” That’s an important point, because on Dec. 23 the FDA approved nusinersen with a broad label for children and adults with all forms of the disease. SMA affects 10,000 to 25,000 people in the U.S., according to the nonprofit SMA Foundation, and comes in type 0, diagnosed in utero, through type 4, diagnosed much later on in life. The disease varies in severity, even from patient to patient. Type 2 patients, for example, typically live longer than those with Type 1 but might need a wheelchair. Type 3 patients can often walk when young but tend to be diagnosed before adolescence, with a deterioration of motor skills to follow. It’s unclear at this point how Anthem will handle reimbursement requests for patients with forms of SMA other than Type 1.
In an e-mailed statement to Xconomy, Anthem said that its reimbursement criteria “closely parallels” the patients who have clearly been helped in Biogen’s randomized, placebo-controlled trial. “Our medical policies are intended to reflect scientific data and clinical thinking,” Anthem said in the statement.
Anthem could change its stance as more clinical data arrives. In explaining its coverage decision, the insurer said that it will review new information when available, but for now there are “no evaluable published data” on the safety or effectiveness of nusinersen in patients whose symptoms arise after they are six months old.
Those data could come soon. Biogen spokesperson Matt Fearer said the company intends to publish data from the Phase 3 trial in Type 2 SMA patients, a study called “Cherish,” at an “upcoming medical conference.” Biogen halted the Cherish trial late last year because the drug was clearly improving motor function in patients compared to placebo. There are multiple ongoing trials of nusinersen that include patients with Type 1 through Type 3 SMA.
“We believe in the clinical efficacy and innovation of [nusinersen] and its potential to benefit a wide range of SMA patients with [SMA], as illustrated by the broad indication granted by the [FDA] and clinical data across multiple patient populations and disease severity,” Fearer said. Biogen is meeting with Anthem and other payers to encourage coverage. Fearer called those talks “encouraging” and said Biogen’s goal is “to ensure that no patient forgoes treatment because of financial limitations or insurance status.”
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