[Updated, 1:57 pm E.T., see below] Another major U.S. health insurer, UnitedHealthCare, has said it would cover the first-ever approved drug for the rare disease spinal muscular atrophy, a closely watched bellwether in the national debate over drug prices. While UHC’s policy is less restrictive than the one disclosed recently by rival insurer Anthem, UHC has nonetheless set limits and will only pay past an initial handful of doses if doctors can prove the drug is working.
UHC, which covers more than 50 million Americans, disclosed that nusinersen (Spinraza), the SMA drug from Biogen (NASDAQ: BIIB) and Ionis Pharmaceuticals (NASDAQ: IONS), is “proven and medically necessary” for patients with Type 1, 2, or 3 SMA under a newly established policy. (There are four primary types in all.)
The declaration is a contrast to Anthem, which a week ago deemed nusinersen medically necessary only for Type 1 SMA patients, even though the FDA approved the drug for children and adults with the disease. Anthem, which covers 40 million Americans, said its stance could change as more clinical data arrives. (Biogen, which has multiple nusinersen trials underway, has said that it plans to publish more data at a future medical meeting.)
A majority of SMA patients are diagnosed with Type 1—infants who may never able to sit up or walk and often die at an early age. But more patients are living with Type 2 and Type 3 SMA. These patients are diagnosed at a later age and their disease progresses more slowly. Type 2 patients may never have the ability to walk, while Type 3 patients may lose the ability to do so later in life.
SMA, which affects anywhere from 10,000 to 25,000 people in the U.S., according to the nonprofit SMA Foundation, varies in severity, even from patient to patient. Evercore ISI analyst John Scotti wrote in a research note last year that an estimated 2,500 patients in the U.S., Europe and Japan have Type 1 SMA, while about 10,000 in those countries have Type 2.
Both UHC and Anthem have instituted a pay-for-performance model for nusinersen. In UHC’s case, the insurer will only cover treatment beyond the first four injections if patients have a documented, “positive clinical response,” such as improvement or maintenance of certain motor skills, compared to the results of a medical test administered prior to nusinersen treatment. UHC says it will require evidence of the drug’s benefit every subsequent year as well. Nusinersen costs $125,000 per infusion, which comes to $750,000 for the first year of treatment and $375,000 each year thereafter. Nusinersen is meant to be taken chronically and slow the progression of SMA.
UHC’s policy doesn’t cover type 4 patients, who are diagnosed much later in life than other SMA patients.
While UHC’s policy “probably looks good to those outside the SMA community,” says patient advocate Khrystal Davis, it precludes … Next Page »