This past Christmas weekend, Ana Memedovich had her best two days in more than a decade.
Her 23-year-old son Mikhail has spinal muscular atrophy, or SMA, a rare genetic disease that has slowly robbed him of the ability to walk and function independently. On Dec. 23, the FDA approved nusinersen (Spinraza), the first-ever drug for the disease. More than a decade after his diagnosis, Mikhail only has the strength to take a few steps on his own. With the approval, Memedovich finally had hope.
But just after Christmas, she saw the price. Cambridge, MA-based Biogen (NASDAQ: [[ticker:BIIB]]), the drug’s developer, priced nusinersen, a chronic treatment, at $750,000 for the first year of treatment, and $375,000 every year thereafter. The numbers blew past analyst estimates, and they push the envelope even further for drugs for rare diseases, which are priced high to begin with because of the lack of alternative treatments and small patient numbers.
Memedovich froze. She sensed the battle ahead with insurers, and knew Mikhail would likely have to wait even longer to get nusinersen. His condition could get worse. “I thought we were finally going to get some help, that the path was clear,” she says with a sigh. “This is going to be a huge problem.”
Mikhail’s wait has already begun. Memedovich has been feverishly calling and e-mailing the Gillette Lifetime Specialty Healthcare Clinic in St. Paul, MN, trying to contact her son’s neurologist to get a nusinersen prescription. Mikhail’s neurologist this week told her that “critical patients”—younger patients, or those with the more severe type 1 or type 2 SMA—would be treated first.
According to his mother, Mikhail might not get a prescription until the end of the summer, meaning he might not actually get nusinersen until next year. Memedovich says Mikhail’s neurologist told her the clinic would have to triage patients because of “bottlenecks.” There is only one doctor at Gillette giving nusinersen injections, she says. Triaging is a common medical practice in emergencies to treat the most severe cases first.
Xconomy asked Gillette to respond to Memedovich’s claims. This is the response a spokesperson sent via email: “The determination of when and to whom this drug is administered is based solely on clinical factors, beginning with a patient’s SMA type and age. Administration of [nusinersen] requires a very specific and complicated clinical schedule involving a series of precisely timed lumbar punctures over a several-month period.”
That doesn’t make it any easier for Memedovich, who must now sit and wait for a phone call. “Why are we supposed to sit back and relax until we hear from them down the road? We are not taking anyone’s place,” she says. “We just want treatment and are flat out being denied one.”
Memedovich and other parents of SMA patients are worried that the high price will add another obstacle to their loved ones getting nusinersen. “The concern that insurance companies will deny coverage has echoed throughout the SMA community,” says Khrystal Davis, whose five-year-old son, Hunter, has type 1 SMA. “However, the concern is not restricted to those with type 3 and type 4 of the disease—we all fear our insurance companies will deny coverage.”
SMA affects 10,000 to 25,000 people in the U.S., according to the SMA Foundation, and comes in a variety of forms—type 0 through type 4. Type 0 is diagnosed in utero. Patients with type 1 are diagnosed within six months of birth, might never walk or even sit up, and often die within a few years. Patients with type 2 through type 4 have a better prognosis, but their lives are still compromised. Type 2 patients live longer but might need a wheelchair. Type 3 patients, like Mikhail, can often walk when young but tend to be diagnosed before adolescence, with a deterioration of motor skills to follow.
Insurers have yet to issue decisions about nusinersen. Most insurers contacted by Xconomy, including Anthem, Aetna, UnitedHealthcare, Molina Healthcare (which administers Medicaid plans), and Harvard Pilgrim Health Care, either
