In a recent webinar on Spinraza, the first approved treatment for spinal muscular atrophy (SMA), the patient advocacy group Cure SMA addressed issues such as access and insurance coverage, and answered questions from people affected by the disease.
Cure SMA, a foundation that promotes SMA research and provides support for patients and their families, is working to bring knowledge of, and access to, Spinraza (nusinersen) to people across the United States.
Kenneth Hobby, Cure SMA president, and Jill Jarecki, PhD, its chief scientific officer, discussed in the Feb. 14 webinar — the group’s second on the treatment — the two main issues that affect access to Spinraza right now: the availability of sites to administer this drug to patients, and trends in commercial and government insurance coverage for the therapy.
The drug, approved in December by the U.S. Food and Drug Administration (FDA), is expensive, as drugs to treat rare diseases usually are. The cost of Spinraza for the individual patient is listed at $125,000 per vial, meaning that the first year of treatment could cost between $625,000 to $750,000 (five or six injections).
Most urgent is bringing the number of hospitals geared to administer the drug up to meet demand: people cannot be treated if they have nowhere to go to get the treatment.
“We’ve got the FDA approval, but now we have to deal with several aspects, several issues … about getting access to this SMA therapy,” Hobby said. “These issues always come in play after you’ve got approval, so they’re not unexpected.”
Cure SMA highlights that there’s a need for an estimated 200-300 sites in the U.S., or several in every state. There are presently only 20-30 (trial sites and care centers of excellence), and they are already running at full capacity. Moreover, these sites already have waiting lists.
Spinraza treatment involves much more than simply injecting the drug several times a year. It is administered intrathecally, meaning it must be done in hospitals or centers whose staff are trained in performing lumbar punctures, and with equipment available for the necessary anesthesia and radiology procedures.
Hospitals will specialize in the kind of patients with SMA that they treat. First both pediatric hospitals and hospitals treating adults need to get on board, but they may also specialize in treating specific types of SMA (it is approved for types 1, 2 or 3).
Cure SMA’s strategy is to contact hospitals across the U.S., asking them to put education and training in place and to become sites offering Spinraza treatment. The idea is to have more hospitals that specialize in different types of patients in each state, rather than fewer hospitals treating a broader patient base. The group encourages families of patients to approach hospitals in their states about it, as well as individual specialists at these hospitals, and to report the contacts they’ve made to CureSMA, so that it can follow up on their initiatives.
It’s not yet clear who will be covered for Spinraza treatment by commercial and government (Medicaid) insurance providers. It’s expected that some insurers may limit coverage only to younger patients, patients with a specific type of SMA, or only to patients who show improvements. But it’s too early to know how insurance coverage — which determines how much of a drug’s a price any given patient feels — will play out.