Spinraza and Key Issues of Access and Insurance: An Update from CureSMA’s Webinar

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.

“The insurance decisions are really where we will start to see whether the drug price is gonna be an issue or not,” Hobby said. “We obviously see this very high drug price, not out of line with orphan diseases, but … we don’t know, though, if it’s going to have an impact on the access on this therapy through whether insurance is going to pay that price or not.”

Insurance companies, he said, are now much more focused on “data gaps … information that is missing for them to judge whether the therapy works or not. … They don’t know if it works in certain areas.”

This last point  — who has access — is critical, because Spinraza has been shown to stop disease progression, an invaluable benefit, but it doesn’t necessarily cause improvement in the condition. Spinraza is approved by the FDA to treat a very wide range of patients with SMA, but hospitals and insurers are likely to narrow down the type of patient they treat or cover.

In the webinar, Cure SMA presented the known, and wildly variable, coverage of three commercial insurance companies, Anthem, United Health and Humana. No official national policy has come from Medicaid, but Cure SMA reports that some patients appear to have been approved for treatment through this coverage.

There are still gaps in information about the success of Spinraza treatment in older patients, ventilator-dependent patients, or patients who’ve had spinal fusions or spinal rods, for example, and different insurers will probably cover different situations according to the data available.

“We have heard that all types so far have been dosed. There are people from type 1, 2 and 3 who have got commercial access to the therapy so far,” Hobby said. “We’ve also heard the ages of people who have been dosed range from a few weeks old, all the way up to late 30s.”

Cure SMA encourages families to go through the process of applying for insurance, no matter what their insurer’s policy is, because denials can make them eligible for other programs to fund Spinraza treatment. Families were also asked in the webinar to report the outcome of their insurance applications to Cure SMA. It also encourages people to visit Biogen’s Spinraza website, and go to Support Services on the site, to find out about financial assistance available from Biogen, the drug’s manufacturer.

Cure SMA plans to continue offering regular webinars on treatment access and insurance coverage, as Spinraza treatment sites and coverage becomes more widespread, and more is known about outcomes in specific groups of patients, as well as insurers’ policies.