Cure SMA Chief Tells Families Seeking Insurance Coverage for Spinraza: ‘Don’t Get Discouraged’

In the roughly four months since the U.S. Food and Drug Administration (FDA) approved Spinraza (nusinersen) to treat all types of spinal muscular atrophy (SMA) in both children and adults, dozens of hospitals and clinics across the nation have begun dosing patients.

Yet insurers are reluctant to pay for Spinraza, which at $125,000 per dose — or $750,000 for six vials covering the first year of treatment — already ranks as one of the world’s most expensive drugs.

“We’ve been very active in the last month and a half getting data and letters out to CEOs and CMOs of all the insurance companies,” said Kenneth Hobby, president of the advocacy organization Cure SMA. “More recently, we’re sending letters with data from clinical trials to all of the states’ Medicaid directors as well as governors, urging them to provide coverage of Spinraza for all ages and types.”

Hobby spoke during a lengthy May 4 webinar for SMA families that covered several topics, including the current state of Spinraza dosing at U.S. administration sites, commercial and government insurance policies, trial results, and community support.

The Cure SMA website now lists 58 sites in 28 states plus the District of Columbia (including six in Arizona, five in Texas, and seven in New York) that have given the organization approval to include them. Yet Hobby said closer to 90 sites across the United States are now “up and running, and dosing” with Spinraza.

In all, Cure SMA has been in touch with 308 potential dosing sites, he said, up from the original 20 to 30 clinical trial locations.

“We’re aiming for 250 to 300 sites eventually to meet the needs of thousands of patients,” he said. “Two months ago, when things kicked off, tens of patients were getting dosed, and now we’re up to 400 or 500. We want to get up into the thousands to have good, secure and quick access.”

Hobby said each site can manage only 25 to 50 patients each due to repeat dosing. Access to the therapy is further limited because Spinraza — which Biogen is licensing from Ionis Pharmaceuticals — must be administered through a spinal tap in specialized facilities.

“That’s why we need such big numbers,” he explained. “Obviously, we’re not yet up to the numbers we need, so there are periods where it’ll take time to get into hospitals. Even with our 250 to 300 sites, there will still be waiting lists.”

Eventually, Cure SMA hopes to have at least one adult Spinraza dosing site and one pediatric site in all 50 states, since “the range [of patients] covers from a few weeks of age to even 70-year-olds who are interested in getting dosed. Finally, we have to make sure states and metropolitan areas with much higher population density have more sites.”

SMA, one of the leading genetic causes of death in infants, occurs in anywhere from 1 in 6,000 to 1 in 10,000 U.S. births. Type 1, its most severe form, accounts for about 60 percent of all SMA cases, and generally kills infants before their second birthday.

Jill Jarecki, Cure SMA’s chief scientific officer, noted the encouraging results of two clinical trials whose results were announced during last month’s 2017 American Academy of Neurology annual meeting in Boston.

In the Phase 3 ENDEAR clinical trial (NCT02193074), 51 percent of infants with type 1 SMA receiving Spinraza met motor developmental milestones, such as being able to sit unassisted, roll and control their heads. CHERISH (NCT02292537), also Phase 3, investigated the effectiveness of Spinraza versus a placebo in 126 children who were diagnosed with SMA after the age of six months.

“In the final analysis, 61 percent of treated infants were alive and did not require ventilation more than 16 hours a day. This is really good and exciting news,” Jarecki said. “And in CHERISH, a study on SMA children ages 2 to 12 years old, the final data had a four-point gain on the Hammersmith Functional Motor Scale.”

Hobby said he hopes results of the CHERISH trial will convince those insurance providers putting restrictions on SMA types 2 and 3, which affect older children and adults.

“Some insurers are approving Spinraza coverage but insisting on monitoring to prove the therapy actually works,” said Hobby. “So one of the key things we have to do is make sure we’re collecting data on individual patients, to see how things have changed over time, and data on what might have happened without the therapy.”

He advised: “Don’t get discouraged. You might see a national policy that says you’re not covered, but we’ve heard of patients who are getting approved anyway. Through an appeals process, we’ve seen those initial denials turned around. It’s very frustrating, but once somebody gets approval, the precedent gets set for everybody else.”

During the webinar, Hobby stressed the importance of pushing back on insurers that decline to cover Spinraza for all types of SMA.

“Where we see a national policy in draft form with restrictions, we provide evidence against those restrictions and why Spinraza should work,” he said. “We have resources available on our website and have produced a booklet to help guide you through this.”

He also urged parents of babies with SMA to be patient.

“Some people have seen a quick impact after three doses, but we don’t know,” he said. “There’s still the potential for a placebo impact that something might be happening when you get on a therapy. But Spinraza takes longer to build up for its main impact. That’s going to take time.”

Dr. John W. Day, a muscular dystrophy expert, acknowledged the frustration of families eager to start their children on Spinraza therapy but facing the prospect of denial.

“There’s a tendency for insurers to focus on the different types of SMA, but SMA is a continuum from more severely affected to less severely affected individuals,” he said. “The FDA understood that. They had access to a lot of data that wasn’t yet public. That’s why they allowed [Spinraza] to treat patients of all ages and levels of severity. We’re very eager to work with insurers so that they understand that.”

He added: “Insurers are asking us to prove the drug works in a set period of time, but that’s difficult. I usually tell families that I don’t expect to see anything I can measure as being different for 6 to 12 months.”

Day stressed the importance of ongoing care for people — especially infants ­— with SMA, even if they manage to get on the new medication.

“Spinraza is not a magic bullet. It does not make SMA go away,” he said. “Patients with SMA deserve and require the same ongoing care we’ve always recommended.”

He also warned against unintended consequences of treatment with Spinraza.

“Patients can show an improvement in stamina or strength that actually gets them in trouble,” he said. “We’ve seen some infants whose parents did not realize they could sit up or turn over. Even in adults, their increase in stamina can mislead them into doing things that actually put them at risk of injury. So it’s important to keep our eyes open and continue to learn best practices as we enter this new era of SMA treatment.”