Equity Concerns as Medicaid for Spinraza, Zolgensma Varies by State

Marisa Wexler MS avatar

by Marisa Wexler MS |

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Within the U.S., there is marked state-to-state variation in guidelines for whether Medicaid will cover Spinraza (nusinersen) or Zolgensma (onasemnogene abeparvovec) treatments, according to a new study.

This finding raises concerns about equitable access to these lifesaving therapies for spinal muscular atrophy (SMA), according to researchers. The scientists proposed several policies that states could implement to help address this problem and make treatment more accessible.

The study, “Coverage of genetic therapies for spinal muscular atrophy across fee-for-service Medicaid programs,” was published in the Journal of Managed Care and Specialty Pharmacy.

Medicaid is the U.S. government program that provides health insurance to low-income people. Because Medicaid programs are responsible for virtually all the medical costs for the people insured under them, and because these state-sponsored programs must cover basically any approved therapy, many states have implemented guidelines to dictate whether or not various treatments will be covered by Medicaid in that state.

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Now, scientists at Johns Hopkins University, in Baltimore, analyzed state Medicaid websites to look at guidelines for covering two approved therapies for SMA: Spinraza, by Biogen, and Zolgensma, by Novartis. Both of these therapies work to increase levels of the SMN1 protein, which is deficient in SMA.

Although both Spinraza and Zolgensma can be lifesaving, their high price tag has raised concerns about accessibility: Spinraza’s list price is $750,000 in the first year and $375,000 every year thereafter, while Zolgensma is a one-time therapy that carries a list price of $2.1 million.

The researchers’ analysis covered 19 states with guidelines for Spinraza, and 17 states with guidelines for Zolgensma. The team noted that the states with available information generally covered a good sample of the U.S.

For some treatment coverage criteria, there was a lot of agreement across states; for example, virtually every state required that patients have a genetic diagnosis of SMA prior to receiving either therapy. Almost all states also required prior authorization — basically meaning that healthcare providers have to check with the insurer prior to giving a patient the therapy.

For other criteria, however, there were notable state-to-state variations. For instance, 42% of analyzed states had requirements for Spinraza based on whether or not the patient needed a ventilator to breathe. For Zolgensma, the proportion of states with such a requirement was significantly higher, at 77%.

For both therapies, some states required that patients had to see a specialist or subspecialist — for example, a pediatric neurologist — prior to gaining access to treatment, while other states had no such requirements. Similarly, some states but not others would not cover Spinraza if the patient had already received Zolgensma, or vice versa.

Restrictions based on a patients’ number of copies of the SMN2 gene also varied by state. Of note, the SMN2 gene can somewhat compensate for mutations in the SMN1 gene that cause SMA, and patients with more copies of SMN2 tend to have milder disease.

“Medicaid coverage policies for [Spinraza] and [Zolgensma] varied across states. This variation raises equity concerns regarding patient access to these drugs,” the researchers wrote.

“This is particularly worrisome given that Medicaid beneficiaries tend to be from disadvantaged populations already facing health care access concerns,” they added.

In further analyses, the researchers assigned each analyzed state a score from 1 to 4, with higher scores indicating generally more restrictive guidelines. Analyses showed that states with more restrictive guidelines had significantly fewer cases in which Spinraza was used; Zolgensma was not included in the analysis.

These results imply that more restrictive guidelines could prevent patients from accessing these therapies, though the researchers stressed that it’s impossible to conclude cause-and-effect relationships from these correlational data.

Illinois and Florida were found to be the two most restrictive states, earning the score of 4. Conversely, Iowa, Nevada, West Virginia, and Louisiana ranked as the least restrictive, earning a score of 1.

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The scientists proposed a number of policies that may help to improve access to these medications. For example, they noted that two or more states could band together to make agreements on purchasing medications from pharmaceutical companies or making policies. This would allow more uniformity state-to-state, and also could give the states more bargaining power in negotiating with manufacturers.

“For example, if states agree on a specific SMA drug coverage policy around a patient’s ventilator status, they could have better bargaining power, and better bargaining power could yield additional rebates from the drug manufacturer,” the researchers wrote.

“Michigan Medicaid entered into a joint purchase agreement with the state of Vermont in 2003,” they added as an example. “This agreement resulted in lower brand-name drug prices, although the magnitude of savings was small.”

Another proposed policy is the subscription-based “Netflix” model, in which states pay a fixed amount to pharmaceutical companies in return for unlimited access to an expensive medication.

Overall, more needs to be done to make medication access more equitable, according to the researchers.

“State policymakers should consider adopting uniform coverage policies to facilitate multistate negotiations, reduce access inequity due to geography, and reduce administrative burden,” the team concluded.

This study was funded by Arnold Ventures, a private investment fund.

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