SMA Treatment Coverage Recommendations for Managed Care Organizations

Managed care organizations (MCOs) should adjust coverage criteria for spinal muscular atrophy (SMA) treatments based on most current clinical data and provider input, according to recent data.

An Institute for Clinical and Economic Review analysis of costs per quality-adjusted life-year (QALY) concluded that two treatments for SMA, nusinersen and onasemnogene abeparvovec-xioi, do not meet typical cost-effectiveness thresholds. However, onasemnogene abeparvovec-xioi is estimated to have a higher cost per QALY gained than nusinersen in patients with pre-symptomatic type 1 SMA.

The recently published supplement noted that cost-effectiveness analyses should be reconsidered for ultra-rare disorders, and MCOs should consider recommendations other than cost-effectiveness for SMA pharmacotherapy coverage criteria. The author emphasized the importance of early diagnosis and treatment to prevent or delay symptom severity.

Recommendations for managed care pharmacists include the following:

• SMA’s diagnosis should be confirmed using one genetic test.
• Patients can be genetically screened for number of Survival of Motor Neuron 2 (SMN2) copies. Pharmacotherapy should be implemented immediately in patients who test for 4 or fewer copies of SMN2, as this number may predict less severe SMA.
• Coverage should not be restricted by age, as “evidence does not support loss of efficacy in patients with SMA type 2-3 at all ages.” Treatment should begin immediately in pre-symptomatic populations.
• Pre-symptomatic patients should not be restricted by any other criteria. The author notes that some MCOs may place restrictions on treatment eligibility based on symptom severity, such as with patients who are immobilized or on permanent ventilation. Advocates note, however, that small motor improvements due to treatment can result in large QALY gains.
• For coverage renewals, MCOs should use a baseline assessment of treatment success with a standard outcome measure. Providers should assess patient improvement or halt of disease progression to justify renewal.
• In the absence of strong clinical data regarding combination or sequential therapy, MCOs should monitor concurrent use of treatments and switching between therapies if current treatment does not yield adequate response.

Additionally, given high costs of treatment and uncertainty of long-term efficacy, MCOs may consider outcomes-based payment contracts to shift some financial risk back onto the manufacturer. The author noted that “outcomes should be quantifiable, widely accepted outcomes” for adequate patient response, which may be difficult to determine with SMA.

“Advances in the ability to genetically screen for SMA, as well as the availability of novel treatment options that should be started before symptom development, offer MCOs and providers an opportunity to address patients with SMA like never before,” wrote the author of the supplement. “By treating SMA before symptom development, there is a potential to mitigate disease progression and improve patient outcomes.”

Reference:
Pannier, AD. Spinal muscular atrophy: An update for managed care pharmacists. Am J Manag Care. 2020;27(suppl 1):S13-S18. https://doi.org/10.37765/ajmc.2021.88593