Global Assessment of Nusinersen Reimbursement Highlights Need for More Transparency

The researchers investigated the role that economic evaluation and budget impact analysis plays in reimbursement decisions for nusinersen, collecting data from countries across the globe.

A global look at the cost-effectiveness of Spinraza (nusinersen) for spinal muscular atrophy (SMA) treatment revealed limited data on budget impact across various countries and highlighted a need for more transparency in the appraisal process.

The researchers investigated the role that economic evaluation and budget impact analysis plays in reimbursement decisions for nusinersen, collecting data from the United States and Canada, as well as various European countries including Sweden, Belgium, England, and France.

Cost-effectiveness analyses across countries showed that for patients with early-onset SMA, a range of €464,891 to €6,399,097 (equivalent to $526,693 to $7,249,792) per quality-adjusted life year (QALY) was gained for nusinersen compared with standard of care. For later-onset disease, the researchers found a range of €493,756 to €10,611,936 (equivalent to $559,395 to $12,022,686) per QALY gained with the treatment.

“Despite economic evaluations indicating that nusinersen was generally not cost-effective and despite limited data on budget impact, all countries under study reimbursed nusinersen. Germany, Belgium, and Italy provided the broadest access, by reimbursing nusinersen for all SMA patients, in line with the European label,” noted the researchers.

“On the other hand, access in Scotland was most narrow, reimbursing nusinersen only for EO (type I) SMA patients. In the US, access depends on the patient’s health insurance provider. The remaining countries provided access to type I, II, and III SMA patients, either with or without age restrictions. In England and Wales, additionally, pre-symptomatic patients were covered,” they continued.

This finding may indicate that decision makers across countries use other determinants to favor reimbursement for nusinersen, suggesting a “grey zone” between the assessment of cost-effectiveness and the final decision in the reimbursement process, leading the researchers to call for more transparency in the process.

According to the authors, the amount and level of cost-effectiveness data available to each country varied. For example, while economic evaluations submitted to health technology assessment agencies in certain countries were base case results from a payer perspective, others took a broader, societal perspective.

“Yet, the reports described similar methodological barriers that may have complicated a proper evaluation and reassessment of the cost-effectiveness of nusinersen from the submitted models,” wrote the researchers. “We believe that these barriers are in alignment with those encountered for orphan drugs in general, which are extensively described in the peer-reviewed literature.”

Barriers cited throughout the study included uncertainties in utility values and the value of the treatment in certain SMA subtypes, including type 0, type 4, and type 3. These reports mirror prior criticisms that methods used to determine utility values are “ill-adapted” for younger patients, such as those with SMA.

In addition, the researchers found that clinical trial data was the primary source for determining cost-effectiveness. These clinical trials included patients with SMA that were healthier than those in real-world settings who have varying comorbidities such as scoliosis and mental health conditions.


Blonda A, Lacosta TB, Toumi M, Simoens S. Assessing the value of nusinersen for spinal muscular atrophy: a comparative analysis of reimbursement submission and appraisal in European countries. Front Pharmacol. Published online January 21, 2022. doi: 10.3389/fphar.2021.750742

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