OBJECTIVES : Nusinersen is an orphan drug intended for the treatment of spinal muscular athrophy (SMA), a severe neuromuscular disorder. Considering the very high costs of orphan drugs and the expected market entry of cell and gene therapies, there is increased interest in the use of health technology assessment (HTA) for orphan drugs. This study explores the role of the economic evaluation and budget impact analysis on the reimbursement of nusinersen.

METHODS : Appraisal reports for nusinersen were retrieved from reimbursement and HTA agencies in Belgium, Canada, France, England and Wales, Germany, Italy, Ireland, Scotland, Sweden, the Netherlands and the United States. Detailed information was extracted on the economic evaluation, the budget impact, the overall reimbursement decision and the managed entry agreement (MEA). Costs were adjusted for inflation and currency.

RESULTS : Overall, the reports included limited data on budget impact, excluding information on the sources of data for cost and patient estimates. Only three jurisdictions reported on total budget impact, estimated between 30 to 40 million euro per year. For early-onset SMA, the ICER ranged from €464,891 to €6,399,097 per qualify-adjusted life year (QALY) gained for nusinersen versus standard of care. For later-onset SMA, the ICER varied from €493,756 to €10,611,936 per QALY. Although none of the jurisdictions found nusinersen to be cost-effective, reimbursement was granted in each jurisdiction. Remarkably, only two reports included arguments in favour of reimbursement. However, the majority of the jurisdictions set up an MEA, which may have promoted a positive reimbursement decision.

CONCLUSIONS : There is a need for more transparency on the appraisal process and conditions included in the MEA. Additionally, by considering all relevant criteria explicitly during the appraisal process, decision-makers are in a better position to justify their allocation of funds among the rising number of orphan drugs that are coming to the market in the near future.