Abstract
Background: Nusinersen is an orphan drug intended for the treatment of spinal muscular atrophy (SMA), a severe genetic 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 and 40 million euros per year. For early-onset SMA, the incremental cost-effectiveness threshold (ICER) ranged from €464,891 to €6,399,097 per quality-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 four reports included arguments in favor of reimbursement. However, the majority of the jurisdictions set up an MEA, which may have promoted a positive reimbursement decision. Conclusion: 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.
Highlights
Spinal muscular atrophy (SMA) linked to chromosome 5q is a rare and life-threatening neuromuscular disorder with an estimated incidence of 1 per 12,000 births, making it the most frequent genetic cause of child mortality (Pearn, 1980; Verhaart et al, 2017)
Biogen obtained utilities for both EO and LO SMA by deriving Pediatric Quality of Life Inventory (PedsQL) data from LO SMA patients enrolled in the CHERISH (SMA II) trial
The probabilistic sensitivity analysis resulted in a mean incremental cost-effectiveness threshold (ICER) of €498,480 for EO SMA, €2,107,108 for LO SMA, and €1,037,003 for LO SMA with caregiver quality-adjusted life year (QALY) included
Summary
Spinal muscular atrophy (SMA) linked to chromosome 5q is a rare and life-threatening neuromuscular disorder with an estimated incidence of 1 per 12,000 births (estimated prevalence of 1–2 per 100,000 persons), making it the most frequent genetic cause of child mortality (Pearn, 1980; Verhaart et al, 2017). The disorder is characterized by a loss in alpha motor neurons in the spinal cord and brain stem, which causes progressive weakness of the proximal and respiratory muscles and motor neuron death (Castro and Iannaccone, 2014). This is a result of a deficiency of the survival motor neuron (SMN) protein, which is responsible for maintenance of these neurons. In >90% of the cases, SMA is caused by a deficiency in survival motor neuron 1 (SMN1) gene, as a result of either a mutation or deletion (Lefebvre et al, 1995). This study explores the role of the economic evaluation and budget impact analysis on the reimbursement of nusinersen
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