Pharmacoeconomic report : (Novartis Pharmaceuticals Canada Inc.) : indication: for the treatment of pediatric patients with 5q spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene and 3 or fewer copies of SMN2 gene; or, infantile-onset SMA. Onasemnogene abeparvovec (Zolgensma) / Canadian Agency for Drugs and Technologies in Health.

Format:

Book

Author/Creator:

Canadian Agency for Drugs and Technologies in Health, author, issuing body.

Language:

English

Subjects (All):

Outcome assessment (Medical care).

Physical Description:

1 online resource

Edition:

Version: final (with redactions).

Place of Publication:

Ottawa (ON) : Canadian Agency for Drugs and Technologies in Health, 2021.

Summary:

Several major limitations were identified but could not be addressed within the sponsor's submitted model. In particular, there is a lack of information on the long-term comparative clinical effectiveness of onasemnogene abeparvovec. The indirect comparison technique used by the sponsor was insufficient to establish the comparative effectiveness of nusinersen. Within the model, 96% and 98% of the quality-adjusted life-year (QALY) benefit compared to best supportive care (BSC) and nusinersen, respectively, was estimated through extrapolation beyond the observed trial data. The cost of nusinersen is the key cost driver but the actual price for participating plans is unknown. Additionally, the sponsor's submission only considered patients with spinal muscular atrophy (SMA) type 1 who were younger than 2 years, who were symptomatic at baseline prior to 6 months of age, and who had 2 copies of the survival motor neuron 2 (SMN2) gene, rather than the full indicated population. The sponsor submitted a scenario analysis suggesting similar results in patients with 3 copies of the SMN2 gene who were pre-symptomatic at baseline, but limitations within the clinical data informing this analysis result in a high degree of uncertainty. CADTH was able to address some of these limitations through reanalysis. The CADTH findings were in line with those of the sponsor. CADTH estimated that onasemnogene abeparvovec was associated with an incremental cost-effectiveness ratio (ICER) of 334,090 per QALY compared to BSC; onasemnogene abeparvovec appeared to dominate nusinersen. Compared with BSC, onasemnogene abeparvovec would not be considered cost-effective at a conventional willingness-to-pay threshold. A price reduction of greater than 90% is required for onasemnogene abeparvovec to be considered the cost-effective strategy compared to BSC at a willingness-to-pay threshold of 50,000 per QALY. Becauseof the limitations of the evidence supporting the comparative efficacy of onasemnogene abeparvovec to nusinersen, these cost-effectiveness findings must be interpreted with a great deal of caution. When considering the key limitations noted above that could not be addressed, the cost- effectiveness of onasemnogene abeparvovec in the indicated population is highly uncertain. Additionally, the cost-effectiveness of onasemnogene abeparvovec is unknown in patients who are older than 6 months or with a single copy of the SMN2 gene.

Contents:

Abbreviations

Executive Summary

Conclusions

Stakeholder Input Relevant to the Economic Review

Economic Review

Economic Evaluation

CADTH Reanalyses of the Economic Evaluation

Issues for Consideration

Overall Conclusions

Appendix 1. Cost Comparison Table

Appendix 2. Submission Quality

Appendix 3. Additional Information on the Submitted Economic Evaluation

Appendix 4. Additional Details on the CADTH Reanalyses and Sensitivity Analyses of the Economic Evaluation

Appendix 5. Submitted Budget Impact Analysis and CADTH Appraisal

References.

Notes:

Includes bibliographical references.

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