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Cost Effectiveness of Nusinersen in the Treatment of Patients with Infantile-Onset and Later-Onset Spinal Muscular Atrophy in Sweden

Author

Listed:
  • Santiago Zuluaga-Sanchez

    (RTI Health Solutions)

  • Megan Teynor

    (Biogen Inc)

  • Christopher Knight

    (RTI Health Solutions)

  • Robin Thompson

    (Biogen International GmbH)

  • Thomas Lundqvist

    (Biogen Sweden)

  • Mats Ekelund

    (Biogen Sweden)

  • Annabelle Forsmark

    (Nordic Health Economics AB)

  • Adrian D. Vickers

    (RTI Health Solutions)

  • Andrew Lloyd

    (Acaster Lloyd Consulting Ltd)

Abstract

Background Spinal muscular atrophy is a rare neuromuscular disorder with a spectrum of severity related to age at onset and the number of SMN2 gene copies. Infantile-onset (≤ 6 months of age) is the most severe spinal muscular atrophy and is the leading monogenetic cause of infant mortality; patients with later-onset (> 6 months of age) spinal muscular atrophy can survive into adulthood. Nusinersen is a new treatment for spinal muscular atrophy. Objective The objective of this study was to evaluate the cost effectiveness of nusinersen for the treatment of patients with infantile-onset spinal muscular atrophy and later-onset spinal muscular atrophy in Sweden. Methods One Markov cohort health-state transition model was developed for each population. The infantile-onset and later-onset models were based on the efficacy results from the ENDEAR phase III trial and the CHERISH phase III trial, respectively. The cost effectiveness of nusinersen in both models was compared with standard of care in Sweden. Results For a time horizon of 40 years in the infantile-onset model and 80 years in the later-onset model, treatment with nusinersen resulted in 3.86 and 9.54 patient incremental quality-adjusted life-years and 0.02 and 2.39 caregiver incremental quality-adjusted life-years and an incremental cost of 21.9 and 38.0 million SEK (Swedish krona), respectively. These results translated into incremental cost-effectiveness ratios (including caregiver quality-adjusted life-years) of 5.64 million SEK (€551,300) and 3.19 million SEK (€311,800) per quality-adjusted life-year gained in the infantile-onset model and later-onset model, respectively. Conclusions Treatment with nusinersen resulted in overall survival and quality-adjusted life-year benefits but with incremental costs above 21 million SEK (€2 million) [mainly associated with maintenance treatment with nusinersen over a patient’s lifespan]. Nusinersen was not cost effective when using a willingness-to-pay threshold of 2 million SEK (€195,600), which has been considered in a recent discussion by the Dental and Pharmaceutical Benefits Agency as a reasonable threshold for rare disease. Nonetheless, nusinersen gained reimbursement in Sweden in 2017 for paediatric patients (below 18 years old) with spinal muscular atrophy type I–IIIa.

Suggested Citation

  • Santiago Zuluaga-Sanchez & Megan Teynor & Christopher Knight & Robin Thompson & Thomas Lundqvist & Mats Ekelund & Annabelle Forsmark & Adrian D. Vickers & Andrew Lloyd, 2019. "Cost Effectiveness of Nusinersen in the Treatment of Patients with Infantile-Onset and Later-Onset Spinal Muscular Atrophy in Sweden," PharmacoEconomics, Springer, vol. 37(6), pages 845-865, June.
    • Handle: RePEc:spr:pharme:v:37:y:2019:i:6:d:10.1007_s40273-019-00769-6
    DOI: 10.1007/s40273-019-00769-6
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    1. Marianna Cavazza & Yllka Kodra & Patrizio Armeni & Marta Santis & Julio López-Bastida & Renata Linertová & Juan Oliva-Moreno & Pedro Serrano-Aguilar & Manuel Posada-de-la-Paz & Domenica Taruscio & Arr, 2016. "Social/economic costs and health-related quality of life in patients with Duchenne muscular dystrophy in Europe," The European Journal of Health Economics, Springer;Deutsche Gesellschaft für Gesundheitsökonomie (DGGÖ), vol. 17(1), pages 19-29, April.
    2. Kathryn J Swoboda & Charles B Scott & Thomas O Crawford & Louise R Simard & Sandra P Reyna & Kristin J Krosschell & Gyula Acsadi & Bakri Elsheik & Mary K Schroth & Guy D'Anjou & Bernard LaSalle & Thom, 2010. "SMA CARNI-VAL Trial Part I: Double-Blind, Randomized, Placebo-Controlled Trial of L-Carnitine and Valproic Acid in Spinal Muscular Atrophy," PLOS ONE, Public Library of Science, vol. 5(8), pages 1-13, August.
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