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Cost-effectiveness of midostaurin in the treatment of newly diagnosed FLT3-mutated acute myeloid leukemia in France

Author

Listed:
  • Gabriel Tremblay

    (Purple Squirrel Economics)

  • Clemence Cariou

    (Novartis Pharmaceuticals)

  • Christian Recher

    (Institut Universitaire du Cancer de Toulouse Oncopole-Centre Hospitalier Université de Toulouse)

  • Mike Dolph

    (Purple Squirrel Economics)

  • Patricia Brandt

    (Novartis Pharmaceuticals)

  • Anne-Sandrine Blanc

    (Novartis Pharmaceuticals)

  • Anna Forsythe

    (Purple Squirrel Economics)

Abstract

Background Midostaurin (MIDO) combined with standard chemotherapy was approved by the European Medicines Agency in 2017 for the treatment of adults with newly diagnosed FLT3-mutated acute myeloid leukemia (AML) based on results from the RATIFY trial. Methods A cost-effectiveness model was developed to compare MIDO and standard-of-care (SOC) to SOC alone in France. Per Haute Autorité de Santé (HAS) guidelines, a partitioned survival model with eight health states was used: diagnosis/induction, complete remission, relapse, hematopoietic stem cell transplantation (HSCT), HSCT recovery, post-HSCT recovery (stabilized after HSCT recovery), post-HSCT relapse, and mortality. A lifetime horizon was used beginning at diagnosis with a “cure model,”, which assumed natural mortality after trial cut-off. Utility values were obtained from a systematic literature review and included disutilities. Resource utilization was based on HAS clinical guidelines and a survey of French physicians and included drugs and administration, adverse events, routine medical care, HSCT, and end-of-life care costs. Results In RATIFY and after extrapolation, MIDO improved survival compared to SOC, translating into MIDO-treated patients gaining 1.12 life years (LYs) and 1.23 quality-adjusted life years (QALYs) versus SOC. The incremental cost-effectiveness ratio (ICER) for MIDO versus SOC was €68,781 per LY and €62,305 per QALY. Sensitivity analyses showed consistency with base case findings. Conclusions MIDO represents a clinically significant advancement in the management of newly diagnosed FLT3-mutated AML. In this analysis, MIDO add-on therapy showed gains in LYs and QALYs versus SOC alone and was found to be a cost-effective option at a €100,000 per QALY threshold for end-of-life treatment.

Suggested Citation

  • Gabriel Tremblay & Clemence Cariou & Christian Recher & Mike Dolph & Patricia Brandt & Anne-Sandrine Blanc & Anna Forsythe, 2020. "Cost-effectiveness of midostaurin in the treatment of newly diagnosed FLT3-mutated acute myeloid leukemia in France," The European Journal of Health Economics, Springer;Deutsche Gesellschaft für Gesundheitsökonomie (DGGÖ), vol. 21(4), pages 543-555, June.
    • Handle: RePEc:spr:eujhec:v:21:y:2020:i:4:d:10.1007_s10198-019-01149-9
    DOI: 10.1007/s10198-019-01149-9
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