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

Gabriel Tremblay, Clemence Cariou, Christian Recher, Mike Dolph, Patricia Brandt, Anne-Sandrine Blanc and Anna Forsythe ()
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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

The European Journal of Health Economics, 2020, vol. 21, issue 4, No 6, 543-555

Abstract: 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.

Keywords: Acute myeloid leukemia; FMS-like tyrosine kinase 3; Cost-effectiveness analysis; Life years; Quality-adjusted life years; Incremental cost-effectiveness ratio (search for similar items in EconPapers)
Date: 2020
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DOI: 10.1007/s10198-019-01149-9

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