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

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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.

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Abbreviations

AML:

Acute myeloid leukemia

FLT3:

FMS-like tyrosine kinase 3

SOC:

Standard of care

HSCT:

Hematopoietic stem cell transplantation

MIDO:

Midostaurin

EQ-5D:

EuroQol 5 dimensions

QLQ-30:

EORTC core quality of life questionnaire

CI:

Confidence interval

QALY:

Quality-adjusted life year

CR:

Complete remission

HRQoL:

Health-related quality of life

ICER:

Incremental cost-effectiveness ratio

OS:

Overall survival

CE:

Cost-effectiveness

HR:

Hazard ratio

EFS:

Event-free survival

NOS:

Not otherwise specified

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Acknowledgements

Some authors contributed to an analysis of the cost-effectiveness of midostaurin from a United Kingdom perspective, published in Cost-Effectiveness and Resource Allocation in October of 2018 [1]. Mapping of utilities used in this analysis was described in full in a 2018 study by Forsythe et al. [2]. We thank Jaclyn Hearnden for her contributions to writing and editing the manuscripts.

Funding

This study was funded by Novartis Pharmaceuticals.

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Authors and Affiliations

  1. Purple Squirrel Economics, New York, USA

    Gabriel Tremblay, Mike Dolph & Anna Forsythe

  2. Novartis Pharmaceuticals, Rueil-Malmaison, France

    Clemence Cariou & Anne-Sandrine Blanc

  3. Service d’Hématologie, Institut Universitaire du Cancer de Toulouse Oncopole-Centre Hospitalier Université de Toulouse, Toulouse, France

    Christian Recher

  4. Novartis Pharmaceuticals, East Hanover, USA

    Patricia Brandt

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  1. Gabriel Tremblay
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Correspondence to Anna Forsythe.

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Clemence Cariou, Christian Recher, Patricia Brandt, and Anne-Sandrine Blanc are employees of Novartis.

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Tremblay, G., Cariou, C., Recher, C. et al. Cost-effectiveness of midostaurin in the treatment of newly diagnosed FLT3-mutated acute myeloid leukemia in France. Eur J Health Econ 21, 543–555 (2020). https://doi.org/10.1007/s10198-019-01149-9

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