Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
. 2020 Jun;21(4):543-555.
doi: 10.1007/s10198-019-01149-9. Epub 2020 Jan 22.

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

Gabriel Tremblay  1 , Clemence Cariou  2 , Christian Recher  3 , Mike Dolph  1 , Patricia Brandt  4 , Anne-Sandrine Blanc  2 , Anna Forsythe  5
Affiliations

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

Gabriel Tremblay et al. Eur J Health Econ. 2020 Jun.

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; Cost-effectiveness analysis; FMS-like tyrosine kinase 3; Incremental cost-effectiveness ratio; Life years; Quality-adjusted life years.

PubMed Disclaimer

References

    1. Visser, O., Trama, A., Maynadie, M., Stiller, C., Marcos-Gragera, R., De Angelis, R., Mallone, S., Tereanu, C., Allemani, C., Ricardi, U., Schouten, H.C.: Incidence, survival and prevalence of myeloid malignancies in Europe. Eur. J. Cancer 48(17), 3257–3266 (2012). https://doi.org/10.1016/j.ejca.2012.05.024 - DOI - PubMed
    1. Khan, I., Altman, J.K., Licht, J.D.: New strategies in acute myeloid leukemia: redefining prognostic markers to guide therapy. Clin. Cancer Res. 18(19), 5163–5171 (2012). https://doi.org/10.1158/1078-0432.CCR-12-0313 - DOI - PubMed - PMC
    1. Sant, M., Minicozzi, P., Mounier, M., Anderson, L.A., Brenner, H., Holleczek, B., Marcos-Gragera, R., Maynadie, M., Monnereau, A., Osca-Gelis, G., Visser, O., De Angelis, R.: Survival for haematological malignancies in Europe between 1997 and 2008 by region and age: results of EUROCARE-5, a population-based study. Lancet Oncol. 15(9), 931–942 (2014). https://doi.org/10.1016/s1470-2045(14)70282-7 - DOI - PubMed
    1. Maynadie, M., De Angelis, R., Marcos-Gragera, R., Visser, O., Allemani, C., Tereanu, C., Capocaccia, R., Giacomin, A., Lutz, J.M., Martos, C., Sankila, R., Johannesen, T.B., Simonetti, A., Sant, M., Group, H.W.: Survival of European patients diagnosed with myeloid malignancies: a HAEMACARE study. Haematologica 98(2), 230–238 (2013). https://doi.org/10.3324/haematol.2012.064014 - DOI
    1. Meyers, J., Yu, Y., Kaye, J.A., Davis, K.L.: Medicare fee-for-service enrollees with primary acute myeloid leukemia: an analysis of treatment patterns, survival, and healthcare resource utilization and costs. Appl. Health Econ. Health Policy 11(3), 275–286 (2013). https://doi.org/10.1007/s40258-013-0032-2 - DOI - PubMed

MeSH terms

LinkOut - more resources