IDEAS home Printed from https://ideas.repec.org/a/spr/eujhec/v21y2020i4d10.1007_s10198-019-01149-9.html
   My bibliography  Save this article

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
    as

    Download full text from publisher

    File URL: http://link.springer.com/10.1007/s10198-019-01149-9
    File Function: Abstract
    Download Restriction: Access to the full text of the articles in this series is restricted.
    File URL: https://libkey.io/10.1007/s10198-019-01149-9?utm_source=ideas
    LibKey link: if access is restricted and if your library uses this service, LibKey will redirect you to where you can use your library subscription to access this item
    ---><---

    As the access to this document is restricted, you may want to search for a different version of it.

    References listed on IDEAS

    as
    1. Don Husereau & Michael Drummond & Stavros Petrou & Chris Carswell & David Moher & Dan Greenberg & Federico Augustovski & Andrew Briggs & Josephine Mauskopf & Elizabeth Loder, 2013. "Consolidated Health Economic Evaluation Reporting Standards (CHEERS) Statement," PharmacoEconomics, Springer, vol. 31(5), pages 361-367, May.
    2. Keith Tolley & Catherine Goad & Yunni Yi & Penny Maroudas & Amin Haiderali & Gwilym Thompson, 2013. "Utility elicitation study in the UK general public for late-stage chronic lymphocytic leukaemia," The European Journal of Health Economics, Springer;Deutsche Gesellschaft für Gesundheitsökonomie (DGGÖ), vol. 14(5), pages 749-759, October.
    3. Juliana Meyers & Yanni Yu & James Kaye & Keith Davis, 2013. "Medicare Fee-for-Service Enrollees with Primary Acute Myeloid Leukemia: An Analysis of Treatment Patterns, Survival, and Healthcare Resource Utilization and Costs," Applied Health Economics and Health Policy, Springer, vol. 11(3), pages 275-286, June.
    4. Ralph Crott & Andrew Briggs, 2010. "Mapping the QLQ-C30 quality of life cancer questionnaire to EQ-5D patient preferences," The European Journal of Health Economics, Springer;Deutsche Gesellschaft für Gesundheitsökonomie (DGGÖ), vol. 11(4), pages 427-434, August.
    Full references (including those not matched with items on IDEAS)

    Most related items

    These are the items that most often cite the same works as this one and are cited by the same works as this one.
    1. Saha, Sanjib & Gerdtham, Ulf-G. & Toresson, Håkan & Minthon, Lennart & Jarl, Johan, 2018. "Economic Evaluation of Interventions for Screening of Dementia," Working Papers 2018:20, Lund University, Department of Economics.
    2. Najmiatul Fitria & Antoinette D. I. Asselt & Maarten J. Postma, 2019. "Cost-effectiveness of controlling gestational diabetes mellitus: a systematic review," The European Journal of Health Economics, Springer;Deutsche Gesellschaft für Gesundheitsökonomie (DGGÖ), vol. 20(3), pages 407-417, April.
    3. Qi Cao & Erik Buskens & Hans L. Hillege & Tiny Jaarsma & Maarten Postma & Douwe Postmus, 2019. "Stratified treatment recommendation or one-size-fits-all? A health economic insight based on graphical exploration," The European Journal of Health Economics, Springer;Deutsche Gesellschaft für Gesundheitsökonomie (DGGÖ), vol. 20(3), pages 475-482, April.
    4. Thomas Grochtdreis & Hans-Helmut König & Alexander Dobruschkin & Gunhild von Amsberg & Judith Dams, 2018. "Cost-effectiveness analyses and cost analyses in castration-resistant prostate cancer: A systematic review," PLOS ONE, Public Library of Science, vol. 13(12), pages 1-25, December.
    5. Saha, Sanjib & Gerdtham, Ulf-G. & Toresson, Håkan & Minthon, Lennart & Jarl, Johan, 2018. "Economic Evaluation of Nonpharmacological Interventions for Dementia Patients and their Caregivers - A Systematic Literature Review," Working Papers 2018:10, Lund University, Department of Economics.
    6. Jesse Elliott & Sasha Katwyk & Bláthnaid McCoy & Tammy Clifford & Beth K. Potter & Becky Skidmore & George A. Wells & Doug Coyle, 2019. "Decision Models for Assessing the Cost Effectiveness of Treatments for Pediatric Drug-Resistant Epilepsy: A Systematic Review of Economic Evaluations," PharmacoEconomics, Springer, vol. 37(10), pages 1261-1276, October.
    7. Don Husereau & Michael Drummond & Stavros Petrou & Dan Greenberg & Josephine Mauskopf & Federico Augustovski & Andrew Briggs & David Moher & Elizabeth Loder & Chris Carswell, 2015. "Reply to Roberts et al.: CHEERS is Sufficient for Reporting Cost-Benefit Analysis, but May Require Further Elaboration," PharmacoEconomics, Springer, vol. 33(5), pages 535-536, May.
    8. Neily Zakiyah & Antoinette D I van Asselt & Frank Roijmans & Maarten J Postma, 2016. "Economic Evaluation of Family Planning Interventions in Low and Middle Income Countries; A Systematic Review," PLOS ONE, Public Library of Science, vol. 11(12), pages 1-19, December.
    9. Kathryn Schnippel & Naomi Lince-Deroche & Theo van den Handel & Seithati Molefi & Suann Bruce & Cynthia Firnhaber, 2015. "Cost Evaluation of Reproductive and Primary Health Care Mobile Service Delivery for Women in Two Rural Districts in South Africa," PLOS ONE, Public Library of Science, vol. 10(3), pages 1-13, March.
    10. Rachel Elliott & Koen Putman & James Davies & Lieven Annemans, 2014. "A Review of the Methodological Challenges in Assessing the Cost Effectiveness of Pharmacist Interventions," PharmacoEconomics, Springer, vol. 32(12), pages 1185-1199, December.
    11. Abualbishr Alshreef & Michelle Jenks & William Green & Simon Dixon, 2016. "Review of Economic Submissions to NICE Medical Technologies Evaluation Programme," Applied Health Economics and Health Policy, Springer, vol. 14(6), pages 623-634, December.
    12. Yue Yin & Yusi Tu & Mingye Zhao & Wenxi Tang, 2022. "Effectiveness and Cost-Effectiveness of Non-Pharmacological Interventions among Chinese Adults with Prediabetes: A Protocol for Network Meta-Analysis and CHIME-Modeled Cost-Effectiveness Analysis," IJERPH, MDPI, vol. 19(3), pages 1-12, January.
    13. Huajie Jin & Paul Tappenden & Stewart Robinson & Evanthia Achilla & David Aceituno & Sarah Byford, 2020. "Systematic review of the methods of health economic models assessing antipsychotic medication for schizophrenia," PLOS ONE, Public Library of Science, vol. 15(7), pages 1-18, July.
    14. Mona Aghdaee & Bonny Parkinson & Kompal Sinha & Yuanyuan Gu & Rajan Sharma & Emma Olin & Henry Cutler, 2022. "An examination of machine learning to map non‐preference based patient reported outcome measures to health state utility values," Health Economics, John Wiley & Sons, Ltd., vol. 31(8), pages 1525-1557, August.
    15. S. Rajsic & H. Gothe & H. H. Borba & G. Sroczynski & J. Vujicic & T. Toell & Uwe Siebert, 2019. "Economic burden of stroke: a systematic review on post-stroke care," The European Journal of Health Economics, Springer;Deutsche Gesellschaft für Gesundheitsökonomie (DGGÖ), vol. 20(1), pages 107-134, February.
    16. B Ekman & H Nero & L S Lohmander & L E Dahlberg, 2020. "Costing analysis of a digital first-line treatment platform for patients with knee and hip osteoarthritis in Sweden," PLOS ONE, Public Library of Science, vol. 15(8), pages 1-12, August.
    17. Stuart Wright & Cheryl Jones & Katherine Payne & Nimarta Dharni & Fiona Ulph, 2015. "The Role of Information Provision in Economic Evaluations of Newborn Bloodspot Screening: A Systematic Review," Applied Health Economics and Health Policy, Springer, vol. 13(6), pages 615-626, December.
    18. Fernando Hoces de la Guardia & Sean Grant & Edward Miguel, 2021. "A framework for open policy analysis," Science and Public Policy, Oxford University Press, vol. 48(2), pages 154-163.
    19. Jason Madan & Meghan Bruce Kumar & Miriam Taegtmeyer & Edwine Barasa & Swaran Preet Singh, 2020. "SEEP-CI: A Structured Economic Evaluation Process for Complex Health System Interventions," IJERPH, MDPI, vol. 17(18), pages 1-12, September.
    20. Jesse Elliott & Bláthnaid McCoy & Tammy Clifford & Beth K. Potter & George A. Wells & Doug Coyle, 2020. "Economic Evaluation of Cannabinoid Oil for Dravet Syndrome: A Cost-Utility Analysis," PharmacoEconomics, Springer, vol. 38(9), pages 971-980, September.

    Corrections

    All material on this site has been provided by the respective publishers and authors. You can help correct errors and omissions. When requesting a correction, please mention this item's handle: RePEc:spr:eujhec:v:21:y:2020:i:4:d:10.1007_s10198-019-01149-9. See general information about how to correct material in RePEc.

    If you have authored this item and are not yet registered with RePEc, we encourage you to do it here. This allows to link your profile to this item. It also allows you to accept potential citations to this item that we are uncertain about.

    If CitEc recognized a bibliographic reference but did not link an item in RePEc to it, you can help with this form .

    If you know of missing items citing this one, you can help us creating those links by adding the relevant references in the same way as above, for each refering item. If you are a registered author of this item, you may also want to check the "citations" tab in your RePEc Author Service profile, as there may be some citations waiting for confirmation.

    For technical questions regarding this item, or to correct its authors, title, abstract, bibliographic or download information, contact: Sonal Shukla or Springer Nature Abstracting and Indexing (email available below). General contact details of provider: http://www.springer.com .

    Please note that corrections may take a couple of weeks to filter through the various RePEc services.

    IDEAS is a RePEc service. RePEc uses bibliographic data supplied by the respective publishers.