IDEAS home Printed from https://ideas.repec.org/a/spr/eujhec/v21y2020i2d10.1007_s10198-019-01122-6.html
   My bibliography  Save this article

Methodology and results of real-world cost-effectiveness of carfilzomib in combination with lenalidomide and dexamethasone in relapsed multiple myeloma using registry data

Author

Listed:
  • M. Campioni

    (Economic Modeling Center of Excellence, Global Health Economics, Amgen (Europe) GmbH)

  • I. Agirrezabal

    (Economic Modeling Center of Excellence, Global Health Economics, Amgen (Europe) GmbH)

  • R. Hajek

    (University of Ostrava)

  • J. Minarik

    (Palacky University Olomouc)

  • L. Pour

    (University Hospital Brno and Faculty of Medicine Masaryk Universit)

  • I. Spicka

    (Charles University in Prague, First Faculty of Medicine and General Teaching Hospital)

  • S. Gonzalez-McQuire

    (Global Health Economics, Amgen (Europe) GmbH)

  • P. Jandova

    (Amgen s.r.o)

  • V. Maisnar

    (Charles University in Prague, First Faculty of Medicine and General Teaching Hospital)

Abstract

Objective To predict the real-world (RW) cost-effectiveness of carfilzomib in combination with lenalidomide and dexamethasone (KRd) versus lenalidomide and dexamethasone (Rd) in relapsed multiple myeloma (MM) patients after one to three prior therapies. Methods A partitioned survival model that included three health states (progression-free, progressed disease and death) was built. Progression-free survival (PFS), overall survival (OS) and time to discontinuation (TTD) data for the Rd arm were derived using the Registry of Monoclonal Gammopathies in the Czech Republic; the relative treatment effects of KRd versus Rd were estimated from the phase 3, randomised, ASPIRE trial, and were used to predict PFS, OS and TTD for KRd. The model was developed from the payer perspective and included drug costs, administration costs, monitoring costs, palliative care costs and adverse-event related costs collected from Czech sources. Results The base case incremental cost effectiveness ratio for KRd compared with Rd was €73,156 per quality-adjusted life year (QALY) gained. Patients on KRd incurred costs of €117,534 over their lifetime compared with €53,165 for patients on Rd. The QALYs gained were 2.63 and 1.75 for patients on KRd and Rd, respectively. Conclusions Combining the strengths of randomised controlled trials and observational databases in cost-effectiveness models can generate policy-relevant results to allow well-informed decision-making. The current model showed that KRd is likely to be cost-effective versus Rd in the RW and, therefore, the reimbursement of KRd represents an efficient allocation of resources within the healthcare system.

Suggested Citation

  • M. Campioni & I. Agirrezabal & R. Hajek & J. Minarik & L. Pour & I. Spicka & S. Gonzalez-McQuire & P. Jandova & V. Maisnar, 2020. "Methodology and results of real-world cost-effectiveness of carfilzomib in combination with lenalidomide and dexamethasone in relapsed multiple myeloma using registry data," The European Journal of Health Economics, Springer;Deutsche Gesellschaft für Gesundheitsökonomie (DGGÖ), vol. 21(2), pages 219-233, March.
  • Handle: RePEc:spr:eujhec:v:21:y:2020:i:2:d:10.1007_s10198-019-01122-6
    DOI: 10.1007/s10198-019-01122-6
    as

    Download full text from publisher

    File URL: http://link.springer.com/10.1007/s10198-019-01122-6
    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/s10198-019-01122-6?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. Kelly Fust & Xiaoyan Li & Michael Maschio & Guillermo Villa & Anju Parthan & Richard Barron & Milton C. Weinstein & Luc Somers & Caroline Hoefkens & Gary H. Lyman, 2017. "Cost-Effectiveness Analysis of Prophylaxis Treatment Strategies to Reduce the Incidence of Febrile Neutropenia in Patients with Early-Stage Breast Cancer or Non-Hodgkin Lymphoma," PharmacoEconomics, Springer, vol. 35(4), pages 425-438, April.
    2. Daniel Grima & Lisa Bernard & Elizabeth Dunn & Philip McFarlane & David Mendelssohn, 2012. "Cost-Effectiveness Analysis of Therapies for Chronic Kidney Disease Patients on Dialysis," PharmacoEconomics, Springer, vol. 30(11), pages 981-989, November.
    3. Neyt, Mattias & Cleemput, Irina & Thiry, Nancy & De Laet, Chris, 2012. "Calculating an intervention's (cost-)effectiveness for the real-world target population: The potential of combining strengths of both RCTs and observational data," Health Policy, Elsevier, vol. 106(2), pages 207-210.
    4. Patricia Guyot & Anthony E. Ades & Matthew Beasley & Béranger Lueza & Jean-Pierre Pignon & Nicky J. Welton, 2017. "Extrapolation of Survival Curves from Cancer Trials Using External Information," Medical Decision Making, , vol. 37(4), pages 353-366, May.
    5. Ruth Brown & Sean Stern & Sujith Dhanasiri & Steve Schey, 2013. "Lenalidomide for multiple myeloma: cost-effectiveness in patients with one prior therapy in England and Wales," The European Journal of Health Economics, Springer;Deutsche Gesellschaft für Gesundheitsökonomie (DGGÖ), vol. 14(3), pages 507-514, June.
    6. Richard Grieve & Neil Hawkins & Mark Pennington, 2013. "Extrapolation of Survival Data in Cost-effectiveness Analyses," Medical Decision Making, , vol. 33(6), pages 740-742, August.
    7. Chantal Gils & Saskia Groot & William Redekop & Miriam Koopman & Cornelis Punt & Carin Uyl-de Groot, 2013. "Real-World Cost-Effectiveness of Oxaliplatin in Stage III Colon Cancer: A Synthesis of Clinical Trial and Daily Practice Evidence," PharmacoEconomics, Springer, vol. 31(8), pages 703-718, August.
    8. Christopher Jackson & John Stevens & Shijie Ren & Nick Latimer & Laura Bojke & Andrea Manca & Linda Sharples, 2017. "Extrapolating Survival from Randomized Trials Using External Data: A Review of Methods," Medical Decision Making, , vol. 37(4), pages 377-390, May.
    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. Zhaojing Che & Nathan Green & Gianluca Baio, 2023. "Blended Survival Curves: A New Approach to Extrapolation for Time-to-Event Outcomes from Clinical Trials in Health Technology Assessment," Medical Decision Making, , vol. 43(3), pages 299-310, April.
    2. Sarah Gooding & I-Jun Lau & Mimi Sheikh & Pamela Roberts & Julia Wong & Emmy Dickens & Ash Bullement & Jamie Elvidge & Dawn Lee & Karthik Ramasamy, 2015. "Double Relapsed and/or Refractory Multiple Myeloma: Clinical Outcomes and Real World Healthcare Costs," PLOS ONE, Public Library of Science, vol. 10(9), pages 1-9, September.
    3. Taihang Shao & Mingye Zhao & Leyi Liang & Lizheng Shi & Wenxi Tang, 2023. "Impact of Extrapolation Model Choices on the Structural Uncertainty in Economic Evaluations for Cancer Immunotherapy: A Case Study of Checkmate 067," PharmacoEconomics - Open, Springer, vol. 7(3), pages 383-392, May.
    4. Fuhmei Wang & Jung-Der Wang & Yu-Wen Hung, 2018. "Universal health insurance, health inequality and oral cancer in Taiwan," PLOS ONE, Public Library of Science, vol. 13(10), pages 1-13, October.
    5. Jing-Shiang Hwang & Tsuey-Hwa Hu, 2020. "Later-Life Exposure to Moderate PM 2.5 Air Pollution and Life Loss of Older Adults in Taiwan," IJERPH, MDPI, vol. 17(6), pages 1-12, March.
    6. Ren-Yeong Huang & Yuh-Feng Lin & Sen-Yeong Kao & Yi-Shing Shieh & Jin-Shuen Chen, 2014. "A Retrospective Case-Control Analysis of the Outpatient Expenditures for Western Medicine and Dental Treatment Modalities in CKD Patients in Taiwan," PLOS ONE, Public Library of Science, vol. 9(2), pages 1-9, February.
    7. Hamraz Mokri & Ingelin Kvamme & Linda Vries & Matthijs Versteegh & Pieter Baal, 2023. "Future medical and non-medical costs and their impact on the cost-effectiveness of life-prolonging interventions: a comparison of five European countries," The European Journal of Health Economics, Springer;Deutsche Gesellschaft für Gesundheitsökonomie (DGGÖ), vol. 24(5), pages 701-715, July.
    8. Mathyn Vervaart & Eline Aas & Karl P. Claxton & Mark Strong & Nicky J. Welton & Torbjørn Wisløff & Anna Heath, 2023. "General-Purpose Methods for Simulating Survival Data for Expected Value of Sample Information Calculations," Medical Decision Making, , vol. 43(5), pages 595-609, July.
    9. Adrian Vickers, 2019. "An Evaluation of Survival Curve Extrapolation Techniques Using Long-Term Observational Cancer Data," Medical Decision Making, , vol. 39(8), pages 926-938, November.
    10. Daniel Gallacher & Peter Kimani & Nigel Stallard, 2022. "Biased Survival Predictions When Appraising Health Technologies in Heterogeneous Populations," PharmacoEconomics, Springer, vol. 40(1), pages 109-120, January.
    11. Nicholas R. Latimer & Kurt Taylor & Anthony J. Hatswell & Sophia Ho & Gabriel Okorogheye & Clara Chen & Inkyu Kim & John Borrill & David Bertwistle, 2024. "An Evaluation of an Algorithm for the Selection of Flexible Survival Models for Cancer Immunotherapies: Pass or Fail?," PharmacoEconomics, Springer, vol. 42(12), pages 1395-1412, December.
    12. Ash Bullement & Mark Edmondson-Jones & Patricia Guyot & Nicky J. Welton & Gianluca Baio & Matthew Stevenson & Nicholas R. Latimer, 2024. "MPES-R: Multi-Parameter Evidence Synthesis in R for Survival Extrapolation—A Tutorial," PharmacoEconomics, Springer, vol. 42(12), pages 1317-1327, December.
    13. Alexina J. Mason & Manuel Gomes & James Carpenter & Richard Grieve, 2021. "Flexible Bayesian longitudinal models for cost‐effectiveness analyses with informative missing data," Health Economics, John Wiley & Sons, Ltd., vol. 30(12), pages 3138-3158, December.
    14. Pieter Baal & Alec Morton & David Meltzer & Werner Brouwer, 2019. "Future unrelated medical costs need to be considered in cost effectiveness analysis," The European Journal of Health Economics, Springer;Deutsche Gesellschaft für Gesundheitsökonomie (DGGÖ), vol. 20(1), pages 1-5, February.
    15. Ash Bullement & Matthew D. Stevenson & Gianluca Baio & Gemma E. Shields & Nicholas R. Latimer, 2023. "A Systematic Review of Methods to Incorporate External Evidence into Trial-Based Survival Extrapolations for Health Technology Assessment," Medical Decision Making, , vol. 43(5), pages 610-620, July.
    16. M. A. Chaudhary & M. Edmondson-Jones & G. Baio & E. Mackay & J. R. Penrod & D. J. Sharpe & G. Yates & S. Rafiq & K. Johannesen & M. K. Siddiqui & J. Vanderpuye-Orgle & A. Briggs, 2023. "Use of Advanced Flexible Modeling Approaches for Survival Extrapolation from Early Follow-up Data in two Nivolumab Trials in Advanced NSCLC with Extended Follow-up," Medical Decision Making, , vol. 43(1), pages 91-109, January.
    17. Philip Cooney & Arthur White, 2023. "Direct Incorporation of Expert Opinion into Parametric Survival Models to Inform Survival Extrapolation," Medical Decision Making, , vol. 43(3), pages 325-336, April.
    18. Thomas Grochtdreis & Christian Brettschneider & Annemarie Wegener & Birgit Watzke & Steffi Riedel-Heller & Martin Härter & Hans-Helmut König, 2015. "Cost-Effectiveness of Collaborative Care for the Treatment of Depressive Disorders in Primary Care: A Systematic Review," PLOS ONE, Public Library of Science, vol. 10(5), pages 1-20, May.

    More about this item

    Keywords

    Carfilzomib; Multiple myeloma; Cost-effectiveness; Real world; ASPIRE; Registry of Monoclonal Gammopathies;
    All these keywords.

    JEL classification:

    • I19 - Health, Education, and Welfare - - Health - - - Other

    Statistics

    Access and download statistics

    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:2:d:10.1007_s10198-019-01122-6. 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.