IDEAS home Printed from https://ideas.repec.org/a/spr/pharme/v43y2025i7d10.1007_s40273-025-01484-1.html
   My bibliography  Save this article

A Hemophilia Joint Health Score-Based Model for the Economic Evaluation of Hemophilia A Prophylaxis Interventions

Author

Listed:
  • Sam Hirniak

    (University of Waterloo)

  • Andrea N. Edginton

    (University of Waterloo)

  • Alfonso Iorio

    (McMaster University
    McMaster University)

  • William W. L. Wong

    (University of Waterloo)

Abstract

Background and Objective Hemophilia A is a costly, lifelong illness with multiple prophylaxis options. Previously, these options were assessed using a Peterson score-based model to simulate joint damage over time. This study built a model for the economic evaluation of hemophilia A with less socioeconomic selection bias utilizing the hemophilia joint health score (HJHS). Methods A mechanistically defined HJHS-based state-transition microsimulation model was implemented for the cost-utility analysis conducted over a lifetime horizon from a Canadian provincial Ministry of Health perspective, with a 1.5% discount rate on (costs and outcomes), to compare the following interventions: standard half-life (SHL), extended half-life (EHL), emicizumab, and efanesocotog alfa (EA). The health states are HJHS levels, waiting for surgery, postoperative time, and death. Individuals experience bleeds, joint bleeds (increasing the HJHS), and surgery in each health state. Disutilities include injections and postoperative time. Model validation included face validity, internal validity, comparison analysis, external validity, and predictive validity. Probabilistic analysis, pricing threshold analysis, and one-way scenario analyses were completed. Results EA showed lower levels of hospitalizations and surgeries and an improved joint damage experience in the simulation. However, EA was not cost-effective against emicizumab, which continued to be the most cost-effective intervention. Pricing threshold analysis indicated that a price decrease would be required for EA to dominate SHL (50% decrement) and emicizumab (55% decrement). Conclusions This is the first cost-effectiveness model incorporating HJHS to apply sequential joint damage to hemophilia A. While EA offers clinical benefits, our analysis suggests it will not be cost-effective from a Canadian provincial Ministry of Health perspective without a significant price decrease.

Suggested Citation

  • Sam Hirniak & Andrea N. Edginton & Alfonso Iorio & William W. L. Wong, 2025. "A Hemophilia Joint Health Score-Based Model for the Economic Evaluation of Hemophilia A Prophylaxis Interventions," PharmacoEconomics, Springer, vol. 43(7), pages 765-778, July.
    • Handle: RePEc:spr:pharme:v:43:y:2025:i:7:d:10.1007_s40273-025-01484-1
    DOI: 10.1007/s40273-025-01484-1
    as

    Download full text from publisher

    File URL: http://link.springer.com/10.1007/s40273-025-01484-1
    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/s40273-025-01484-1?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.

    More about this item

    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:pharme:v:43:y:2025:i:7:d:10.1007_s40273-025-01484-1. 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.

    We have no bibliographic references for this item. You can help adding them by using 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.