Health economic models in hemophilia A and utility assumptions from a clinician's perspective

Michele Valente, Paolo A. Cortesi, Giuseppe Lassandro, Prasad Mathew, Jennifer Pocoski, Angelo C. Molinari, Lorenzo G. Mantovani, Paola Giordano

Research output: Contribution to journalArticle

Abstract

Background: The clinical benefits of prophylaxis in patients with hemophilia are well-established and include the following: reduced bleeding episodes, prevention of joint damage, decreased inhibitor development, and improved health-related quality of life. However, the cost-effectiveness of prophylaxis is still not clear. Procedures: We reviewed the published hemophilia prophylaxis economic models focusing on utility assumptions. Results: We found six cost-utility studies that compared prophylaxis and on-demand regimens. These studies reported remarkably different results, using utility values based on different assumptions and data sources. Conclusions: We suggest that cooperation among key stakeholders (clinicians, patient organizations and health-care decision makers) as a means of collecting evidence-based and experiential data to represent both the utility and the quality of life changes for patients with Hemophilia A who are treated with prophylaxis or receive on-demand treatments may represent a winning strategy with which to resolve the outstanding issues related to health technology assessments in the care of patients with hemophilia. Pediatr Blood Cancer 2015;62:1826-1831.

Original languageEnglish
Pages (from-to)1826-1831
Number of pages6
JournalPediatric Blood and Cancer
Volume62
Issue number10
DOIs
Publication statusPublished - Oct 1 2015

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Keywords

  • Cost Utility Analysis (CUA)
  • Health Technology Assessment (HTA)
  • Health-related quality of life (HRQoL)
  • Hemophilia
  • Prophylaxis
  • Quality-adjusted life years (QALY)

ASJC Scopus subject areas

  • Oncology
  • Pediatrics, Perinatology, and Child Health
  • Hematology
  • Medicine(all)

Cite this

Valente, M., Cortesi, P. A., Lassandro, G., Mathew, P., Pocoski, J., Molinari, A. C., Mantovani, L. G., & Giordano, P. (2015). Health economic models in hemophilia A and utility assumptions from a clinician's perspective. Pediatric Blood and Cancer, 62(10), 1826-1831. https://doi.org/10.1002/pbc.25543