Making economic evaluations more helpful for treatment choices in haemophilia

Michael Frank Drummond, N. Houwing, U. Slothuus, P. Giangrande

Research output: Contribution to journalReview articlepeer-review


Aim: Poorly conducted economic evaluations have the potential to mislead both clinicians, leading to inappropriate treatment choices, and payers who must decide on the reimbursement of treatment costs. This paper reviews the methods used in economic evaluations in haemophilia and proposes standards for conducting and reporting such evaluations in the future. Methods: A systematic review of economic evaluations in haemophilia published since 2008 was conducted. The reporting and methods of the studies were assessed using the recently published Consolidated Health Economic Evaluation Reporting Guidelines (CHEERS) checklist. The key methodological deficiencies in the studies were recorded. Results: Twenty-one studies met the inclusion criteria, classified as follows: prophylaxis vs. treatment on-demand (five studies); use of bypassing therapy (six); immune tolerance induction (four); and other topics (six). In general, the quality of reporting was good. However, it was poorest for the CHEERS item of patient heterogeneity, with most studies lacking discussion of heterogeneity in the patient population. The main recurring methodological deficiencies were the evaluation of single episodes of care rather than entire treatment strategies; inadequate control for confounders when comparing treatment options; the frequent use of expert opinion to determine drug doses and treatment patterns; lack of consideration of patient heterogeneity; failure to identify patient subgroups; and the inadequate exploration of uncertainty in estimates. Conclusions: A set of twelve standards for future reporting and conduct of economic evaluations within haemophilia is proposed, with the objective of making such evaluations more relevant and reliable for those making treatment and reimbursement decisions in the future.
Original languageEnglish
Pages (from-to)e58-e66
Number of pages9
JournalHaemophilia Journal
Issue number2
Early online date22 Jan 2017
Publication statusE-pub ahead of print - 22 Jan 2017

Bibliographical note

© 2017 John Wiley & Sons Ltd. This is an author-produced version of the published paper. Uploaded in accordance with the publisher’s self-archiving policy. Further copying may not be permitted; contact the publisher for details.


  • bypassing therapy
  • cost-effectiveness analysis
  • haemophilia
  • immune intolerance induction
  • methodological standards
  • prophylaxis

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