Revisiting the cost‐effectiveness of primary prophylaxis with clotting factor for the treatment of severe haemophilia A
- 24 June 2009
- journal article
- Published by Wiley in Haemophilia
- Vol. 15 (4), 881-887
- https://doi.org/10.1111/j.1365-2516.2009.02019.x
Abstract
Severe haemophilia A is a lifelong condition that requires treatment with exogenous clotting factor. While primary prophylaxis is the clinically preferred method of delivering treatment, its provision is costly. A 2002 evaluation of primary prophylaxis suggested an incremental cost-effectiveness of approximately 50,000 pounds per additional quality-adjusted life-year (QALY). However, since this time, preferable evaluative methods have been developed and means of assessing the value of future research also now exist. Thus, the primary aims of this study were to update a previously published cost-effectiveness analysis of primary prophylaxis vs. treating on-demand in terms of methods and to estimate the value of undertaking further primary research. The base case incremental cost-effectiveness ratio was shown to be approximately 37,000 pounds, 10,000 pounds lower than the value published in 2002. The main reason for this difference was the use of different structural assumptions and methods to fit the various model parameters. At a willingness to pay per additional QALY threshold of 30,000 pounds, the probability prophylaxis is cost-effective was 13%. However, this increased to over 90% when alternative structural assumptions were employed, such as the rate at which future QALYs are discounted. The value of further research to increase the precision of this newly calculated cost-effectiveness estimate was high at a threshold willingness to pay values of 30,000- 40,000 pounds per QALY, particularly for the utilities associated with the health states. Thus, there is considerable value in conducting further primary research related to economic aspects of primary prophylaxis.Keywords
This publication has 19 references indexed in Scilit:
- Decision analysis for resource allocation in health careJournal of Health Services Research & Policy, 2008
- Guideline on the selection and use of therapeutic products to treat haemophilia and other hereditary bleeding disordersHaemophilia, 2008
- Cost‐utility analysis of Canadian tailored prophylaxis, primary prophylaxis and on‐demand therapy in young children with severe haemophilia AHaemophilia, 2008
- Prophylactic Treatment for Prevention of Joint Disease in Hemophilia — Cost versus BenefitNew England Journal of Medicine, 2007
- Willingness to pay for on‐demand and prophylactic treatment for severe haemophilia in SwedenHaemophilia, 2004
- Costs of on‐demand and prophylactic treatment for severe haemophilia in Norway and SwedenHaemophilia, 2004
- Probabilistic Analysis of Cost-Effectiveness Models: Choosing between Treatment Strategies for Gastroesophageal Reflux DiseaseMedical Decision Making, 2002
- Primary prophylaxis for individuals with severe haemophilia: how many hospital visits could treatment prevent?Journal of Internal Medicine, 2000
- Assessing health-related quality-of-life in patients with severe haemophilia A and BPsychology, Health & Medicine, 1999
- Twenty‐five years' experience of prophylactic treatment in severe haemophilia A and BJournal of Internal Medicine, 1992