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The health and economic burden of haemophilia in Belgium: a rare, expensive and challenging disease

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Henrard, Séverine [UCL] Devleesschauwer, Brecht [UCL] Beutels, Philippe [University of Antwerp] Callens, Michael [National Alliance of Christian Sickness Funds] De Smet, Frank [National Alliance of Christian Sickness Funds] Hermans, Cedric [UCL] Speybroeck, Niko [UCL]

BACKGROUND: Haemophilia is a rare hereditary haemorrhagic disease that requires regular intravenous injections of clotting factor (CF) concentrates. This study sought to estimate the health and economic burden of haemophilia in Belgium. This is the first study of its type to be conducted, and reflects the Belgian authorities' growing interest for haemophilia as part of their priority planning for rare and chronic diseases. METHODS: A probabilistic model was developed in order to estimate the lifetime haemophilia burden for the 2011 birth-year Belgian cohort. The health burden was initially expressed in terms of disability-adjusted life years (DALYs), the number of healthy life years lost due to living with disability and dying prematurely. An incidence perspective was used in line with World Health Organization recommendations. The economic burden calculated from direct and indirect haemophilia-related costs was expressed in euros. Data were drawn from the literature if none were available from federal institutions or health insurance. Disability weights for DALY calculation were derived using generic quality-of-life tools such as SF-6D from the SF-36 (36-item Short-Form Health Survey; for adults) and KINDL (generic quality-of-life instrument; for children) compared to population norms. Analyses were stratified according to haemophilia type and severity. RESULTS: In Belgium, haemophilia resulted in 145 undiscounted and unweighted DALYs in total (95% credible interval [CrI] = 90-222), which represents an average of 11 DALYs per incident case with haemophilia (95% CrI = 8-15) during his life, varying according to haemophilia severity (17 DALYs for severe haemophilia, 12 DALYs for moderate, and 4 DALYs for mild). Mean total lifetime costs reached €7.8 million per people with haemophilia, 94.3% being direct costs and 5.7% indirect costs. Clotting factors accounted for 82.5% of direct costs. CONCLUSIONS: Haemophilia represents both an economic and health burden, especially regarding individual health on an individual patient level. Initiatives to counteract this burden should be clearly identified and given full support, as this burden is likely to increase in the future, especially from an economic perspective. Our study may also contribute towards a better global evaluation of haemophilia in the future.

Document type Article de périodique (Journal article) – Article de recherche
Access type Accès restreint
Publication date 2014
Language Anglais
Journal information "Orphanet Journal of Rare Diseases" - Vol. 9, no.1, p. 39 (2014)
Peer reviewed yes
Publisher BioMed Central Ltd. ((United Kingdom) London)
issn 1750-1172
e-issn 1750-1172
Publication status Publié
Affiliations UCL - SSS/IRSS - Institut de recherche santé et société
UCL - (SLuc) Service d'hématologie
UCL - SSS/IREC/CARD - Pôle de recherche cardiovasculaire
Keywords Burden ; Cost ; Haemophilia ; Disability-adjusted life year
Links
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Bibliographic reference Henrard, Séverine ; Devleesschauwer, Brecht ; Beutels, Philippe ; Callens, Michael ; De Smet, Frank ; et. al. The health and economic burden of haemophilia in Belgium: a rare, expensive and challenging disease. In: Orphanet Journal of Rare Diseases, Vol. 9, no.1, p. 39 (2014)
Permanent URL http://hdl.handle.net/2078.1/146929