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

Henrard, Séverine [UCL] Devleesschauwer, Brecht [Gent Universiteit] Beutels, Philippe [Universiteit Antwerpen] Callens, Michael [Mutualités chrétiennes] De Smet, Frank [Mutualités chrétiennes] 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 disability-adjusted life years (DALYs) lost, this being the number of lost healthy years due to both disability and premature mortality. 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 euro. Data was drawn from the literature if none was available from federal institutions or health insurance. Disability weights for DALY calculation were derived using generic quality-of-life tools such as 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 healthy years lost due to morbidity, invalidity or early death (or DALYs) in total (95% credible interval [CrI]=90-222), which represents an average of 11 DALYs per incident case with haemophilia (PWH) (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 euro per PWH, 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 on an individual patient level and especially for severe PWH. Initiatives to counteract this burden should be clearly identified and given full support, as this burden is likely to further increase in the future, especially from an economic perspective. Our study may also contribute towards a better global evaluation of haemophilia in the future.