Dataset Information

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

ABSTRACT:

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.

SUBMITTER: Henrard S

PROVIDER: S-EPMC3998193 | biostudies-literature | 2014 Mar

REPOSITORIES: biostudies-literature

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Publications

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

Henrard Séverine S Devleesschauwer Brecht B Beutels Philippe P Callens Michael M De Smet Frank F Hermans Cedric C Speybroeck Niko N

Orphanet journal of rare diseases 20140321

<h4>Background</h4>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.<h4>Methods</h4>A probabilistic model was developed in order to est ...[more]

PMID: 24655371

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Dataset Information

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

Background

Methods

Results

Conclusions

Publications

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

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