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dc.contributor.authorO’Hara, Jamie*
dc.contributor.authorHughes, David*
dc.contributor.authorCamp, Charlotte*
dc.contributor.authorBurke, Tom*
dc.contributor.authorCarroll, Liz*
dc.contributor.authorDiego, Daniel-Anibal G.*
dc.date.accessioned2017-06-01T09:23:43Z
dc.date.available2017-06-01T09:23:43Z
dc.date.issued2017-05-31
dc.identifier.citationO’Hara, J., et. al. (2017). The cost of severe haemophilia in Europe: the CHESS study. Orphanet Journal of Rare Diseases, 12(1), 106. DOI: 10.1186/s13023-017-0660-y
dc.identifier.doi10.1186/s13023-017-0660-y
dc.identifier.urihttp://hdl.handle.net/10034/620521
dc.description.abstractBackground Severe haemophilia is associated with major psychological and economic burden for patients, caregivers, and the wider health care system. This burden has been quantified and documented for a number of European countries in recent years. However, few studies have taken a standardised methodology across multiple countries simultaneously, and sought to amalgamate all three levels of burden for severe disease. The overall aim of the ‘Cost of Haemophilia in Europe: a Socioeconomic Survey’ (CHESS) study was to capture the annualised economic and psychosocial burden of severe haemophilia in five European countries. A cross-section of haemophilia specialists (surveyed between January and April 2015) provided demographic and clinical information and 12-month ambulatory and secondary care activity for patients via an online survey. In turn, patients provided corresponding direct and indirect non-medical cost information, including work loss and out-of-pocket expenses, as well as information on quality of life and adherence. The direct and indirect costs for the patient sample were calculated and extrapolated to population level. Results Clinical reports for a total of 1,285 patients were received. Five hundred and fifty-two patients (43% of the sample) provided information on indirect costs and health-related quality of life via the PSC. The total annual cost of severe haemophilia across the five countries for 2014 was estimated at EUR 1.4 billion, or just under EUR 200,000 per patient. The highest per-patient costs were in Germany (mean EUR 319,024) and the lowest were in the United Kingdom (mean EUR 129,365), with a study average of EUR 199,541. As expected, consumption of clotting factor replacement therapy represented the vast majority of costs (up to 99%). Indirect costs are driven by patient and caregiver work loss. Conclusions The results of the CHESS study reflect previous research findings suggesting that costs of factor replacement therapy account for the vast majority of the cost burden in severe haemophilia. However, the importance of the indirect impact of haemophilia on the patient and family should not be overlooked. The CHESS study highlights the benefits of observational study methodologies in capturing a ‘snapshot’ of information for patients with rare diseases.
dc.language.isoenen
dc.publisherBioMed Centralen
dc.relation.urlhttp://ojrd.biomedcentral.com/articles/10.1186/s13023-017-0660-yen
dc.rights.urihttp://creativecommons.org/licenses/by/4.0/en
dc.subjectHaemophiliaen
dc.subjectHealth Economicsen
dc.titleThe cost of severe haemophilia in Europe: the CHESS studyen
dc.typeArticleen
dc.identifier.eissn1750-1172
dc.contributor.departmentUniversity of Chester; HCD Economics, Daresbury; The Haeomophelia Society, London; FedHemo, Madrid
dc.identifier.journalOrphanet Journal of Rare Diseases
dc.language.rfc3066en
dc.rights.holderThe Author(s).
dc.date.updated2017-06-01T03:45:24Z
dc.date.accepted2017-05-22
or.grant.openaccessYesen
rioxxterms.funderUnknownen
rioxxterms.identifier.projectUnknownen
rioxxterms.versionVoRen
rioxxterms.licenseref.startdate2017-05-31
refterms.dateFCD2019-07-16T13:39:48Z
refterms.versionFCDVoR
refterms.dateFOA2018-08-13T14:41:20Z
html.description.abstractBackground Severe haemophilia is associated with major psychological and economic burden for patients, caregivers, and the wider health care system. This burden has been quantified and documented for a number of European countries in recent years. However, few studies have taken a standardised methodology across multiple countries simultaneously, and sought to amalgamate all three levels of burden for severe disease. The overall aim of the ‘Cost of Haemophilia in Europe: a Socioeconomic Survey’ (CHESS) study was to capture the annualised economic and psychosocial burden of severe haemophilia in five European countries. A cross-section of haemophilia specialists (surveyed between January and April 2015) provided demographic and clinical information and 12-month ambulatory and secondary care activity for patients via an online survey. In turn, patients provided corresponding direct and indirect non-medical cost information, including work loss and out-of-pocket expenses, as well as information on quality of life and adherence. The direct and indirect costs for the patient sample were calculated and extrapolated to population level. Results Clinical reports for a total of 1,285 patients were received. Five hundred and fifty-two patients (43% of the sample) provided information on indirect costs and health-related quality of life via the PSC. The total annual cost of severe haemophilia across the five countries for 2014 was estimated at EUR 1.4 billion, or just under EUR 200,000 per patient. The highest per-patient costs were in Germany (mean EUR 319,024) and the lowest were in the United Kingdom (mean EUR 129,365), with a study average of EUR 199,541. As expected, consumption of clotting factor replacement therapy represented the vast majority of costs (up to 99%). Indirect costs are driven by patient and caregiver work loss. Conclusions The results of the CHESS study reflect previous research findings suggesting that costs of factor replacement therapy account for the vast majority of the cost burden in severe haemophilia. However, the importance of the indirect impact of haemophilia on the patient and family should not be overlooked. The CHESS study highlights the benefits of observational study methodologies in capturing a ‘snapshot’ of information for patients with rare diseases.


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