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The European Journal of Health Economics
Published in: The European Journal of Health Economics 1/2016

01-04-2016 | Original Paper

Social/economic costs and quality of life in patients with haemophilia in Europe

Authors: Marianna Cavazza, Yllka Kodra, Patrizio Armeni, Marta De Santis, Julio López-Bastida, Renata Linertová, Juan Oliva-Moreno, Pedro Serrano-Aguilar, Manuel Posada-de-la-Paz, Domenica Taruscio, Arrigo Schieppati, Georgi Iskrov, László Gulácsi, Johann Matthias Graf von der Schulenburg, Panos Kanavos, Karine Chevreul, Ulf Persson, Giovanni Fattore, BURQOL-RD Research Network

Published in: The European Journal of Health Economics | Special Issue 1/2016

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Abstract

Objective

The aim of this study was to determine the economic burden from a societal perspective and the health-related quality of life (HRQOL) of patients with haemophilia in Europe.

Methods

We conducted a cross-sectional study of patients with haemophilia from Bulgaria, France, Germany, Hungary, Italy, Spain Sweden and the UK. Data on demographic characteristics, health resource utilisation, informal care, loss of labour productivity and HRQOL were collected from the questionnaires completed by patients or their caregivers. HRQOL was measured with the EuroQol 5-domain (EQ-5D) questionnaire. The costs have been estimated from a societal perspective adopting a bottom-up approach.

Results

A total of 401 questionnaires were included in the study, of which 339 were collected from patients with haemophilia and 62 from caregivers. The lowest average annual cost per person was reported in Bulgaria (€6,660) and the highest in Germany (€194,490). Our results demonstrate both a large difference from country to country in the average annual cost per patient in 2012 and the driving role of drugs in costs. Drugs represent nearly 90 % of direct healthcare costs in a majority of the countries analysed (Hungary, Italy, Spain and Germany). In Bulgaria, France and Sweden, however, healthcare services (visits, tests and hospitalisations) prevail. Costs are also shown to differ between children and adults. The mean EQ-5D index score for adult patients was 0.69 and mean EQ-5D VAS was 66.6. The mean EQ-5D index score for carers was 0.87 and mean EQ-5D VAS was 75.5. In the disability score, 60 % showed no disability and measuring caregiver burden with the Zarit Index produced an overall mean score of 25.3.

Conclusion

We have shown that haemophilia is associated with a substantial economic burden and impaired HRQOL. Studies on cost of illness and HRQOL are important for haemophilia as the future of this disease is likely to change with the development of new innovative treatments. The introduction of these treatments will most likely impact future costs related to haemophilia.
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Literature
1.
Rosendal, F.R., Briet, E.: The increasing prevalence of haemophilia. Thromb. Haemostasis 63, 145 (1990)
2.
3.
Evatt, B.L.: Demographics of haemophilia in developing countries. Semin. Thromb. Hemostasis 31, 489–494 (2005)CrossRef
4.
Stonebraker, J.S., Bolton-Maggs, P.H.B., Soucie, M.J., et al.: A study of variations in the reported haemophilia A prevalence around the world. Haemophilia 16, 20–32 (2010)CrossRefPubMed
5.
DeJager, T., Pericleous, L., KoKot-Kierapa, M., et al.: Burden and management of factor XIII deficiency. Haemophilia 20, 733–740 (2014)CrossRef
6.
Sherry, D.D.: Avoiding the impact of muscolo-skeletal pain on quality of life in children with haemophilia. Orthop. Nurse 27, 103–108 (2008)CrossRef
7.
Antunes, S.V., Vicari, P., Cavalheiro, S., Bordin, J.O.: Intracranial haemorrhage among a population of haemophilic patients in Brazil. Haemophilia 9, 573–577 (2003)CrossRefPubMed
8.
Siddiqi, A.E., Ebrahim, S.H., Soucie, J.M., Parker, C.S., Atrash, H.K.: Burden of disease resulting from hemophilia in the U.S. Am. J. Prev. Med. 38(4 Suppl), S482–S488 (2010)CrossRefPubMed
9.
Nilsson, I.M., Berntorp, E., Löfqvist, T., Pettersson, H.: Twenty-five years’ experience of prophylactic treatment in severe haemophilia A and B. J. Intern. Med. 232, 25–32 (1992)CrossRefPubMed
10.
Berntorp, E., Boulyjenkov, V., Brettler, D., et al.: Modern treatment of haemophilia. Bull. World Health Organ. 73, 691–701 (1995)PubMedPubMedCentral
11.
12.
Manco-Johnson, M.J., Abshire, T.C., Shapiro, A.D., et al.: Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia. N. Engl. J. Med. 357, 535–544 (2007)CrossRefPubMed
13.
Santagostino, E., Lents, S.R., Busk, A.K., Regnault, A., Iorio, A.: Assessment of the impact of treatment on quality of life of patients with haemophilia A at different ages: insights from two clinical trials on turoctocog alfa. Haemophilia 20, 527–534 (2014)CrossRefPubMedPubMedCentral
14.
15.
van Dijk, K., Fischer, K., van der Born, J.G., Scheibel, E., Ingersley, J., van den Berg, H.M.: Can long-term prophylaxis for severe haemophilia be stopped in adulthood? Results from Denmark and the Netherlands. Br. J. Haematol. 130, 107–112 (2005)CrossRefPubMed
16.
Valentino, L.A., Mamonov, V., Hellmann, A., Prophylaxis Study Group, et al.: A randomized comparison of two prophylaxis regimens and a paired comparison of on-demand and prophylaxis treatments in hemophilia A management. J. Thromb. Haemost. 10, 359–367 (2012)CrossRefPubMedPubMedCentral
17.
von Mackensen, S., Gringeri, A., Siboni, S.M., et al.: Health-related quality of life and psychological well-being in elderly patients with haemophilia. Haemophilia 18, 345–352 (2012)CrossRef
18.
Tagliaferri, A., Rivolta, G.F., Iorio, A., et al.: Mortality and causes of death in Italian persons with haemophilia, 1990–2007. Haemophilia 16, 437–446 (2010)CrossRefPubMed
19.
Johnson, K., Zhou, Z.Y.: Costs of care in hemophilia and possible implications of health care reform. Hematology 1, 413–418 (2011)CrossRef
20.
Miners, A.H., Sabin, C.A., Tolley, K.H., et al.: Cost-utility analysis of primary prophylaxis versus treatment on-demand for individuals with severe haemophilia. Pharmacoeconomics 20, 759–774 (2002)CrossRefPubMed
21.
Miners, A.H.: Economic evaluations of prophylaxis with clotting factor for people with severe haemophilia: why do the results vary so much? Haemophilia 19, 174–180 (2013)CrossRefPubMed
22.
Carlsson, S.K., Höjgård, S., Glomstein, A., et al.: On-demand vs. prophylactic treatment for severe haemophilia in Norway and Sweden: differences in treatment characteristics and outcome. Haemophilia 9, 555–566 (2003)CrossRef
23.
Brown, S.A., Aledort, L.M.: The Round Table Group: economic challenges in haemophilia. Haemophilia 10(Suppl. 1), 44–49 (2004)
24.
Gringeri, A., Lundin, B., von Mackensen, S., Mantovani, L., Mannucci, P.M., ESPRIT Study Group: A randomized clinical trial of prophylaxis in children with hemophilia A (the ESPRIT Study). J. Thromb. Haemost. 9, 700–710 (2011)CrossRefPubMed
25.
Lindvall, K., von Mackensen, S., Berntorp, E.: Quality of life in adult patients with haemophilia—a single centre experience from Sweden. Haemophilia 18, 527–531 (2012)CrossRefPubMed
26.
Carvalhosa, M., Henrard, S., Lambert, C., Hermans, C.: Physical and mental quality of life in adult patients with haemophilia in Belgium: the impact of financial issues. Haemophilia 20, 479–485 (2014)CrossRefPubMed
27.
Royal, S., Schramm, W., Berntorp, E., Giangrande, P., Gringeri, A., Ludlam, C., Kroner, B., Szucs, T., European haemophilia economics study group: Quality-of-life differences between prophylactic and on-demand factor replacement therapy in European haemophilia patients. Haemophilia 8, 44–50 (2002)CrossRefPubMed
28.
Fischer, K., van der Bom, J.G., Molho, P.: Prophylactic versus on-demand treatment strategies for severe haemophilia: a comparison of costs and long-term outcome. Haemophilia 8, 745–752 (2002)CrossRefPubMed
29.
Klamroth, R., Pollmann, H., Hermans, C.: The relative burden of haemophilia A and the impact of target joint develop on health related quality of life: results from the ADVATE post-authorisation safety surveillance (PASS) study. Haemophilia 17, 412–421 (2011)CrossRefPubMed
30.
Henrard, S., Devleesschauwer, B., Beutels, P., Callens, M.: The health and economic burden of haemophilia in Belgium: a rare, expensive and challenging disease. Orphanet J Rare Dis 9, 39 (2014)CrossRefPubMedPubMedCentral
31.
Nerich, V., Tissot, E., Faradji, A.: Cost-of-illness study of severe haemophilia A and B in five French haemophilia treatment centres. Pharm. World Sci. 30, 287–292 (2008)CrossRefPubMed
32.
Auerswald, G., Von Depka Prondzinski, M., Ehlken, B.: Treatment patterns and cost-of-illness of severe haemophilia in patients with inhibitors in Germany. Haemophilia 10, 499–508 (2004)CrossRefPubMed
33.
Gringeri, A., Mantovani, L.G., Scalone, L.: Cost of care and quality of life for patients with hemophilia complicated by inhibitors: the COCIS Study Group. Blood 102, 2358–2363 (2003)CrossRefPubMed
34.
35.
Drummond, M.F., O’Brien, B., Stoddart, G.L., Torrance, G.W.: Methods for the economic evaluation of health care programmes, 2nd edn. Oxford University Press, Oxford (1997)
36.
McDaid, D.: Estimating the costs of informal care for people with Alzheimer’s disease: methodological and practical challenges. Int. J. Geriatr. Psychiatry 16, 400–520 (2001)CrossRefPubMed
37.
van den Berg, B., Brouwer, W., Koopmanschap, M.: Economic valuation of informal care: an overview of methods and applications. Eur. J. Health Econ. 5, 36–45 (2004)CrossRefPubMed
38.
Hodgson, T.A., Meiners, M.R.: Cost-of-illness methodology: a guide to assessment practices and procedures. Milbank Mem. Fund Q. 60, 429–491 (1982)CrossRef
39.
Dolan, P.: Modeling valuations for EuroQol health states. Med. Care 735, 1095–1108 (1997)CrossRef
40.
Szende, A., Janssen, B., Cabases, J. (eds.): Self-reported population health: an international perspective based on EQ-5D. Springer, Netherlands (2014)
41.
Mahoney, F.I., Barthel, D.W.: Functional evaluation: the Barthel index. Md. State Med. 14, 61–65 (1965)
42.
Shah, S., Vanclay, F., Cooper, B.: Improving the sensitivity of the Barthel index for stroke rehabilitation. J. Clin. Epidemiol. 42, 703–709 (1989)CrossRefPubMed
43.
Hérbert, R., Bravo, G., Préville, M.: Reliability, validity, and reference values of the Zarit Burden Interview for assessing informal caregivers of community-dwelling older persons with dementia. Can. J. Aging 19, 494–507 (2000)CrossRef
44.
O’Mahony, B., Noone, D., Giangrande, P.L.F., Prihodova, L.: Haemophilia care in Europe—a survey of 35 countries. Haemophilia 2013(19), e239–e247 (2013)CrossRef
45.
O’Mahony, B., Noone, D., Giangrande, P.L.F., Prihodova, L.: Haemophilia care in Europe: a survey of 19 countries. Haemophilia 17, 35–40 (2011)CrossRefPubMed
46.
Pocoski, J., Benjamin, K., Michaels, L.A., Flood, E., Sasane, R.: An overview of current trends and gaps in patient-reported outcome measures used in haemophilia. Eur. J. Haematol. 93 Suppl 75, 1–8 (2014)CrossRefPubMed
47.
Tarricone, R.: Cost-of-illness analysis: what room in health economics? Health Policy 77, 51–63 (2006)CrossRefPubMed
48.
Drummond, M., Sculpher, M.J., Torrance, G.W., et al.: Methods for economic evaluation of health care programmes, 3rd edn. Oxford University Press, Oxford (2005)
49.
50.
Taruscio, D., Gentile, A.E., Evangelista, T., Frazzica, R.G., Bushby, K.: Centres of Expertise and European Reference Networks: key issues in the field of rare diseases. The EUCERD recommendations. Blood Transfus. Suppl 3, 621–625 (2014)
Metadata
Title
Social/economic costs and quality of life in patients with haemophilia in Europe
Authors
Marianna Cavazza
Yllka Kodra
Patrizio Armeni
Marta De Santis
Julio López-Bastida
Renata Linertová
Juan Oliva-Moreno
Pedro Serrano-Aguilar
Manuel Posada-de-la-Paz
Domenica Taruscio
Arrigo Schieppati
Georgi Iskrov
László Gulácsi
Johann Matthias Graf von der Schulenburg
Panos Kanavos
Karine Chevreul
Ulf Persson
Giovanni Fattore
BURQOL-RD Research Network
Publication date
01-04-2016
Publisher
Springer Berlin Heidelberg
Published in
The European Journal of Health Economics / Issue Special Issue 1/2016
Print ISSN: 1618-7598
Electronic ISSN: 1618-7601
DOI
https://doi.org/10.1007/s10198-016-0785-2

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