Hostname: page-component-78c5997874-lj6df Total loading time: 0 Render date: 2024-11-15T07:57:19.613Z Has data issue: false hasContentIssue false
  • English
  • Français

Hemophilia Home Treatment: Economic Analysis and Implications for Health Policy

Published online by Cambridge University Press:  10 March 2009

Dennis Ross-Degnan ,
Stephen B. Soumerai ,
Jerry Avorn ,
Rhonda L. Bohn ,
Rosalie Bright  and
Louis M. Aledort
Dennis Ross-Degnan
Affiliation:
Harvard Medical School and Harvard Community Health Plan
Stephen B. Soumerai
Affiliation:
Harvard Medical School and Harvard Community Health Plan
Jerry Avorn
Affiliation:
Harvard Medical School and Women's Hospital
Rhonda L. Bohn
Affiliation:
Harvard Medical School and Women's Hospital
Rosalie Bright
Affiliation:
U.S. Food and Drug Administration
Louis M. Aledort
Affiliation:
Mount Sinai Medical Center
Get access Rights & Permissions [Opens in a new window]

Abstarct

This analysis describes the development of technology for home self-infusion of factor VIII in the treatment of hemophilia and its clinical, economic, and social consequences, and uses the case study of such home care treatment to illustrate the potentials and pitfalls of formal economic analyses of programs to treat chronically ill children. A comprehensive review of all original data on hemophilia programs, their related costs, and outcomes, conducted from 1966 through 1993, examined the economic outcomes for two hypothetical cohorts, one aged 0–4 years and the other aged 30–34 years. Including the measurement of treatment effects on the productivity of parental caregivers substantially increases the benefit-cost relationship of an intervention directed at chronically ill children. Increased economic productivity and societal return resulting from such a program for young adults exceeds those for a cohort of children, primarily due to assumptions related to discounting. However, estimation of quality-adjusted life years favors the younger age cohort, since children survive for a longer period of time and with each year survived comes a higher quality of life. Unlike simpler instances in which economic benefits can be shown to outweigh resource costs, policy decisions concerning services for chronically ill children raise an additional set of complex analytic issues. Inclusion of the benefits in productivity experienced by family caregivers provides an important added dimension to such analyses. The development of cost-benefit or cost-effectiveness analyses of these programs illustrates the importance of careful measurement of outcomes and explicit statements of underlying assumptions. Such an analysis of home care for children with hemophilia therefore demonstrates both the strengths and the limitations of this approach.

Type
General Essays
Information
International Journal of Technology Assessment in Health Care , Volume 11 , Issue 2 , Spring 1995 , pp. 327 - 344
Copyright
Copyright © Cambridge University Press 1995

Access options

Get access to the full version of this content by using one of the access options below. (Log in options will check for institutional or personal access. Content may require purchase if you do not have access.)

References

REFERENCES

1.Aledort, L. M.Lesson from hemophilia (editorial). New England Journal of Medicine, 1982, 306, 607–08.CrossRefGoogle Scholar
2.Aledort, L. M., & Branch., I. Cost of care for hemophiliacs. In Hilgartner, M. and Pochedley, C. (eds.), Hemophilia in the child and adult. New York: Raven Press Ltd., 1989, 137–47.Google Scholar
3.Aledort, L. M., Cohen, M., Hilgartner, M. W., & Lipton., R. Treatment of hemophiliacs with inhibitors: Cost and effect on blood resources. In Hoyer, L. (ed.), Factor VIII inhibitors. New York: Alan R. Liss, Inc., 1984, 353–65.Google Scholar
4.Avorn, J.Benefit and cost analysis in geriatric care: Turning age discrimination into health policy. New England Journal of Medicine, 1984, 10, 1294–301.CrossRefGoogle Scholar
5.Berwick, D. M., Cretin, S., & Keeler, E. B.Cholesterol, children, and heart disease: An analysis of alternatives. New York: Oxford University Press, 1980.Google Scholar
6.Boon, R. A., & Roberts, D. F.The social impact of haemophilia. Journal of Biosocial Science, 1970, 2, 237–64.CrossRefGoogle ScholarPubMed
7.Braunwald, E., Isselbacher, K. J., Petersdorf, R. G., et al. (eds.), Harrison's principles of internal medicine, 11th ed.New York: McGraw-Hill Book Company, 1987.Google Scholar
8.Bronks, I. G., & Blackburn, E. K. A socio-medical study of haemophilia and related states. British Journal of Preventative Social Medicine, 1968, 22, 68–72.Google Scholar
9.Butler, J. A., Budetti, P., McManus, M. A., et al. Health care expenditures for children with chronic illnesses. In Hobbs, N. and Perrin, J. M.. Issues in the care of children with chronic illness. San Francisco: Jossey-Bass Publishers, 1985.Google Scholar
10.Cook, T. D., & Campbell, D. T.Quasi–experimentation: Design and analysis issues for field settings. Boston: Houghton-Mifflin, 1979.Google Scholar
11.Dinkel, R. H.Cost-benefit analysis: A helpful tool for decision makers? Health Policy, 1985, 4, 321–30.CrossRefGoogle ScholarPubMed
12.Eyster, M. E., Lewis, J. H., Shapiro, S. S., et al. The Pennsylvania Hemophilia Program 1973–1978. American Journal of Hematology, 1980, 9, 277–86.CrossRefGoogle ScholarPubMed
13.Fundenberg, H. H.The dollar benefits of biomedical research: A cost analysis. Journal of Laboratory and Clinical Medicine, 1972, 79, 353–63.Google Scholar
14.Griffiths, A.Cost-effectiveness and cost-benefit analysis of health services: The methodology and its application. Health Policy, 1988, 9, 251–65.CrossRefGoogle ScholarPubMed
15.Hernandez, J., Gray, D., & Lineberger, H. P.Social and economic indicators of well-being among hemophiliacs over a 5-year period. General Hospital Psychiatry, 1989, 11, 241–47.CrossRefGoogle ScholarPubMed
16.Hilgartner, M. W., Aledort, L., & Giardina, P. J. V. Thalassemia and hemophilia. In Hobbs, N. and Perrin, J. M. (eds.), Issues in the care of children with chronic illness. San Francisco: Jossey-Bass Publishers, 1985.Google Scholar
17.Hilgartner, M. W., & Sergis, E.Current therapy for hemophiliacs: Home care and therapeutic complications. Mount Sinai Journal of Medicine, 1977, 44, 316–31.Google ScholarPubMed
18.Ingram, G. I. C., Dykes, S. R., Creese, A. L., et al. Home treatment in haemophilia: Clinical, social and economic advantages. Clinical and Laboratory Haematology, 1979, 1, 13–27.CrossRefGoogle ScholarPubMed
19.Johnson, R. E., Lawrence, D. N., Evatt, B. L., et al. Acquired immunodeficiency syndrome among patients attending hemophilia treatment centers and mortality experience of hemophiliacs in the United States. American Journal of Epidemiology, 1985, 121, 797–810.CrossRefGoogle ScholarPubMed
20.Jones, P. K., & Ratnoff, O. D.The changing prognosis of classic hemophilia (factor VIII deficiency). Annals of Internal Medicine, 1991, 114, 641–48.CrossRefGoogle ScholarPubMed
21.Klarman, H. E.Application of cost-benefit analysis to health systems technology. Journal of Occupational Medicine, 1974, 16, 172–86.Google ScholarPubMed
22.Koop, C. E.Surgeon General's report: children with special health care needs, campaign 87, commitment to family centered coordinated care for children with special health car needs. Rockville, MD: U.S. Department of Health and Human Services, 1987.Google Scholar
23.Levine, P. H.Delivery of health care in hemophilia. Annals of the New York Academy of Sciences, 1975, 240, 201–07.CrossRefGoogle ScholarPubMed
24.Linney, D. R., & Lazerson, J.Hemophilia: Cost considerations for prescribing therapeutic materials. Transfusion, 1979, 19, 57–59.CrossRefGoogle ScholarPubMed
25.Markova, I., Forbes, C. D., Rowlands, A., et al. The haemophilic patient’s self-perception of changes in health and life-style arising from self-treatment. International Journal of Rehabilitation Research, 1983, 6, 11–18.CrossRefGoogle ScholarPubMed
26.McMillan, C. W., Greene, W. B., Blatt, P. M., et al. The management of musculoskeletal problems in hemophilia. Instructors Course Lectures, 1983, 32, 210–16.Google ScholarPubMed
27.Newacheck, P. W.The costs of caring for chronically ill children. Business and Health, 1987, 4, 18–24.Google ScholarPubMed
28.Perry, M.C.Mortality and morbidity in hemophilia. Missouri Medicine, 1984, 81,133–35.Google ScholarPubMed
29.Roberts, H. R.Hemophiliacs with inhibitors: Therapeutic options (editorial). New England Journal of Medicine, 1981, 305, 757–58.CrossRefGoogle Scholar
30.Rosendaal, F. R., Smith, C., & Briet, E.Hemophilia treatment in historical perspective: A review of medical and social developments. Annals of Hematology, 1991, 62, 5–15.CrossRefGoogle ScholarPubMed
31.Rosendaal, F. R., Varekamp, I., Smith, C., et al. Mortality and causes of death in Dutch haemophiliacs, British Journal of Haematology, 1989, 71, 71–76.CrossRefGoogle ScholarPubMed
32.Rossi, P. H., Freeman, H. E., & Wright, S. R.Evaluation: A systemic approach. Beverly Hills, Sage Publications, Inc., 1979.Google Scholar
33.Salkever, D. S. Parental opportunity costs and other economic costs of children’s disabling conditions. In Hobbs, N. and Perrin, J. M. (eds.), Issues in the care of children with chronic illness. San Francisco: Jossey-Bass Publishers, 1985.Google Scholar
34.Sjamsoedin, L. J. M., Heijnen, L., Mauser-Bunschoten, E. P., et al. The effect of activated prothrombin-complex concentrate (FEIBA) on joint and muscle bleeding in patients with hemophilia A and antibodies to factor VIII. New England Journal of Medicine, 1981, 305, 717–21.CrossRefGoogle ScholarPubMed
35.Smith, C., Rosendaal, F. R., Varekamp, I., et al. Physical condition, longevity, and social performance of Dutch haemophiliacs, 1972–1985. British Medical Journal, 1989, 298, 235–38.CrossRefGoogle Scholar
36.Smith, P. S., & Goldman, D. S.Care of the young hemophiliac. American Journal of Pediatric Hematology and Oncology, 1985, 7, 165–74.Google ScholarPubMed
37.Smith, P. S., Keyes, N. C., & Forman, E. N.Socioeconomic evaluation of a state-funded comprehensive hemophilia-care program. New England Journal Medicine, 1982, 306, 575–79.CrossRefGoogle ScholarPubMed
38.Stason, W. B., & Weinstein, M. C.Allocation of resources to manage hypertension. New England Journal of Medicine, 1977, 296, 732–39.CrossRefGoogle ScholarPubMed
39.Steinbrook, R., & Lo, B.The Oregon Medicaid Demonstration Project—Will it provide adequate medical care? New England Journal of Medicine, 1992, 326, 340–44.CrossRefGoogle ScholarPubMed
40.Strawczynski, H., Stachewitsch, A., Morgenstern, G., & Shaw, M. E.Delivery of care to hemophilic children: Home care versus hospitalization. Pediatrics, 1973, 51, 986–91.CrossRefGoogle ScholarPubMed
41.Stuart, J., Forbes, C. D., Jones, P., et al. Improving prospects for employment of the haemophiliac. British Medical Journal, 1980, 280, 1169–72.CrossRefGoogle ScholarPubMed
42.Thompson, M. S.Benefit-cost analysis for program evaluation. Beverly Hills: Sage Publications, Inc., 1980.Google Scholar
43. U.S. Bureau of Economic Analysis. No. 678. Personal income and its disposition: 1970 to 1986. The national income and product accounts of the United States, 1929–1982, and survey of current business, July 1987. In U.S. Bureau of the Census, Statistical Abstract of the United States: 1988, 108th ed.Washington, DC: Government Printing Office, 1987.Google Scholar
44. U.S. Bureau of Labor Statistics. No. 730. Annual percent change in selected price indexes: 1960 to 1986. Monthly Labor Review. In U.S. Bureau of the Census, Statistical Abstract of the United States: 1988,108th ed.Washington, DC: Government Printing Office, 1987.Google Scholar
45.Weighton, W.Mortality in hemophiliac populations. Transactions of the Association of Life Insurance Medical Directors of America, 1982, 66, 129–29.Google Scholar
46.Weinstein, M. C., Fineberg, H. V.Clinical Decision Analysis. Philadelphia: W. B. Saunders Company, 1980.Google Scholar
47.Weinstein, M. C., & Stason, W. B.Foundations of cost-effectiveness analysis for health and medical practices. New England Journal of Medicine, 1977, 296, 716–21.CrossRefGoogle ScholarPubMed
48.Wilson, R. B.The Missouri Hemophilia Program. Missouri Medicine, 1984, 81, 130–32, 135.Google ScholarPubMed