, Volume 15, Issue 2, pp 179–195 | Cite as

Cost Effectiveness of Recombinant Human Insulin-Like Growth Factor I Therapy in Patients with ALS

  • Stacey J. Ackerman
  • Erin M. Sullivan
  • Kathleen M. Beusterien
  • Howard M. Natter
  • Deborah F. Gelinas
  • Donald L. Patrick
Original Research Article Cost Effectiveness of rhIGF-I Therapy in ALS


Objective: Amyotrophic lateral sclerosis (ALS) is a fatal, degenerative neuromuscular disease characterised by a progressive loss of voluntary motor activity. Recombinant human insulin-like growth factor I (rhIGF-I) has been shown to be useful in treating ALS. The purpose of this study was to examine the cost effectiveness of rhIGF-I therapy in patients who have ALS.

Design: We performed a cost-effectiveness analysis from the societal perspective on 177 patients who received treatment with rhIGF-I or placebo in a North American randomised clinical trial. We estimated the incremental cost-effectiveness ratio of rhIGF-I using resource utilisation and functional status measurements from the clinical trial. Costs were estimated from 1996 US Medicare reimbursement schedules. Utility weights were elicited from ALS healthcare providers using the standard gamble technique.

Main outcome measures and results: The overall cost per quality-adjusted lifeyear (QALY) gained for rhIGF-I therapy compared with placebo was $US67 440. For the subgroups of patients who were progressing rapidly or were in earlier stages of disease at enrolment, rhIGF-I cost $US52 823 and $US43 197 per QALY gained, respectively.

Conclusions: Treatment with rhIGF-I is most cost effective in ALS patients who are either in earlier stages of the disease or progressing rapidly. The cost effectiveness of rhIGF-I therapy compares favourably with treatments for other chronic progressive diseases.


Amyotrophic Lateral Sclerosis Adis International Limited Utility Weight Hospice Care Sporadic Amyotrophic Lateral Sclerosis 
These keywords were added by machine and not by the authors. This process is experimental and the keywords may be updated as the learning algorithm improves.


Unable to display preview. Download preview PDF.

Unable to display preview. Download preview PDF.


  1. 1.
    Incidence & Prevalence (Database Online). Mountain view: timely data resources. 1997; Nov 1996Google Scholar
  2. 2.
    Klein LM, Forshew DA. The economic impact of ALS. Neurology 1996; 47 Suppl. 2: 126S–9SCrossRefGoogle Scholar
  3. 3.
    Lewis ME, Neff NT, Contreras PC, et al. Insulin-like growth factor-I for treatment of motor neuronal disorders. Exp Neurol 1993; 124: 73–88PubMedCrossRefGoogle Scholar
  4. 4.
    D’Ercole AJ, Ye P, Calikoglu AS, et al. The role of insulin-like growth factors in the central nervous system. Mol Neurobiol 1996; 13: 227–55PubMedCrossRefGoogle Scholar
  5. 5.
    The Pink Sheet. FDC Reports 1996; June 24: T&G 2–3Google Scholar
  6. 6.
    Lai EC, Felice KJ, Festoff BW, et al. Effect of recombinant human insulin-like growth factor I on progression of ALS: a placebo-controlled study. Neurology 1997; 49: 1621–30PubMedCrossRefGoogle Scholar
  7. 7.
    Oddone EZ, Cowper P, Hamilton JD, et al. Cost effectiveness analysis of early zidovudine treatment of HIV infected patients. BMJ 1993; 307: 1322–5PubMedCrossRefGoogle Scholar
  8. 8.
    Garner TI, Dardis R. Cost-effectiveness analysis of end-stage renal disease treatments. Med Care 1987; 25–34Google Scholar
  9. 9.
    Kattan MW, Inoue Y, Giles FJ, et al. Cost-effectiveness of interferon-alpha and conventional chemotherapy in chronic myelogenous leukemia. Ann Intern Med 1996; 125: 541–8PubMedGoogle Scholar
  10. 10.
    Haverkamp LJ, Appel V, Appel SH. Natural history of amyotrophic lateral sclerosis in a database population. Validation of a scoring system and a model for survival prediction. Brain 1995; 118: 707–9PubMedCrossRefGoogle Scholar
  11. 11.
    Appel V, Stewart SS, Smith G, et al. A rating scale for amyotrophic lateral sclerosis: description and preliminary experience. Ann Neurol 1987; 22: 328–33PubMedCrossRefGoogle Scholar
  12. 12.
    Bergner M, Bobbitt RA, Carter WB, et al. The Sickness Impact Profile: development and final revision of a health status measure. Med Care 1981; 19: 787–805PubMedCrossRefGoogle Scholar
  13. 13.
    McGuire D, Garrison L, Armon C, et al. Relationship of the Tufts Quantitative Neuromuscular Exam (TQNE) and the Sickness Impact Profile (SIP) in measuring progression of ALS. SSNJV/CNTF ALS Study Group. Neurology 1996; 46: 1442–4PubMedCrossRefGoogle Scholar
  14. 14.
    Schulman KA, Rubenstein LE, Glick HA, et al. Relationships between sponsors and investigators in pharmacoeconomic and clinical research. Pharmacoeconomics 1995; 7: 206–20PubMedCrossRefGoogle Scholar
  15. 15.
    Hillman AL, Eisenberg JM, Pauly MV, et al. Avoiding bias in the conduct and reporting of cost-effectiveness research sponsored by pharmaceutical companies. N Engl J Med 1991; 324: 1362–5PubMedCrossRefGoogle Scholar
  16. 16.
    Lipscomb J, Weinstein MC, Torrance GW. Time preference. In: Gold MR, Weinstein MC, editors. Cost-effectiveness in health and medicine. New York: Oxford University Press, 1996: 214–35Google Scholar
  17. 17.
    Norris FH. Care of the amyotrophic lateral sclerosis patient. In: H Mitsumoto, FH Norris, editors. Amyotrophic lateral sclerosis. A comprehensive guide to management. New York: Demos Publications, 1994: 39Google Scholar
  18. 18.
    O’Brien BJ, Drummond MF, Labelle RJ, et al. In search of power and significance: Issues in the design and analysis of stochastic cost-effectiveness studies in health care. Med Care 1994; 32: 150–63CrossRefGoogle Scholar
  19. 19.
    Sacristan JA, Day SJ, Navarro O, et al. JM. Use of confidence intervals and sample size calculations in health economics studies. Ann Pharmacother 1995; 29: 719–25PubMedGoogle Scholar
  20. 20.
    Gardiner J, Hogan A, Holmes-Rovner M, et al. Confidence intervals for cost-effectiveness ratios. Med Decis Making 1995; 15: 254–63PubMedCrossRefGoogle Scholar
  21. 21.
    Fenn P, McGuire A, Phillips V, et al. The analysis of censored treatment cost data in economic evaluation. Med Care 1995; 33: 851–63PubMedCrossRefGoogle Scholar
  22. 22.
    Ganiats TG, Miller CJ, Kaplan RM. Comparing the quality-adjusted life-year output of two treatment arms in a randomized trial. Med Care 1995; 33: 245AS–54ASGoogle Scholar
  23. 23.
    Lee ET. Analytical estimation procedures for survival distributions. In: Statistical methods for survival data analysis. Wiley series in probability and mathematical statistics. New York: John Wiley and Sons, Inc; 1992Google Scholar
  24. 24.
    Glasziou PP, Simes RJ, Gelber RD. Quality adjusted survival analysis. Stat Med 1990; 9: 1259–76PubMedCrossRefGoogle Scholar
  25. 25.
    Sonnenberg FA, Beck JR. Markov models in medical decision making: a practical guide. Med Decis Making 1993; 13: 322–38PubMedCrossRefGoogle Scholar
  26. 26.
    Drummond MF. Discussion: Torrance’s ‘Utility approach to measuring health-related quality of life.’ J Chron Dis 1987; 40: 601–3CrossRefGoogle Scholar
  27. 27.
    Patrick DL, Starks HE, Cain KC, et al. Measuring preferences for health states worse than death. Med Decis Making 1994; 14: 9–18PubMedCrossRefGoogle Scholar
  28. 28.
    Torrance GW. Designing and conducting cost-utility analyses. In: Spilker B, editor. Quality of life and pharmacoeconomics in clinical trials. 2nd ed. Philadelphia: Lippincott-Raven Publishers, 1996Google Scholar
  29. 29.
    Health Care Financing Administration. 1996 Medicare program: Revisions to payment policies and adjustments to the relative value units under the physician fee schedule for calendar year 1996. Federal Register 1996Google Scholar
  30. 30.
    Samet JH, Libman H, Steger KA, et al. Compliance with zidovudine therapy in patients infected with human immunodeficiency virus, type 1: a cross-sectional study in a municipal hospital clinic. Am J Med 1992; 92: 495–502PubMedCrossRefGoogle Scholar
  31. 31.
    Briggs A, Sculpher M, Buxton M. Uncertainty in the economic evaluation of health care technologies: the role of sensitivity analysis. Health Econ 1994; 3: 95–104PubMedCrossRefGoogle Scholar
  32. 32.
    Mooney CZ, Duval RD. Bootstrapping: a nonparametric approach to statistical inference. Newbury Park; Sage Publications Inc. 1993Google Scholar
  33. 33.
    Coyle D. Statistical analysis in pharmacoeconomic studies. Pharmacoeconomics 1996; 9: 506–16PubMedCrossRefGoogle Scholar
  34. 34.
    Ringle SP, Murphy JR, Alderson MK, et al. The natural history of amyotrophic lateral sclerosis. Neurology 1993; 43: 1316–22CrossRefGoogle Scholar
  35. 35.
    Munsat TL, Andres PL, Finison L, et al. The natural history of motoneuron loss in amyotrophic lateral sclerosis. Neurology 1988; 38: 409–13PubMedCrossRefGoogle Scholar
  36. 36.
    Gubbay SS, Kahana E, Zilber N, et al. Amyotrophic lateral sclerosis. A study of its presentation and prognosis. Neurology 1985; 232: 295–300CrossRefGoogle Scholar
  37. 37.
    Norris F, Shepherd R, Denys E UK, et al. Onset, natural history and outcome in idiopathic adult motor neuron disease. J Neurol Sci 1993; 118: 48–55PubMedCrossRefGoogle Scholar
  38. 38.
    Caroscio JT, Mulvihill MN, Sterling R, et al. Amyotrophic lateral sclerosis. It’s natural history. Neurol Clin 1987; 5: 1–8PubMedGoogle Scholar
  39. 39.
    Rosen AD. Amyotrophic lateral sclerosis. Clinical features and prognosis. Arch Neurol 1978; 35: 638–42PubMedCrossRefGoogle Scholar
  40. 40.
    Welch HG, Larson EB. Cost-effectiveness of bone marrow transplantation in acute nonlymphocytic leukemia. N Engl J Med 1989; 321: 807–12PubMedCrossRefGoogle Scholar
  41. 41.
    Evans RW. Cost-effectiveness analysis of transplantation. Surg Clin North Am 1986; 66: 603–16PubMedGoogle Scholar
  42. 42.
    US Bureau of the Census. Statistical abstract of the United States 1997. 117th ed. Washington, DC: US Bureau of the Census, 1997Google Scholar
  43. 43.
    Torrance GW. Utility approach to measuring health-related quality of life. J Chron Dis 1987; 40: 593–600PubMedCrossRefGoogle Scholar
  44. 44.
    Laupacis A, Feeny D, Detsky AS, et al. How attractive does a new technology have to be to warrant adoption and utilization? Tentative guidelines for using clinical and economic evaluations. Can Med Assoc J 1992; 146: 473–81Google Scholar
  45. 45.
    Gold MR, Patrick DL, Torrance GW, et al. Identifying and valuing outcomes. In: Gold MR, Siegel JE, Russell LB, et al., editors. Cost-effectiveness in health and medicine. New York: Oxford University Press, 1996: 82–123Google Scholar
  46. 46.
    McDonald ER. Psychosocial-spiritual overview. In: H Mitsumoto, FH Norris, editors. Amyotrophic lateral sclerosis: a comprehensive guide to management. New York: Demos Publications, 1994: 206–22Google Scholar

Copyright information

© Adis International Limited 1999

Authors and Affiliations

  • Stacey J. Ackerman
    • 1
  • Erin M. Sullivan
    • 1
  • Kathleen M. Beusterien
    • 1
  • Howard M. Natter
    • 2
  • Deborah F. Gelinas
    • 3
  • Donald L. Patrick
    • 4
  1. 1.Covance Health Economics and Outcomes Services Inc.WashingtonUSA
  2. 2.Allegheny University, Hahnemann HospitalALS Clinical Services CenterPhiladelphiaUSA
  3. 3.Forbes Norris MDA/ALS CenterSan FranciscoUSA
  4. 4.University of WashingtonSeattleUSA

Personalised recommendations