, Volume 13, Issue 6, pp 659–666 | Cite as

Common Errors and Controversies in Pharmacoeconomic Analyses

Leading Article Errors and Controversies in Pharmacoeconomic Analyses


The need to demonstrate the cost effectiveness of healthcare interventions has led to a rapid increase in the use of economic tools within pharmaceutical evaluations. Pharmacoeconomics is employed at many stages of the evaluation process, helping to predict which products are likely to be economically viable at an early stage, and providing information to aid price and reimbursement negotiations as well as formulary and purchasing decisions in conjunction with phase III and IV clinical trials.

The ability of economic evaluations to accurately determine the best use of society—s scarce resources, however, is strongly influenced by the existence of areas of confusion, controversy and dispute which hinder the researcher at every step. A good economic evaluation requires a number of ingredients including: (i) relevant, good quality clinical data, raising issues of trial design, sample size and perspective; (ii) relevant costs and outcomes, measured, valued and discounted credibly and accurately; (iii) appropriate methods of data analysis (statistical, incremental and sensitivity); and, once the trial is over, (iv) presentation of the results in a way which maximises the generalisability of the results and, hence, the usefulness of the research. None of these areas are trouble-free but with understanding and openness, mistakes can be minimised.


Adis International Limited Economic Evaluation Indirect Cost Pharmacoeconomic Analysis Pharmacoeconomic Study 
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  1. 1.
    Andersson F. Why is the pharmaceutical industry investing increasing amounts in health economic evaluations? Int J Technol Assess Health Care 1995; 11 (4): 750–61PubMedCrossRefGoogle Scholar
  2. 2.
    Drummond MF, Menzin J, Oster G. Problems in undertaking pharmacoeconomic assessments in phase III clinical trials: the case of colony-stimulating factors. In: Spilker B, editor. Quality of life and pharmacoeconomics in clinical trials. Philadelphia: Lippincott-Raven, 1995Google Scholar
  3. 3.
    Milne RJ. Evaluation of the pharmacoeconomic literature. Pharmacoeconomics 1994; 6 (4): 337–45PubMedGoogle Scholar
  4. 4.
    Jolicoeur LM, Jones-Grizzle AJ, Boyer JG. Guidelines for performing a pharmacoeconomic analysis. Am J Hosp Pharm 1992; 49: 1741–7PubMedGoogle Scholar
  5. 5.
    Drummond M. Common mistakes in the design of economic evaluations of medicines. Br J Med Econ 1991; 1: 5–14Google Scholar
  6. 6.
    Woolf SH, Battista RN, Anderson GM, et al. Assessing the clinical effectiveness of preventive manoeuvres: analytic principles and systematic methods in reviewing evidence and developing clinical practice recommendations. J Clin Epidemiol 1990; 43: 891–905PubMedCrossRefGoogle Scholar
  7. 7.
    Commonwealth Department of Human Services and Health. Guidelines for the pharmaceutical industry on preparation of submissions to the pharmaceutical benefits advisory committee: including major submissions involving economic analysis. Canberra: Australia Government Publishing Service, 1995Google Scholar
  8. 8.
    Drummond MF. The future of pharmacoeconomics: bridging science and practice. Clin Ther 1996; 18 (5): 969–78PubMedCrossRefGoogle Scholar
  9. 9.
    Hilleman DE, Mohiuddin SM, Lucas BD, et al. Cost-minimization analysis of initial antihypertensive therapy in patients with mild-to-moderate essential diastolic hypertension. Clin Ther 1994; 16 (1): 88–102PubMedGoogle Scholar
  10. 10.
    Weinstein MC. Principles of cost-effective resource allocation in health care organizations. Int J Technol Assess Health Care 1990; 6: 93–103PubMedCrossRefGoogle Scholar
  11. 11.
    Schwartz D, Lellouch J. Explanatory and pragmatic attitudes in therapeutic trials. J Chronic Dis 1976; 20: 637–48CrossRefGoogle Scholar
  12. 12.
    De Graeve D, Nonneman W. Pharmacoeconomic studies: pitfalls and problems. Int J Technol Assess Health Care 1996; 12 (1): 22–30PubMedCrossRefGoogle Scholar
  13. 13.
    Siegel JE, Torrance GW, Russell LB, et al. Guidelines for pharmacoeconomic studies: recommendations from the panel on cost effectiveness in health and medicine. Pharmacoeconomics 1997; 11 (2): 159–68PubMedCrossRefGoogle Scholar
  14. 14.
    Sculpher M, Drummond M, Buxton M. The iterative use of economic evaluation as part of the process of health technology assessment. J Health Serv Res Policy 1997; 2 (1): 26–30PubMedGoogle Scholar
  15. 15.
    Gray AM, Marshall M, Lockwood A, et al. Problems in conducting economic evaluations alongside clinical trials. Br J Psychiatry 1997; 170: 47–52PubMedCrossRefGoogle Scholar
  16. 16.
    Drummond M, OBrien B. Clinical importance, statistical significance and the assessment of economic and quality of life outcomes. Health Econ 1993; 2: 205–12PubMedCrossRefGoogle Scholar
  17. 17.
    Coyle D. Statistical analysis in pharmacoeconomic studies: a review of current issues and standards. Pharmacoeconomics 1996; 9 (6): 506–16PubMedCrossRefGoogle Scholar
  18. 18.
    Johannesson M. A note on the depreciation of the societal perspective in economic evaluation of health care. Health Policy 1995; 33: 59–66PubMedCrossRefGoogle Scholar
  19. 19.
    Lee JT, Sanchez LA. Interpretation of ‘cost—effective’ and soundness of economic evaluations in the pharmacy literature. Am J Hosp Pharm 1991; 48: 2622–7PubMedGoogle Scholar
  20. 20.
    Mynors-Wallis L, Davies I, Gray A, et al. A randomised controlled trial and cost analysis of problem-solving treatment for emotional disorders given by community nurses in primary care. Br J Psychiatry 1997; 170: 113–9PubMedCrossRefGoogle Scholar
  21. 21.
    Ontario Ministry of Health. Ontario guidelines for economic analysis of pharmaceutical products. Toronto: Ontario Ministry of Health, 1994Google Scholar
  22. 22.
    Canadian Co-ordinating Office for Health Technology Assessment (CCOHTA). Guidelines for economic evaluation of pharmaceuticals. 1st ed. Ottawa: CCOHTA, 1994Google Scholar
  23. 23.
    England and Wales Department of Health. Guidelines on good practice in the conduct of economic evaluation of medicines. London: Department of Health, 1994Google Scholar
  24. 24.
    Davidoff AJ, Powe NR. The role of perspective in defining economic measures for the evaluation of medical technology. Int J Technol Assess Health Care 1996; 12 (1): 9–21PubMedCrossRefGoogle Scholar
  25. 25.
    Koopmanschap MA, Rutten FFH. A practical guide for calculating indirect costs of disease. Pharmacoeconomics 1996; 10 (5): 460–6PubMedCrossRefGoogle Scholar
  26. 26.
    Drummond MF, Stoddart GL, Torrance GW. Methods for the economic evaluation of health care programmes. Oxford: Oxford University Press, 1987Google Scholar
  27. 27.
    Paladino JA, Rainstein MA, Serrianne DJ, et al. Ampicillinsulbactam versus cefoxitin for prophylaxis in high-risk patients undergoing abdominal surgery. Pharmacotherapy 1994; 14 (6): 734–9PubMedGoogle Scholar
  28. 28.
    Luce BR, Elixhauser A. Estimating costs in the economic evaluation of medical technologies. Int J Technol Assess Health Care 1990; 6: 57–75PubMedCrossRefGoogle Scholar
  29. 29.
    Luce BR, Simpson K. Methods of cost-effectiveness analysis: areas of consensus and debate. Clin Ther 1995; 17 (1): 109–25PubMedCrossRefGoogle Scholar
  30. 30.
    Drummond M, Brandt A, Luce B, et al. Standardizing methodologies for economic evaluation in health care. Int J Technol Assess Health Care 1993; 9 (1): 26–36PubMedCrossRefGoogle Scholar
  31. 31.
    Kind P. The design and construction of quality of life measures. York: University of York, Centre for Health Economics, 1988. Discussion paper no.: 43Google Scholar
  32. 32.
    Ware JE, Sherbourne CD. The MOS 36-item short-form health survey (SF36). Med Care 1992; 30 (6): 473–83PubMedCrossRefGoogle Scholar
  33. 33.
    EuroQol Group. EuroQol: a new facility for the measurement of health-related quality of life. Health Policy 1990; 16: 199–208CrossRefGoogle Scholar
  34. 34.
    Rosser RM, Kind P. A scale of valuations of states of illness: is there a social consensus? Int J Epidemiol 1978; 7 (4): 347–58PubMedCrossRefGoogle Scholar
  35. 35.
    Loomes G, McKenzie L. The use of QALYs in health care decision making. Soc Sci Med 1989; 28 (4): 299–308PubMedCrossRefGoogle Scholar
  36. 36.
    Drummond M. Output measurement for resource-allocation decisions in health care. In: McGuire A, Fenn P, Mayhew K, editors. Providing health care: the economics of alternative systems of finance and delivery. Oxford: Oxford University Press, 1991Google Scholar
  37. 37.
    Lawrence K, McWhinnie D, Goodwin A, et al. Randomised controlled trial of laparoscopic versus open repair of inguinal hernia: early results. BMJ 1995; 311: 981–5PubMedCrossRefGoogle Scholar
  38. 38.
    Gandhi SK, Kong SX. Quality-of-life measures in the evaluation of antihypertensive drug therapy: reliability, validity and quality-of-life domains. Clin Ther 1996; 18 (6): 1276—95Google Scholar
  39. 39.
    Krahn M, Gafni A. Discounting in the economic evaluation of health care interventions. Med Care 1993; 31 (5): 403–18PubMedCrossRefGoogle Scholar
  40. 40.
    Mennie ME, Gilfillan A, Compton M, et al. Prenatal screening for cystic fibrosis. Lancet 1992; 340 (8813): 214–6PubMedCrossRefGoogle Scholar
  41. 41.
    Donaldson C, Shackley P, Abdalla M, et al. Willingness to pay for antenatal screening for cystic fibrosis. Aberdeen: Health Economics Research Unit, 1993. Discussion paper no.: 04/93Google Scholar
  42. 42.
    Silcock J, Torgerson D. Evaluation of costs [letter]. Lancet 1994; 344 (8924): 755–6PubMedCrossRefGoogle Scholar
  43. 43.
    Mangtani P, Hall AJ, Normand CE. Hepatitis B vaccination: the cost-effectiveness of alternative strategies in England and Wales. J Epidemiol Community Health 1995; 49 (3): 238–44PubMedCrossRefGoogle Scholar
  44. 44.
    Drummond M. Economic analysis alongside controlled trials: an introduction for clinical researchers. London: UK Department of Health, 1994Google Scholar
  45. 45.
    Kort MA, Bootman JL. The economics of benign hyperplasia treatment: a literature review. Clin Ther 1996; 18 (6): 1227–41CrossRefGoogle Scholar
  46. 46.
    Martens LL, Guibert R. Cost-effectiveness analysis of lipid modifying therapy in Canada: comparison of HMG-CoA reductase inhibitors in the primary prevention of coronary heart disease. Clin Ther 1994; 16 (6): 1052–6PubMedGoogle Scholar
  47. 47.
    Weinstein MC. Economic assessments of medical practices and technologies. Med Decis Making 1981; 1 (4): 309–30PubMedCrossRefGoogle Scholar
  48. 48.
    Eisenberg JM. Clinical economics: a guide to the economic analysis of clinical practices. JAMA 1989; 262 (20): 2879–86PubMedCrossRefGoogle Scholar
  49. 49.
    Task Force on Principles for Economic Analysis of Health Care Technology. Economic analysis of health care technology: a report on principles. Ann Intern Med 1995; 122: 61–70Google Scholar
  50. 50.
    Agro KE, Bradley CA, Mittman N, et al. Sensitivity analysis in health economic and pharmacoeconomic studies: an appraisal of the literature. Pharmacoeconomics 1997; 11 (1): 75–86PubMedCrossRefGoogle Scholar
  51. 51.
    Briggs A, Sculpher M. Sensitivity analysis in economic evaluation: a review of published studies. Health Econ 1995; 4: 355–71PubMedCrossRefGoogle Scholar
  52. 52.
    Office of Health economics (OHE). Handling uncertainty in the results of economic evaluation. London: OHE, 1995. OHE briefing no.: 32Google Scholar
  53. 53.
    OBrien BJ, Drummond MF. Statistical versus quantitative significance in the socioeconomic evaluation of medicines. Pharmacoeconomics 1994; 5 (5): 389–98PubMedCrossRefGoogle Scholar
  54. 54.
    Creed F, Mbaya P, Lancashire S, et al. Cost-effectiveness of day and inpatient psychiatric treatment: results of a randomised controlled trial. BMJ 1997; 314: 1381–5PubMedCrossRefGoogle Scholar
  55. 55.
    Rutten-Van Molken M, Van Doorslaer E, Van Vliet R. A statistical analysis of cost outcomes in a randomized controlled clinical trial. Health Econ 1994; 3: 333–45CrossRefGoogle Scholar
  56. 56.
    Polsky D, Glick HA, Willke R, et al. Confidence intervals for cost-effectiveness ratios: a comparison of four methods. Health Econ 1997; 6: 243–52PubMedCrossRefGoogle Scholar
  57. 57.
    Mason J. The generalisability of pharmacoeconomic studies. Pharmacoeconomics 1997; 11 (6): 503–14PubMedCrossRefGoogle Scholar
  58. 58.
    Gold MR, Siegel JE, Russell LB, et al. Cost-effectiveness in health and medicine. New York: Oxford University Press, 1996Google Scholar
  59. 59.
    Sloan FA. Valuing health care: costs, benefits and effectiveness of pharmaceutical and other medical technologies. Cambridge: Cambridge University Press, 1995Google Scholar
  60. 60.
    Drummond MF. Methodological principles for economic evaluation of pharmaceuticals. Br J Med Econ 1993; 6B: 1–18Google Scholar

Copyright information

© Adis International Limited 1998

Authors and Affiliations

  1. 1.Centre for Health EconomicsUniversity of YorkEngland

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