Abstract
Pharmacoeconomic data may be obtained within the context of randomised clinical trials (RCTs) and from effectiveness studies in the ‘real world’. The differences between the 2 types of study design have implications for the types of data that can be obtained and the interpretation of the resulting findings. Because RCTs are designed to assess the safety and efficacy of pharmaceuticals, and because the study design of RCTs emphasises internal validity over generalisability, the pharmacoeconomic data collected from them are limited. The data may not be applicable to the more heterogeneous patients encountered in actual clinical practice, and cost estimates may be inaccurate because of protocol requirements. Effectiveness studies, in which treatments are studied under real-world conditions, remedy some of these limitations. Generalisability to actual users is generally enhanced in effectiveness designs, but data may be biased in other ways.
This brief review compares the 2 study designs as they relate to pharmacoeconomic evaluations in terms of the research questions they address, design differences and their implications for study bias, data collection and data analysis and the generalisability of their results.
Similar content being viewed by others
References
Ray WA, Griffin MR, Avorn J. Evaluating drugs after their approval for clinical use. N Engl J Med 1993; 329: 2029–32
Burnam MA. Measuring outcomes of care for substance abuse and mental disorders. New Dir Ment Health Serv 1996; 71: 3–17
Drummond MF, Davies LM. Economic analysis alongside clinical trials: revisiting the methodological issues. Int J Technol Assess Health Care 1991; 7: 561–73
Revicki DA, Luce BR. Methods of pharmacoeconomic evaluation of new medical treatments in psychiatry. Psychopharm Bull 1995; 31: 249–58
Hargreaves WA, Shumway M, Hu T, et al. Cost-outcome methods for mental health. San Diego CA: Academic Press, 1998
Donabedian A. The seven pillars of quality. Arch Pathol Lab Med 1990; 114: 1115–8
Revicki DA. Methods of pharmacoeconomic evaluation of psychopharmacologic therapies for patients with schizophrenia. J Psychiatry Neurosci 1997; 22: 256–66
Simon G, Wagner E, Von Korff M. Cost-effectiveness comparisons using real world randomized trials. J Clin Epidemiol 1995; 48: 363–73
Schwarz D, Lellouch J. Explanatory and pragmatic attitudes in clinical trials. J Chronic Dis 1967; 20: 637–48
Diamond G, Denton TA. Alternative perspectives on the biased foundations of medical technology assessment. Ann Intern Med 1993; 118: 455–64
Rosenbaum P. Discussing hidden bias in observational studies. Ann Intern Med 1991; 115: 901–5
Simon GE, Von Korff M, Heiligenstein JH, et al. Initial antidepressant choice in primary care. Effectiveness and cost of fluoxetine vs. tricyclic antidepressants. JAMA 1996; 175: 1897–902
Simon GE, Heiligenstein JH, Revicki DA, et al. Long-term outcomes of initial antidepressant choice in a ‘real world’ randomized trial. Arch Fam Med. In press
Rosenheck R, Cramer J, Xu W, et al. A comparison of clozapine and haloperidol in hospitalized patients with refractory schizophrenia. N Engl J Med 1997; 337: 809–15
Essocks SM, Hargreaves WA, Covell NH, et al. Clozapine’s effectiveness for patients in state hospitals: results of a randomized trial. Psychopharmacol Bull 1996; 32: 683–97
Mahmoud RA, Englehart LM, Oster G, et al. Risperidone vs. conventional antipsychotics: a prospective randomized naturalistic effectiveness trial of outcomes in chronic schizophrenia. 36th Annual Meeting of the American College of Neuropsychopharmacology; 1997 Dec 8-12: Kamuela, Hawaii
Oster G, Borok GM, Menzin J, et al. A randomized trial to assess effectiveness and cost in clinical practice: rational and design of the CRIS. Control Clin Trials 1995; 16: 3–16
Oster G, Borok GM, Menzin J, et al. Cholesterol-reduction intervention study (CRIS). A randomized trial to assess effectiveness and costs in clinical practice. Arch Intern Med 1996; 156: 731–9
Kaplan-Machlis B, Spiegler GE, Revicki DA. Health-related quality of life in primary care patients with gastroesophageal reflux disease. Charleston (WV): School of Pharmacy, West Virginia University, 1998
Revicki DA, Hirschfeld RMA, Keck PE, et al. Cost-effectiveness of divalproex sodium vs lithium in long-term treatment of bipolar disorder. 37th Annual Meeting of the American College of Neuropsychopharmacology; 1998 Dec 14-18: Las Croabas, Puerto Rico
Cook TD, Campbell DT. Quasi-experimentation. design and analysis issues for field settings. Hopewell (NJ): Houghton Mifflin Company, 1979
Sackett D, Gent M. Controversy in counting and attributing events in clinical trials. New Engl J Med 1979; 801: 1410–2
Feinstein A. An additional basic science for clinical medicine. II: the limitations of randomized trials. Ann Intern Med 1983; 99: 544–50
Fletcher RH, Fletcher SW, Wagner EH. Clinical epidemiology. The essentials. 3rd ed. Baltimore: Williams and Wilkins, 1996
Fowler FJ, Cleary PD, Magaziner J, et al. Methodological issues in measuring patient-reported outcomes: the agenda of the work group on outcomes assessment. Med Care 1994; 32: JS65–76
Luce BR, Hillman AL. When is a cost-effectiveness claim valid? How much should the FDA care? Am J Manage Care 1997; 3: 1660–6
Popper KR. The logic of scientific discovery. New York: Basic Books, 1959
Moses LE. Measuring effects without randomized trials? Options, problems, challenges. Med Care 1995; 33 (4) Suppl.: AS8–14
Osterhaus JT, Townsend RJ. Incorporating pharmacoeconomic research into clinical trials. In: Bootman JL, Townsend RJ, McGhan WF, editors. Principles of pharmacoeconomics. 2nd ed. Cincinnatti (OH): Harvey Whitney Books, 1996
Revicki DA. Pharmacoeconomic studies of atypical antipsychotics for the treatment of schizophrenia. Schizophr Res 1999; 35: S101–9
Eddy DM. Clinical decision making: from theory to practice. What do we do about costs? JAMA 1990; 264: 1161–7
Simon GE, Von Korff M, Revicki DA. Telephone assessment of depression severity. J Psychiatr Res 1993; 27: 247–52
Revicki DA, Tohen M, Gyulai L, et al. Telephone versus in-person clinical and health status assessment interviews in bipolar disorder patients. Harv Rev Psychiatry 1997; 5: 75–81
O’Brien B, Drummond M, Labelle R, 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: 150–63
Polsky D, Glick HA, Willke R, et al. Confidence intervals for cost-effectiveness ratios: a comparison of four methods. Health Econ 1997; 6: 243–52
Sacristan JA, Day SJ, Navarro O, et al. Use of confidence intervals and sample size calculations in health economic studies. Ann Pharmacother 1995; 29: 719–25
Lavori PW, Dawson R, Shera D. A multiple imputation strategy for clinical trials with truncation of patient data. Stat Med 1995; 14: 1913–25
Little RJA. Modeling the drop-out mechanism in repeatedmeasures studies. J Am Stat Assoc 1995; 90: 1112–21
Heyting A, Tolboom JTBM, Essers JGA. Statistical handling of dropouts in longitudinal clinical trials. Stat Med 1992; 11: 2043–61
Revicki DA, Schwartz C, Gold K, et al. Imputing health status scores missing due to mortality: comparison of multiple techniques. Presented at the annual meeting of the International Society for Quality of Life Research; 1997 Nov: Vienna, Austria
Bootman JL, Townsend RJ, McGhan WF, editors. Principles of pharmacoeconomics. 2nd ed. Cincinnatti (OH): Harvey Whitney Books, 1996
Johnson NE, Hirsch JD, Nash DB, et al. Pharmacoeconomic research in medical centers. In: Bootman JL, Townsend RJ, McGhan WF, editors. Principles of pharmacoeconomics. 2nd ed. Cincinnatti (OH): Harvey Whitney Books, 1996
Heithoff KA, Lohr KN, editors. Effectiveness and outcomes in health care. Proceedings of an invitational conference by the Institute of Medicine Division of Health Care Services. Washington DC: National Academy Press, 1990
Author information
Authors and Affiliations
Corresponding author
Rights and permissions
About this article
Cite this article
Revicki, D.A., Frank, L. Pharmacoeconomic Evaluation in the Real World. Pharmacoeconomics 15, 423–434 (1999). https://doi.org/10.2165/00019053-199915050-00001
Published:
Issue Date:
DOI: https://doi.org/10.2165/00019053-199915050-00001