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Logo of annrheumdAnnals of the Rheumatic DiseasesCurrent TOCInstructions for authors
Ann Rheum Dis. Nov 1999; 58(Suppl 1): I82–I85.
PMCID: PMC1766572

Pharmacoeconomic evaluation of new treatments: efficacy versus effectiveness studies?


The juxtaposition of economic and clinical evaluation raises new issues in the design of clinical trials. Recent pharmacoeconomic guidelines provide some direction, but do not deal with the appropriate timing of economic evaluations in the drug developmental process. Ideally, pharmacoeconomic data should be available at the time of the regulatory and formulary decision making. Current pivotal phase III trials do not provide these data; they are designed to test safety and efficacy (does the drug work under optimal circumstances?) and not to answer questions about the effectiveness of a drug, the more relevant question for economic analysis (does the drug work in usual care?). The use of more "naturalistic" designs for some phase III randomised trials has been suggested. These so called "effectiveness trials" more closely reflect routine clinical practice. They use a more flexible dosage regimen, and a "usual care" instead of a placebo comparator. Patients randomised are more representative of actual practice and outcomes include quality of life and utility measures. They are more suited to provide the data needed to estimate the real benefit of the treatment in actual care. When costs are applied and compared with these benefits, you can estimate the efficiency of allocating resources to this new drug. Increasing the use of effectiveness trials in phase III would decrease the need for economic modelling.

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Selected References

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  • Fryback DG. QALYs, HYEs, and the loss of innocence. Med Decis Making. 1993 Oct-Dec;13(4):271–272. [PubMed]
  • Richardson J. Cost utility analysis: what should be measured? Soc Sci Med. 1994 Jul;39(1):7–21. [PubMed]
  • Cairns JA. Valuing future benefits. Health Econ. 1994 Jul-Aug;3(4):221–229. [PubMed]
  • Krahn M, Gafni A. Discounting in the economic evaluation of health care interventions. Med Care. 1993 May;31(5):403–418. [PubMed]
  • Redelmeier DA, Heller DN. Time preference in medical decision making and cost-effectiveness analysis. Med Decis Making. 1993 Jul-Sep;13(3):212–217. [PubMed]
  • O'Brien BJ, Drummond MF, Labelle RJ, Willan A. In search of power and significance: issues in the design and analysis of stochastic cost-effectiveness studies in health care. Med Care. 1994 Feb;32(2):150–163. [PubMed]
  • van Hout BA, Al MJ, Gordon GS, Rutten FF. Costs, effects and C/E-ratios alongside a clinical trial. Health Econ. 1994 Sep-Oct;3(5):309–319. [PubMed]
  • Russell LB, Gold MR, Siegel JE, Daniels N, Weinstein MC. The role of cost-effectiveness analysis in health and medicine. Panel on Cost-Effectiveness in Health and Medicine. JAMA. 1996 Oct 9;276(14):1172–1177. [PubMed]
  • Siegel JE, Weinstein MC, Russell LB, Gold MR. Recommendations for reporting cost-effectiveness analyses. Panel on Cost-Effectiveness in Health and Medicine. JAMA. 1996 Oct 23;276(16):1339–1341. [PubMed]
  • Siegel JE, Torrance GW, Russell LB, Luce BR, Weinstein MC, Gold MR. Guidelines for pharmacoeconomic studies. Recommendations from the panel on cost effectiveness in health and medicine. Panel on cost Effectiveness in Health and Medicine. Pharmacoeconomics. 1997 Feb;11(2):159–168. [PubMed]
  • Weinstein MC, Siegel JE, Gold MR, Kamlet MS, Russell LB. Recommendations of the Panel on Cost-effectiveness in Health and Medicine. JAMA. 1996 Oct 16;276(15):1253–1258. [PubMed]
  • Drummond MF, Jefferson TO. Guidelines for authors and peer reviewers of economic submissions to the BMJ. The BMJ Economic Evaluation Working Party. BMJ. 1996 Aug 3;313(7052):275–283. [PMC free article] [PubMed]
  • Evans RG. Manufacturing consensus, marketing truth: guidelines for economic evaluation. Ann Intern Med. 1995 Jul 1;123(1):59–60. [PubMed]
  • Hillman AL, Eisenberg JM, Pauly MV, Bloom BS, Glick H, Kinosian B, Schwartz JS. Avoiding bias in the conduct and reporting of cost-effectiveness research sponsored by pharmaceutical companies. N Engl J Med. 1991 May 9;324(19):1362–1365. [PubMed]
  • Kassirer JP, Angell M. The journal's policy on cost-effectiveness analyses. N Engl J Med. 1994 Sep 8;331(10):669–670. [PubMed]
  • Mason J, Drummond M. Reporting guidelines for economic studies. Health Econ. 1995 Mar-Apr;4(2):85–94. [PubMed]
  • Drummond MF, Davies L. Economic analysis alongside clinical trials. Revisiting the methodological issues. Int J Technol Assess Health Care. 1991;7(4):561–573. [PubMed]
  • Maetzel A, Ferraz MB, Bombardier C. The cost-effectiveness of misoprostol in preventing serious gastrointestinal events associated with the use of nonsteroidal antiinflammatory drugs. Arthritis Rheum. 1998 Jan;41(1):16–25. [PubMed]
  • Verhoeven AC, Bibo JC, Boers M, Engel GL, van der Linden S. Cost-effectiveness and cost-utility of combination therapy in early rheumatoid arthritis: randomized comparison of combined step-down prednisolone, methotrexate and sulphasalazine with sulphasalazine alone. COBRA Trial Group. Combinatietherapie Bij Reumatoïde Artritis. Br J Rheumatol. 1998 Oct;37(10):1102–1109. [PubMed]
  • Schwartz D, Lellouch J. Explanatory and pragmatic attitudes in therapeutical trials. J Chronic Dis. 1967 Aug;20(8):637–648. [PubMed]
  • Feinstein AR. An additional basic science for clinical medicine: II. The limitations of randomized trials. Ann Intern Med. 1983 Oct;99(4):544–550. [PubMed]

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