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Beware of Surrogate Outcome Measures

Published online by Cambridge University Press:  10 March 2009

Peter C. Gøtzsche
Affiliation:
The Nordic Cochrane Centre
Alessandro Liberati
Affiliation:
The Italian Cochrane Centre
Valter Torri
Affiliation:
The Italian Cochrane Centre
Luca Rossetti
Affiliation:
Clinica Oculistica

Abstract

Surrogate outcome measures may speed up clinical research if they can be measured earlier in a study than the primary outcome of interest. We review the justification for their use and conclude that reliance on them may be harmful. Results obtained with surrogates should therefore be regarded as preliminary. Large, definitive trials with clinically relevant outcomes should always be performed before new interventions are accepted.

Type
Special Section: The Quality of the Medical Evidence: Is It Good Enough?
Copyright
Copyright © Cambridge University Press 1996

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References

REFERENCES

1.Aboulker, J. P., & Swart, A. M., Concorde Coordinating Committee. Preliminary analysis of the Concorde trial. Lancet, 1993, 341, 890–91.Google Scholar
2.Antman, E. M., Lau, J., Kupelnick, B., et al. A comparison of results of meta-analyses of randomized control trials and recommendations of clinical experts: Treatments for myocardial infarction. Journal of the American Medical Association, 1992, 268, 240–48.Google Scholar
3.Bentzon, M. W., Gad, I., & Halberg, P.Influence of previous gold treatment and other patient variables on outcome of treatment with disease modifying anti-rheumatic drugs (DMARD) in patients with rheumatoid arthritis. Clinical Rheumatology, 1986, 5, 3948.Google Scholar
4.Boissel, J-P., Collett, J-P., Moleur, P., & Haugh, M.Surrogate endpoints: A basis for a rational approach. European Journal of Clinical Pharmacology, 1992, 43, 235–44.Google Scholar
5.The CAST investigators. Preliminary report: Effect of encainide and flecainide on mortality in a randomized trial of arrhythmia suppression after myocardial infarction. New England Journal of Medicine, 1989, 321, 406–12.Google Scholar
6.Chalmers, T. C., Frank, C. S., & Reitman, D.Minimizing the three stages of publication bias. Journal of the American Medical Association, 1990, 263, 1392–95.Google Scholar
7.The Coronary Drug Project Research Group. Influence of adherence to treatment and response of cholesterol on mortality in the coronary drug project. New England Journal of Medicine, 1980, 303, 1038–41.Google Scholar
8.The Diabetes Control and Complication Trials Research Group. The effect of intensive treatment of diabetes on the development and progression of long-term complications in insulin-dependent diabetes mellitus. New England Journal of Medicine, 1993, 329, 977–86.Google Scholar
9.Gruppo Italiano per lo Studiodella Soprawivenza nell’Infarto Miocardico. GISSI-3:effects of lisinopril and transdermal glyceryl trinitrate singly and together on 6-week mortality and ventricular function after acute myocardial infarction. Lancet, 1994, 343, 1115–22.Google Scholar
10.Harris, S. T., Watts, N. B., Jackson, R. D., et al. Four-year study of intermittent cyclic etidronate treatment of postmenopausal osteoporosis: Three years of blinded therapy followed by one year of open therapy. American Journal of Medicine, 1993, 95, 557–67.Google Scholar
11.Hillis, A.Surrogate end-points in clinical trials: Ophthalmologic disorders. Statistics in Medicine, 1989, 8, 427–30.Google Scholar
12.Impact Research Group. International mexiletine and placebo antiarrhythmic coronary trial, I: Report on arrhythmia and other findings. Journal of the American College of Cardiology, 1984, 4, 1148–63.Google Scholar
13.Lewis, E. J., Hunsicker, L. G., Lan, S-P., et al. for the Lupus Nephritis Collaborative Study Group. A controlled trial of plasmapheresis therapy in severe lupus nephritis. New England Journal of Medicine, 1992, 326, 1373–79.Google Scholar
14.Miller, F. W., Leitman, S. F., Cronin, M. E., et al. Controlled trial of plasma exchange and leuka-pheresis in polymyositis and dermatomyositis. New England Journal of Medicine, 1992, 326, 1380–84.Google Scholar
15.Mullen, M., Mullen, B., & Carey, M.The association between beta-agonist use and death from asthma: A meta-analytic integration of case-control studies. Journal of the American Medical Association, 1993, 270, 1842–45.Google Scholar
16.Riggs, B. L., Hodgson, S. F., O’Fallon, W. M., et al. Effect of fluoride treatment on the fracture rate in postmenopausal women with osteoporosis. New England Journal of Medicine, 1990, 322, 802–09.Google Scholar
17.Rossetti, L., Marchetti, I., Orzalesi, N., et al. Randomized clinical trials on medical treatment of glaucoma: Are they appropriate to guide clinical practice? Archives of Ophthalmology, 1993, 111, 96103.Google Scholar
18.Smith, G. D., Song, F., & Sheldon, T. A.Cholesterol lowering and mortality: The importance of considering the initial level of risk. British Medical Journal, 1993, 306, 1367–73.Google Scholar
19.Sommer, A., Tielsch, J., Katz, J., et al. Relationship between intraocular pressure and primary open-angle glaucoma among white and black Americans: The Baltimore Eye Survey. Archives of Ophthalmology, 1991, 109, 1090–95.Google Scholar
20.Sponsel, W. E.Tonometry in question: Can visual screening play a more decisive role in glaucoma diagnosis and management? Survey of Ophthalmology, 1989, 33(suppl.), 291300.Google Scholar
21.Tiné, F., Liberati, A., Craxi, V., et al. Interferon treatment in patients with chronic hepatitis B: A meta-analysis of the published literature. Journal of Hepatology, 1993, 18, 154–62.Google Scholar
22.Torri, V., Simon, R., Russek-Cohen, E., et al. Statistical model to determine the relationship of response and survival in patients with advanced ovarian cancer treated with chemotherapy.Journal of the National Cancer Institute, 1992, 84, 407–14.Google Scholar
23.Ward, J.Prevention of invasive Haemophilus influenzae type b disease: Lessons from vaccine efficacy trials. Vaccine, 1991, 9, S17–S24.Google Scholar
24.Wulff, H., Pedersen, S. A., & Rosenberg, R.Philosophy of medicine. Oxford: Blackwell, 1986.Google Scholar