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Randomized Clinical Trials in Surgery

Published online by Cambridge University Press:  10 March 2009

Bucknam McPeek ,
Frederick Mosteller  and
Martin McKneally
Bucknam McPeek
Affiliation:
Harvard University and Massachusetts General Hospital
Frederick Mosteller
Affiliation:
Harvard University
Martin McKneally
Affiliation:
Albany Medical College
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Extract

When it is well conducted, a randomized clinical provides the strongest evidence available for evaluating the comparative effectiveness of the interventions tested. Over the last two generations, we have learned much about various devices for strengthening them and about methods of avoiding between in their design, execution, analysis, and reporting. In a trial, we seek evidence for a causal link between treatment and observed outcomes. Becaues the controlled trial depends on an argument based on exculsion (i.e., no other causes or differences affected the experimental groups), we strengthen its inference by taking steps to exclude any such differences.

This article discusses a number of issues that deserve consideration: problems of multiplicity and generalizability, devices for strengthening trials, issues of power and sample size, the relationship between study design and reported gains, when to undertake a trial, the role of collaborative trials, and ways to make trials more feasible in clinical settings.

Type
Special Section: Technology Assessment and Surgical Policy
Information
International Journal of Technology Assessment in Health Care , Volume 5 , Issue 3 , July 1989 , pp. 317 - 332
Copyright
Copyright © Cambridge University Press 1989

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References

REFERENCES

Amberson, J. B. Jr, McMahon, B. T., & Pinner, M. Aclinical trial of sanocryson in pulmonary tuberculosis. American Review of Tuberculosis, 1931, 24, 401–35.Google Scholar
Colditz, G. A., Miller, J. N., & Mosteller, F.The effect of study design on gain in evaluations of new treatments in medicine and surgery. Drug Information Journal, 1988, 22, 343–52.CrossRefGoogle Scholar
Colditz, G. A., Miller, J. N., & Mosteller, F. How study design affects outcome in comparisons of therapy. I. Medical. In: Statistics in Medicine. In press.Google Scholar
Eddy, D. TAPSS: A Technology Assessment Priority-Setting System. Center for Health Policy Research and Education, Duke University, 1987. Unpublished.Google Scholar
Emerson, J. D., McPeek, B., & Mosteller, F.Reporting clinical trials in general surgical journals. Surgery, 1984, 95, 572–79.Google ScholarPubMed
Fisher, R. A.The design of experiments Edinburgh: Oliver & Boyd, 1935.Google Scholar
Fisher, R. A., & MacKenzie, W. A.Studies in crop variation: II. The manurial response of different potato varieties. Journal of Agricultural Science, 1923, 13, 311–20.CrossRefGoogle Scholar
Freiman, J. A., Chalmers, T. C., & Smith, H.The importance of beta, the type II error, and sample size in the design and interpretation of the randomized controlled trial. New England Journal of Medicine, 1978, 290, 690–94.CrossRefGoogle Scholar
Gilbert, J. P., McPeek, B., Mosteller, F. Progress in surgery and anesthesia: Benefits and risks of innovative therapy. In Bunker, J. P., Barnes, B. A., & Mosteller, F. (Eds.), Costs, risks, and benefits of surgery. New York: Oxford University Press, 1977, 124–69.Google Scholar
Gilbert, J. P., McPeek, B., & Mosteller, F.Statistics and ethics in surgery and anesthesia. Science, 1977, 198, 684–89.CrossRefGoogle ScholarPubMed
Goligher, J.The skeptical chirurgeon. Annals of the Royal College of Surgeons of England, 1984, 66, 207–10.Google ScholarPubMed
Goligher, J. C., Pulvertaft, C. N., & Watkinson, G.Controlled trial of vagotomy and gastroenterostomy, vagotomy and antrectomy, and subtotal gastrectomy in elective treatment of duodenal ulcer: Interim report. British Medical Journal, 1964, 1, 455–60.CrossRefGoogle ScholarPubMed
Hoaglin, D. C., Light, R. J., McPeek, B., Mosteller, F. et al. Data for decisions. Cambridge, MA: Abt, 1982, 997–9.Google Scholar
Medical Research Council. Streptomycin treatment of pulmonary tuberculosis. British Medical Journal, 1948, 2, 769–82.CrossRefGoogle Scholar
Meinert, C. L., Clinical trials—Design, conduct, and analysis. New York: Oxford University Press, 1986.CrossRefGoogle Scholar
Miller, J. N., Colditz, G. A., & Mosteller, F. How study design affects outcomes in comparisons of therapy. II. Surgical. Statistics in Medicine. In press.Google Scholar
Mosteller, F., Gilbert, J. P., & McPeek, B.Reporting standards and research strategies for controlled trials. Controlled Clinical Trials, 1980, 1, 37–58.CrossRefGoogle Scholar
Neuhauser, D. The Metro firm trials and ongoing patient randomization. In Tanur, J. M. et al. (Eds.) Statistics, a guide to the unknown, 3rd Edition. Pacific Grove, CA: Brooks/Cole. In press.Google Scholar
O'Young, J., & McPeek, B.Quality of life variables in surgical trials. Journal of Chronic Disease, 1987, 40, 513–22.CrossRefGoogle ScholarPubMed
Packard, F. R., Life and times of Ambrose Pare, 1510–1590. New York: Paul B. Hoeber, 1921.Google Scholar
Pernick, M. S.A calculus of suffering. New York: Columbia University Press, 1985.Google Scholar
Peto, R.Clinical trial methodology. Biomedicine, 1978, 28, 24–36.Google ScholarPubMed
Pocock, S.Clinical trials—a practical approach. New York: Wiley, 1983.Google Scholar
Rand Corporation. A million random digits with 100,000 normal deviates. New York: 1955.Google Scholar
Sackett, D. L., & Gent, M.Controversy in counting and attributing events in clinical trials. New England Journal of Medicine, 1979, 301, 1410–12.CrossRefGoogle ScholarPubMed
Schwartz, D., Flamant, R., & Lellouch, J.Clinical trials. New York: Academic, 1980.Google Scholar
Schwartz, D., & Lellouch, J.Explanatory and pragmatic attitudes in clinical trials. Journal of Chronic Disease, 1967, 20, 637–48.CrossRefGoogle Scholar
Shapiro, S., & Louis, T. A. (Eds.). Clinicaltrials: Issues and approaches. New York: Marcel Decker, 1982.Google Scholar
Winsor, C. P.Probability and Listerism, Human Biology, 1948, 169, 161–9.Google Scholar
Wood-Dauphinee, S., & Troidl, H. Endpoints for clinical studies: Conventional and innovative variables. In Troidl, H., Spitzer, W. O., McPeek, B., McKneally, M. F., & Mulder, D. S. (Eds.), Principles and practice of research. New York: Springer-Verlag, 1986, 53–68.CrossRefGoogle Scholar
Zelen, M. Anew design for randomized clinical trials. New England Journal of Medicine, 1979, 300, 1242–5.CrossRefGoogle ScholarPubMed
Zelen, M., Gehan, E., & Glibwell, O. Biostatistics in cancer research. In Hoogstralen, B. (Ed.), Importance of cooperative groups. New York: Mason, 1980, 291–312.Google Scholar