Abstract
In biomedical research, meaningful conclusions can only be drawn based on data collected from a valid scientific design using appropriate statistical methods. Therefore, the selection of an appropriate study design is important in order to provide an unbiased and scientific evaluation of the research questions. In this chapter, the different kinds of experimental studies commonly used in biology and medicine are introduced. A brief survey of basic experimental study designs, randomization, blinding, possible biases, issues in data analysis, and interpretation of the study results are mainly provided.
Access this chapter
Tax calculation will be finalised at checkout
Purchases are for personal use only
References
Grodstein, F., and Stampfer, M. (1995) The epidemiology of coronary heart disease and estrogen replacement in menopausal women. Prog. Cardiovasc. Dis. 38, 199–210.
Manson, J. E., Hsia, J., Johnson, K. C., Rossouw, J. E., Assaf, A. R., Lasser, N. L., Trevisan, M., Black, H. R., Heckbert, S. R., Detrano, R., Strickland, O. L., Wong, N. D., Crouse, J. R., Stein, E., and Cushman, M. (2003) Estrogen plus progestin and the risk of coronary heart disease. N. Engl. J. Med. 349, 523–534.
Women’s Health Initiative Study. (2002) Risks and benefits of estrogen plus progestin in healthy postmenopausal women. JAMA 288, 321–333.
Lipid Research Clinics Program. (1984) The lipids research clinics coronary primary prevention trials results. I. Reduction in incidence of coronary heart disease. JAMA 251, 351–364.
Poole, C. V., Carlton, D., Bimbo, L., and Allon, M. (2004) Treatment of catheterrelated bacteraemia with an antibiotic lock protocol: effect of bacterial pathogen. Nephrol. Dial. Transplant 19, 1237–1244.
Graff-Lonnevig, V., and Browaldh, L. (1990) Twelve hours bronchodilating effect of inhaled formoteral in children with asthma: a double-blind cross over study versus salbutamol. Crit. Exp. Allergy 20, 429–432.
Stampfer, M. J., Buring, J. E., Wilett, W. Rosner, B., Eberleiner, K., and Hennekens, C. H. (1985) The 2 × 2 factorial design: its application to a randomized trial of aspirin and carotene in US physicians. Stat. Med. 4, 111–116.
Women’s Health Initiative Study Protocol. (1994) Bethesda, National Institutes of Health.
Vijayaraghavan K., Radhaiah, G., Prakasam, B. S., Sarma, K. V. R., and Reddy, V. (1990) Effect of massive dose vitamin A on morbidity and mortality in Indian children. Lancet 336, 1342–1345.
Peterson, A. V., Mann, S. L., Kealey, K. A., and Marek, P. M. (2000) Experimental design and methods for school-based randomized trials: experience from the Hutchinson Smoking Prevention Project (HSPP). Control. Clin. Trials 21, 144–165.
Liaw, Y., Sung, J., Chow, W. C., Farrell, G., Lee, C., Yuen, H., Tanwandee, T., Tao, O., Shue, K., Keene, O. N., Dixon, J. S., Gray, D. F., and Sabbat, J. (2004) Lamivudine for patients with chronic hepatitis B and advanced liver disease. N. Engl. J. Med. 351, 1521–1531.
Efron, B. (1971) Forcing a sequential experiment to be balanced. Biometrika 58, 403–417.
Wei, L. J. (1977) A class of designs for sequential clinical trials. J. Am. Stat. Assoc. 72, 382–386.
Wei, L. J. (1978) An application of an urn model to the design of sequential controlled clinical trials. J. Am. Stat. Assoc. 73, 559–563.
Pocock, S. J., and Simon, R. (1975) Sequential treatment assignment with balancing for prognostic factors in the controlled clinical trials. Biometrics 35, 102–115.
Minert, C. L. (1986) Clinical Trials. New York, Oxford University Press.
Fisher, C. J., Dixon, D. O., Herson, J., Frankowski R. F., Hearron, M. S., and Peace, K. E. (1990) Intend-to-treat in clinical trials. In: Peace, K. E. Statistical Issues in Drug Research and Development. New York, Marcel Dekker.
Sheiner, L. B., and Rdubin, D. B. (1995) Intention-to-treat analysis and the goals of clinical trials. Clin. Pharmacol. Ther. 57, 6–15.
Coronary Drug Project Research Group. (1980) Influence of adherence to treatment and response of cholesterol on mortality in the Coronary Drug Project. N. Engl. J. Med. 303, 1038–1041.
Author information
Authors and Affiliations
Editor information
Editors and Affiliations
Rights and permissions
Copyright information
© 2007 Humana Press Inc., Totowa, NJ
About this protocol
Cite this protocol
Lim, H.J., Hoffmann, R.G. (2007). Study Design: The Basics. In: Ambrosius, W.T. (eds) Topics in Biostatistics. Methods in Molecular Biology™, vol 404. Humana Press. https://doi.org/10.1007/978-1-59745-530-5_1
Download citation
DOI: https://doi.org/10.1007/978-1-59745-530-5_1
Publisher Name: Humana Press
Print ISBN: 978-1-58829-531-6
Online ISBN: 978-1-59745-530-5
eBook Packages: Springer Protocols