Appendix B
Clinical Trial Designs
TABLE B-1 Brief Summary of Some Advantages and Disadvantages of Various Clinical Trial Designs
Design | Structure | Advantages | Disadvantages |
---|---|---|---|
Traditional RCT |
|
|
|
Design | Structure | Advantages | Disadvantages |
---|---|---|---|
Cluster Randomized Trials
(Campbell et al., 2004; Donner and Klar, 2004; Edwards et al., 1999) |
|
|
|
Design | Structure | Advantages | Disadvantages |
---|---|---|---|
Stepped Wedge |
|
|
|
Design | Structure | Advantages | Disadvantages |
---|---|---|---|
|
Design | Structure | Advantages | Disadvantages |
---|---|---|---|
Multiarm, Multistage Trial with a Common Control |
|
|
|
Design | Structure | Advantages | Disadvantages |
---|---|---|---|
Delayed Start |
|
|
|
Design | Structure | Advantages | Disadvantages |
---|---|---|---|
Adaptive Platform |
|
|
|
Design | Structure | Advantages | Disadvantages |
---|---|---|---|
Single Arm with Comparisons to Historical Controls |
|
|
|
Design | Structure | Advantages | Disadvantages |
---|---|---|---|
Uncontrolled Case Series | A group or series of case reports involving patients who were given similar treatment. Reports of case series usually contain detailed information about the individual patients before and after an intervention but with no control group.
|
Some appropriate settings for the use of the case series study design:
|
|
REFERENCES
Brown, C. A., and R. J. Lilford. 2006. The stepped wedge trial design: A systematic review. BMC Medical Research Methodology 6(1):54.
Campbell, M. K., D. R. Elbourne, and D. G. Altman. 2004. CONSORT statement: Extension to cluster randomised trials. BMJ 328(7441):702–708.
D’Agostino, R. B. 2009. The delayed-start study design. New England Journal of Medicine 361(13):1304–1306.
Donner, A., and N. Klar. 2004. Pitfalls of and controversies in cluster randomization trials. American Journal of Public Health 94(3):416–422.
Edwards, S. J. L., D. A. Braunholtz, R. J. Lilford, and A. J. Stevens. 1999. Ethical issues in the design and conduct of cluster randomised controlled trials. BMJ 318(7195):1407–1409.
Evans, S. R. 2010. Clinical trial structures. Journal of Experimental Stroke & Translational Medicine 3(1):8–18.
Ford, D. E. 2010. Study design case series and cross-sectional. Johns Hopkins Institute for Clinical and Translational Research. http://ictr.johnshopkins.edu/wp-content/uploads/import/1274-Ford%20Final%20Rev%20Cross%20Sectional%20July%2012%202011.pdf (accessed January 25, 2017).
Glasziou, P., I. Chalmers, M. Rawlins, and P. McCulloch. 2007. When are randomised trials unnecessary? Picking signal from noise. BMJ 334(7589):349–351.
Hughes, J. P. 2007. Stepped wedge design. In Wiley Encyclopedia of Clinical Trials: 1–8. Hoboken, NJ: John Wiley & Sons, Inc.
Jaki, T. 2015. Multi-arm clinical trials with treatment selection: What can be gained and at what price? Clinical Investigation 5(4):393–399.
Kempen, J. H. 2011. Appropriate use and reporting of uncontrolled case series in the medical literature. American Journal of Ophthalmology 151(1):7–10.e11.
Quinlan, J., B. Gaydos, J. Maca, and M. Krams. 2010. Barriers and opportunities for implementation of adaptive designs in pharmaceutical product development. Clinical Trials 7(2):167–173.
Saville, B. R., and S. M. Berry. 2016. Efficiencies of platform clinical trials: A vision of the future. Clinical Trials 13(3):358–366.
Suresh, K. P. 2011. An overview of randomization techniques: An unbiased assessment of outcome in clinical research. Journal of Human Reproductive Sciences 4(1):8–11.
Velengtas, P., P. Mohr, and D. A. Messner. 2012. Making informed decisions: Assessing the strengths and weaknesses of study designs and analytic methods for comparative effectiveness research. Washington, DC: National Pharmaceutical Council. http://www.npcnow.org/publication/making-informed-decisions-assessing-strengths-and-weaknessesstudy-designs-and-analytic (accessed January 25, 2017).
Wason, J., D. Magirr, M. Law, and T. Jaki. 2016. Some recommendations for multi-arm multistage trials. Statistical Methods in Medical Research 25(2):716–727.