Clinical trial designs: a simplified overview
DOI:
https://doi.org/10.18203/2349-3259.ijct20232153Keywords:
Clinical trial design, Adaptive clinical trial design, Types of clinical trial, Randomized control clinical trial, Placebo control clinical trialAbstract
Clinical trials aim to invent new, better, safer, more effective ways to prevent, detect, or treat health conditions and to compare them to existing standards of care as applicable. This scientific inquiry prompts researchers to design and develop proposed clinical trial protocols to evaluate preliminary information about the product and develop research questions and objectives. The average budget for new product development is enormous, and this eventuality is because almost 90 percent of a trial drugs never get approved. Hence, having an appropriate clinical trial design is crucial for successful product development. A trial's ability to deliver the proposed indication relies on its suitable design, background information, the trial justification to sample size, interim monitoring rules, and ways to review and analyze study data. In this article, we presented an overview of clinical trial designs emphasizing adaptive designs in a simplified way for field-based clinical research professionals.
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References
Office of the Commissioner & Office of the Commissioner. 2018. Drug Study Designs. U.S. Food and Drug Administration. Available at: https://www.fda.gov/regulatory-information/search-fda-guidance-documents/drug-study-designs. Accessed on 09 March 2023.
Nair B. Clinical Trial Designs. Indian Dermatol Online J. 2019;10(2):193.
Enck P, Klosterhalfen S. Placebos and the Placebo Effect in Drug Trials. Handbook Exp Pharmacol. 2019;399-431.
Chidambaram AG, Josephson MB. Clinical research study designs: The essentials. Pediatric Investig. 2019;3(4):245-52.
Efird JT. Blocked Randomization with Randomly Selected Block Sizes. Int J Env Res Public Health. 2010;8(1):15-20.
Higgins JPT, Eldridge S, Li T. Chapter 23: Including variants on randomized trials. Cochrane Handbook for Systematic Reviews of Interventions version 6.3 (updated February 2022). Cochrane. 2022.
Jaikumar V. Cluster Randomized Trials: Concepts - Tutorials and Fundamentals. Students 4 Best Evidence. 2022.
Simon GE, Shortreed SM, DeBar L. Zelen design clinical trials: why, when, and how. Trials. 2021;22(1).
Jin M, Polis AB, Hartzel J. Algorithms for minimization randomization and the implementation with an R package. Communications in Statistics - Simulation and Computation. 2021;50(10).
Turner JJ. Crossover Design. Springer eBooks. 2013;521.
Krzywinski M, Altman N. Two-factor designs. Nature Methods. 2014;11(12):1187-8.
Chen Y, Gesser RM, Luxembourg A. A seamless Phase IIB/III adaptive outcome trial: Design rationale and implementation challenges. Clinical Trials. 2015;12(1):84-90.
Center for Drug Evaluation and Research. Adaptive designs for clinical trials of drugs and Biologics guidance. U.S. Food and Drug Administration. 2019. Available at: https://www.fda.gov/regulatory-information/search-fda-guidance-documents/ adaptive-design-clinical-trials-drugs-and-biologics-guidance-industry. Accessed on 09 March 2023.
Dodd LE, Proschan MA, Neuhaus J, Koopmeiners JS, Neaton JD, Beigel JH, et al. Design of a Randomized Controlled Trial for Ebola Virus Disease Medical Countermeasures: PREVAIL II, the Ebola MCM Study. J Infect Dis. 2016;213(12):1906-13.
FDA-NIH Biomarker Working Group. Best (biomarkers, endpoints, and other tools) resource. National Center for Biotechnology Information. 2016.
Fisher LD. Self-designing clinical trials. Statistics Medicine. 1998;17(14):1551-62.
Friede T, Kieser M. Blinded sample size reassessment in non-inferiority and equivalence trials. Statistics in Medicine. 2003;22(6):995-1007.
Glimm E, Maurer W, Bretz F. Hierarchical testing of multiple endpoints in group-sequential trials. Statistics in Medicine. 2010;29(2):219-28.
Gould A. Sample size re-estimation without unblinding for normally distributed outcomes with unknown variance. 1992. Available at: https://www.semanticscholar.org/paper/Sample-size-re-estimation-without-unblinding-for-Gould-Shih/219e5dc9baa20fc879c6629cbf5925111fc5df4. Accessed on 09 March 2023.
Stallard N, Todd S. Seamless phase II/III designs. Statistical Methods in Medical Research. 2011;20(6):623-34.
Cui L, Hung HMJ, Wang S. Modification of Sample Size in Group Sequential Clinical Trials. Biometrics. 1999;55(3):853-7.
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