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What is trial design and why is it important in drug development?

Trial design is the protocol that is developed for a clinical trial or study to achieve an answer to a question of interest, such as determining whether an investigational compound is effective. It includes factors such as how many participants will be needed to arrive at a valid answer and what types of controls will be necessary. To learn more about trial design and why it is important for drug development, click on the links below.

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Relevant publications

• Green: Design of Randomized Trials
• Krause and Howard: What Random Assignment Does and Does Not Do
• Mayo and Gajewski: Bayesian Sample Size Calculations in Phase II Clinical Trials Using Informative Conjugate Priors
• Lin and Shih: Adaptive Two-Stage Designs for Single-Arm Phase IIA Cancer Clinical Trials
• Fazzari et al.: The Phase II/III Transition. Towards Proof of Efficacy in Cancer Clinical Trials
• Marschner: Optimal Design of Clinical Trials Comparing Several Treatments with a Control
• Pangeas et al.: An Optimal Two-Stage Phase II Design Utilizing Complete and Partial Response Information Separately
• Sambucini: A Bayesian Predictive Two-Stage Design for Phase II A Clinical Trials
• Stallard and Todd: Sequential Designs for Phase III Clinical Trials Incorporating Treatment Selection
• Taylor et al.: Comparing an Experimental Agent to a Standard Agent
• FDA: Guidance for Industry – Choice of Control Groups and Related Issues in Clinical Trials
• IOM: Small Clinical Trials: Issues and Challenges

Related Tools

• OOPD and ORDR: The Science of Small Clinical Trials