Related Experiment Video
Updated: May 30, 2026

A Clinical Trial Assessing the Safety, Efficacy, and Delivery of Olive-Oil-Based Three-Chamber Bags for Parenteral Nutrition
Published on: September 20, 2019
Statistical considerations for the next generation of clinical trials
Wenting Wu1, Qian Shi, Daniel J Sargent
1Division of Biomedical Statistics and Informatics, Department of Health Science Research, Mayo Clinic and Mayo Foundation, Rochester, MN 55905, USA. wu.wenting@mayo.edu
Abstract:
In this article, we address some of the statistical issues associated with the next generation of oncology clinical trials. Specifically, we focus on two critical aspects of oncologic clinical trials: study endpoints and study design. In our discussion of study endpoints, we provide an overview of endpoints relevant to each phase of clinical trials (phases I, II, and III) and discuss some of the trends in selecting endpoints in recent years. For phase I designs, the traditional assumption that increasing dose will always lead to increasing efficacy, appropriate for cytotoxic agents, is not applicable to novel therapeutics such as in colorectal carcinoma. We emphasize the role of surrogate endpoints in modern clinical trials. In our discussion of study design, we are particularly interested in the essential role of randomization, and discuss recently developed randomized phase II designs. We consider the role biomarkers play in the design of clinical trials and introduce study designs for biomarker evaluations. Finally, we briefly discuss the application of adaptive designs in clinical trials.
Related Concept Videos
Clinical Trials
There are four phases in a clinical trial. A phase one...
Clinical Trials: Overview
Types of Biopharmaceutical Studies: Controlled and Non-Controlled Approaches
Non-controlled studies, commonly employed for initial exploration, lack a control group, rendering them susceptible to biases and external influences. In contrast, controlled...
Bioavailability Study Design: Healthy Subjects Versus Patients
Study Design in Statistics
Does aspirin reduce the risk of heart attacks? Is one brand of fertilizer more effective at growing roses than another? Is fatigue as dangerous to a driver as the influence of alcohol? Questions like these are answered using randomized experiments with proper...
Statistical Software for Data Analysis and Clinical Trials
