Course overview
The aim of this course is to enable students to understand and apply the principles of design and analysis of experiments, with a particular focus on randomised controlled trials (RCTs), to a level where they are able to contribute effectively as a statistician to the planning, conduct and reporting of a standard RCT. Topics include: ethical considerations; principles and methods of randomisation in controlled trials; treatment allocation, blocking, stratification and allocation concealment; parallel, factorial and crossover designs including n-of-1 studies; practical issues in sample size determination; intention-to-treat principle; phase I dose finding studies; phase II safety and efficacy studies; interim analyses and early stopping; multiple outcomes/endpoints, including surrogate outcomes, multiple tests and subgroup analyses, including adjustment of significance levels and p-values; missing data; reporting trial results and use of the CONSORT statement.
Course learning outcomes
- See Studies Guides at: https://url.au.m.mimecastprotect.com/s/jyzMCmO5QMCjMl1XJtJi1URPVES?domain=bca.edu.au/
- Identify the benefits of randomisation as a mechanism for reducing bias, and implement a variety of randomisation schemes.
- Demonstrate knowledge of the principles behind the common experimental designs.
- Describe the efficiency advantages of crossover designs, and be able to design and interpret the two-period crossover study.
- Demonstrate an understanding of the principles underlying Phase I, II, III and IV studies, as well as an appreciation of the scientific basis underlying issues in clinical studies including intention-to-treat, blinding, interim analyses, subgroup analyses and the handling of missing data.
- Appreciate the importance of sample size in clinical studies, and perform sample size calculations for a variety of trial designs with different outcomes.