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Unit of study_

CLTR5001: Trial Design and Methods

This unit of study will focus on the strengths and weaknesses of different clinical study designs. Designs considered will include cohort (retrospective and prospective), cross-sectional, case-control and randomized controlled designs. The different phases of clinical trial designs in the development of therapies will also be examined including phase I (first in man), phase II/pilot and phase III comparative designs. Extension and adaptation of randomized designs will also be covered including cluster and factorial designs and adaptive pilot studies. Students will gain the skills necessary to choose between these designs for best practice. Types of outcomes (continuous, categorical, time-to-event) will be discussed. Methods of allocating participants to interventions (randomization), as well blinding and allocation concealment will be covered together with aspects of protocol development. On completion of this unit, the student will be familiar with the differences between study types and study designs, as well as the principles and practice of randomisation. It is also expected that the candidate will be able to develop stratified randomisation schemes for their own studies.

Code CLTR5001
Academic unit NHMRC Clinical Trials Centre
Credit points 6

At the completion of this unit, you should be able to:

  • LO1. understand which phase and/or type of study is required to address the primary question of any trial
  • LO2. understand basic statistical concepts of a trial design, such as sample size calculations and randomisation
  • LO3. understand the basic principles of protocol design and the key aspects that go into it
  • LO4. understand the different study designs (observational and experimental), the advantages and disadvantages of each, and when they are appropriate to use
  • LO5. understand the different types of study outcomes
  • LO6. understand how outcomes relate to study design
  • LO7. understand basic principles for sample size calculations
  • LO8. understand outcomes which are specific to oncology studies
  • LO9. understand the purpose, design and analysis, and interpretation of phase 1 and 2 clinical trials (including dose-finding)
  • LO10. show a basic understanding of phase 3 trials
  • LO11. understand the rationale for phase 4 clinical trials
  • LO12. understand the rationale for and the principle of phase 3 studies and RCTs
  • LO13. understand different study designs (parallel, crossover, etc.) and when they would be appropriate to use
  • LO14. understand the difference between superiority, non-inferiority and equivalence studies
  • LO15. understand the theory behind basic methods of randomisation and why randomisation is important
  • LO16. understand the role of less common approaches to randomisation
  • LO17. understand the important of blinding and the different types
  • LO18. understand methods of allocation concealment
  • LO19. understand the basic format, importance and purpose of a protocol
  • LO20. understand how the topics in this unit relate to the sections of a protocol.

Unit outlines

Unit outlines will be available 1 week before the first day of teaching for the relevant session.