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Advanced Clinical Study Design

Drug Development
Workshop profile:
This workshop will be of most value to those who already have some experience in clinical development and have become familiar with different kinds of clinical trial. Terms such as crossover, double dummy, baseline, endpoint, active control, intent to treat, per protocol, and surrogate endpoints will be used extensively but will not be explained. The content is not immediately sequential to the content of the beginner's workshop on Basic Study Design. You should have a minimum of 2 years of clinical development experience or at least a sound science background to obtain maximum benefit from this workshop.
Study Reports and Clinical Summaries all demand critical discussion of design issues and the choice of the control groups. Participants should become confident drafting the relevant sections of these documents.
The workshop is based on regulatory guidelines covering confirmatory trials of efficacy. The format is classroom-style. The topics covered will be firstly crossover and parallel-group design together with a brief introduction to novel strategies such as factorial design, flexible design and designs for small populations. Secondly, control group options such as placebo, ‘ethical’ placebo, multiple dose and active controls (equivalence and non-inferiority), will be covered. Differences between the EMA and the FDA in the weight attached to studies using active controls on the one hand and placebo on the other, will be highlighted. The final section will be a review of the critical issues that must be discussed in a report and summary documents. There will be group exercises on evaluating the period effects and adequacy of conduct of a hypothetical crossover trial and selecting an appropriate control group depending on the objective of a trial. Based on your assessment of the strengths and weaknesses of the design and conduct of the study, the post homework assignment will require you to critically evaluate a hypothetical study report. Overall the approach will be intuitive, not statistical.