Statistics in clinical trials.

Teacher(s)

Legrand Catherine; Robert Annie;

Language

French

Main themes

The following topics will be discussed: - International guidelines in clinical trials. - Phase 1: pharmacokinetics and pharmacodynamics. - Phase 1: dose determination: the continual reassessment method. - Phases 2 & 3: hypothesis tests in efficacy, superiority or equivalence trials. - Phases 2 & 3: power and sample size computation, randomisation and blinding. Application to sequential trials. - Phases 2 & 3: cross-over and factorial designs. - Phase 4: pharmacovigilance. Rare events and risk factors. - Reporting in clinical trials.

Learning outcomes

At the end of this learning unit, the student is able to :
1	Objectives The goal of this course is to propose a broad overview of the statistical aspects of phase 1, 2, 3 and 4 clinical trials.

Content

The following topics will be discussed: - International guidelines in clinical trials. - Phase 1: pharmacokinetics and pharmacodynamics. - Phase 1: dose determination: the continual reassessment method. - Phases 2 & 3: hypothesis tests in efficacy, superiority or equivalence trials. - Phases 2 & 3: power and sample size computation, randomisation and blinding. Application to sequential trials. - Phases 2 & 3: cross-over and factorial designs. - Phase 4: pharmacovigilance. Rare events and risk factors. - Reporting in clinical trials.

Teaching methods

The course consists of lectures and discussion of documents distributed during the course.
Practical works are also organised. They aim to: - deepen concepts introduced during the course, - analyse real data using tools presented during the course.
Depending on the evolution of the situation, the course will be given either in presential or remotely.

Evaluation methods

Closed-book written exam. 
Depending on the evolution of the situation, the written exam could be replaced by a closed-book oral exam organised remotely. 

Other information

Prerequisites: Bases of probability and descriptive and inferential statistics, basic knowledge of SAS and R.

Online resources

All necessary resources will be made available via Moodle.

Bibliography

Redmond, C. K. and Colton T. (2001), Biostatistics ub Clinical Trials, Wiley.
Fleiss J. (1986), The Design and Analysis of Clinical Experiments. Wiley.

Teaching materials

• transparents sur moodle

Faculty or entity

LSBA