N of 1 trials are trials in which individual patients are repeatedly treated with experimental and control treatments in a deliberate and designed manner using principles of control, randomisation and replication. Their uses include personalising treatment and increasing efficiency by reducing the number of patients it is necessary to study.
In chronic diseases, sets of n-of-1 trials (in which a limited number of patients follow an n-of-1 protocol) have great potential as phase IV trials for understanding components of variation but may also constitute possible Phase III programmes for rare diseases. They can also be used as phase II studies for proof of concept and dose-finding. However, they are often poorly analysed and, indeed, much of the published advice is poor.
This course will present the latest thinking on n-of-1 trials and cover not only their analysis through SAS®, R® GenStat® and meta-analysis packages but also approaches to design. They will also be critically examined as to their potential use in a) establishing average effects of treatment b) studying the extent to which such effects vary from patient to patient and c) optimising treatment for individual patients.
This course will be given by Professor Stephen Senn, of the Luxembourg Institute of Health, who is well- known for his work on the design and analysis of clinical trials and the application of statistics in drug development.
Uses of n-of-1 trials and purposes of analysis
Showing the treatment can work
Understanding variation in effect
Randomisation in cycles
Randomisation in patient blocks
Graphical presentation of results
Analysis of variance
Block structure and the Wilkinson & Roger notation
Main effect models
Allowing for interaction
Summary measures approaches
Best linear unbiased predictors (shrunk estimates)
PSI, the European Federation of Statisticians in the Pharmaceutical Industry (EFSPI) and the Biopharmaceutical Section of the American Statistical Association (ASA) are jointly organising a webinar on Estimands in Practice. Speakers from regulatory authorities (FDA and EMA) and industry will present on their experience on this topic to date.
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