The Design and Analysis of N of 1 Trials

Introduction

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.

Topics covered

Uses of n-of-1 trials and purposes of analysis

Showing the treatment can work
Understanding variation in effect
Predicting effects

Design

Randomisation in cycles
Randomisation in patient blocks

Graphical presentation of results

Trellis plots
Dot plots

Causal analysis

Analysis of variance

Block structure and the Wilkinson & Roger notation
Main effect models
Allowing for interaction

Summary measures approaches

Mixed models

Estimation
Best linear unbiased predictors (shrunk estimates)

Meta-analysis approaches

Fixed effects
Random effects

Binary data

Planning issues

Early
(up to and including 6^th June)

Late
(after 6^th June)

Academic (includes non voting reduced membership)

£245 + VAT

£295 + VAT

PSI member

£295 + VAT

£345 + VAT

Non-member (includes PSI membership for one year)

£375 +VAT

£425 + VAT

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