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 EIWG Webinar: PIONEERing estimands in Clinical Research (Session 2)
Bringing estimands to life through a real case study. Members of the estimands Implementation Working Group (EIWG) will describe the background and study objectives, intercurrent events of interest and chosen estimands in the PIONEER study.
Our annual PSI Medical Statistics Careers Event, will be held online using the Career Fair Plus app on Wednesday 3rd March 2021. The event will include a live online panel discussion and a virtual exhibition session.
Discover your extraordinary potential by working as a Covance AD, Biostatistics / Senior Principal Biostatistician. You will consult in the design of complex and innovative studies & clinical trial programs as well as Leading DMC/DSMB processes.
By joining our company, in this role you will apply your skills and experience in support of developing scientifically based arguments aiming to provide fair accessibility to our company’s drugs and vaccines making a real difference in patient’s lives.
Senior Principal / Principal Biostatistician (Oncology) – FSP
Join our team as a Covance FSP Senior Principal or Principal Biostatistician. You will be assigned to one Sponsor working on Phase I and II oncology studies. Previous Lead experience essential. Office or home-based anywhere in Europe or South Africa.