WP6 – Design of pharmacogenetic small population group trials
including cross-over trials, n-of-1 trials and enrichment trials
Individual response to treatment is often poorly studied and understood. It is necessary to isolate the various components of variation: pure between patient, pure within patients and patient by treatment interaction. This work-package is particularly concerned with developing approaches to identifying such components.
The following objectives will be met:
- Identification of conditions that have to be met for personalising therapy to be worthwhile, paying particular attention to so-called theranostic (combined diagnostic and therapeutic) approaches
- Development of practical and useable n-of-1 (or repeated cross-over) templates for those disease areas where repeated treatment of patients is possible
- Development of suitable approaches to analysing subgroups for those diseases where cross-over trials are not possible
- Establishing approaches to sample-size determination that take account of the value of information
It is envisaged that each of the tasks will lead to a paper, which will include recommendations and guidance, and that tasks 1,2 and 4 will involve writing code that will be made publicly available.