Université Paris Descartes (UPD)
WP6 aims to establish standards for the design of randomised trials in personalised medicine through the involvement of multiple stakeholders including medicines agencies and HTAs.
WP6 will explore the added value of the personalised approach vs. non-personalised standard of care. It will address questions related to: stratification in the context of new trial designs (umbrella, basket, platform, trials within cohort); the corresponding statistical analysis; the cost-effectiveness and socio-economic impact of personalised medicine; and the involvement of patients in trial design.
Description of work:
A workshop will be organised with stakeholders, investigators and sponsors having designed, planned and conducted complex personalised medicine trials. The expected output will be a report on innovative trial designs for personalised medicine.
Moreover, the work package will develop a report describing the methodology of comparative effectiveness research on personalised medicine. It will also describe the level of evidence expected by HTAs to validate the personalised medicine approach and to define an optimal level of stratification. Finally, it will describe the methods used to assess the cost-effectiveness of personalised medicine.