Joint modelling of the disease process and the treatment allocation process in long term follow-up studie



Juni Palmgren
Rolf Nevanlinna Institute, Section of Biometry
00014 University of Helsinki
Finland


Abstract

Assessment of the effect of 'treatment actually received' on a disease endpoint is obscured by the fact that (i) those who 'adhere to treatment', i.e. the compliers, may differ from the noncompliers on important risk factors, and (ii) the disease endpoint itself may influence the treatment process. Using the notion of potential outcomes we specify a model for the treatment process and for the disease process and for the association between these. We relate observed data to the potential outcomes and discuss estimation strategies for retrieving 'causal' parameters of interest. We illustrate the argument on a randomized placebo controlled study by relating the total active dose received to a survival endpoint. We discuss implications of the randomization.