ABSTRACT

Compliance is important. Better adherence to treatment regimes leads to lesshealthcare resource utilization overall, as fewer illness recurrence or medicationerrors leading to side-effects take place. Traditionally, we have seen a conflict between assessments of the efficacy of treatments and the assessment of effectiveness of treatments. Some trials are designed to test the maximal effect of a treatment (that is, efficacy), which others argue is unrealistic and therefore misleading. This group argues for trials based on ‘real-world’ scenarios (that is, effectiveness). This conflict is natural and inevitable as both objectives are valid, assuming that the trial environment can be replicated in a meaningful way in routine medical care. Furthermore, the conflict can be minimized by accepting that both objectives can be assessed in a competent development plan for a new treatment, answering all critical questions.