Numerous problems may be encountered when evaluating and interpreting estimates of comparative clinical efficacy as highlighted by an evaluation of pharmacoeconomic analyses assessed by the Australian Pharmaceutical Benefits Scheme. Such problems include the availability or quality of trials, analysis or interpretation of results, use of appropriate outcome measures, and determination of therapeutic dose and equivalence. Aspects of the assessment of treatment effectiveness were addressed in recent major work; however, a number of areas specific to pharmaceutical evaluation and reimbursement were not addressed there. These include equivalence, non-inferiority, and the use and interpretation of surrogate and composite clinical outcomes. In this chapter we first present general discussions of randomised trials, systematic reviews, and meta-analysis of randomised trials. We next consider special topics not previously addressed elsewhere.
Evaluating Pharmaceuticals for Health Policy and Reimbursement p. 24-45