Trial-based Economic Evaluations: Methodological and Applied Studies

Due to high and rising healthcare costs in combination with budget constraints and rising demands for healthcare, resources for healthcare are scarce. Therefore, healthcare decision-makers need to allocate these scarce resources as efficiently as possible with the aim to maximize health benefits within the available budget. To inform such decisions, they need information on the relative efficiency of alternative healthcare interventions, which can be provided by economic evaluations. A prerequisite for using results of so-called trial-based economic evaluations in day-to-day decision-making, is that they are valid and reliable. Using suboptimal methods can lead to biased conclusions and thus potentially to a waste of scarce resources in healthcare. Within this thesis, optimal statistical methods for trial-based economic evaluations are identified by summarizing recommendations in the health economic literature, evaluating their statistical performance in methodological studies and demonstrating their use in applied trial-based economic evaluations. In order to facilitate researchers in employing optimal statistical methods in trial-based economic evaluations, this thesis includes statistical software code.