Methodische Aspekte bei der Durchführung von Arzneimittelsicherheits- und Utilisationsstudien basierend auf Routinedaten der gesetzlichen Krankenversicherung

At the time of approval, the safety and utilization of drugs in routine clinical practice cannot be fully assessed. Therefore, active post-marketing surveillance including the determination of the safety but also the utilization of the drugs as part of risk management plans is required by regulatory agencies. Pharmacoepidemiological studies based on claims data from statutory health insurance (SHI) providers are increasingly used to address these questions. These studies require a comprehensive knowledge of the healthcare system including reimbursement policies as well as specifics of the data source. Based on several safety and utilization studies using claims data, this thesis discusses methodological aspects relevant to conducting these studies. Considering regulations of the German health care system, the assessment of exposure, outcomes, and confounders as well as the determination of potential indications are described. Further, this thesis discusses approaches for addressing confounders which either cannot be measured in databases or if, only inadequately. Since even large electronic healthcare databases may not provide a sufficient sample size to examine rare outcomes or rare exposures, multinational database studies including databases with different structures are conducted. This thesis characterizes challenges arising from the analysis of these different databases. Pharmacoepidemiological studies based on SHI claims data are an important instrument to assess the safety and utilization of drugs following approval. The development of pharmacoepidemiology is characterized by the implementation of suitable data sources as well as the development of methodological instruments. This thesis depicts the current status and outlines future challenges.