What is a good decision in medicine regulation? Unravelling the regulatory thought process

Before a medicine can be marketed and prescribed to patients, its quality, efficacy, and safety need to be assessed by a regulatory authority such as the European Medicines Agency (EMA). Regulatory assessment is a complex task, which can be influenced by both technical and social factors. The goal of this research was to gain more insight in the ‘regulatory thought’ process that shapes decision making by using an ethnographic approach to study decision making in practice at the Dutch Medicines Evaluation Board (MEB).

When assessing the benefit-risk balance of a drug, regulators may value several aspects of the drug (e.g., uncertainty about efficacy or safety) and its context (e.g., unmet need of the patients) differently. This means that there are multiple kinds of good decisions, depending on what one values. Based on what MEB regulators strived for in practice, this dissertation provides three answers to the question: what is a good decision in medicine evaluation, and how can decision making be improved? These three answers focus on the regulator’s concern with 1) the scientific credibility of the claimed effects of a drug; 2) the value of a drug for clinical practice and 3) the consistency of their decision making. This dissertation illustrates how regulators may strive for multiple, potentially conflicting, values in daily practice. Analyzing regulatory decision making like this allows one to compare and contrast what decisions (could) look like when prioritizing different values, which creates opportunities for reflection and change.