Post-Approval Regulatory Requirements

Health authorities throughout the world have regulations for requesting additional research in the post-approval setting. This chapter focuses on the regulations in the USA and European Union (EU). The history of post-approval studies can be traced through changing regulations enforced by the US Food and Drug Administration (FDA) and the EU European Medicines Agency (EMA). Post-approval studies are either clinical trials (interventional) or observational (non-interventional) studies. Choosing a study design may be influenced by the strengths and weaknesses of the design options and available data sources. Imposed post-approval studies are reviewed for compliance by the regulatory agencies. For clinical trials that are ongoing at the time of approval, often these are classified as post-marketing commitment (PMC) in the USA or post-authorization measure (PAM) in the EU. Findings of these trials can be submitted to the health authorities for addition to the prescribing information. The FDA and EMA both track progress on PMC/PMRs and PAMs, respectively. Post-approval studies are necessary to continually gather data on the safety and effectiveness of approved drugs. These studies are regulated by health authorities, included in registries (e.g., ClinicalTrials.gov, ENCePP), and tracked to completion. This chapter reviews the history of the regulations, terminology, study designs, and systematic reviews of the published post-approval studies.