Real-World Data in the Postapproval Setting as Applied by the EMA and the US FDA

Purpose: This article provides an analysis of the various regulatory decisions available in the public domain that suggest the use of real-world data (RWD) for postmarketing surveillance activities of products that have a marketing authorization approved by the European Medicines Agency (EMA) and the US Food and Drug Administration (FDA). The study focuses on the cases in which RWD was used for postapproval commitments or requirements (and to a lesser extent label extensions, as this has been previously published) of medicinal products comprising small molecules and biologics to support efficacy claims or confirm an acceptable safety profile.

Methods: Clarivate Analytics was commissioned to collect data from cases in which RWD was used in the postapproval settings submitted to the EMA (data were found covering the last 14 years) and the FDA (data were found spanning 23 previous years). The query resulted in 165 cases in which regulatory approval was associated with RWD. The data were then categorized and expanded with supporting information gathered from public databases and company websites.

Findings: The use of RWD to support regulatory decision-making in the postmarketing surveillance setting has increased in recent years. Most postmarketing surveillance activities are legally enforced requests on the marketing authorization holder to further document the product's safety profile. Data drawn from registries tend to be the most common source in this regard.

Implications: RWD have increasingly been used in recent years, both for new product approvals and line extensions and, as shown in this study, in the postapproval setting. There is now a growing appreciation of the potential of RWD as a source in its own right to support regulatory decision-making on the benefits and risks associated with clinical interventions.