Real World Data en de vergunnings-levenscyclus voor het in de handel brengen van geneesmiddelen – Op zoek naar een geschikt EU wettelijk kader

For a medicinal product to be authorised onto the EU market, an applicant needs to prove the product's efficacy and safety. Evidence for this is traditionally generated through clinical trials. However, clinical trials have flaws. They arguably take (too) much time, are expensive, and are insufficiently generalisable. Therefore, stakeholders involved in the marketing authorisation lifecycle for medicinal products are exploring a different source for evidence generation namely Real World Data (RWD). My research defines RWD as 'data relating to people's health status or the delivery of healthcare from sources other than clinical trials.' This source of evidence has its flaws too, primarily linked to the risk of reduced proven causality given the absence of safeguards such as randomisation and blinding. Nevertheless, European and U.S. authorities increasingly refer to RWD, occasionally in legislation, and abundantly in policy documents. This dissertation investigates whether EU law in a suitable way enables the availability of RWD, and whether by doing so, it complies with the principle of legal certainty. Given its nature, RWD is at the crossroad of two distinct EU legal frameworks, specifically EU health law and EU data law. Therefore, this dissertation firstly assesses the EU health law framework's applicability to RWD. For each phase of the marketing authorisation lifecycle for medicinal products, it analyses whether EU pharmaceutical law specifically strikes the right precautionary balance to assess the suitability of RWD availability. Further, I assess those EU data laws with rules tailored to health-related data in a research context. Each data law has an impact on RWD accessibility (defined in line with the FAIR principles), again granting insights on RWD availability. The research demonstrates that the lack of alignment between EU health law with EU data law is detrimental to legal certainty. In addition, neither of the EU legal frameworks truly benefits from the introduction of the concept 'RWD' in the semantics. My research proposes to provide a clearer framework for non-interventional clinical studies, tied to an overarching RWD framework at policy level, including a more explicit evidence hierarchy in EU pharmaceutical legislation, supported by clearer potential routes for secondary RWD use as authorised by current EU data laws.