Masterstudiengang "Drug Regulatory Affairs"

Master-Thesis

Effectiveness of Risk Minimisation Measures in the EU - Evaluation Strategies and Impact on Regulatory Decisions ***

Jelena Katic (Abschlußjahr: 2022)

Summary
Language: English
Risk minimisation measures (RMMs) are essential pharmacovigilance tools for optimising the benefit-risk balance of medicinal products for human use, with the ultimate purpose to prevent or reduce the frequency and severity of adverse health outcomes. Since the implementation of the current pharmacovigilance legislation of the European Union (EU) in 2012, it is obligatory for marketing authorisation holders (MAHs) to monitor the results of RMMs included in the risk management plan (RMP) or imposed as a condition of the marketing authorisation. While extensive experience has been gained ever since, respective methodological and regulatory guidelines are continuously evolving. Thus, the main goal of this master thesis was to present a qualitative review of studies evaluating RMM effectiveness that have been recorded in the European Union electronic Register of Post-Authorisation Studies (EU PAS Register) up to 3 April 2022. Furthermore, the impact of studies evaluating the effectiveness of RMMs on regulatory decisions in the EU is described based on illustrative examples from safety referral procedures retrieved from the European Medicines Agency´s website.
Strategies of RMM effectiveness evaluations are discussed with respect to the type of evaluated RMMs, employed data sources, indicators of RMM effectiveness, time of evaluation, data analysis and representation of EU member states. The review also reflects on the data availability and study registration details in the EU PAS Register. Overall, 83 out of 429 screened studies were included in the analysis based on pre-defined criteria. While the findings were in many aspects consistent with the published literature, this review identified relevant differences between category 1 and category 3 studies in terms of evaluated RMMs, employed data sources, indicators of RMM effectiveness and time of evaluation.
Overall, the analysed dataset from publicly available sources was adequate to meet the objectives of this master thesis, based on which it can be concluded that studies evaluating the effectiveness of RMMs may have a relevant impact on regulatory decision in the EU concerning the initiation, assessment and outcome of benefit-risk evaluations for medicinal products. The effectiveness evaluation of RMMs has evidently become an integral part of pharmacovigilance activities in the EU representing a valuable asset for improving the risk minimisation strategy and safety of medicines.
Pages 76
Annexes: 3, Pages: 19