(027) More-EUROPA (More Effectively Using Registries) – A cross-sectional survey assessing evidence needs around the introduction of new medicines

Background: Several decisions are taken by different stakeholders along the life cycle of pharmaceuticals. These decisions have implications for the availability, coverage, and use of medicines and they should be based on the best available evidence. In recent years, new opportunities to generate real-world evidence (RWE) have arisen through the development of data sources and novel methods. There is limited knowledge on how RWE is being valued in decision-making.

Objectives: To assess the attitudes and experiences among different stakeholders toward different types of evidence when making decisions regarding the introduction of new medicines.

Methods: This study was conducted as part of the More-EUROPA project that explores perspectives of regulators and other stakeholders on accepting RWE in the assessment of new medicines. A cross-sectional survey was distributed to 1210 European regulators and other stakeholders (academia, HTA-agencies, payers and healthcare professionals) involved in decisions along the life cycle of pharmaceuticals. The survey included questions on their experience and opinions on RWE. Data collection took place November 2023-January 2024.

Results: In total, 191 participants (110 regulators, 24 HTA/payers and 57 other stakeholders) (response rate: 18%) completed the survey. Most respondents were between 41-60 years, 53% had >10 years work experience, and 66% were women. All stakeholders considered randomization and clinically relevant endpoints as the most important aspects when assessing the clinical effect of a medicine. Country specific data and patient preferences were seen as the least important. Other stakeholders were generally more positive than regulators in using RWE to address remaining uncertainties during the life cycle of a medicine. RWE was considered likely to be accepted as supplementary evidence for clinical effect of a medicine by all stakeholders for example in situations such as orphan diseases, paediatric investigations, and situations of high unmet clinical need, but other stakeholders were generally more optimistic that RWE could also be accepted as pivotal evidence compared to regulators. Differences between stakeholders emerged in accepting RWE as evidence, particularly concerning orphan diseases, unmet clinical needs, and the impracticality of traditional RCTs.

Conclusions: Our findings highlight some opportunities and challenges in accepting RWE in decision-making processes around the marketing authorisation of new medicines. We believe it may contribute to a dialogue between stakeholders on how to best integrate RWE with traditional RCTs to generate the best evidence for decision-making and when during a medicinal products life cycle it will be most suitable to use.