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Abstract
Introduction A review of European Union (EU)-funded initiatives linked to ‘Real World Evidence’ (RWE) was performed to determine whether their outputs could be used for the generation of real-world data able to support the European Medicines Agency (EMA)’s regulatory decision-making on medicines.
Method The initiatives were identified from publicly available websites. Their topics were categorised into five areas: ‘Data source’, ‘Methodology’, ‘Governance model’, ‘Analytical model’ and ‘Infrastructure’. To assess their immediate relevance for medicines evaluation, their therapeutic areas were compared with the products recommended for EU approval in 2016 and those included in the EMA pharmaceutical business pipeline.
Results Of 171 originally identified EU-funded initiatives, 65 were selected based on their primary and secondary objectives (35 ‘Data source’ initiatives, 15 ‘Methodology’, 10 ‘Governance model’, 17 ‘Analytical model’ and 25 ‘Infrastructure’). These 65 initiatives received over 734 million Euros of public funding. At the time of evaluation, the published outputs of the 40 completed initiatives did not always match their original objectives. Overall, public information was limited, data access was not explicit and their sustainability was unclear. The topics matched 8 of 14 therapeutic areas of the products recommended for approval in 2016 and 8 of 15 therapeutic areas in the 2017–2019 pharmaceutical business pipeline. Haematology, gastroenterology or cardiovascular systems were poorly represented.
Conclusions This landscape of EU-funded initiatives linked to RWE which started before 31 December 2016 highlighted that the immediate utilisation of their outputs to support regulatory decision-making is limited, often due to insufficient available information and to discrepancies between outputs and objectives. Furthermore, the restricted sustainability of the initiatives impacts on their downstream utility. Multiple projects focussing on the same therapeutic areas increase the likelihood of duplication of both efforts and resources. These issues contribute to gaps in generating RWE for medicines and diminish returns on the public funds invested.
- EU-funded initiatives
- real world evidence
- data sources
- methodologies
- governance models
- analytical models
- infrastructure
- sustainability
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Footnotes
Contributors AC and XK conceived the idea of the study. All authors were involved in the study design. KP, PM and AP performed the analysis and interpretation of results. All authors contributed to writing the manuscript and approved the final draft.
Funding The authors have not declared a specific grant for this research from any funding agency in the public, commercial or not-for-profit sectors.
Disclaimer The views expressed in this article are the personal views of the authors and may not be understood or quoted as being made on behalf of or reflecting the position of the European Medicines Agency or one of its committees or working parties.
Competing interests None declared.
Patient consent Not required.
Provenance and peer review Not commissioned; externally peer reviewed.
Data sharing statement All the information provided in this manuscript other than the data on the EMA business pipeline is publically available online.