Study on the use of real-world data (RWD) for research, clinical care, regulatory decision-making, health technology assessment, and policy making

PPMI is currently finalising a study on the use of real-world data (RWD) which is one of the European Commission’s key initiatives aiming to translate RWD into meaningful health outcomes. RWD refers to a subspecies of big data that denotes health-related data not collected within the context of a typical clinical research setting. A variety of initiatives funded by the European Commission and other stakeholders worldwide are aimed at improving the use of RWD by concerned stakeholder groups. This study analyses outcomes and lessons learned from these initiatives to support the development of EU-level guidance for enabling the wider use of RWD. This analysis builds upon an extensive mapping of RWD initiatives which either use RWD for healthcare purposes or develop tools and methods to facilitate the use of RWD for other stakeholders. Over the course of the study, we delved into five key use cases of RWD, including research, clinical care, regulatory decision-making, health technology assessment, and policy-making.

The study involved different methods featuring elements of both qualitative and quantitative data analysis. Capitalising on machine learning and other cutting-edge techniques, the study team scanned over 35,000 projects along with nearly 460,000 monitoring documents to identify 192 RWD-specific initiatives. Although most of these initiatives were EU-funded projects, the sample also included highly relevant non-EU funded projects which have proven to be highly impactful either at the national, EU or global level. Through an extensive mapping exercise and EU-wide stakeholder consultations, we identified the key stakeholders and their needs, types of RWD sources and outputs produced along with projects’ complementarities per thematic research area. Multivariate cluster and network analyses were also used to display how the analysed projects can be clustered together.

Towards the end of the study, a needs-gap analysis and interviews with selected EC officials were carried out to identify future research that would make RWD more valuable, and to derive recommendations on how to enable the wider use of RWD by all concerned stakeholder groups. Through a series of six virtual workshops, we validated the future strategies for widening the use of RWD with more than 150 participants from the sectors of academia, industry and government as well as various patient organisations.

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