Abstract
CONCLUSIONS
There is a lack of harmonisation in assessing RWE among HTA and regulatory agencies. The available RWE guidance documents provide limited guidance on real-world hospital data. Considering their unique nature and to unlock their potential for HTA, we emphasise the need for more in-depth guidance tailored to the hospital context.
RESULTS
We identified 19 guidance documents providing recommendations for RWE. Of these, four documents explicitly provided recommendations tailored to hospital data, while two others did so implicitly. The scope, definition and applications of RWE vary among guidance. Recommendations across all agencies mainly address the clinical-effectiveness domain, with limited guidance on quality-of-life and patient-reported outcomes, and none on real-world cost. The interviews identified seven themes playing a role in using hospital data: data-related, generalisability, ethical/legal, organisational, communication, governance and technology-related. Barriers included data availability, access, timeliness, quality, validation and heterogeneity. HTA experts emphasised the need for standardised policies.
OBJECTIVES
Hospital data can inform decision-makers with real-time evidence, yet it remains underutilised. This study aims to compare international health technology assessment (HTA) and regulatory real-world evidence (RWE) guidance, focusing on their applicability to hospital data.
STUDY DESIGN, SETTING AND PARTICIPANTS
We used a two-step sequential qualitative design: a scoping review and semi-structured interviews with HTA experts. We searched for RWE guidance for HTA in 12 countries: the UK, Germany, Italy, Spain, France, Finland, the Netherlands, Portugal, Denmark, Canada, the US and Australia, along with the European Medicines Agency and Food and Drug Administration. The expert interviews aimed to validate document selection and assess their applicability to hospital data. We analysed the interviews thematically.