Patient and public involvement and engagement in real world data and evidence research across the medicines development cycle: a rapid review of peer-reviewed literature and NICE technology appraisals.

Abstract

Objectives: The objective of this rapid review is to understand the reporting, role and quality of patient and public involvement and engagement (PPIE) in real world data and evidence (RWDE) research across the medicines development cycle.

Methods: We comprehensively searched, with no date restrictions, Medline and Embase databases for peer-reviewed literature and conference abstracts (Embase only) reporting PPIE in RWD studies. We also assessed PPIE in a sample of 100 NICE technology appraisals (TAs) comprising both single technology appraisals (STAs) and highly specialized technologies (HSTs). We used standard methods for screening and data extraction. In addition, we used the Patient Focused Medicines Development (PFMD)'s Patient Engagement Quality Guidance (PEQG) as a framework to assess the quality of PPIE. We planned to conduct narrative synthesis of included studies, however there were insufficient studies and data reported.

Results: We included three RWD studies that reported PPIE from the peer-reviewed literature and two NICE HSTs. One of the HSTs included data from one of the peer-reviewed journal articles. Reporting of PPIE in included studies was limited. No studies reported a PPIE framework and it was unclear how integrated and meaningful PPIE was. Four out of seven of PFMD's quality criteria for PPIE were poorly reported by included studies. This suggests reporting and/or conduct of PPIE requires improvement in RWD studies.

Conclusions: Our review found that PPIE was rarely reported in RWDE research and uncovers a need for consistent reporting. For most publications there was insufficient information to judge the extent to which patients and carers, were considered meaningful partners. However, our review provided preliminary evidence that PPIE can influence protocol development, recruitment, and retention methods in RWD studies. More inclusive approaches to PPIE would help interpretation of RWE regarding relevance and importance to patients and carers.