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

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. 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 specialised 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. 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. 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. Patient and public involvement and engagement (PPIE) in research is increasingly important, ensuring that patients are partners in all stages of research and that research focuses on issues relevant to patients. PPIE happens a lot in clinical trials research but is much newer in real world data studies (RWD). As RWD is important to understand real world issues, understanding the type and meaningfulness of PPIE in these studies is important. Working with patients, we started by looking at what had already been published. We looked in medical journal databases for scientific publications of RWD studies and RWE included in National Institute for Health Care Excellence (NICE) technology appraisals. We selected studies where PPIE was reported and looked at the process for PPIE and how meaningful it was. From looking at these sources we found that there were only 3 studies and 2 NICE technology appraisals that clearly reported PPIE. It was difficult to understand how meaningful PPIE was, as this information was not included in most studies. Our research uncovers a need for an increase in PPIE in RWDE research or more clear reporting. This is important, because where PPIE was mentioned, it showed that it can influence study protocol development, recruitment and retention in the study.