Co-creating tools for embedding meaningful patient and public involvement and engagement in real-world data and evidence research in the pharmaceutical industry setting: a multistakeholder participatory co-design study

Objectives
This study aimed to (1) gauge patients understanding and expectations of real-world data and evidence (RWDE) research and (2) use this understanding and patients lived experience to co-create resources and a framework for embedding meaningful patient and public involvement and engagement (PPIE) in RWDE research within the pharmaceutical industry setting.

Setting and participants
An academic organisation, a pharmaceutical company and a PPIE panel of 12 patients or carers partnered to form the project team. The PPIE panel was purposively selected to maximise diversity.

Design
Participatory and co-design methods were used to engender an understanding of the PPIE perspective on RWDE research and the PPIE role within that. Interactive workshops explored understanding and expectations of RWDE research as well as perceived barriers and facilitators of PPIE within each stage of the RWDE research cycle. Workshops were audio and video recorded, with notes captured. Summaries were analysed thematically and shared back with the PPIE panel for validation and further reflection.

Results
We identified a lack of trust and understanding of real-world data, its collection and use and the need to educate the public and researchers. Four themes were identified for meaningful PPIE in RWDE research; equality, diversity and inclusion; feeling valued; ownership and understanding and evaluating impact. We co-created learning resources (video, infographic) and a novel PPIE framework, incorporating potential PPIE activities, resources and support needs for use by researchers conducting RWDE research.

Conclusions
To our knowledge, this is the first project to explore the practicalities of PPIE in RWDE research from the perspective of patients and carers. Some findings confirm PPIE experience and guidance derived from other areas, with some specific insights into the pharmaceutical industry. These underpin the PPIE framework to enable robust and meaningful PPIE in RWDE research. This article includes a plain language summary in the supplement.

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Febbraio 2025

Inhaled methoxyflurane (Penthrox) use in the outpatient and ambulatory setting: a systematic review

Objective
We assessed whether Penthrox (methoxyflurane) can provide better pain relief for elective, outpatient interventional procedures compared with other methods of pain control.

Design
Systematic review.

Data sources
Cochrane Central Register of Controlled Trial, MEDLINE, Emcare, Embase, ClinicalTrials.gov and the Cumulative Index to Nursing and Allied Health Literature were searched from inception to 6 November 2023.

Eligibility criteria
All published studies including randomised controlled trials (RCTs), cohort studies and case–control studies investigating Penthrox for pain relief, during and/or after an interventional procedure, were included.

Data extraction and synthesis
Two independent reviewers identified eligible studies and retrieved data. Risk of bias was assessed using Cochrane Collaboration Risk of Bias V.2.0 and Risk of Bias in Non-randomised Studies of Interventions (ROBINS I) tools. A meta-analysis was not possible due to heterogeneity.

Results
The literature search yielded 1189 records. 12 studies were eligible for inclusion: 5 RCTs and seven non-randomised studies of interventions (NRSIs). Penthrox was used for a range of procedures across five medical specialities: gastroenterology, gynaecology, haematology, orthopaedics and urology. Two RCTs showed a significant reduction in intraprocedural pain when Penthrox was compared with placebo. However, three RCTs where Penthrox was compared with either placebo or intravenous sedation showed no significant differences in pain or comfort level. Similarly, the NRSIs showed variable results. Most patients were satisfied (63% to >98%; seven studies) with Penthrox, and seven out of eight studies reported that the majority of participants would use it again (46.8%–95%; eight studies). No serious adverse events were reported.

Conclusion
Penthrox (methoxyflurane) shows promise as an analgesic for invasive, elective interventional procedures in the outpatient setting, although the relative benefits appear to vary depending on intervention and comparator pain control measures. There is a pressing need for robustly conducted, large, generalisable multicentre RCTs to evaluate the effectiveness of Penthrox.

PROSPERO registration number
CRD42023404574.

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Febbraio 2025

[Articles] Severe maternal morbidity in the high income setting: a systematic review of composite definitions

Despite common approaches to defining SMM, there are opportunities to improve comparability. No two definitions for use with administrative data in different settings involved a similar incidence and set of components and involved a similar distribution of components among cases. Harmonization of the purpose, constituent codes, and sources of data would facilitate comparisons between maternity systems.

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Febbraio 2025