RESUMO
OBJECTIVES: Real-world evidence (RWE) is essential for the development of pharmaceutical and medical technologies and informs treatment-related decisions by regulatory agencies, payers, healthcare providers, and patients. Given that planning RWE studies present diverse challenges, we developed the RWE Framework, a concise, visual, interactive tool designed to align multidisciplinary stakeholders toward common goals and encourage a methodical approach to RWE study planning. METHODS: A search of published literature and internet-based resources was performed to identify guidance on RWE study planning with decision and/or visual aids. A conceptual framework for a study design tool was developed based on best practices for RWE studies, enhanced with an infographic design, and refined by multidisciplinary input from RWE researchers. RESULTS: The searches confirmed an unmet need for a concise tool to support a broad range of RWE study designs: only two sources with decision/visual aids were identified. The novel RWE Framework comprises sequential decision steps with instructions to guide users through consideration of research objectives, product approval status, study setting, outcomes of interest, data availability in routine practice, need for primary data collection and/or randomization, study type and methodology, and applicable regulatory standards. Pilot testing using case studies of pharmaceutical assets demonstrated the utility of RWE Framework and applicability for use in team environments. CONCLUSIONS: The RWE Framework is a novel, concise, visual, and interactive tool to inform RWE study planning. It addresses a broad range of real-world study types and research objectives and was found to enhance RWE decision-making by multidisciplinary teams. Further validation is warranted.
RESUMO
PURPOSE: Inadequate relief of pain is common in prehospital and hospital emergency department (ED) settings. We investigated pain treatments and timelines in patients receiving pre-hospital and hospital ED care to provide insight into potential approaches to reduce the burden of trauma-related pain. PATIENTS AND METHODS: In this observational, retrospective chart review, patients had received emergency care for musculoskeletal trauma injuries and analgesic treatment for moderate-to-severe pain in Belgium, France, Germany, Italy, Spain or Sweden. As inhaled low-dose methoxyflurane (LDM) is used extensively in Australia but was not widely available in Europe at the time of this analysis, data from Australia were collated to provide insight into the potential utility of this analgesic in Europe. The primary endpoint was time to administration of first pain relief treatment following arrival of paramedic/ED care. RESULTS: Randomly selected physicians (n=189) collated data from 856 patients (Europe: n=585; Australia: n=271) via an online survey. Time to first pain relief treatment varied between countries and was significantly longer across Europe versus Australia (mean [SD] 38.1 [34.7] vs 29.9 [35.5] mins; P=0.0017). Patients from Australia who received LDM experience a shorter mean (SD) time to first pain treatment following arrival of emergency care versus patients who received other analgesics (propensity score matched [n=85] per group: 21.7 [24.2] vs 39.1 [43.0] mins; P=0.0013). Across all countries, mean (SD) time to first analgesic was shorter when treatment was administered by paramedics versus hospital ED staff (15.7 [14.7] vs 49.1 [38.4] mins). CONCLUSIONS: While there was a large variation in analgesia timelines across countries, mean times are shorter in Australia compared with Europe overall. In Australia, use of LDM was associated with a significantly shorter time from emergency assistance to first pain treatment compared with non-LDM treatments. Further studies are needed to investigate the utility of LDM in Europe.
