How methodological frameworks are being developed: evidence from a scoping review

Background: Although the benefits of using methodological frameworks are increasingly recognised, to date, there is no formal definition of what constitutes a ‘methodological framework’, nor is there any published guidance on how to develop one. For the purposes of this study we have defined a methodological framework as a structured guide to completing a process or procedure. This study’s aims are to: (a) map the existing landscape on the use of methodological frameworks; (b) identify approaches used for the development of methodological frameworks and terminology used; and (c) provide suggestions for developing future methodological frameworks. We took a broad view and did not limit our study to methodological frameworks in research and academia. Methods: A scoping review was conducted, drawing on Arksey and O’Malley’s methods and more recent guidance. We systematically searched two major electronic databases (MEDLINE and Web of Science), as well as grey literature sources and the reference lists and citations of all relevant papers. Study characteristics and approaches used for development of methodological frameworks were extracted from included studies. Descriptive analysis was conducted. Results: We included a total of 30 studies, representing a wide range of subject areas. The most commonly reported approach for developing a methodological framework was ‘Based on existing methods and guidelines’ (66.7%), followed by ‘Refined and validated’ (33.3%), ‘Experience and expertise’ (30.0%), ‘Literature review’ (26.7%), ‘Data synthesis and amalgamation’ (23.3%), ‘Data extraction’ (10.0%), ‘Iteratively developed’ (6.7%) and ‘Lab work results’ (3.3%). There was no consistent use of terminology; diverse terms for methodological framework were used across and, interchangeably, within studies. Conclusions: Although no formal guidance exists on how to develop a methodological framework, this scoping review found an overall consensus in approaches used, which can be broadly divided into three phases: (a) identifying data to inform the methodological framework; (b) developing the methodological framework; and (c) validating, testing and refining the methodological framework. Based on these phases, we provide suggestions to facilitate the development of future methodological frameworks

Conceptual models in health economic evaluation: a new role

Healthcare budgets are limited, and decisions must be made about which healthcare technologies should be funded from these limited budgets. Decision makers rely on clinical and cost-effectiveness evidence on which to base their decisions, the gold standard vehicle for this evidence is a clinical trial. Guidance exists for conducting economic evaluations alongside clinical trials using standard treatment arm-based comparisons to assess cost-effectiveness, however because of differences in the purposes of the clinical effectiveness and cost-effectiveness analyses this conventional treatment arm-based economic evaluation is often uncertain. Furthermore, economic evaluations focus on the final cost-effectiveness summary measure without consideration of what is driving this measure. There is scope for providing a more detailed understanding of the conventional cost-effectiveness results. Conceptual models are simplified versions of real-life systems in a visual format, illustrating how key components of the system are linked and interact within it. In the field of economic evaluation conceptual models are recommended for and used as a guide to establish the structure of decision analytic models, in other fields they are also used as a communication tool, to aid understanding of the system and to give direction to research. The aim of this thesis was to expand the role of conceptual models in economic evaluation. This thesis proposed a new role for conceptual models to provide additional understanding to decision makers, extending the conventional economic evaluation analysis beyond a treatment arm-based analysis. This role was demonstrated using two case studies as illustrative examples, aiming to show how the role could be applied. This thesis demonstrates the value of an additional conceptual model driven analysis to supplement the conventional treatment arm-based analysis, adding further insight into the trial mechanism and what is driving the economic evaluation results. The contribution of this thesis to the field of economic evaluation is twofold; a new role for conceptual models in economic evaluation and a methodological framework for developing conceptual models in this new role

Interventions in practice:reframing policy approaches to consumer behaviour

This report introduces a novel approach to sustainability policy— a practice perspective. We argue that social practices are a better target of intervention for sustainability policy than ‘behaviour’, ‘choice’ or technical innovation alone. Understanding the dynamics of practices offers us a window into transitions towards sustainability. We consume resources as part of the practices that make up everyday life—showering, doing the laundry, cooking or driving—what we might call inconspicuous or ordinary consumption. While we may have degrees of choice in how we perform these practices, access to resources (economic, social, cultural), norms of social interaction, as well as infrastructures and institutional organisation constrain our autonomy. Practices are social phenomena—their performance entails the reproduction of cultural meanings, socially learnt skills and common tools, technologies and products. This shift of perspective places practices, not individuals or infrastructures, at the centre stage of analysis. Taking practices as the unit of analysis moves policy beyond false alternatives—beyond individual or social, behaviour or infrastructure. A practice perspective re-frames the question from “How do we change individuals’ behaviours to be more sustainable?” to “How do we shift everyday practices to be more sustainable?” After all, ‘behaviours’ are largely individuals’ performances of social practices

Further evolution in the pharmaceutical sector : changes in the division of labour and the markets for technology

The pharmaceutical sector has undergone many changes, particularly in the past several decades. The purpose of this research was to ascertain the existence of further changes to the division of labour and changes in the markets for technology within the sector. This research was also undertaken to understand the specific issues that may be impacting the division of labour and the changes in the markets for technology including the role of finance and the role of a surplus of unexploited knowledge. The division of labour between large and small new firms was initially more pronounced as the fully integrated firms continued to develop, manufacture and market drugs while 'classical biotechnology' firms pursued an exploratory business model of supplying knowledge and early stage drug candidates to these fully integrated companies (McKelvey, 2008). However, firms are changing in this sector and changes may be evident that have not been discussed in the literature to date. A new type of firm is evident within this sector, the No Research Development Only (NRDO) firm, as well as changes in the existing firms. This has impacted markets for technology as changes are also apparent in the way in which firms exchange products and knowledge. A combined quantitative and qualitative study was used to answer the research questions. A random sample of 100 EU and US companies that own and develop drug products was generated. Descriptive statistics were gathered to form a database of information and case studies were compiled to provide in-depth data related to a sample of eight firms. The newly identified NRDO firms do not possess internal capabilities to discover their own products; surprising given the historically research intensive nature of the types of small firms that operate in this sector. There also appears to be changes in the markets for technology as large firms are selling drug candidates to these hitherto research-intensive discovery and development (DD) firms who are willing to in-license these drug candidates to bolster pipelines and financial valuations. Markets for knowledge in this sector have undoubtedly evolved and a more complex set of arrangements are evident. The roles of finance and a surplus of unexploited knowledge have played an important part in these changes as the sustained level of exploration in the sector has resulted in a greater number of exploitation opportunities. Overall there is evidence to support further evolution in the sector.EThOS - Electronic Theses Online ServiceGBUnited Kingdo

Health economic evaluation of lung cancer screening using a 2 diagnostic blood test : the Early detection of Cancer of the Lung 3 Scotland (ECLS)

Funding: Funding for the ECLS study was received from Oncimmune Ltd and the Scottish Government Health & Social Care Directorate of the Chief Scientist Office (CSO).Background: Diagnostic blood tests have the potential to identify lung cancer in people at high risk. We assessed the cost-effectiveness of a lung cancer screening intervention, using the EarlyCDT®-Lung Test (ECLS) with subsequent X-ray and low-dose chest CT scans (LDCT) for patients with a positive test result, compared to both usual care and LDCT screening for the target population. Methods: We conducted a model-based lifetime analysis from a UK NHS and personal social services perspective. We estimated incremental net monetary benefit (NMB) for the ECLS intervention compared to no screening and to LDCT screening. Results: The incremental NMB of ECLS intervention compared to no screening was GBP 33,179 (95% CI: −GBP 81,396, GBP 147,180) and GBP 140,609 (95% CI: -GBP 36,255, GBP 316,612), respectively, for a cost-effectiveness threshold of GBP 20,000 and GBP 30,000 per quality-adjusted life year. The same figures compared with LDCT screening were GBP 162,095 (95% CI: GBP 52,698, GBP 271,735) and GBP 52,185 (95% CI: −GBP 113,152, GBP 220,711). Conclusions: The ECLS intervention is the most cost-effective screening alternative, with the highest probability of being cost-effective, when compared to no screening or LDCT screening. This result may change with modifications of the parameters, suggesting that the three alternatives considered in the main analysis are potentially cost-effective.Peer reviewe

The conceptualisation of cardiometabolic disease policy model in the UK

Background: Decision models are increasingly used to inform policy-making processes, and there is a need to improve their credibility. The estimation of health and economic outcomes generated from decision models is influenced by the development process itself. This paper aims to present the conceptual model development process of cardiometabolic disease (CMD) policy models in the UK setting. Methods: This conceptual model followed the International Society of Pharmacoeconomics and Outcomes Research-Society of Medical Decision Making (ISPOR-SMDM) Modelling Good Research Practices Task Force-2. Results: First, for the conceptualisation of the problem, the CMD disease staging, progression and current clinical guidelines were summarised, followed by a systematic review of published policy models. We critically appraised policy models such as cardiovascular disease and type 2 diabetes. Key messages from the review emphasised the importance of understanding various determinants influencing model development, including risk factors, model structure, models’ parameters, data utilisation, economic perspective, equality/equity consideration, transparency and validation process. Second, as a sequential process, is model conceptualisation, to determine which modelling types and their attributes best represent the defined problem. Expert opinions, including a clinician and experienced modellers, provided input on the state transition model to ensure the structure is clinically relevant. From this stage, the consideration and agreement to establish a disease state in a state transition model was discussed. Conclusion: This conceptual model serves as a basis for representing the systematic process for structuring a CMD policy model to enhance its transparency and credibility

Health economic evaluation of lung cancer screening using a diagnostic blood test: the Early detection of Cancer of the Lung Scotland (ECLS)

Background: Diagnostic blood tests have the potential to identify lung cancer in people at high risk. We assessed the cost-effectiveness of a lung cancer screening intervention, using the Early CDT®-Lung Test (ECLS) with subsequent X-ray and low-dose chest CT scans (LDCT) for patients with a positive test result, compared to both usual care and LDCT screening for the target population. Methods: We conducted a model-based lifetime analysis from a UK NHS and personal social services perspective. We estimated incremental net monetary benefit (NMB) for the ECLS intervention compared to no screening and to LDCT screening. Results: The incremental NMB of ECLS intervention compared to no screening was GBP 33,179 (95% CI: −GBP 81,396, GBP 147,180) and GBP 140,609 (95% CI: -GBP 36,255, GBP 316,612), respectively, for a cost-effectiveness threshold of GBP 20,000 and GBP 30,000 per quality-adjusted life year. The same figures compared with LDCT screening were GBP 162,095 (95% CI: GBP 52,698, GBP 271,735) and GBP 52,185 (95% CI: −GBP 113,152, GBP 220,711). Conclusions: The ECLS intervention is the most cost-effective screening alternative, with the highest probability of being cost-effective, when compared to no screening or LDCT screening. This result may change with modifications of the parameters, suggesting that the three alternatives considered in the main analysis are potentially cost-effective

Recent evidence on rates and factors influencing smoking behaviours after release from smokefree prisons: a scoping review

Purpose: Smoke-free prison policies have been introduced in some countries, in part to address very high levels of tobacco use in people in prison. However, relapse rates post-release remain high. This papers aims to improve understanding of post-release smoking and/or vaping behaviour is necessary to inform support for a priority population. Design/methodology/approach: The authors searched health, social science and criminal justice databases for studies about smoking/vaping behaviours among people released from smoke-free prisons. Studies were included if they reported primary data and were published between January 2017 and March 2024 in English; the population was adults/young people (16 yr+) imprisoned or formerly imprisoned, in prisons with comprehensive smoke-free policies; and at least one of the following was reported: pre-release intention to smoke, vape or remain abstinent post-release; smoking/vaping behaviour post-release and factors influencing smoking/vaping behaviour; attempts to quit again following post-release smoking/vaping relapse. Findings: Nine studies met our criteria. The evidence base is small and mainly from the USA or Australia. Evidence continues to suggest that most people resume smoking after leaving a smoke-free prison. No new interventions have been successful in reducing relapse rates. No studies report on vaping post-release, although two studies report on perceived factors affecting smoking relapse post-release from prisons allowing vaping. Research limitations/implications: Given very high rates of relapse, there remains a significant need to better understand what approaches are feasible and acceptable for reducing return to smoking post-release. Originality/value: This review updates the limited evidence on smoking behaviours after leaving a smoke-free prison

Cost effectiveness of commercial portable ex vivo lung perfusion at a low-volume US lung transplant center

Background: Portable ex vivo lung perfusion during lung transplantation is a resource-intensive technology. In light of its increasing use, we evaluated the cost-effectiveness of ex vivo lung perfusion at a low-volume lung transplant center in the USA. Methods: Patients listed for lung transplantation (2015–2021) in the United Network for Organ Sharing database were included. Quality-of-life was approximated by Karnofsky Performance Status scores 1-year post-transplant. Total transplantation encounter and 1-year follow-up costs accrued by our academic center for patients listed from 2018 to 2021 were obtained. Cost-effectiveness was calculated by evaluating the number of patients attaining various Karnofsky scores relative to cost. Results: Of the 13 930 adult patients who underwent lung transplant in the United Network for Organ Sharing database, 13 477 (96.7%) used static cold storage and 453 (3.3%) used ex vivo lung perfusion, compared to 30/58 (51.7%) and 28/58 (48.3%), respectively, at our center. Compared to static cold storage, median total costs at 1 year were higher for ex vivo lung perfusion (918 000 dollars vs. 516 000 dollars; p = 0.007) along with the cost of living 1 year with a Karnofsky functional status of 100 after transplant (1 290 000 dollars vs. 841 000 dollars). In simulated scenarios, each Karnofsky-adjusted life year gained by ex vivo lung perfusion was 1.00–1.72 times more expensive. Conclusions: Portable ex vivo lung perfusion is not currently cost-effective at a low-volume transplant centers in the USA, being 1.53 times more expensive per Karnofsky-adjusted life year. Improving donor lung and/or recipient biology during ex vivo lung perfusion may improve its utility for routine transplantation.</p