Understanding multi-stakeholder needs, preferences and expectations to define effective practices and processes of patient engagement in medicine development: A mixed-methods study.

BACKGROUND: The holistic evolution of patient engagement in medicines development requires a more detailed understanding of the needs of all involved stakeholders, and one that better accounts for the specific needs of some potentially vulnerable patient populations and key stages in medicines development. OBJECTIVE: The purpose of this convergent mixed-methods study was to better understand the needs of different stakeholders concerning patient engagement at three key stages in medicines development: research priority setting, clinical trial design and early dialogues with Health Technology Assessment bodies and regulators. DESIGN: This study brought together findings from three sources: i) an online questionnaire, ii) face-to-face consultations with two potentially vulnerable patient populations, a workshop with Health Technology Assessment bodies, and iii) three-step modified Delphi methodology. RESULTS: Overall stakeholders still need additional varied support mechanisms to undertake, sustain or measure value of patient engagement. Health Technology Assessment bodies need better rationale for patient engagement in early dialogue and tools to support its implementation. Improved awareness and understanding of the need and value that involving patients, who are often considered as potentially vulnerable, can bring is needed, as is better accommodation of their specific needs. Similarly, weighted Delphi categories were as follows: aims and objectives, and sustainability. Several additional themes were common across the three key stages in medicines development. CONCLUSION: This broad-reaching study provides the blocks needed to build a framework for patient engagement in medicines development. PATIENT OR PUBLIC CONTRIBUTION: Patients were involved in review and interpretation of data.

How will Brexit affect health services in the UK? An updated evaluation.

All forms of Brexit are bad for health, but some are worse than others. This paper builds on our 2017 analysis using the WHO health system building blocks framework to assess the likely effects of Brexit on the National Health Service (NHS) in the UK. We consider four possible scenarios as follows: a No-Deal Brexit under which the UK leaves the EU on March 29, 2019, without any formal agreement on the terms of withdrawal; a Withdrawal Agreement, as negotiated between the UK and EU and awaiting (possible) formal agreement, which provides a transition period until the end of December, 2020; the Northern Ireland Protocol's backstop coming into effect after the end of that period; or the Political Declaration on the Future Relationship between the UK and EU. Our analysis shows that a No-Deal Brexit is substantially worse for the NHS than a future involving the Withdrawal Agreement, which provides certainty and continuity in legal relations while the Political Declaration on the Future Relationship is negotiated and put into legal form. The Northern Ireland backstop has varying effects, with continuity in some areas, such as health products, but no continuity in others. The Political Declaration on the Future Relationship envisages a relationship that is centred around a free-trade agreement, in which wider health-related issues are largely absent. All forms of Brexit, however, involve negative consequences for the UK's leadership and governance of health, in both Europe and globally, with questions about the ability of parliament and other stakeholders to scrutinise and oversee government actions.

Public involvement in the governance of population-level biomedical research: unresolved questions and future directions.

Population-level biomedical research offers new opportunities to improve population health, but also raises new challenges to traditional systems of research governance and ethical oversight. Partly in response to these challenges, various models of public involvement in research are being introduced. Yet, the ways in which public involvement should meet governance challenges are not well understood. We conducted a qualitative study with 36 experts and stakeholders using the World Café method to identify key governance challenges and explore how public involvement can meet these challenges. This brief report discusses four cross-cutting themes from the study: the need to move beyond individual consent; issues in benefit and data sharing; the challenge of delineating and understanding publics; and the goal of clarifying justifications for public involvement. The report aims to provide a starting point for making sense of the relationship between public involvement and the governance of population-level biomedical research, showing connections, potential solutions and issues arising at their intersection. We suggest that, in population-level biomedical research, there is a pressing need for a shift away from conventional governance frameworks focused on the individual and towards a focus on collectives, as well as to foreground ethical issues around social justice and develop ways to address cultural diversity, value pluralism and competing stakeholder interests. There are many unresolved questions around how this shift could be realised, but these unresolved questions should form the basis for developing justificatory accounts and frameworks for suitable collective models of public involvement in population-level biomedical research governance.

Evaluating the "return on patient engagement initiatives" in medicines research and development: A literature review.

BACKGROUND: Showing how engagement adds value for all stakeholders can be an effective motivator for broader implementation of patient engagement. However, it is unclear what methods can best be used to evaluate patient engagement. This paper is focused on ways to evaluate patient engagement at three decision-making points in the medicines research and development process: research priority setting, clinical trial design and early dialogues with regulators and health technology assessment bodies. OBJECTIVE: Our aim was to review the literature on monitoring and evaluation of patient engagement, with a focus on indicators and methods. SEARCH STRATEGY AND INCLUSION CRITERIA: We undertook a scoping literature review using a systematic search, including academic and grey literature with a focus on evaluation approaches or outcomes associated with patient engagement. No date limits were applied other than a cut-off of publications after July 2018. DATA EXTRACTION AND SYNTHESIS: Data were extracted from 91 publications, coded and thematically analysed. MAIN RESULTS: A total of 18 benefits and 5 costs of patient engagement were identified, mapped with 28 possible indicators for their evaluation. Several quantitative and qualitative methods were found for the evaluation of benefits and costs of patient engagement. DISCUSSION AND CONCLUSIONS: Currently available indicators and methods are of some use in measuring impact but are not sufficient to understand the pathway to impact, nor whether interaction between researchers and patients leads to change. We suggest that the impacts of patient engagement can best be determined not by applying single indicators, but a coherent set of measures.

Frameworks for supporting patient and public involvement in research: Systematic review and co-design pilot.

BACKGROUND: Numerous frameworks for supporting, evaluating and reporting patient and public involvement in research exist. The literature is diverse and theoretically heterogeneous. OBJECTIVES: To identify and synthesize published frameworks, consider whether and how these have been used, and apply design principles to improve usability. SEARCH STRATEGY: Keyword search of six databases; hand search of eight journals; ancestry and snowball search; requests to experts. INCLUSION CRITERIA: Published, systematic approaches (frameworks) designed to support, evaluate or report on patient or public involvement in health-related research. DATA EXTRACTION AND SYNTHESIS: Data were extracted on provenance; collaborators and sponsors; theoretical basis; lay input; intended user(s) and use(s); topics covered; examples of use; critiques; and updates. We used the Canadian Centre for Excellence on Partnerships with Patients and Public (CEPPP) evaluation tool and hermeneutic methodology to grade and synthesize the frameworks. In five co-design workshops, we tested evidence-based resources based on the review findings. RESULTS: Our final data set consisted of 65 frameworks, most of which scored highly on the CEPPP tool. They had different provenances, intended purposes, strengths and limitations. We grouped them into five categories: power-focused; priority-setting; study-focused; report-focused; and partnership-focused. Frameworks were used mainly by the groups who developed them. The empirical component of our study generated a structured format and evidence-based facilitator notes for a "build your own framework" co-design workshop. CONCLUSION: The plethora of frameworks combined with evidence of limited transferability suggests that a single, off-the-shelf framework may be less useful than a menu of evidence-based resources which stakeholders can use to co-design their own frameworks.

How will Brexit affect health and health services in the UK? Evaluating three possible scenarios.

The process of leaving the European Union (EU) will have profound consequences for health and the National Health Service (NHS) in the UK. In this paper, we use the WHO health system building blocks framework to assess the likely effects of three scenarios we term soft Brexit, hard Brexit, and failed Brexit. We conclude that each scenario poses substantial threats. The workforce of the NHS is heavily reliant on EU staff. Financing of health care for UK citizens in the EU and vice versa is threatened, as is access to some capital funds, while Brexit threatens overall economic performance. Access to pharmaceuticals, technology, blood, and organs for transplant is jeopardised. Information used for international comparisons is threatened, as is service delivery, especially in Northern Ireland. Governance concerns relate to public health, competition and trade law, and research. However, we identified a few potential opportunities for improvement in areas such as competition law and flexibility of training, should the UK Government take them. Overall, a soft version of Brexit would minimise health threats whereas failed Brexit would be the riskiest outcome. Effective parliamentary scrutiny of policy and legal changes will be essential, but the scale of the task risks overwhelming parliament and the civil service.

Beyond Adoption: A New Framework for Theorizing and Evaluating Nonadoption, Abandonment, and Challenges to the Scale-Up, Spread, and Sustainability of Health and Care Technologies.

BACKGROUND: Many promising technological innovations in health and social care are characterized by nonadoption or abandonment by individuals or by failed attempts to scale up locally, spread distantly, or sustain the innovation long term at the organization or system level. OBJECTIVE: Our objective was to produce an evidence-based, theory-informed, and pragmatic framework to help predict and evaluate the success of a technology-supported health or social care program. METHODS: The study had 2 parallel components: (1) secondary research (hermeneutic systematic review) to identify key domains, and (2) empirical case studies of technology implementation to explore, test, and refine these domains. We studied 6 technology-supported programs-video outpatient consultations, global positioning system tracking for cognitive impairment, pendant alarm services, remote biomarker monitoring for heart failure, care organizing software, and integrated case management via data sharing-using longitudinal ethnography and action research for up to 3 years across more than 20 organizations. Data were collected at micro level (individual technology users), meso level (organizational processes and systems), and macro level (national policy and wider context). Analysis and synthesis was aided by sociotechnically informed theories of individual, organizational, and system change. The draft framework was shared with colleagues who were introducing or evaluating other technology-supported health or care programs and refined in response to feedback. RESULTS: The literature review identified 28 previous technology implementation frameworks, of which 14 had taken a dynamic systems approach (including 2 integrative reviews of previous work). Our empirical dataset consisted of over 400 hours of ethnographic observation, 165 semistructured interviews, and 200 documents. The final nonadoption, abandonment, scale-up, spread, and sustainability (NASSS) framework included questions in 7 domains: the condition or illness, the technology, the value proposition, the adopter system (comprising professional staff, patient, and lay caregivers), the organization(s), the wider (institutional and societal) context, and the interaction and mutual adaptation between all these domains over time. Our empirical case studies raised a variety of challenges across all 7 domains, each classified as simple (straightforward, predictable, few components), complicated (multiple interacting components or issues), or complex (dynamic, unpredictable, not easily disaggregated into constituent components). Programs characterized by complicatedness proved difficult but not impossible to implement. Those characterized by complexity in multiple NASSS domains rarely, if ever, became mainstreamed. The framework showed promise when applied (both prospectively and retrospectively) to other programs. CONCLUSIONS: Subject to further empirical testing, NASSS could be applied across a range of technological innovations in health and social care. It has several potential uses: (1) to inform the design of a new technology; (2) to identify technological solutions that (perhaps despite policy or industry enthusiasm) have a limited chance of achieving large-scale, sustained adoption; (3) to plan the implementation, scale-up, or rollout of a technology program; and (4) to explain and learn from program failures.

Maximising value from a United Kingdom Biomedical Research Centre: study protocol.

BACKGROUND: Biomedical Research Centres (BRCs) are partnerships between healthcare organisations and universities in England. Their mission is to generate novel treatments, technologies, diagnostics and other interventions that increase the country's international competitiveness, to rapidly translate these innovations into benefits for patients, and to improve efficiency and reduce waste in healthcare. As NIHR Oxford BRC (Oxford BRC) enters its third 5-year funding period, we seek to (1) apply the evidence base on how best to support the various partnerships in this large, multi-stakeholder research system and (2) research how these partnerships play out in a new, ambitious programme of translational research. METHODS: Organisational case study, informed by the principles of action research. A cross-cutting theme, 'Partnerships for Health, Wealth and Innovation' has been established with multiple sub-themes (drug development, device development, business support and commercialisation, research methodology and statistics, health economics, bioethics, patient and public involvement and engagement, knowledge translation, and education and training) to support individual BRC research themes and generate cross-theme learning. The 'Partnerships' theme will support the BRC's goals by facilitating six types of partnership (with patients and citizens, clinical services, industry, across the NIHR infrastructure, across academic disciplines, and with policymakers and payers) through a range of engagement platforms and activities. We will develop a longitudinal progress narrative centred around exemplar case studies, and apply theoretical models from innovation studies (Triple Helix), sociology of science (Mode 2 knowledge production) and business studies (Value Co-creation). Data sources will be the empirical research studies within individual BRC research themes (who will apply separately for NHS ethics approval), plus documentary analysis and interviews and ethnography with research stakeholders. This study has received ethics clearance through the University of Oxford Central University Research Ethics Committee. DISCUSSION: We anticipate that this work will add significant value to Oxford BRC. We predict accelerated knowledge translation; closer alignment of the innovation process with patient priorities and the principles of responsible, ethical research; reduction in research waste; new knowledge about the governance and activities of multi-stakeholder research partnerships and the contexts in which they operate; and capacity-building that reflects the future needs of a rapidly-evolving health research system.

Research impact in the community-based health sciences: an analysis of 162 case studies from the 2014 UK Research Excellence Framework.

BACKGROUND: The 2014 UK Research Excellence Framework (REF2014) generated a unique database of impact case studies, each describing a body of research and impact beyond academia. We sought to explore the nature and mechanism of impact in a sample of these. METHODS: The study design was manual content analysis of a large sample of impact case studies (producing mainly quantitative data), plus in-depth interpretive analysis of a smaller sub-sample (for qualitative detail), thereby generating both breadth and depth. For all 162 impact case studies submitted to sub-panel A2 in REF2014, we extracted data on study design(s), stated impacts and audiences, mechanisms of impact, and efforts to achieve impact. We analysed four case studies (selected as exemplars of the range of approaches to impact) in depth, including contacting the authors for their narratives of impact efforts. RESULTS: Most impact case studies described quantitative research (most commonly, trials) and depicted a direct, linear link between research and impact. Research was said to have influenced a guideline in 122 case studies, changed policy in 88, changed practice in 84, improved morbidity in 44 and reduced mortality in 25. Qualitative and participatory research designs were rare, and only one case study described a co-production model of impact. Eighty-two case studies described strong and ongoing linkages with policymakers, but only 38 described targeted knowledge translation activities. In 40 case studies, no active efforts to achieve impact were described. Models of good implementation practice were characterised by an ethical commitment by researchers, strong institutional support and a proactive, interdisciplinary approach to impact activities. CONCLUSION: REF2014 both inspired and documented significant efforts by UK researchers to achieve impact. But in contrast with the published evidence on research impact (which depicts much as occurring indirectly through non-linear mechanisms), this sub-panel seems to have captured mainly direct and relatively short-term impacts one step removed from patient outcomes. Limited impacts on morbidity and mortality, and researchers' relatively low emphasis on the processes and interactions through which indirect impacts may occur, are concerns. These findings have implications for multi-stakeholder research collaborations such as UK National Institute for Health Research Collaborations for Leadership in Applied Health Research and Care, which are built on non-linear models of impact.

Training for managing impacted fetal head at caesarean birth: multimethod evaluation of a pilot.

BACKGROUND: Implementation of national multiprofessional training for managing the obstetric emergency of impacted fetal head (IFH) at caesarean birth has potential to improve quality and safety in maternity care, but is currently lacking in the UK. OBJECTIVES: To evaluate a training package for managing IFH at caesarean birth with multiprofessional maternity teams. METHODS: The training included an evidence-based lecture supported by an animated video showing management of IFH, followed by hands-on workshops and real-time simulations with use of a birth simulation trainer, augmented reality and management algorithms. Guided by the Kirkpatrick framework, we conducted a multimethod evaluation of the training with multiprofessional maternity teams. Participants rated post-training statements about relevance and helpfulness of the training and pre-training and post-training confidence in their knowledge and skills relating to IFH (7-point Likert scales, strongly disagree to strongly agree). An ethnographer recorded sociotechnical observations during the training. Participants provided feedback in post-training focus groups. RESULTS: Participants (N=57) included 21 midwives, 25 obstetricians, 7 anaesthetists and 4 other professionals from five maternity units. Over 95% of participants agreed that the training was relevant and helpful for their clinical practice and improving outcomes following IFH. Confidence in technical and non-technical skills relating to managing IFH was variable before the training (5%-92% agreement with the pre-training statements), but improved in nearly all participants after the training (71%-100% agreement with the post-training statements). Participants and ethnographers reported that the training helped to: (i) better understand the complexity of IFH, (ii) recognise the need for multiprofessional training and management and (iii) optimise communication with those in labour and their birth partners. CONCLUSIONS: The evaluated training package can improve self-reported knowledge, skills and confidence of multiprofessional teams involved in management of IFH at caesarean birth. A larger-scale evaluation is required to validate these findings and establish how best to scale and implement the training.

Priority setting and cross-country learning: the relevance of TO-REACH for primary care.

AIM: To inform the primary care community about priorities for research in primary care as came up from the European project TO-REACH and to discuss transferability of service and policy innovations between countries. BACKGROUND: TO-REACH stands for Transfer of Organizational innovations for Resilient, Effective, equitable, Accessible, sustainable and Comprehensive Health services and systems. This EU-funded project has put health systems and services research higher on the European agenda and has led to the current development of a European 'Partnership Transforming Health and Care Systems'. METHODS: To identify research priorities, both qualitative and quantitative approaches were used. Policy documents and strategic roadmaps were searched, and priorities were mapped. Stakeholders were involved through national roundtable consultations and online consultations. Regarding transferability, we carried out a review of the literature, guided by a conceptual framework, and using a snowballing approach. FINDINGS: Primary care emerged as an important priority from the inventory, as are areas that are conducive to strengthening primary care, such as workforce policies. The large variation in service organisation and policy around primary care in Europe is a huge potential for cross-country learning. However, the simple transfer of primary care service and policy arrangements from one health system to another has a big chance to fail, unless known conditions for successful transfer are taken into account and gaps in our knowledge about transfer are resolved.

Impact on the NHS and health of the UK's trade and cooperation relationship with the EU, and beyond.

The UK's relationship with the European Union (EU) is now embodied in two principal legal instruments: the EU-UK Trade and Cooperation Agreement, which formally entered into force on 1 May 2021; and the Withdrawal Agreement, with its Protocol on Ireland/Northern Ireland, which continues to apply. Using a 'building blocks' framework for analysis of national health systems derived from the World Health Organisation, this article examines the likely impacts in the UK of this legal settlement on the National Health Service (NHS), health and social care. Specifically, we determine the extent to which the trade, cooperation and regulatory aspects of those legal measures support positive impacts for the NHS and social care. We show that, as there is clear support for positive health and care outcomes in only one of the 17 NHS 'building blocks', unless mitigating action is taken, the likely outcomes will be detrimental. However, as the legal settlement gives the UK a great deal of regulatory freedom, especially in Great Britain, we argue that it is crucial to track the effects of proposed new health and social care-related policy choices in the months and years ahead.

Assessing the potential impact on health of the UK's future relationship agreement with the EU: analysis of the negotiating positions.

While policy attention is understandably diverted to COVID-19, the end of the UK's post-Brexit 'transition period' remains 31 December 2020. All forms of future EU-UK relationship are worse for health than EU membership, but analysis of the negotiating texts shows some forms are better than others. The likely outcomes involve major negative effects for NHS staffing, funding for health and social care, and capital financing for the NHS; and for UK global leadership and influence. We expect minor negative effects for cross border healthcare (except in Northern Ireland); research collaboration; and data sharing, such as the Early Warning and Response System for health threats. Despite political narratives, the legal texts show that the UK seeks de facto continuity in selected key areas for pharmaceuticals, medical devices, and equipment [including personal protective equipment (PPE)], especially clinical trials, pharmacovigilance, and batch-testing. The UK will be excluded from economies of scale of EU membership, e.g. joint procurement programmes as used recently for PPE. Above all, there is a major risk of reaching an agreement with significant adverse effects for health, without meaningful oversight by or input from the UK Parliament, or other health policy stakeholders.

The Bright Elusive Butterfly of Value in Health Technology Development Comment on "Providing Value to New Health Technology: The Early Contribution of Entrepreneurs, Investors, and Regulatory Agencies".

The current system of health technology development is characterised by multiple misalignments. The "supply" side (innovation policy-makers, entrepreneurs, investors) and the "demand" side (health policy-makers, regulators, health technology assessment, purchasers) operate under different - and conflicting - logics. The system is less a "pathway" than an unstable ecosystem of multiple interacting sub-systems. "Value" means different things to each of the numerous actors involved. Supply-side dynamics are built on fictions; regulatory checks and balances are designed to assure quality, safety and efficacy, not to ensure that technologies entering the market are either desirable or cost-effective. Assessment of comparative and cost-effectiveness usually comes too late in the process to shape an innovation's development. We offer no simple solutions to these problems, but in the spirit of commencing a much-needed public debate, we suggest some tentative ways forward. First, universities and public research funders should play a more proactive role in shaping the system. Second, the role of industry in forging long-term strategic partnerships for public benefit should be acknowledged (though not uncritically). Third, models of "responsible innovation" and public input to research priority-setting should be explored. Finally, the evidence base on how best to govern inter-sectoral health research partnerships should be developed and applied.

Patient Mobility in Times of Austerity: A Legal and Policy Analysis of the Petru Case.

The case-law of the Court of Justice (ECJ) on patient mobility was recently challenged by a ruling that a patient could go to Germany for treatment when facilities in Romanian hospitals were inadequate. Given the reported impact of austerity measures in the field of health care this raises the question; what is the impact of the ECJ's ruling on how Member States can manage expenditure and limit outflows of patients and how should such measures be legally evaluated? The objective of this article is to analyse potential impact on health systems in the context of increasing pressure on public financing for health. While the ECJ mainly referred to the requirement of treatment in due time, we also analyse possible austerity reductions of the basket of care against the background of EU law (i.e., EGJ case-law, patient mobility directive, Charter of Fundamental rights and social security regulation).

SCALS: a fourth-generation study of assisted living technologies in their organisational, social, political and policy context.

INTRODUCTION: Research to date into assisted living technologies broadly consists of 3 generations: technical design, experimental trials and qualitative studies of the patient experience. We describe a fourth-generation paradigm: studies of assisted living technologies in their organisational, social, political and policy context. Fourth-generation studies are necessarily organic and emergent; they view technology as part of a dynamic, networked and potentially unstable system. They use co-design methods to generate and stabilise local solutions, taking account of context. METHODS AND ANALYSIS: SCALS (Studies in Co-creating Assisted Living Solutions) consists (currently) of 5 organisational case studies, each an English health or social care organisation striving to introduce technology-supported services to support independent living in people with health and/or social care needs. Treating these cases as complex systems, we seek to explore interdependencies, emergence and conflict. We employ a co-design approach informed by the principles of action research to help participating organisations establish, refine and evaluate their service. To that end, we are conducting in-depth ethnographic studies of people's experience of assisted living technologies (micro level), embedded in evolving organisational case studies that use interviews, ethnography and document analysis (meso level), and exploring the wider national and international context for assisted living technologies and policy (macro level). Data will be analysed using a sociotechnical framework developed from structuration theory. ETHICS AND DISSEMINATION: Research ethics approval for the first 4 case studies has been granted. An important outcome will be lessons learned from individual co-design case studies. We will document the studies' credibility and rigour, and assess the transferability of findings to other settings while also recognising unique aspects of the contexts in which they were generated. Academic outputs will include a cross-case analysis and progress in theory and method of fourth-generation assisted living technology research. We will produce practical guidance for organisations, policymakers, designers and service users.

Governing the public-private-partnerships of the future: learnings from the experiences in pandemic times

No-one is safe until everyone is safe. But what can be done when a country fails to take measures to control a pandemic virus? It poses a threat to its own people but also to its neighbours and beyond. Countries do pool sovereignty, working through supra-national structures, such as international agencies, or using processes set out in treaties, recognising the mutual benefits of the international rules-based system. Here we review the ways in which governments have, or have not worked together on other issues that pose a threat to global health and discuss the implications for pandemic responses.