Patient Group Representatives Research Articles

Engaging stakeholders in embedded newborn health services/systems research in Kenya; a continuous process involving multiple actors.

Background Engaging relevant stakeholders throughout the research cycle is increasingly recognised as critical to conducting quality health systems research. There are few descriptions and analyses of stakeholder engagement in practice for embedded health systems research especially those that must navigate multi-level decentralised health systems. We describe and reflect on the stakeholder engagement activities of an international multi-disciplinary programme of research focused on newborn care in hospitals in Kenya. Methodology Our experienced project stakeholder engagement group coordinated engagement activities across multiple stakeholders with differing interests in the research. We conducted a stakeholder mapping and analysis using an engagement matrix to include national and county-level policymakers, professional communities, associations and regulators, health managers, frontline healthcare workers, patients, families and patient representative groups. Our engagement group maintained a log of engagement activities and had regular programme feedback meetings and discussions. As part of our analysis of stakeholder engagement, we drew on project documents and meeting minutes, and on a conceptual framework which distinguishes between the moral, strategic and practicaldimensions of stakeholder engagement. Results Stakeholder engagement was essential but complex in practice. Although we had significant prior engagement experience and relationships at the hospitals and the counties, introducing new staff into NBUs triggered complexities that required careful consultation along the bureaucracies at the counties. Despite the counties having similar hierarchical architectures, engagement processes varied and achieved different outcomes across counties. There were also multiple officeholder transitions over the research period, occasioned by factors in our external environment, often necessitating engaging afresh. Conclusion Even with a carefully developed stakeholder engagement plan, an experienced team, and a landscape backed by long-term embeddedness, health research stakeholder engagement can unfold in unexpected ways and requires continuous effort, resources, and adaptability. Meeting the moral, strategic, and practical potential of engagement requires flexibility, responsiveness, and commitment, including adequate resources.

Top Ten epilepsy research priorities: A UK priority setting partnership.

Research into epilepsy has experienced decades of chronic underfunding compared to other neurological conditions despite its prevalence and seriousness. To evidence the need for greater investment, the Epilepsy Research Institute (formerly Epilepsy Research UK) funded, led and managed a James Lind Alliance (JLA) Priority Setting Partnership (PSP). This "industry standard" methodology brings together healthcare professionals, patients, carers and patient group representatives to identify and prioritise research uncertainties within a defined area of health or care. The UK Epilepsy PSP is a once-in-a-generation, national consensus that collated and ranked the research priorities of the UK epilepsy and associated condition community. Following JLA methodology, this 18-month project engaged over 100 patient groups and 5000 people affected by and working in epilepsy, including medics and allied healthcare professionals, from across the UK. Over 5400 priorities were received, with anti-seizure medication, sudden unexpected death in epilepsy (SUDEP) and epilepsy in women among the most frequently reported themes. The responses received were categorised and translated into distinct, researchable questions. Questions were excluded if deemed to be "answered" following an evidence check, while research uncertainties (i.e. unanswered and partially answered questions) formed the basis of a second, shortlisting survey. The shortlisted questions were then discussed and debated at the final workshop by participants that broadly represented the UK epilepsy and associated condition community. The final ranking and Top Ten priorities for research into epilepsy were then agreed. The aim of the UK Epilepsy PSP is to encourage and inspire researchers to investigate the research areas prioritised by those most affected by the condition and provide the evidence of need to aid future policy making discussions and support research funding applications.

Protocol for a mixed-methods study to explore implementation outcomes of the Phone-based Interventions under Nurse Guidance after Stroke (PINGS-II) across 10 hospitals in Ghana

BackgroundStroke survivors are at a substantially higher risk for adverse vascular events driven partly by poorly controlled vascular risk factors. Mobile health interventions supported by task shifting strategies have been...

Advocating Values through Meaningful Participation: Introducing a Method to Elicit and Analyze Values for Enriching Data Donation Practices in Healthcare

The secondary use of routinely collected patient data made possible by the broad consent form is seen as a prerequisite for developing data-driven health technologies. In Germany, relevant stakeholder groups (e.g., ethics committees and data protection authorities) specified the broad consent form; however, only one group of patient representatives was consulted, which may indicate asymmetries in engagement. This situation informed our research on medical data donation and emphasized foregrounding patient values. Drawing on participatory design, value sensitive design, and emerging research on value-led participation, we propose a method consisting of (1) a workshop concept for participatory value elicitation composed of four carefully coordinated phases and (2) an analysis procedure to examine the empirical data collected. This analysis allowed us to derive design requirements for medical data donation user interfaces. We conducted three workshops with patient advocates of vulnerable groups and patients in residential care of a psychosomatic unit. Our findings provide new directions to improve user interfaces for medical data donation: First, user interfaces need to enhance patients' reflective thinking about the potential consequences of their data donation; second, a decision facilitator supporting patients' value-based decision-making (e.g., by providing simple language or tailoring descriptions to patient needs); and finally, a data intermediary relieving patients' decision-making and giving them control over their data after donation. Moreover, we emphasize the need to increase the use of participatory approaches in health technology development.

Developing generic templates to shape the future for conducting integrated research platform trials

BackgroundInterventional clinical studies conducted in the regulated drug research environment are designed using International Council for Harmonisation (ICH) regulatory guidance documents: ICH E6 (R2) Good Clinical Practice—scientific guideline, first published in 2002 and last updated in 2016. This document provides an international ethical and scientific quality standard for designing and conducting trials that involve the participation of human subjects. Recently, there has been heightened awareness of the importance of integrated research platform trials (IRPs) designed to evaluate multiple therapies simultaneously. The use of a single master protocol as a key source document to fulfill trial conduct obligations has resulted in a re-examination of the templates used to fulfill the dynamic regulatory and modern drug development environment challenges.MethodsRegulatory medical writing, biostatistical, and other members of EU Patient-cEntric clinicAl tRial pLatforms (EU-PEARL) developed the suite of templates for IRPs over a 3.5-year period. Stakeholders contributing expertise included academic hospitals, pharmaceutical companies, non-governmental organizations, patient representative groups, and small and medium-sized enterprises (SMEs).ResultsThe suite of templates for IRPs based on TransCelerate’s Common Protocol Template (CPT) and statistical analysis plan (SAP) should help authors navigate relevant guidelines as they create study design content relevant for today’s IRP studies. It offers practical suggestions for adaptive platform designs which offer flexible features such as dropping treatments for futility or adding new treatments to be tested during a trial. The EU-PEARL suite of templates for IRPs comprises a preface, followed by the actual resource. The preface clarifies the intended use and underlying principles that inform resource utility. The preface lists references contributing to the development of the resource. The resource includes TransCelerate CPT guidance text, and EU-PEARL-derived guidance text, distinguished from one another using shading. Rationale comments are used throughout for clarification purposes. In addition, a user-friendly, functional, and informative Platform Trials Best Practices tool to support the setup, design, planning, implementation, and conduct of complex and innovative trials to support multi-sourced/multi-company platform trials is also provided. Together, the EU-PEARL suite of templates and the Platform Trials Best Practices tool constitute the reference user manual.ConclusionsThis publication is intended to enhance the use, understanding, and dissemination of the EU-PEARL suite of templates for designing IRPs. The reference user manual and the associated website (http://www.eu-pearl) should facilitate the designing of IRP trials.

Clinical practice recommendations for kidney involvement in tuberous sclerosis complex: a consensus statement by the ERKNet Working Group for Autosomal Dominant Structural Kidney Disorders and the ERA Genes & Kidney Working Group.

Tuberous sclerosis complex (TSC) is an autosomal dominant disorder characterized by the presence of proliferative lesions throughout the body. Management of TSC is challenging because patients have a multifaceted systemic illness with prominent neurological and developmental impact as well as potentially severe kidney, heart and lung phenotypes; however, every organ system can be involved. Adequate care for patients with TSC requires a coordinated effort involving a multidisciplinary team of clinicians and support staff. This clinical practice recommendation was developed by nephrologists, urologists, paediatric radiologists, interventional radiologists, geneticists, pathologists, and patient and family group representatives, with a focus on TSC-associated kidney manifestations. Careful monitoring of kidney function and assessment of kidney structural lesions by imaging enable early interventions that can preserve kidney function through targeted approaches. Here, we summarize the current evidence and present recommendations for the multidisciplinary management of kidney involvement in TSC.

Abstract P1-08-10: A web-based Patient Decision Aid Toolkit for unaffected BRCA mutation carriers

Abstract Women with a pathogenic mutation in the BRCA1 or BRCA2 genes have an elevated lifetime risk of developing breast and ovarian cancer. To address this risk, women are managed with a combination of surveillance and/or risk-reduction strategies. Decisions about risk management strategies can be complex, personal and multifactorial. Women often struggle with the decision-making process. In addition, within the clinical environment, there may be variations in recommendations between clinicians that can leave women uncertain and less able to choose a risk management pathway. The overall aim of this project is the development of a web-based patient decision aid toolkit for BRCA mutation carriers that will improve the decision-making process by providing the user with information about their cancer risk, options for risk management and potential benefits and side effects. Development of the patient decision aid was guided by the International Patient Decision Aid Standards (IPDAS). With appropriate ethical approval, a mixed methods approach was used to identify suitable content for the decision aid. A decision-making needs assessment was conducted to identify the information needs of women with a BRCA mutation. Semi-structured interviews were held with cancer unaffected BRCA mutation carriers (n = 16) and key stakeholders including healthcare professionals, policy makers and patient group representatives (n= 10). Data were analysed by thematic analysis. Systematic scoping reviews were conducted to synthesise relevant evidence on risk-management options, benefits, harms and the development and testing of patient decision aids in general. Content for the decision aid was refined using a Delphi process to build consensus on items for inclusion in the decision aid amongst a diverse panel of experts (n=13). A prototype patient decision aid was developed which included written information as well as visual depictions of risk, videos and photographs to enhance the patient’s information experience. A ‘values clarification’ activity was included to enable women to work through their own values and preferences relating to risk management interventions and their associated benefits and side-effects. Initial ‘sandpit’ testing of the decision aid prototype was performed by the research team and advisory group. Usability testing was conducted with BRCA mutation carries (n = 8) and healthcare professionals (n = 8) using both qualitative interviews and quantitative surveys. The research team made final revisions to the decision aid based on participant feedback and committee consensus. This evidence-based patient decision aid can be used by BRCA mutation carriers unaccompanied or during a clinical consultation. We envisage that this decision aid will improve the decision-making process by assisting women and clinicians during shared decision-making regarding cancer risk management. Citation Format: Sarah A. McGarrigle, Carol Spillane, Niamh Byrne, Manria Polus, Geraldine Prizeman, Amanda Drury, Elizabeth Connolly, Anne-Marie Brady, Yvonne Hanhauser. A web-based Patient Decision Aid Toolkit for unaffected BRCA mutation carriers [abstract]. In: Proceedings of the 2022 San Antonio Breast Cancer Symposium; 2022 Dec 6-10; San Antonio, TX. Philadelphia (PA): AACR; Cancer Res 2023;83(5 Suppl):Abstract nr P1-08-10.

Patient-based evidence: its role in decision making on end-of-life, orphan, and ultra-orphan medicines.

The Scottish Medicines Consortium (SMC) conducts early health technology assessment (HTA) of new medicines on behalf of NHSScotland. Assessment of end-of-life (EoL), orphan, and ultra-orphan medicines includes a process to gather evidence from patients and carers during Patient and Clinician Engagement (PACE) meetings. The output of PACE meetings is a consensus statement describing the medicine's added value from the perspective of patients/carers and clinicians. The PACE statement is used by SMC committee members in decision making. This study compared how PACE participants and SMC committee members rate the importance of information in PACE statements for these medicines. A survey was undertaken of patient group (PG) representatives and clinicians who participated in PACE meetings, and SMC committee members. PACE participants who responded (26 PG representatives and 14 clinicians) rated health benefits and ability to take part in normal life as important/very important. Convenience of administration and treatment choice received the lowest rating. Hope for the future received the most diverse response. PACE participants generally rated the importance of quality of life themes higher than committee members (n=20) but the rank order was similar. Differences between the proportion of PACE participants and committee members who rated themes as important/very important were greatest for treatment choice and hope for the future. In general, PACE themes and subthemes that were rated highly by PACE participants were also considered important by SMC committee members, indicating that information captured during PACE meetings is relevant when making decisions on EoL, orphan, and ultra-orphan medicines.

Defining patient-centered research priorities in pediatric dermatology.

Patient and caregiver perspectives are critical in understanding dermatologic disease impact, presentation, and management in children. The Pediatric Dermatology Research Alliance (PeDRA) Patient Advisory Committee (PtAC), a group of patient representatives and parents of children with cutaneous disease, pursued a multistep, iterative, consensus-building process to identify comprehensive, high-priority research needs. Building on discussions at the 2020 PeDRA Annual Conference, a research prioritization survey was developed and completed by PtAC members. Survey themes were aggregated and workshopped by the PtAC through a series of facilitated calls. Emerging priorities were refined in collaboration with additional PeDRA patient community members at the 2021 PeDRA Annual Conference. Subsequently, a final actionable list was agreed upon. Fourteen PtAC members (86.7% female) representing patients with alopecia areata, atopic dermatitis, vascular birthmarks, congenital melanocytic nevi, ectodermal dysplasias, epidermolysis bullosa, Gorlin syndrome, hidradenitis suppurativa, ichthyosis, pemphigus, psoriasis, Sturge-Weber syndrome, and pachyonychia congenita completed the survey. Following serial PtAC meetings, 60 research needs were identified from five domains: psychosocial challenges, health care navigation/disease management, causes/triggers, treatments to preserve or save life, and treatments to preserve or save quality of life. Many pediatric dermatology research priorities align across affected communities and may drive meaningful, patient-centric initiatives and investigations.

Experiences of living with Lynch Syndrome: A reflexive thematic analysis.

Lynch Syndrome is one of the most common hereditary cancer syndromes, arising from DNA mismatch repair. Lynch Syndrome carriers are at increased lifetime risk of developing certain cancers, such as colorectal and endometrial. This increased risk can result in adverse psychological outcomes. The present qualitative study explores the experiences of individuals with Lynch Syndrome when accessing and managing healthcare in the period after learning of their Lynch Syndrome status. Twelve interviews were conducted with Lynch Syndrome carriers in Ireland, with recruitment occurring predominantly online through closed social media platforms. This was coordinated by Lynch Syndrome Ireland, a patient representative group. Reflexive thematic analysis was used to analyse the data. There was significant Public and Patient Involvement in this study, with the committee members (N=2) of Lynch Syndrome Ireland acting on the panel. The involvement of the PPI panel began from initial project idea conception and continued throughout the study. Lynch Syndrome carriers highlighted the lack of adequate information from medical professionals regarding their diagnosis. Furthermore, participants spoke of the significant lack of knowledge amongst medical professionals about Lynch Syndrome. A theme depicting guilt was also noted regarding passing Lynch Syndrome to their children, and the worry experienced when children underwent genetic testing. This study highlighted the experiences of having a Lynch Syndrome diagnosis and demonstrates a need for further psychological and medical support for the Lynch Syndrome community, including a clear need for improvements in genetic cancer services in this field.

P230 Feasibility study of mobile phone photography as a possible outcome measure of systemic sclerosis-related digital ulcers

Abstract Background/Aims Digital ulcers cause pain and disability in patients with systemic sclerosis (SSc). They are often used as outcome measures in clinical trials; however, trials have been hampered by a lack of reliable outcome measures of healing. Photographic monitoring of digital ulcers using smartphones could overcome this difficulty. Our objective was to assess the feasibility of patients collecting mobile-phone images of their digital ‘lesions’ as a first step in a programme of research to develop a smartphone-based outcome measure. Methods An imaging protocol was developed with input from a patient representative group. Patients with SSc-related digital lesions were recruited and instructed to photograph one or more finger lesions each day over 30 days using their smartphone, at the same time each day. Instructions on how to take the photographs were given either face-to-face or remotely. An adhesive dot, placed adjacent to the lesion(s), provided a 1cm reference scale to help extract accurate measurements. Images were uploaded by each patient to a secure Dropbox folder. Image quality was assessed using six criteria: blurriness, shadow, uniform lighting, dot location, dot angle, and central positioning of the lesion. After image collection, patients were asked to complete a feedback questionnaire, with most questions on a scale of 1 (very easy) to 10 (very difficult). Results Twelve patients (10 female, age 37 to 72 years, disease duration 1 to 27 years) returned 331 photographs of 19 lesions (maximum three lesions per patient). Each patient sent a median of 30 photographs (IQR 14 to 33), with a median of 15 photographs per lesion (IQR 2 to 31). Of the 331 photographs, 24 were ‘duplicates’ taken on the same day, meaning that there were 263 ‘missing’ photographs (i.e., 570 photographs would have been taken if 19 lesions had been photographed every day). Image quality results included the following: 255 (77%) of photographs were sufficiently in focus; 268 (81%) had some shadow; lighting was even in 55 (17%); dot location was acceptable in 231 (70%); dot angle was ideal in 107 (32%); the lesion was centred in 254 (77%). Patient feedback showed 9/10 responses would be willing to record images at least 1-3 times per week, and the overall patient experience taking photographs was good, with a median rating of 3 (IQR 3 to 4). Conclusion Capturing photographs of digital lesions with a smartphone was feasible for most patients. They were able to take photographs in focus and with lesions central in the image. Support will be required to improve lighting, shadow, and dot positioning while minimising discomfort. These encouraging results will inform the next research phase to develop a smartphone app for monitoring finger lesions. Disclosure A.K. Davison: None. H. Patrick: None. P. New: None. G. Dinsdale: None. V.P. Taxiarchi: None. W.G. Dixon: None. A. Vail: None. A.K. Murray: None. M. Dickinson: None. C. Taylor: None. A.L. Herrick: None.

Editor's Foreword

Welcome to the fifth issue of the Journal of Vascular Societies Great Britain and Ireland (JVSGBI). We are really delighted with the success of the journal, and this issue marks the first anniversary of the launch at the Vascular Societies GB&I Annual Scientific meeting last year. The increasing interest in the JVSGBI has been demonstrated by the growing number of submitted articles and the impressive website stats (over 3,000 views). This issue includes three editorials. The first by Andrew Garnham (VSGBI President Elect) and Rachael Forsythe (Rouleaux President) provides a thoughtful reflection on the problem of bullying, undermining and harassment in (vascular) surgical training highlighted as a persistent problem by a Rouleaux Club paper in this edition. The second editorial from Dr Una Adderley, National Wound Care Strategy Programme (NWCSP) Director, outlines past achievements and future plans for the programme. Finally, Duygu Yenidogan-Schmidt, Chair of the Executive Committee of AAA UK, a newly formed patient representative group giving national representation to AAA patients and relatives, highlights the origins, purpose and aims of this group. I am sure these editorials will be of great interest to readers. There are four original research articles, the first from the Rouleaux Club, the UK Vascular Surgery Trainees’ Association, reports the findings from their recent bullying, undermining and harassment survey. The second from the Vascular and Endovascular Research Network (VERN) reports the findings from the Tier 3 COVID-19 Vascular Service (COVER) study. The final two original articles present the findings from the James Lind Alliance Priority Setting Partnership from the Vascular Services and Venous Conditions Groups. It is fantastic to see vascular trainees continuing to undertake meaningful and impactful research and choose the JVSGBI to disseminate their work. This edition also contains a research protocol from Ravindhran et al for a systematic review which aims to study changes in functional health status following open abdominal aortic aneurysm repair. Finally, in this issue there is a short educational report entitled ‘Intraoperative management of REBOA’ and a case report entitled ‘Carotid web: an important cause of stroke in young people’. The National Wound Care Strategy Programme Guidelines for lower limb wounds are published as a supplement to this issue and can be located on the journal website. This is our fifth issue, and we would like to take the opportunity to thank all the authors who have submitted articles during the last year, which is building on the success of the journal – your contribution has been really appreciated and I hope you are delighted with your publication. We would also like to thank all the reviewers for their timely and thorough contributions. Finally, please do continue to share your work by submitting articles for publication.

A framework for action to improve patient and public involvement in health technology assessment.

Patient and public involvement (PPI) in the Brazilian Health Technology Assessment (HTA) process occurs in response to a legislative mandate for "social participation." This resulted in some limited patient participation activities, and, therefore, a more systematic approach was needed. The study describes the development of a suggested framework for action to improve PPI in HTA. This work used formal methodology to develop a PPI framework based on three-phase mixed-methods research with desktop review of Brazilian PPI activities in HTA; workshop, survey, and interviews with Brazilian stakeholders; and a rapid review of international practices to enact effective patient involvement. Patient partners reviewed the draft framework. According to patient group representatives, their involvement in the Brazilian HTA process is important but could be improved. Different stakeholders perceived barriers, identified values, and made suggestions for improvement, such as expansion of communication, capacity building, and transparency, to support more meaningful patient involvement. The international practices identified opportunities for earlier, more active, and collaborative PPI during all HTA stages, based on values and principles that are relevant for Brazilian patients and the public. These findings were synthesized to design a framework that defines and systematizes actions to support PPI in Brazil, highlighting the importance of evaluating these strategies. Since the publication of this framework, some of its suggestions are being implemented in the Brazilian HTA process to improve PPI. We encourage other HTA organizations to consider a systematic and planned approach with regular evaluation when pursuing or strengthening involvement practices.

Medical cannabis for severe treatment resistant epilepsy in children: a case-series of 10 patients

ObjectivesTo report the findings of a case-series of 10 children suffering with intractable epilepsies in the UK to determine the feasibility for using whole-plant cannabis medicines to treat seizures in...

OP242 Patient-based Evidence: A Comparison Of The Views Of Patient And Clinical Engagement Participants And Committee Members

IntroductionThe Scottish Medicines Consortium (SMC) conducts early health technology assessment (HTA) of new medicines on behalf of NHSScotland. Evidence from patients and carers on end-of-life and orphan medicines is gathered during Patient and Clinician Engagement (PACE) meetings. The output is a consensus statement describing a medicine's added value from the perspective of patients/carers and clinicians, which is used by SMC committee members in decision-making. This study compared the importance of factors in the PACE statement to PACE participants and committee members.MethodsA survey of ninety-eight PACE participants (consisting of forty-two patient group (PG) representatives and fifty-six clinicians) investigated the importance of quality of life (QoL) themes (family/carer impact, health benefits, tolerability, psychological benefit, hope, normal life, treatment choice and convenience) identified from an earlier thematic analysis of PACE statements. The findings from PG representatives and clinicians were compared, and the overall results were further compared with those from a previous survey of committee members (n = 26).ResultsAmong PACE participants who responded (twenty-six PG representatives and fourteen clinicians), 100 percent rated ‘health benefits’ and ‘ability to take part in normal life’ as important / very important. ‘Convenience of administration’ and ‘treatment choice’ received the lowest rating with fifteen percent and nineteen percent respectively of PG representatives versus seven percent of clinicians rating each as very important. ‘Hope for the future’ received the most diverse response with fifty-eight percent of PG representatives and fourteen of clinicians rating this as very important.In general, PACE participants rated importance of QoL themes higher than committee members (n = 21) but the rank order was similar. Differences between the proportion of PACE participants and committee members who rated themes important/very important was greatest for ‘treatment choice’ (sixty-seven percent versus twenty percent respectively) and ‘hope for the future’ (eighty-two percent versus fifty-three percent).ConclusionsThe findings demonstrate some alignment between PACE participants’ and committee members’ responses, supporting the value of the PACE output in decision-making. Areas for further research are highlighted.

Million Hearts Cardiac Rehabilitation Think Tank: Accelerating New Care Models.

This article describes the October 2020 proceedings of the Million Hearts Cardiac Rehabilitation Think Tank: Accelerating New Care Models, convened with representatives from professional organizations, cardiac rehabilitation (CR) programs, academic institutions, federal agencies, payers, and patient representative groups. As CR delivery evolves, terminology is evolving to reflect not where activities occur (eg, center, home) but how CR is delivered: in-person synchronous, synchronous with real-time audiovisual communication (virtual), or asynchronous (remote). Patients and CR staff may interact through ≥1 delivery modes. Though new models may change how CR is delivered and who can access CR, new models should not change what is delivered-a multidisciplinary program addressing CR core components. During the coronavirus disease 2019 (COVID-19) public health emergency, Medicare issued waivers to allow virtual CR; it is unclear whether these waivers will become permanent policy post-public health emergency. Given CR underuse and disparities in delivery, new models must equitably address patient and health system contributors to disparities. Strategies for implementing new CR care models address safety, exercise prescription, monitoring, and education. The available evidence supports the efficacy and safety of new CR care models. Still, additional research should study diverse populations, impact on patient-centered outcomes, effect on long-term outcomes and health care utilization, and implementation in diverse settings. CR is evolving to include in-person synchronous, virtual, and remote modes of delivery; there is significant enthusiasm for implementing new care models and learning how new care models can broaden access to CR, improve patient outcomes, and address health inequities.

Can we learn lessons from the FDA's approval of aducanumab?

On 7 June 2021, aducanumab was granted accelerated approval for the treatment of Alzheimer disease (AD) by the FDA on the basis of amyloid-lowering effects considered reasonably likely to confer clinical benefit. This decision makes aducanumab the first new drug to be approved for the treatment of AD since 2003 and the first drug to ever be approved for modification of the course of AD. Many have questioned how scientific evidence, expert advice and the best interests of patients and families were considered in the approval decision. In this article, we argue that prior to approval, the FDA and Biogen's shared interpretation of clinical trial data - that high-dose aducanumab was substantially clinically effective - avoided conventional scientific scrutiny, was prominently advanced by patient representative groups who had been major recipients of Biogen funds, and raised concerns that safeguards were insufficient to mitigate regulatory capture within the FDA. Here, we reflect on events leading to the FDA's decision on 7 June 2021 and consider whether any lessons can be learned for the field.

Developing a National Set of Health Equity Indicators Using a Consensus Building Process.

Considerable health inequities documented in Israel between communities, populations and regions, undermine the rights of all citizens to optimal health. The first step towards health equity is agreement on a set of national indicators, reflecting equity in healthcare provision and health outcomes, and allowing monitoring of the impact of interventions on the reduction of disparities. We describe the process of reaching a consensus on a defined set of national equity indicators. The study was conducted between January 2019 and June 2020, in a multistage design: (A) Identifying appropriate and available inequity measures via interviews with stakeholders. (B) Agreement on the screening criteria (public health importance; gap characteristics; potential for change; public interest) and relative weighting. (C) Constructing the consultation framework as an online, 3-round Delphi technique, with a range of experts recruited from the health, welfare and education sectors. Participants were of diverse age, gender, geographic location, religion and ethnicity, and came from academia, healthcare provision, government ministries and patient representative groups. Thirty measures of inequity, presented to participants, represented the following domains: Health promotion (11 indicators), acute and chronic morbidity (11), life expectancy and mortality (2), health infrastructures and affordability of care (4), education and employment (2). Of the 77 individuals contacted, 75 (97%) expressed willingness to participate, and 55 (73%) completed all three scoring rounds. The leading ten indicators were: Diabetes care, childhood obesity, adult obesity, distribution of healthcare personnel, fatal childhood injuries, cigarette smoking, infant mortality, ability to afford care, access to psychotherapy and distribution of hospital beds. Agreement among raters, measured as intra-class correlation coefficient (ICC), was 0.75. A diverse range of consultants reached a consensus on the most important national equity indicators, including both clinical and system indicators. Results should be used to guide governmental decision-making and inter-sectoral strategies, furthering the pursuit of a more equitable healthcare system.

Taking charge of eczema self-management: a qualitative interview study with young people with eczema

ObjectivesTo explore young people’s experiences of eczema self-management and interacting with health professionals.DesignSecondary qualitative data analysis of data sets from two semistructured interview studies. Data were analysed using inductive thematic...

Representing the \u2018Voice\u2019 of Patients: How Third Sector Organisations Conceptualise and Communicate Experiential Knowledge in Health Service Development

The growing prominence of patient and public involvement in health services has led to the increased use of experiential knowledge alongside medical and professional knowledge bases. Third sector organisations, which position themselves as representatives of collective patient groups, have established channels to communicate experiential knowledge to health services. However, organisations may interpret and communicate experiential knowledge in different ways, and due to a lack of inherent authority, it can be dismissed by health professionals. Thus, drawing on individual interviews with organisation representatives, we explore the definitions and uses of as well as the ‘filters’ placed upon experiential knowledge. The analysis suggests that whilst experiential knowledge is seen as all-encompassing, practical and transformative, the organisations need to engage in actions that can tame experiential knowledge and try to balance between ensuring that the critical and authentic elements of experiential knowledge were not lost whilst retaining a position as collaborators in health care development processes.