coreSCD: multi-stakeholder consensus on core outcomes for sickle cell disease clinical trials

Background With the dramatic increase in the pipeline for new sickle cell disease (SCD) therapies in recent years, the time is ripe to ensure a robust body of evidence is available for decision making by regulators, payers, clinicians, and patients. Harmonization of the outcomes selected across interventional trials enables optimal post-trial appraisal and decision making through valid pooled analyses and indirect comparisons. We employed a structured, multi-stakeholder consensus process to develop core outcome sets (COS) for use in clinical trials of SCD interventions. Methods CoreSCD utilized a modified Delphi method adapted from the standards recommended by the Core Outcome Measures in Effectiveness Trials (COMET) Initiative. An initial list of candidate outcomes was developed through a targeted literature review and input from an 11-member advisory committee. A 44-member multi-stakeholder Delphi Panel was established and included patients and family members, advocates, clinicians, researchers, payers, health technology assessors, representatives from government agencies, and industry representatives. Patients/advocates comprised 25% of the Delphi Panel and orientation and training was provided prior to the consensus process to ensure all were prepared to participate meaningfully. Panelists completed three rounds of an online survey to rate the importance of candidate outcomes for inclusion in the COS. Summary data was provided between each voting round and an in-person consensus meeting was held between the second and third round of voting. Consensus rules were applied following each round of voting to eliminate outcomes that did not meet predetermined criteria for retention. Results Consensus was reached for two core outcome sets. The final COS for trials of disease-modifying therapies includes ten outcomes and the COS for trials of acute interventions includes six outcomes. Both core sets include clinical outcomes as well as outcomes related to functioning/quality of life, resource utilization, and survival/mortality. Conclusions Use of the COS in clinical development programs for SCD will help to ensure that relevant, consistent outcomes are available for decision making across the product lifecycle.

Standardised Outcomes in Nephrology – Chronic Kidney Disease (SONG-CKD): a protocol for establishing a core outcome set for adults with chronic kidney disease who do not require kidney replacement therapy

Background Globally, over 1.2 million people die from chronic kidney disease (CKD) every year. Patients with CKD are up to 10 times more likely to die prematurely than progress to kidney failure requiring kidney replacement therapy. The burden of symptoms and impaired quality of life in CKD may be compounded by comorbidities and treatment side effects. However, patient-important outcomes remain inconsistently and infrequently reported in trials in patients with CKD, which can limit evidence-informed decision-making. The Standardised Outcomes in Nephrology – Chronic Kidney Disease (SONG-CKD) aims to establish a consensus-based core outcome set for trials in patients with CKD not yet requiring kidney replacement therapy to ensure outcomes of relevance to patients, caregivers and health professionals are consistently reported in trials. Methods SONG-CKD involves four phases: a systematic review to identify outcomes (domains and measures) that have been reported in randomised controlled trials involving adults with CKD who do not require kidney replacement therapy; stakeholder key informant interviews with health professionals involved in the care of adults with CKD to ascertain their views on establishing core outcomes in CKD; an international two-round online Delphi survey with patients, caregivers, clinicians, researchers, policy makers and industry representatives to obtain consensus on critically important outcome domains; and stakeholder consensus workshops to review and finalise the set of core outcome domains for trials in CKD. Discussion Establishing a core outcome set to be reported in trials in patients with CKD will enhance the relevance, transparency and impact of research to improve the lives of people with CKD. Trial registration Not applicable. This study is registered with the Core Outcome Measures in Effectiveness Trials (COMET) database: http://www.comet-initiative.org/Studies/Details/1653.

Outcome Measures Following Critical Illness in Children With Disabilities: A Scoping Review

Children with disabilities compose a substantial portion of admissions and bed-days in the pediatric intensive care unit (PICU) and often experience readmissions over time. Impacts of a PICU admission on post-discharge health status may be difficult to distinguish from pre-existing disability in this population. Efforts to standardize outcome measures used for children with disabilities may help identify morbidities associated with PICU hospitalizations. Although a scoping review of outcome measures to assess children after episodes of critical illness has recently been published, it is not known to what extent these measures are appropriate for use in children with disabilities. This limits our ability to effectively measure long-term outcomes following critical illness in this important patient population. Through mixed methodology of scoping review and multi-stakeholder consensus, we aimed to identify and describe instruments previously utilized for this purpose and to explore additional tools for consideration. This yielded 51 measures across a variety of domains that have been utilized in the PICU setting and may be appropriate for use in children with disabilities. We describe characteristics of these instruments, including the type of developmental domains assessed, availability of population data, validation and considerations regarding administration in children with disabilities, and ease of availability of the instrument to researchers. Additionally, we suggest needed alterations or accommodations for these instruments to augment their utility in these populations, and highlight areas for future instrument development.

Branding of Ternate City

City branding has become a massive effort by many cities as tourist destinations to promote the city nationally and internationally. Ambon City, the Capital of Maluku Province, known as the “City of Music,” and the City of Solo with “Solo the Spirit of Java,” are examples of city branding. In North Maluku Province, Ternate City has a long history of colonialism, international trade, and a developing city. Unfortunately, inconsistencies still exist in how the city should be known. This study aims to identify and define city branding by using the constructivist grounded theory method. The themes identified were quality tourism, economic value, community engagement, stakeholder consensus, legendary ancient leadership, and historic and renowned commodity. Six main themes are theoretically based on how to branding Ternate city and involving seven important participants. These themes are then used to define the branding of Ternate City. Current research offers a testable model to brand a place or city based on the identified subjects. At the same time, potential future studies are also suggested regarding the weaknesses of the current study.

Quality indicators development and prioritisation for emergency medical call centres: a stakeholder consensus

IntroductionEmergency medical regulation is a risky activity. In France, emergency medical societies have proposed activity and performance indicators, but their lists are non-exhaustive, unstructured and used heterogeneously among emergency medical call centres (Centres de Réception et de Régulation des Appels, CRRA). Our objective was to build by means of regional stakeholder consensus an operational quality dashboard for CRRAs.MethodsWe conducted an observational step in a French CRRA from June to September 2018 and at the same time listed existing activity and quality indicators through a rapid international literature review. We adapted and classified all indicators identified in a structured table. We prioritised them from April to September 2019 by seeking consensus with one regulator physician and one medical regulation assistant from the 13 CRRAs of the largest French region. We used an adapted Delphi method with a prioritisation scale from 1 to 9.ResultsThe rapid review of literature included 33 studies among the 414 identified and, with the first observational step, resulted in a list of 360 quality indicators covering the following areas: material resources, human resources, quality approach, call handling and postcall support. 15 of the 26 members participated in the entire process. Seventy indicators were considered as priorities with strong agreement among participants. We built an operational dashboard of quality indicators deemed high priority and provided 70 descriptive indicator sheets.ConclusionOur study allowed to build an operational quality dashboard for CRRAs as a ready-to-use support for an internal audit, for prioritisation of quality approach actions and for national and international benchmarking.

Advancing Efficient and Timely Community Access to SARS-CoV-2 Serology Testing in Europe: a Multi-stakeholder Consensus

Background: Currently there is no clear consensus on the use, value, benefits, and impact of serology testing as part of a comprehensive SARS-CoV-2 testing strategy. The lack of clarity on the use of this strategy in policies and guidelines may have serious implications on the efforts to curb the pandemic. The aim of this paper is to elaborate an experts and community consensus on the use of serology testing as an effective method to respond to and mitigate the impact of the pandemic. The recommendations herein can help build community awareness and guide advocacy strategies. Methods: A desk review was conducted to inform a working document that was subject to a multistage process of validation and feedback by a group of renowned experts. The multi-stakeholder group of experts, representing the European and international levels, convened to inform and validate the recommendations. Results: The consensus offered eight policy recommendations organized in two main themes. The first group of recommendations provides guidance on the role and value of serology testing to contain and understand the COVID-19 pandemic. The second group targets health system strengthening aspects necessary to support the appropriate delivery of serology testing. Conclusions: Recommendations seek to indicate how SARS-CoV-2 serology testing may positively impact national health systems, country economies and local communities. The pertinence of the recommendations is to communities in Europe, and beyond, and relevant to multiple stakeholders. Given the rapidly changing scenario, this set of recommendations should be considered a live document.

Advancing Efficient and Timely Community Access to SARS-CoV-2 Serology Testing in Europe: a Multi-stakeholder Consensus

Background: Currently there is no clear consensus on the use, value, benefits, and impact of serology testing as part of a comprehensive SARS-CoV-2 testing strategy. The lack of clarity on the use of this strategy in policies and guidelines may have serious implications on the efforts to curb the pandemic. The aim of this paper is to elaborate an experts and community consensus on the use of serology testing as an effective method to respond to and mitigate the impact of the pandemic. The recommendations herein can help build community awareness and guide advocacy strategies. Methods: A desk review was conducted to inform a working document that was subject to a multistage process of validation and feedback by a group of renowned experts. The multi-stakeholder group of experts, representing the European and international levels, convened to inform and validate the recommendations. Results: The consensus offered eight policy recommendations organized in two main themes. The first group of recommendations provides guidance on the role and value of serology testing to contain and understand the COVID-19 pandemic. The second group targets health system strengthening aspects necessary to support the appropriate delivery of serology testing. Conclusions: Recommendations seek to indicate how SARS-CoV-2 serology testing may positively impact national health systems, country economies and local communities. The pertinence of the recommendations is to communities in Europe, and beyond, and relevant to multiple stakeholders. Given the rapidly changing scenario, this set of recommendations should be considered a live document.

Stakeholder consensus for decision making in eye-gaze control technology for children, adolescents and adults with cerebral palsy service provision: findings from a Delphi study

Background Limited research exists to guide clinical decisions about trialling, selecting, implementing and evaluating eye-gaze control technology. This paper reports on the outcomes of a Delphi study that was conducted to build international stakeholder consensus to inform decision making about trialling and implementing eye-gaze control technology with people with cerebral palsy. Methods A three-round online Delphi survey was conducted. In Round 1, 126 stakeholders responded to questions identified through an international stakeholder Advisory Panel and systematic reviews. In Round 2, 63 respondents rated the importance of 200 statements generated by in Round 1. In Round 3, 41 respondents rated the importance of the 105 highest ranked statements retained from Round 2. Results Stakeholders achieved consensus on 94 of the original 200 statements. These statements related to person factors, support networks, the environment, and technical aspects to consider during assessment, trial, implementation and follow-up. Findings reinforced the importance of an individualised approach and that information gathered from the user, their support network and professionals are central when measuring outcomes. Information required to support an application for funding was obtained. Conclusion This Delphi study has identified issues which are unique to eye-gaze control technology and will enhance its implementation with people with cerebral palsy.