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Journal of Pediatric Surgery Oct 2023Registries are important in rare disease research. The Anorectal Malformation Network (ARM-Net) registry is a well-established European patient registry collecting...
BACKGROUND
Registries are important in rare disease research. The Anorectal Malformation Network (ARM-Net) registry is a well-established European patient registry collecting demographic, clinical, and functional outcome data. We assessed the quality of this registry through review of the structure, data elements, collected data, and user experience.
MATERIAL AND METHODS
Design and data elements were assessed for completeness, consistency, usefulness, accuracy, validity, and comparability. An intra- and inter-user variability study was conducted through monitoring and re-registration of patients. User experience was assessed via a questionnaire on registration, design of registry, and satisfaction.
RESULTS
We evaluated 119 data elements, of which 107 were utilized and comprised 42 string and 65 numeric elements. A minority (37.0%) of the 2278 included records had complete data, though this improved to 83.5% when follow-up elements were excluded. Intra-observer variability demonstrated 11.7% incongruence, while inter-observer variability was 14.7%. Users were predominantly pediatric surgeons and typically registered patients within 11-30 min. Users did not experience any significant difficulties with data entry and were generally satisfied with the registry, but preferred more longitudinal data and patient-reported outcomes.
CONCLUSIONS
The ARM-Net registry presents one of the largest ARM cohorts. Although its collected data are valuable, they are susceptible to error and user variability. Continuous evaluations are required to maintain relevant and high-quality data and to achieve long-term sustainability. With the recommendations resulting from this study, we call for rare disease patient registries to take example and aim to continuously improve their data quality to enhance the small, but impactful, field of rare disease research.
LEVEL OF EVIDENCE
V.
Topics: Child; Humans; Rare Diseases; Registries; Data Accuracy; Surveys and Questionnaires; Anorectal Malformations; Data Collection
PubMed: 37045715
DOI: 10.1016/j.jpedsurg.2023.02.049 -
Journal of Pharmacy & Pharmaceutical... 2023Patient support programs (PSPs) offer a unique opportunity to collect real-world data that can contribute to improving patient care and informing healthcare decision...
Patient support programs (PSPs) offer a unique opportunity to collect real-world data that can contribute to improving patient care and informing healthcare decision making. In this perspective article, we explore the collection of data through PSPs in Canada, current advances in data collection methods, and the potential for generating acceptable real-world evidence (RWE). With PSP infrastructure already in place for most specialized drugs in Canada, adding and strengthening data collection capacities has been a focus in recent years. However, limitations in PSP data, including challenges related to quality, bias, and trust, need to be acknowledged and addressed. Forward-thinking PSP developers have been taking steps to strengthen the PSP datasphere, such as engaging third parties for data analysis, publishing peer-reviewed studies that utilize PSPs as a data source and incorporating quality controls into data collection processes. This article illustrates the current state of PSP data collection by examining six PSP RWE studies and outlining their data characteristics and the health outcomes collected from the PSP. A framework for collecting real-world data within a PSP and a checklist to address issues of trust and bias in PSP data collection is also provided. Collaboration between drug manufacturers, PSP vendors, and data specialists will be crucial in elevating PSP data to a level acceptable to healthcare decision makers, including health technology assessors and payers, with the ultimate beneficiary being patients.
Topics: Humans; Data Collection; Delivery of Health Care; Canada
PubMed: 37901362
DOI: 10.3389/jpps.2023.11877 -
Qualitative Health Research Dec 2023Identifying and recruiting key informants is a widely used sampling strategy in applied qualitative health research. Key informants were first conceptualized within...
Identifying and recruiting key informants is a widely used sampling strategy in applied qualitative health research. Key informants were first conceptualized within ethnography, but there is little methodological guidance about how to use this technique outside of that research tradition. The objective of this article is to offer practical suggestions about how existing methods for data collection with key informants could be translated to methodologies commonly used in applied qualitative health research. This article delineates how key informants could be conceptualized and sampled and how data sufficiency can be approached. The article prompts deeper consideration of the politics of representation and epistemic power that are inherent to the use of key informants in applied qualitative health research.
Topics: Humans; Qualitative Research; Anthropology, Cultural; Research Design; Politics; Data Collection
PubMed: 37902082
DOI: 10.1177/10497323231198796 -
PloS One 2023To ensure uncertainty in match outcomes, professional sporting leagues have used various competitive balance policies, including player salary caps, revenue sharing...
To ensure uncertainty in match outcomes, professional sporting leagues have used various competitive balance policies, including player salary caps, revenue sharing among teams and player drafts. The Australian Football League (AFL) introduced a player draft in 1986, and to refine its operation, a draft value index (DVI) was introduced in 2015. The DVI allocates a numeric value to each individual player draft pick, with these values determined by the AFL using historic player compensation or wage and salary data. The AFL DVI plays an essential role in the operation of its player draft; however, other research has questioned the validity of such indexes. This paper aims to produce an alternative to the AFL DVI. The former index uses career compensation as the determinant of value, whereas we use other measures of player performance. First, various models were developed to predict on-field performance, such as games played (both in a recruit's career and season) after a draftee was selected for the first time by a team. This was then retrofitted to the pick used to select these draftees to create the new DVIs. Even though the predicted DVI followed an inverse monotonic function like the existing index, the decline in value for the DVI produced here was less steep, unlike the AFL's. This allowed us to conclude that players' salaries did not always strongly correlate to performance. The change in performance between players selected at different points in the draft did not vary as much as their wages. Though this scheme is applied to the AFL, the underlying concept could be directly exported to other player drafts.
Topics: Australia; Data Collection; Athletic Performance; Team Sports
PubMed: 37788286
DOI: 10.1371/journal.pone.0292395 -
Journal of Biomedical Informatics Sep 2023Adverse outcome pathway (AOP) networks are versatile tools in toxicology and risk assessment that capture and visualize mechanisms driving toxicity originating from...
Optimization of an adverse outcome pathway network on chemical-induced cholestasis using an artificial intelligence-assisted data collection and confidence level quantification approach.
BACKGROUND
Adverse outcome pathway (AOP) networks are versatile tools in toxicology and risk assessment that capture and visualize mechanisms driving toxicity originating from various data sources. They share a common structure consisting of a set of molecular initiating events and key events, connected by key event relationships, leading to the actual adverse outcome. AOP networks are to be considered living documents that should be frequently updated by feeding in new data. Such iterative optimization exercises are typically done manually, which not only is a time-consuming effort, but also bears the risk of overlooking critical data. The present study introduces a novel approach for AOP network optimization of a previously published AOP network on chemical-induced cholestasis using artificial intelligence to facilitate automated data collection followed by subsequent quantitative confidence assessment of molecular initiating events, key events, and key event relationships.
METHODS
Artificial intelligence-assisted data collection was performed by means of the free web platform Sysrev. Confidence levels of the tailored Bradford-Hill criteria were quantified for the purpose of weight-of-evidence assessment of the optimized AOP network. Scores were calculated for biological plausibility, empirical evidence, and essentiality, and were integrated into a total key event relationship confidence value. The optimized AOP network was visualized using Cytoscape with the node size representing the incidence of the key event and the edge size indicating the total confidence in the key event relationship.
RESULTS
This resulted in the identification of 38 and 135 unique key events and key event relationships, respectively. Transporter changes was the key event with the highest incidence, and formed the most confident key event relationship with the adverse outcome, cholestasis. Other important key events present in the AOP network include: nuclear receptor changes, intracellular bile acid accumulation, bile acid synthesis changes, oxidative stress, inflammation and apoptosis.
CONCLUSIONS
This process led to the creation of an extensively informative AOP network focused on chemical-induced cholestasis. This optimized AOP network may serve as a mechanistic compass for the development of a battery of in vitro assays to reliably predict chemical-induced cholestatic injury.
Topics: Humans; Adverse Outcome Pathways; Artificial Intelligence; Cholestasis; Risk Assessment; Data Collection
PubMed: 37541407
DOI: 10.1016/j.jbi.2023.104465 -
Circulation Sep 2023The evolution of the electronic health record, combined with advances in data curation and analytic technologies, increasingly enables data sharing and harmonization.... (Review)
Review
The evolution of the electronic health record, combined with advances in data curation and analytic technologies, increasingly enables data sharing and harmonization. Advances in the analysis of health-related and health-proxy information have already accelerated research discoveries and improved patient care. This American Heart Association policy statement discusses how broad data sharing can be an enabling driver of progress by providing data to develop, test, and benchmark innovative methods, scalable insights, and potential new paradigms for data storage and workflow. Along with these advances come concerns about the sensitive nature of some health data, equity considerations about the involvement of historically excluded communities, and the complex intersection of laws attempting to govern behavior. Data-sharing principles are therefore necessary across a wide swath of entities, including parties who collect health information, funders, researchers, patients, legislatures, commercial companies, and regulatory departments and agencies. This policy statement outlines some of the key equity and legal background relevant to health data sharing and responsible management. It then articulates principles that will guide the American Heart Association's engagement in public policy related to data collection, sharing, and use to continue to inform its work across the research enterprise, as well as specific examples of how these principles might be applied in the policy landscape. The goal of these principles is to improve policy to support the use or reuse of health information in ways that are respectful of patients and research participants, equitable in impact in terms of both risks and potential benefits, and beneficial across broad and demographically diverse communities in the United States.
Topics: Humans; United States; American Heart Association; Information Dissemination; Data Collection
PubMed: 37646159
DOI: 10.1161/CIR.0000000000001173 -
Orphanet Journal of Rare Diseases Jul 2023Rare diseases (RDs) affect approximately 8% of all people or > 400 million people globally. The Australian Government's National Strategic Action Plan for Rare... (Review)
Review
BACKGROUND
Rare diseases (RDs) affect approximately 8% of all people or > 400 million people globally. The Australian Government's National Strategic Action Plan for Rare Diseases has identified the need for a national, coordinated, and systematic approach to the collection and use of RD data, including registries. Rare disease registries (RDRs) are established for epidemiological, quality improvement and research purposes, and they are critical infrastructure for clinical trials. The aim of this scoping review was to review literature on the current state of RDRs in Australia; to describe how they are funded; what data they collect; and their impact on patient outcomes.
METHODS
We conducted a literature search on MEDLINE, EMBASE, CINAHL and PsychINFO databases, in addition to Google Scholar and grey literature. Dissertations, government reports, randomised control trials, conference proceedings, conference posters and meeting abstracts were also included. Articles were excluded if they did not discuss RDs or if they were written in a language other than English. Studies were assessed on demographic and clinical patient characteristics, procedure or treatment type and health-related quality of life captured by RDRs or databases that have been established to date.
RESULTS
Seventy-four RDRs were identified; 19 were global registries in which Australians participated, 24 were Australian-only registries, 10 were Australia and New Zealand based, and five were Australian jurisdiction-based registries. Sixteen "umbrella" registries collected data on several different conditions, which included some RDs, and thirteen RDRs stored rare cancer-specific information. Most RDRs and databases captured similar types of information related to patient characteristics, comorbidities and other clinical features, procedure or treatment type and health-related quality of life measures. We found considerable heterogeneity among existing RDRs in Australia, especially with regards to data collection, scope and quality of registries, suggesting a national coordinated approach to RDRs is required.
CONCLUSION
This scoping review highlights the current state of Australian RDRs, identifying several important gaps and opportunities for improvement through national coordination and increased investment.
Topics: Humans; Rare Diseases; Quality of Life; Australia; Registries; Data Collection
PubMed: 37501152
DOI: 10.1186/s13023-023-02823-1 -
Revista Brasileira de Enfermagem 2023to reflect on the impacts of the General Personal Data Protection Law on Nursing practice.
OBJECTIVES
to reflect on the impacts of the General Personal Data Protection Law on Nursing practice.
METHODS
reflection article, through the intentional collection of materials relating to the topic.
RESULTS
legislation regulates confidentiality, processing and data sharing, requiring institutional protection measures. The nursing team is responsible for acting preventively, both in care and in the management role, in order to avoid the misuse of the patient's personal data. The law allows academic research to be carried out as long as the purpose is clear, data collection occurs with an explicit purpose and data is anonymized.
FINAL CONSIDERATIONS
although the General Personal Data Protection Law requires greater care in relation to data processing, it is established on precepts of good faith and respect for the rights of the individual, concepts aligned with the nursing code of ethics.
Topics: Humans; Confidentiality; Data Collection; Computer Security
PubMed: 38055531
DOI: 10.1590/0034-7167-2023-0126 -
Systematic Reviews Jul 2023Case study research is generating interest to evaluate complex interventions. However, it is not clear how this is being utilized by occupational therapists or how... (Review)
Review
BACKGROUND
Case study research is generating interest to evaluate complex interventions. However, it is not clear how this is being utilized by occupational therapists or how feasible it is to contribute to the evidence base. This scoping review explores case study research within occupational therapy in terms of how it is defined, the methodological characteristics adopted, such as data collection and analysis, and the range of practice contexts in which it is applied. We consider the viability of case study research for contributing to our evidence base.
METHODS
Opinion, text and empirical studies within an occupational therapy practice context were included. A three-step extensive search following Joanna Briggs Institute methodology was conducted in June 2020 and updated in July 2021 across ten databases, websites, peer-reviewed and grey literature from 2016 onwards. Study selection was completed by two independent reviewers. A data extraction table was developed and piloted and data charted to align with research questions. Data extraction was completed by one reviewer and a 10% sample cross checked by another.
RESULTS
Eighty-eight studies were included in the review consisting of (n = 84) empirical case study and (n = 4) non-empirical papers. Case study research has been conducted globally, with a range of populations across different settings. The majority were conducted in a community setting (n = 48/84; 57%) with populations experiencing neurodevelopmental disorder (n = 32/84; 38%), stroke (n = 14/84;17%) and non-diagnosis specific (n = 13/84; 15%). Methodologies adopted quantitative (n = 42/84; 50%), mixed methods (n = 22/84; 26%) and qualitative designs (n = 20/84; 24%). However, identifying the methodology and 'case' was a challenge due to methodological inconsistencies.
CONCLUSIONS
Case study research is useful when large-scale inquiry is not appropriate; for cases of complexity, early intervention efficacy, theory testing or when small participant numbers are available. It appears a viable methodology to contribute to the evidence base for occupation and health as it has been used to evaluate interventions across a breadth of occupational therapy practice contexts. Viability could be enhanced through consistent conduct and reporting to allow pooling of case data. A conceptual model and description of case study research in occupational therapy is proposed to support this.
SYSTEMATIC REVIEW REGISTRATION
Open Science Framework 10.17605/OSF.IO/PCFJ6.
Topics: Humans; Qualitative Research; Occupational Therapy; Data Collection
PubMed: 37525266
DOI: 10.1186/s13643-023-02292-4 -
Health Expectations : An International... Dec 2023Co-production is gaining increasing recognition as a good way of facilitating collaboration among different stakeholders, including members of the public. However, it...
INTRODUCTION
Co-production is gaining increasing recognition as a good way of facilitating collaboration among different stakeholders, including members of the public. However, it remains an ambiguous concept as there is no definitive or universal model of co-production or clarity on what constitutes a good co-production approach. This paper draws on the reflections of the academic researchers, practitioners and public advisors involved in co-producing a priority-setting exercise. The exercise was conducted by the Primary and Community Health Services (PCHS) Theme of the National Institute for Health and Care Research Applied Research Collaboration for Kent, Surrey and Sussex (NIHR ARC KSS).
METHODS
We collected data through written and verbal reflections from seven collaborators involved in the PCHS priority-setting exercise. We used Gibbs' model of reflection to guide the data collection. We then analysed the data through an inductive, reflexive thematic analysis.
RESULTS
A common thread through our reflections was the concept of 'sharing'. Although co-production is inherently shared, we used the virtuous cycle to illustrate a sequence of sharing concepts during the research cycle, which provides the underpinnings of positive co-production outcomes. We identified six themes to denote the iterative process of a shared approach within the virtuous cycle: shared values, shared understanding, shared power, shared responsibilities, shared ownership and positive outcomes.
CONCLUSION
Our results present a virtuous cycle of co-production, which furthers the conceptual underpinnings of co-production. Through our reflections, we propose that positive co-production outcomes require foundations of shared values and a shared understanding of co-production as a concept. These foundations facilitate a process of shared power, shared responsibilities and shared ownership. We argue that when these elements are present in a co-production exercise, there is a greater potential for implementable outcomes in the communities in which the research serves and the empowerment of collaborators involved in the co-production process.
PUBLIC MEMBERS' CONTRIBUTIONS
Three members of the public who are public advisors in the NIHR ARC KSS were involved in the priority-setting exercise that informed this paper. The public advisors were involved in the design of the priority-setting exercise and supported participants' recruitment. They also co-facilitated the focus groups during data collection and were involved in the data analysis, interpretation and preparation of the priority-setting report. For this current manuscript, two of them are co-authors. They provided reflections and contributed to the writing and reviewing of this manuscript.
Topics: Humans; Empowerment; Focus Groups; Exercise
PubMed: 37602918
DOI: 10.1111/hex.13851