RESUMO
BACKGROUND: Juvenile idiopathic arthritis (JIA) refers to a heterogeneous group of rheumatic conditions in children. Novel drugs have greatly improved disease outcomes; however, outcomes are impacted by limited awareness of the importance of early diagnosis and adequate treatment, and by differences in access across health systems. As a result, patients with JIA continue to be at risk for short- and long-term morbidity, as well as impacts on virtually all aspects of life of the child and family. MAIN BODY: Literature on the socioeconomic burden of JIA is largely focused on healthcare costs, and the impact of JIA on patients, families, and communities is not well understood. High quality evidence on the impact of JIA is needed to ensure that patients are receiving necessary support, timely diagnostics, and adequate treatment, and to inform decision making and resource allocation. This commentary introduces the European Joint Programme on Rare Diseases: Producing an Arthritis Value Framework with Economic Evidence: Paving the Way for Rare Childhood Diseases (PAVE) project, which will co-develop a patient-informed value framework to measure the impact of JIA on individuals and on society. With a patient-centered approach, fundamental to PAVE is the involvement of three patient advocacy organizations from Canada, Israel, and Europe, as active research partners co-designing all project phases and ensuring robust patient and family engagement. The framework will build on the findings of projects from six countries: Canada, Germany, Switzerland, Spain, Israel, and Belgium, exploring costs, outcomes (health, well-being), and unmet needs (uveitis, mental health, equity). CONCLUSION: This unique international collaboration will combine evidence on costs (from family to societal), outcomes (clinical, patient and family outcomes), and unmet needs, to co-design and build a framework with patients and families to capture the full impact of JIA. The framework will support the development of high-quality evidence, encompassing economic and clinical considerations, unmet needs, and patient perspectives, to inform equitable resource allocation, health system planning, and quality of care better aligned with the needs of children with JIA, their families, and communities. Knowledge gained from this novel approach may pave the way forward to be applied more broadly to other rare childhood diseases.
Assuntos
Artrite Juvenil , Assistência Centrada no Paciente , Doenças Raras , Humanos , Artrite Juvenil/economia , Assistência Centrada no Paciente/economia , Criança , Doenças Raras/economia , Efeitos Psicossociais da Doença , Europa (Continente)RESUMO
OBJECTIVES: Juvenile idiopathic arthritis (JIA) is the most common type of arthritis among children. It can cause joint pain and permanent physical damage, which affects mobility and daily activities. The EQ-5D-Y-3L self-report version has been validated in JIA, but the validity of EQ-5D-Y-5L remains unknown. We examined the psychometric properties of the EQ-5D-Y-5L parent-proxy version among children with JIA. METHODS: We used data from the Understanding Childhood Arthritis Network Canadian-Dutch collaboration study cohort, including patients with new-onset JIA, and those starting or stopping biologics. Clinical data and the parent-proxy version of the childhood health assessment questionnaire (CHAQ) and EQ-5D-Y-5L were collected. We evaluated the ceiling and floor effect; convergent and divergent validity using Spearman's rank correlation; known-group validity using one-way ANOVA (Analysis of Variance) and effect size; and informativity using Shannon's evenness index. RESULTS: 467 patient visits representing 407 patients were analyzed. The EQ-5D-Y-5L had no ceiling/floor effect. The EQ-5D-Y-5L showed good convergent (e.g., EQ-5D-Y-5L pain/discomfort dimension vs. CHAQ pain index (Spearman's r = 0.74, 95% confidence interval (C.I.): 0.69-0.79)), divergent (e.g., EQ-5D-Y-5L pain/discomfort dimension vs. CHAQ eating dimension (Spearman's r = 0.19, 95% C.I.: 0.09-0.29)) and known-group validity (e.g., mean EQ-5D-Y-5L level summary score for patients with inactive versus active disease status, 6.34 vs. 10.52 (p < 0.001, effect size = 1.20 (95% C.I.: 0.95-1.45)). Shannon's evenness index ranged from 0.52 to 0.88, suggesting most dimensions had relatively even distributions. CONCLUSIONS: In this patient sample, EQ-5D-Y-5L parent-proxy version exhibited construct validity and informativity, suggesting the EQ-5D-Y-5L can be used to measure the quality of life of children with JIA.
Juvenile idiopathic arthritis is the most common type of arthritis affecting children. It can cause pain and permanent physical damage to joints and affects mobility and daily activities. While there is no cure yet, new therapies like biologics are effective. However, biologics are expensive and can have side effects. To decide when is the best time to use these biologics, we need to understand their cost and impact on patients. EQ-5D-Y-5L is a common tool to measure how the disease affects a patient's life. It is unclear whether EQ-5D-Y-5L works well for patients with juvenile idiopathic arthritis. In this study, we compared the EQ-5D-Y-5L to another tool that measures how the illness impacts functional ability. We looked to see if the EQ-5D-Y-5L could tell the difference between children who were more or less sick. We also assessed whether the EQ-5D-Y-5L has the ability to describe patients with different severity in health status. This study indicates that the EQ-5D-Y-5L is a good tool to measure the health of patients with juvenile idiopathic arthritis. Findings from this study support the use of the EQ-5D-Y-5L among this patient population in future clinical trials and research studies.
RESUMO
There was an error in our original publication [...].
RESUMO
BACKGROUND AND OBJECTIVES: Rare diseases have a significant impact on patients, families, the health system, and society. Measuring the socioeconomic burden is crucial to valuing interventions for rare diseases. Healthcare system costs are significant, but so are costs to other government sectors, patients, families, and society. To understand the breadth of costs captured in rare disease studies, we examined the cost categories and elements of socioeconomic burden captured in published studies. METHODS: A scoping review was conducted using five electronic databases to identify English language economic evaluations and cost-of-illness studies of interventions for rare diseases (2011-21). We mapped costs using a previously developed evidence-informed framework of socioeconomic burden costs for rare disease. RESULTS: Of 4890 studies identified, 48 economic evaluations and 22 cost-of-illness studies were included. While 18/22 cost-of-illness studies utilized a societal perspective, only 7/48 economic evaluations incorporated societal costs. Most reported cost categories related to medical costs, with medication and hospitalizations being the most common elements for both study designs. Costs borne by patients, families, and society were reported less among economic evaluations than cost-of-illness studies. These included: productivity (10% vs 77%), travel/accommodation (6% vs 68%), government benefits (4% vs 18%), and family impacts (0% vs 50%). CONCLUSIONS: Contrary to cost-of-illness analyses, most of the included economic evaluations did not account for the hidden burden of rare diseases, that is, costs borne by patients, families, and societies. Including these types of costs in future studies would provide a more comprehensive picture of the burden of disease, providing empirical data to inform how we value and make decisions regarding rare disease interventions, health policy, and resource allocation.
Assuntos
Custos de Cuidados de Saúde , Doenças Raras , Humanos , Análise Custo-Benefício , Doenças Raras/terapia , Atenção à Saúde , Fatores SocioeconômicosRESUMO
BACKGROUND: As Canada and other high-income countries continue to welcome newcomers, we aimed to 1) understand newcomer parents' attitudes towards routine-childhood vaccinations (RCVs), and 2) identify barriers newcomer parents face when accessing RCVs in Alberta, Canada. METHODS: Between July 6th-August 31st, 2022, we recruited participants from Alberta, Canada to participate in moderated focus group discussions. Inclusion criteria included parents who had lived in Canada for < 5 years with children < 18 years old. Focus groups were transcribed verbatim and analyzed using content and deductive thematic analysis. The capability opportunity motivation behaviour model was used as our conceptual framework. RESULTS: Four virtual and three in-person focus groups were conducted with 47 participants. Overall, parents were motivated and willing to vaccinate their children but experienced several barriers related to their capability and opportunity to access RCVs. Five main themes emerged: 1) lack of reputable information about RCVs, 2) language barriers when looking for information and asking questions about RCVs, 3) lack of access to a primary care provider (PCP), 4) lack of affordable and convenient transportation options, and 5) due to the COVID-19 pandemic, lack of available vaccine appointments. Several minor themes were also identified and included barriers such as lack of 1) childcare, vaccine record sharing, PCP follow-up. CONCLUSIONS: Our findings highlight that several barriers faced by newcomer families ultimately stem from issues related to accessing information about RCVs and the challenges families face once at vaccination clinics, highlighting opportunities for health systems to better support newcomers in accessing RCVs.
Assuntos
COVID-19 , Pandemias , Humanos , Criança , Adolescente , Alberta , Cuidado da Criança , VacinaçãoRESUMO
OBJECTIVES: Care for JIA patients has been transformed in the biologics era; however, biologics carry important (although rare) risks and are costly. Flares after biological withdrawal are seen frequently, yet there is little clinical guidance to identify which patients in clinical remission can safely have their biologic discontinued (by stopping or tapering). We examined what characteristics of the child or their context are important to pediatric rheumatologists when making the decision to discuss withdrawal of biologics. METHODS: We conducted a survey including a best-worst scaling (BWS) exercise in pediatric rheumatologists who are part of the UCAN CAN-DU network to assess the relative importance of 14 previously identified characteristics. A balanced incomplete block design was used to generate choice tasks. Respondents evaluated 14 choice sets of 5 characteristics of a child with JIA and identified for each set which was the most and least important in the decision to offer withdrawal. Results were analyzed using conditional logit regression. RESULTS: Fifty-one (out of 79) pediatric rheumatologists participated (response rate 65%). The three most important characteristics were how challenging it was to achieve remission, history of established joint damage, and time spent in remission. The three least important characteristics were history of temporomandibular joint involvement, accessibility of biologics, and the patient's age. CONCLUSIONS: These findings give quantitative insight about factors important to pediatric rheumatologists' decision-making about biologic withdrawal. In addition to high quality clinical evidence, further research is needed to understand the perspective of patients and families to inform shared decision-making about biologic withdrawal for JIA patients with clinically inactive disease. Key Points â What is already known on this topic-there is limited clinical guidance for pediatric rheumatologists in making decisions about biologic withdrawal for patients with juvenile idiopathic arthritis who are in clinical remission. â What this study adds-this study quantitatively examined what characteristic of the child in clinical remission, or of their context, are most important to pediatric rheumatologists in deciding whether to offer withdrawal of biologics. â How this study might affect research, practice or policy-understanding of these characteristics can provide useful information to other pediatric rheumatologists in making their decisions, and may guide areas to focus on for future research.
Assuntos
Antirreumáticos , Artrite Juvenil , Produtos Biológicos , Humanos , Criança , Artrite Juvenil/tratamento farmacológico , Antirreumáticos/uso terapêutico , Reumatologistas , Inquéritos e Questionários , Produtos Biológicos/uso terapêuticoRESUMO
BACKGROUND AND OBJECTIVE: Rare diseases place a significant burden on patients, families, the healthcare system, and society. Evidence on the socioeconomic burden of rare disease is limited and mostly reflects diseases where treatments are available. We developed a framework encompassing recommended cost elements for studies of the socioeconomic burden of rare diseases. METHODS: A scoping review, conducted in five databases (Cochrane Library, EconLit, Embase, MEDLINE, and APA PsycINFO), identified English language publications from 2000 to 2021 presenting frameworks developed for determining, measuring or valuing costs for rare or chronic diseases. Cost elements were extracted and used to develop a literature-informed framework. Structured feedback was gathered from experts in rare diseases, health economics/health services, and policy research to revise the framework. RESULTS: Of 2990 records identified, eight papers were included and informed our preliminary framework; three focused on rare disease and five on chronic disease. Following expert input, we developed a framework consisting of nine cost categories (inpatient, outpatient, community, healthcare products/goods, productivity/education, travel/accommodation, government benefits, family impacts, and other), with several cost elements within each category. Our framework includes unique costs, added from the expert feedback, including genetic testing to inform treatment, use of private laboratories or out-of-country testing, family involvement in foundations and organizations, and advocacy costs for special access programs. CONCLUSIONS: Our work is the first to identify a comprehensive list of cost elements for rare disease for use by researchers and policy makers to fully capture socioeconomic burden. Use of the framework will increase the quality and comparability of future studies. Future work should focus on measuring and valuing these costs through onset, diagnosis, and post-diagnosis.
Assuntos
Atenção à Saúde , Doenças Raras , Humanos , Doenças Raras/terapia , Doença Crônica , Fatores SocioeconômicosRESUMO
OBJECTIVE: To determine whether and how often the information to measure a set of key performance indicators (KPIs) in juvenile idiopathic arthritis (JIA) is found in data collected routinely in a Pediatric Rheumatology Clinic. METHODS: A retrospective electronic chart review and administrative data analysis was conducted for a cohort of 140 patients with JIA at a tertiary Pediatric Rheumatology Clinic between 2016-2020. The set of KPIs include measuring patient outcomes (joint assessment, physician's global assessment of disease activity, assessment of functional ability, composite disease activity measurement), access to care (waiting time between referral and first visit, visit with the rheumatologist within the first year of diagnosis, annual follow-up visits with the rheumatologist), and safety (tuberculosis screening, and laboratory monitoring). Documentation was assessed as a binary variable indicating whether the required information was ever found. Documentation frequency for each KPI was assessed with counts and percentages of the number of times the required information was documented for each clinic visit. Compliance with the safety KPI definitions was assessed using administrative databases. RESULTS: Data for each KPI were found at least once in the cohort and documentation varied in frequency and consistency. Access to care and safety KPIs were documented more frequently than patient outcome KPIs. A joint assessment was documented at every visit for 95% of patients, 46% for an assessment of pain, and none for a physician's global assessment of disease activity, an assessment of functional ability, or a composite disease activity measurement. CONCLUSION: Although feasible to measure, there is an opportunity for improving the consistency of documentation. Having an active system of monitoring KPIs and tools to simplify measurement is a key step in the process toward improved patient care outcomes. Streamlining the collection of KPI data can increase the likelihood of compliance. Next steps should involve replicating this study in various centres.
Assuntos
Artrite Juvenil , Criança , Humanos , Artrite Juvenil/diagnóstico , Artrite Juvenil/terapia , Estudos Retrospectivos , Dor , Encaminhamento e Consulta , ReumatologistasRESUMO
BACKGROUND: Juvenile idiopathic arthritis (JIA) is a childhood autoimmune disease that causes swelling and pain in at least one joint. Young people with JIA experience symptoms that persist into adulthood, and thus will undergo a transition including the o transfer of care from a pediatric rheumatologist an adult rheumatologist. Missing from the literature is research that centres the transition experience of young people with JIA in Canada. This goal of this patient-led research was to explore the experience young people with JIA through the process of transition. METHODS: Qualitative study using the Patient and Community Engaged Research (PaCER) approach. Trained patient-researchers conducted three focus groups using the Set, Collect and Reflect PaCER process. Participants, recruited via purposive and snowball sampling using research/personal networks and social media, were young people with JIA in Canada between 18 and 28 years who had experienced with the process of transition to adult care. Recordings were transcribed verbatim. Patient researchers individually coded overlapping sections of the data, and thematic analysis was conducted. RESULTS: In total, nine individuals participated in one or more focus groups. Three themes were identified, with sub-themes: preparedness for transition (readiness for the transfer of care, developing self-advocacy skills), continuity and breadth of care (changing relationships, culture shock, new responsibilities), need for support (social support, mental health support, and ongoing support needs - beyond the transfer of care. Peer support was a connecting concept in the support sub-themes. Transition was more than a change in primary physician but also a change in the care model and breadth of care provided, which was challenging for young people especially if they had insufficient information. CONCLUSIONS: Transition from pediatric to adult care in rheumatology is a significant period for young people living with JIA, and this patient-led study provided insight into the experience from the perspective of young people with JIA which is critical to informing the development of supports for patients through the process. Patients, caregivers, pediatric and adult rheumatologists and members of the multi-disciplinary care team need to collaborate in terms of resources preparing for transfer, and support throughout the transition process to ensure a successful transition process.
RESUMO
BACKGROUND: Few studies have examined pediatric rheumatologists' approaches to treatment decision making for biologic therapy for patients with juvenile idiopathic arthritis (JIA). This study presents the qualitative research undertaken to support the development of a Best-Worst Scaling (BWS) survey for tapering in JIA. The study objectives were to (1) describe the treatment decision-making process of pediatric rheumatologists to initiate and taper biologics; and (2) select attributes for a BWS survey. METHODS: Pediatric rheumatologists across Canada were recruited to participate in interviews using purposeful sampling. Interviews were conducted until saturation was achieved. Interview recordings were transcribed verbatim and transcripts were analyzed using deductive thematic analysis. Initial codes were organized into themes and subthemes using an iterative process. Attributes for the BWS survey were developed from these themes and a literature review was conducted in parallel to inform survey development. Further refinement of the attributes was done through consultation with the research team. RESULTS: Five pediatric rheumatologists participated in the interviews. Shared decision making was part of the approach to initiating and tapering biologics in their practice. Tapering approaches differed; some pediatric rheumatologists preferred to stop biologics immediately, while others tapered by reducing dose and/or increasing the dose interval over time. A total of 14 attributes were developed for the BWS. Thirteen attributes were selected from the themes that emerged from the qualitative interviews and one attribute was included after review with the research team. Attributes related to patient characteristics included JIA subtype, time in remission, history or presence of joint damage or erosive disease, how challenging it was to achieve remission, and history of flares. Contextual attributes included accessibility of biologics and willingness to taper biologics. CONCLUSION: This study contributes to the limited literature on pediatric rheumatologists' approaches to treatment decision making for biologics in JIA and identifies attributes that affect the decision to both initiate and taper. Further research is planned to implement the BWS survey to understand the importance of the attributes identified. Additional investigation is required to determine if these characteristics align with patient and parent preferences.
Assuntos
Artrite Juvenil , Produtos Biológicos , Artrite Juvenil/tratamento farmacológico , Produtos Biológicos/uso terapêutico , Criança , Humanos , Pesquisa Qualitativa , Reumatologistas , Inquéritos e QuestionáriosRESUMO
BACKGROUND: Approximately half of patients with hip and knee osteoarthritis have tried non-surgical management before surgical consultation. Understanding the many factors affecting the uptake of recommended strategies is important to inform future development of such management strategies. OBJECTIVES: The aim of this study was to explore and identify factors that patients with osteoarthritis consider when choosing non-surgical management for hip and knee osteoarthritis, as formative research for a study of patient preferences for non-surgical management programs for osteoarthritis. METHODS: A qualitative research design was used. Participants were recruited using a combination of stratified and convenience sampling. Interviews were conducted, using a semi-structured interview guide, with English-speaking patients who had self-reported hip and/or knee osteoarthritis and at least one joint that had not undergone replacement surgery. Data were thematically analyzed. RESULTS: Thirteen patients participated in these interviews. Sixteen factors that participants considered when choosing non-surgical osteoarthritis management were identified. Eleven were extrinsic, relating to features of programs and services, and are categorized as types of interventions, general program and service details, and program-specific details. Five were intrinsic to the individual and influenced how decisions for osteoarthritis management were approached and the options available to choose from. Three novel factors included participants' desire for further management, their views about joint replacement surgery, and whether they felt personal choice was available in osteoarthritis management strategies. CONCLUSION: Key factors were identified that patients considered when making decisions about non-surgical management for their osteoarthritis that will be used to inform a discrete choice experiment (DCE) that aims to measure preferences for these factors.
Assuntos
Osteoartrite do Quadril , Osteoartrite do Joelho , Tomada de Decisões , Humanos , Osteoartrite do Quadril/cirurgia , Osteoartrite do Joelho/cirurgia , Preferência do Paciente , Pesquisa QualitativaRESUMO
BACKGROUND: Osteoarthritis management aims to reduce pain and improve function. Many factors affect whether patients follow recommended strategies. Locus of control refers to individual beliefs around who and what influences health. Locus of control is related to the treatment strategies patients prefer. Currently, no studies explore locus of control in non-surgical management of osteoarthritis. OBJECTIVES: To explore patients' beliefs about the influences on their osteoarthritis symptoms and disease progression. METHODS: Semi-structured interviews were conducted with individuals experiencing self-reported hip and/or knee osteoarthritis who had at least one joint that had not undergone replacement surgery. We used a qualitative description approach and the Braun and Clarke method for thematic analysis. Participants' locus of control classifications-internal, chance, doctors, or other people-were based on the Multidimensional Health Locus of Control (MHLC) Scales Form C score. RESULTS: Locus of control was discussed in relation to aetiology, progression, and symptoms. Participants' opinions varied on whether their osteoarthritis progression could be influenced. 46% of participants attributed control to other people. Most participants believed that a previous injury had caused their osteoarthritis and that both themselves and others had some influence over their osteoarthritis symptoms, regardless of their locus of control classification. CONCLUSION: This research highlights the need for education about: the aetiology of osteoarthritis, the link between management and progression, and patient management of osteoarthritis. Further research is required to discern why expected patterns were not observed between participants' beliefs and locus of control classifications.
Assuntos
Osteoartrite do Quadril , Osteoartrite do Joelho , Humanos , Osteoartrite do Quadril/cirurgia , Osteoartrite do Joelho/terapiaRESUMO
BACKGROUND: After a national policy change in 2013 disallowing body checking in Pee Wee ice hockey games, the rate of injury was reduced by 50% in Alberta. However, the effect on associated health care costs has not been examined previously. HYPOTHESIS: A national policy removing body checking in Pee Wee (ages 11-12 years) ice hockey games will reduce injury rates, as well as costs. STUDY DESIGN: Cost-effectiveness analysis alongside cohort study. LEVEL OF EVIDENCE: Level 3. METHODS: A cost-effectiveness analysis was conducted alongside a cohort study comparing rates of game injuries in Pee Wee hockey games in Alberta in a season when body checking was allowed (2011-2012) with a season when it was disallowed after a national policy change (2013-2014). The effectiveness measure was the rate of game injuries per 1000 player-hours. Costs were estimated based on associated health care use from both the publicly funded health care system and privately paid health care cost perspectives. Probabilistic sensitivity analysis was conducted using bootstrapping. RESULTS: Disallowing body checking significantly reduced the rate of game injuries (-2.21; 95% CI [-3.12, -1.31] injuries per 1000 player-hours). We found no statistically significant difference in public health care system (-$83; 95% CI [-$386, $220]) or private health care costs (-$70; 95% CI [-$198, $57]) per 1000 player-hours. The probability that the policy of disallowing body checking was dominant (with both fewer injuries and lower costs) from the perspective of the public health care system and privately paid health care was 78% and 92%, respectively. CONCLUSION: Given the significant reduction in injuries, combined with lower public health care system and private costs in the large majority of iterations in the probabilistic sensitivity analysis, our findings support the policy change disallowing body checking in ice hockey in 11- and 12-year-old ice hockey leagues.
Assuntos
Traumatismos em Atletas , Concussão Encefálica , Hóquei , Criança , Estudos de Coortes , Análise Custo-Benefício , Hóquei/lesões , Humanos , Incidência , Fatores de RiscoRESUMO
Sport-related injuries are the leading cause of injury in youth and are costly to the healthcare system. When body checking is disallowed in non-elite levels of Bantam (ages 13-14 years) ice hockey, the injury rate is reduced, but the impact on costs is unknown. This study compared rates of game injuries and costs among non-elite Bantam ice hockey leagues that disallow body checking to those that did not. Methods: An economic evaluation was conducted alongside a prospective cohort study comparing 608 players from leagues where body checking was allowed in games (Calgary/Edmonton 2014-2015, Edmonton 2015-2016) with 396 players from leagues where it was not allowed in games (Vancouver, Kelowna 2014-2015, Calgary in 2015-2016). The effectiveness measure was rate of game injuries per 1000 player-hours. Costs were estimated based on associated healthcare use within the publicly funded healthcare system as well as privately paid healthcare costs. Probabilistic sensitivity analysis was conducted using bootstrapping. Results: Disallowing body checking reduced the rate of injuries by 4.32 per 1000 player-hours (95% CI -6.92, -1.56) and reduced public and total healthcare system costs by $1556 (95% CI -$2478, -$559) and $1577 (95% CI -$2629, -$500) per 1000 player-hours, respectively. These finding were robust in over 99% of iterations in sensitivity analyses in the public healthcare and the total healthcare system perspectives. There was no statistically significant difference in privately paid healthcare costs (-$65 per 1000 player-hours (95% CI -$220, $99)). Interpretation: Disallowing body checking in non-elite 13-14-year-old ice hockey nationally would prevent injuries and reduce public healthcare costs.
Assuntos
Traumatismos em Atletas , Concussão Encefálica , Hóquei , Adolescente , Traumatismos em Atletas/epidemiologia , Análise Custo-Benefício , Humanos , Incidência , Políticas , Estudos ProspectivosRESUMO
BACKGROUND: Discrete choice experiments (DCEs) are a common method used to describe and quantitatively assess preferences in health applications. Increasingly, DCEs have been used to elicit preferences from children and adolescents and generate evidence to inform policies affecting this population. OBJECTIVES: The aim of this review was to summarize and describe the application of DCEs conducted with children and adolescents and describe author-reported age-specific considerations in design, implementation, and analysis. METHODS: A scoping review was conducted using a 'pearl-growing' technique whereby the reference lists of existing systematic reviews of DCEs were used to identify potential studies conducted with children or adolescents as respondents published between 1990 and 2017. This list was supplemented with an updated electronic search using the same strategy as the initial reviews to identify studies from 2017 to 2020. RESULTS: Of 480 studies identified, 19 were included; topics included vaccines (32%), drugs/medical devices (26%), treatment or health promotion interventions/programs (21%), warning labels on cigarettes/nicotine products (10%), and preferences for physical activity and healthy food choices (10%). The youngest reported age for independent DCE completion was 8 years. Approaches to assessing validity and reliability of choices were consistent with best practices for the conduct of DCEs. Reported age-specific considerations included use of visual aids, age-appropriate language, reducing task complexity and cognitive burden, and exploration of interpretation of willingness-to-pay. CONCLUSION: The number of DCEs conducted with children and adolescents has increased in recent years. Detailed explanation of why reported age-specific considerations were necessary, how they could be used to interpret results, or to understand the appropriateness of this methodology for different age groups was limited. Despite a recognition of the need for special consideration when conducting DCEs in this population, the unique issues in the context of age-specific considerations are largely unexplored, and further research is required. Moving forward, stated preference research conducted with children and adolescents should report in more detail methods of recruitment, results of validity assessments, and provide specific reflection on the extent to which modeled results are consistent with expectations and underlying theory.
Assuntos
Preferência do Paciente , Vacinas , Adolescente , Criança , Comportamento de Escolha , Humanos , Reprodutibilidade dos Testes , Projetos de PesquisaRESUMO
PURPOSE: We provide a description of the diagnostic odyssey for a cohort of children seeking diagnosis of a rare genetic disorder in terms of the time from initial consultation to most recent visit or receipt of diagnosis, the number of tests per patient, and the types of tests received. METHODS: Retrospective chart review of 299 children seen at the Alberta Children's Hospital (ACH) Genetics Clinic (GC) for whom the result of at least one single-gene test, gene panel, or chromosome microarray analysis (CMA) was recorded. RESULTS: Of 299 patients, 90 (30%) received a diagnosis in the period of the review. Patients had an average of 5.4 tests each; 236 (79%) patients received CMA; 172 (58%) patients received single-gene tests and 34 (11%) received gene panels; 167 (56%) underwent imaging/electrical activity studies. The mean observation period was 898 days (95% confidence interval [CI] 791, 1004). Among patients with visits recorded prior to visiting ACH GC, 43% of the total observation time occurred prior to the GC. CONCLUSION: As genomic technologies expand, the nature of the diagnostic odyssey will change. This study has outlined the current standard of care in the ACH GC, providing a baseline against which future changes can be assessed.
Assuntos
Testes Genéticos , Genômica , Alberta , Criança , Humanos , Análise em Microsséries , Estudos RetrospectivosRESUMO
BACKGROUND: The evaluation of quality of care in juvenile idiopathic arthritis (JIA) is critical for advancing patient outcomes but is not currently part of routine care across all centers in Canada. The study objective is to review the current landscape of JIA quality measures and use expert panel consensus to define key performance indicators (KPIs) that are important and feasible to collect for routine monitoring in JIA care in Canada. METHODS: Thirty-seven candidate KPIs identified from a systematic review were reviewed for inclusion by a working group including 3 pediatric rheumatologists. A shortlist of 14 KPIs was then assessed using a 3-round modified Delphi panel based on the RAND/UCLA Appropriateness Method. Ten panelists across Canada participated based on their expertise in JIA, quality measurement, or lived experience as a parent of a child with JIA. During rounds 1 and 3, panelists rated each KPI on a 1-9 Likert scale on themes of importance, feasibility, and priority. In round 2, panelists participated in a moderated in-person discussion that resulted in minor modifications to some KPIs. KPIs with median scores of ≥ 7 on all 3 questions without disagreement were included in the framework. RESULTS: Ten KPIs met the criteria for inclusion after round 3. Five KPIs addressed patient assessments: pain, joint count, functional status, global assessment of disease activity, and the clinical Juvenile Arthritis Disease Activity Score (cJADAS). Three KPIs examined access to care: wait times for consultation, access to pediatric rheumatologists within 1 year of diagnosis, and frequency of clinical follow-up. Safety was addressed through KPIs on tuberculous screening and laboratory monitoring. KPIs examining functional status using the Childhood Health Assessment Questionnaire (CHAQ), quality of life, uveitis, and patient satisfaction were excluded due to concerns about feasibility of measurement. CONCLUSIONS: The proposed KPIs build upon existing KPIs and address important processes of care that should be measured to improve the quality of JIA care. The feasibility of capturing these measures will be tested in various data sources including the Understanding Childhood Arthritis Network (UCAN) studies. Subsequent work should focus on development of meaningful outcome KPIs to drive JIA quality improvement in Canada and beyond.
Assuntos
Artrite Juvenil/terapia , Melhoria de Qualidade/normas , Indicadores de Qualidade em Assistência à Saúde/normas , Qualidade de Vida , Canadá , Criança , Consenso , Técnica Delphi , Humanos , Melhoria de Qualidade/estatística & dados numéricos , Indicadores de Qualidade em Assistência à Saúde/estatística & dados numéricos , Revisões Sistemáticas como Assunto/métodos , Revisões Sistemáticas como Assunto/normasRESUMO
OBJECTIVES: Guideline compliant CPR is associated with improved survival for patients with cardiac arrest. Conventional Basic Life Support (BLS) training results in suboptimal CPR competency and skill retention. We aimed to compare the effectiveness of distributed CPR training with real-time feedback to conventional BLS training for CPR skills in pediatric healthcare providers. METHODS: Healthcare providers were randomized into receiving annual BLS training (control) or distributed training with real-time feedback (intervention). The intervention group was asked to practice CPR for 2â¯min on mannequins while receiving real-time CPR feedback, at least once per month. Control group participants were not asked to practice CPR during the study period. Excellent CPR was defined as 90% guideline-compliance for depth, rate and recoil of chest compressions. CPR performance of participants was assessed (on infant and adult-sized mannequins) every 3 months for a duration of 12 months. CPR performance was compared between the 2 groups. RESULTS: A total of 87 healthcare providers were included in the analyses (control nâ¯=â¯41, intervention nâ¯=â¯46). Baseline assessment showed no significant difference in CPR performance across the 2 groups. The intervention group has a significantly greater proportion of participants with excellent CPR compared with the control group on an adult sized mannequin (14.6% vs. 54.3%, pâ¯<â¯0.001) and infant-sized mannequin (19.5% vs. 71.7%, pâ¯<â¯0.001) at the end of the study. In the intervention group, all CPR metrics except infant depth were improved and retained over the course of the study. CONCLUSION: Distributed CPR training with real-time feedback improves the compliance of AHA guidelines of quality of CPR.
Assuntos
Reanimação Cardiopulmonar , Educação/métodos , Pessoal de Saúde/educação , Parada Cardíaca , Pediatria , Adulto , Reanimação Cardiopulmonar/educação , Reanimação Cardiopulmonar/métodos , Reanimação Cardiopulmonar/normas , Criança , Feminino , Feedback Formativo , Parada Cardíaca/mortalidade , Parada Cardíaca/terapia , Humanos , Masculino , Manequins , Pediatria/educação , Pediatria/métodos , Pediatria/normas , Competência Profissional , Melhoria de QualidadeRESUMO
CONTEXT: Simulation-based medical education (SBME) is now ubiquitous at all levels of medical training. Given the substantial resources needed for SBME, economic evaluation of simulation-based programmes or curricula is required to demonstrate whether improvement in trainee performance (knowledge, skills and attitudes) and health outcomes justifies the cost of investment. Current literature evaluating SBME fails to provide consistent and interpretable information on the relative costs and benefits of alternatives. CONTENT: Economic evaluation is widely applied in health care, but is relatively scarce in medical education. Therefore, in this paper, using a focus on SBME, we define economic evaluation, describe the key components, and discuss the challenges associated with conducting an economic evaluation of medical education interventions. As a way forward to the rigorous and state of the art application of economic evaluation in medical education, we outline the steps to gather the necessary information to conduct an economic evaluation of simulation-based education programmes and curricula, and describe the main approaches to conducting an economic evaluation. CONCLUSION: A properly conducted economic evaluation can help stakeholders (i.e., programme directors, policy makers and curriculum designers) to determine the optimal use of resources in selecting the modality or method of assessment in simulation. It also helps inform broader decision making about allocation of scarce resources within an educational programme, as well as between education and clinical care. Economic evaluation in medical education research is still in its infancy, and there is significant potential for state-of-the-art application of these methods in this area.
Assuntos
Competência Clínica/normas , Análise Custo-Benefício , Educação Médica/métodos , Treinamento por Simulação/métodos , Currículo , HumanosRESUMO
BACKGROUND: Discrete choice experiments are increasingly used to assess preferences for vaccines and vaccine service delivery. OBJECTIVES: To synthesize and critically assess the application of discrete choice experiments in childhood/adolescent vaccines, to describe how discrete choice experiments have been applied to understand preferences, and to evaluate the use of discrete choice experiment data to inform estimates of vaccine uptake. METHODS: We conducted a systematic review of six electronic databases. Included studies were discrete choice experiments and conjoint analyses published from 2000 to 2016 related to childhood/adolescent vaccines where respondents were parents, children/adolescents, or service providers. Validity assessment was used to assess study quality and risk of bias. RESULTS: In total, 27 articles were included, representing 21 different studies. A majority of articles were published between 2011 and 2016. Vaccines studied included human papillomavirus (24%), influenza (19%), meningococcal vaccines (14%), childhood vaccines (14%), hypothetical vaccines (10%), hepatitis B (5%), and diphtheria, tetanus, pertussis, hepatitis B, poliomyelitis, and Haemophilus influenzae type b (5%). Most studies assessed parent preferences (67%). The most common attributes were risk (24%), degree/duration of protection (21%), and cost (15%). Commonly reported outcome measures were estimates of uptake (33%), willingness-to-pay (22%), and other marginal rates of substitution (14%). Validity assessments yielded high scores overall. Areas of weakness included low response rates, inefficient experimental design, and failure to conduct formative qualitative work and a pilot of the discrete choice experiment. CONCLUSION: This is the first systematic review of childhood/adolescent vaccine-related discrete choice experiments. In future, special attention should be paid to ensuring that choice context and discrete choice experiment design are compatible to generate reliable estimates of uptake.