Validating Health Economic Models With the Probabilistic Analysis Check dashBOARD.

Objectives: Health economic (HE) models are often considered as "black boxes" because they are not publicly available and lack transparency, which prevents independent scrutiny of HE models. Additionally, validation efforts and validation status of HE models are not systematically reported. Methods to validate HE models in absence of their full underlying code are therefore urgently needed to improve health policy making.

Quantifying hospital-associated costs, and accompanying travel costs and productivity losses, before and after withdrawing TNF-α inhibitors in juvenile idiopathic arthritis.

Objective: To quantify differences in hospital-associated costs, and accompanying travel costs and productivity losses, before and after withdrawing TNF-α inhibitors (TNFi) in JIA patients.

Methods: This was a retrospective analysis of prospectively collected data from electronic medical records of paediatric JIA patients treated with TNFi, which were immediately discontinued, spaced (increased treatment interval) or tapered (reduced subsequent doses). Costs of hospital-associated resource use (consultations, medication, radiology procedures, laboratory testing, procedures under general anaesthesia, hospitalization) and associated travel costs and productivity losses were quantified during clinically inactive disease until TNFi withdrawal (pre-withdrawal period) and compared with costs during the first and second year after withdrawal initiation (first and second year post-withdrawal).

Managing juvenile idiopathic arthritis within the context of their life: What we learnt from children and youth living with juvenile idiopathic arthritis and their parents.

Introduction: Juvenile idiopathic arthritis (JIA) is the most common rheumatic disease in children and causes short- and long-term disability. Optimal management requires pharmacologic and non-pharmacologic interventions. Few studies have explored the youth and family experience of the management of JIA.

Withdrawing biologics in non-systemic JIA: what matters to pediatric rheumatologists?

Objective: Approximately one third of children with JIA receive biologic therapy, but evidence on biologic therapy withdrawal is lacking. This study aims to increase our understanding of whether and when pediatric rheumatologists postpone a decision to withdraw biologic therapy in children with clinically inactive non-systemic JIA.

Methods: A survey containing questions about background characteristics, treatment patterns, minimum treatment time with biologic therapy, and 16 different patient vignettes, was distributed among 83 pediatric rheumatologists in Canada and the Netherlands.

What matters most to pediatric rheumatologists in deciding whether to discontinue biologics in a child with juvenile idiopathic arthritis? A best-worst scaling survey.

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.

Two point-of-care test-based approaches for the exclusion of deep vein thrombosis in general practice: a cost-effectiveness analysis.

Background: In the diagnostic work-up of deep vein thrombosis (DVT), the use of point-of-care-test (POCT) D-dimer assays is emerging as a promising patient-friendly alternative to regular D-dimer assays, but their cost-effectiveness is unknown. We compared the cost-effectiveness of two POCT-based approaches to the most common, laboratory-based, situation.

Methods: A patient-level simulation model was developed to simulate the diagnostic trajectory of patients presenting with symptoms of DVT at the general practitioner (GP).

The societal impact of implementing an at-home blood sampling device for chronic care patients: patient preferences and cost impact.

Background: Diabetes mellitus, cardiovascular diseases, chronic kidney disease, and thyroid diseases are chronic diseases that require regular monitoring through blood tests. This paper first investigates the experiences of chronic care patients with venipuncture and their expectations of an at-home blood-sampling device, and then assesses the impact on societal costs of implementing such a device in current practice.

Methods: An online survey was distributed among chronic care patients to gain insight into their experience of blood sampling in current practice, and their expectations of an at-home blood-sampling device.

Factors associated with care- and health-related quality of life of caregivers of children with juvenile idiopathic arthritis.

Objective: This study investigates the relationship of child, caregiver, and caring context measurements with the care-related quality of life (CRQoL) and health-related quality of life (HRQoL) of caregivers of children with juvenile idiopathic arthritis (JIA).

Methods: We performed a cross-sectional analysis of baseline data on caregivers of children with JIA from Canada and the Netherlands collected for the "Canada-Netherlands Personalized Medicine Network in Childhood Arthritis and Rheumatic Diseases" study from June 2019 to September 2021. We used the CRQoL questionnaire (CarerQoL), adult EQ-5D-5L, and proxy-reported Youth 5-Level version of EuroQoL (EQ-5D-5L-Y) to assess caregiver CRQoL, caregiver HRQoL, and child HRQoL, respectively.

Real-world data reveals the complexity of disease modifying anti-rheumatic drug treatment patterns in juvenile idiopathic arthritis: an observational study.

Objective: Pharmacological treatment is a cornerstone of care for children with juvenile idiopathic arthritis (JIA). The objective of this study is to evaluate prescription patterns of conventional and biologic disease modifying anti-rheumatic drugs (c-DMARDs and b-DMARDs) for patients with JIA.

Methods: We conducted a retrospective cohort study of children diagnosed with JIA at a rheumatology pediatric clinic.

Perspectives of Pediatric Rheumatologists on Initiating and Tapering Biologics in Patients with Juvenile Idiopathic Arthritis: A Formative Qualitative Study.

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.

Monitoring of rheumatoid arthritis: a patient survey on disease insight and possible added value of an innovative inflammation monitoring device.

To enable patients with rheumatoid arthritis (RA) and their healthcare professionals to choose the optimal treatment, it is crucial to accurately assess the current state of inflammatory activity. The objectives of this study were to (1) investigate the perspective of RA patients on their insight into the current status of their disease, and to (2) investigate the patients' perspective on the possible added value of a monitoring device based on optical spectral transmission-called the HandScan-that measures the location and severity of joint inflammation. A survey was distributed online among patients with RA in the Netherlands.

Evaluation of Real-World Healthcare Resource Utilization and Associated Costs in Children with Juvenile Idiopathic Arthritis: A Canadian Retrospective Cohort Study.

Introduction: Juvenile idiopathic arthritis (JIA) is a chronic rheumatic disease, whose multifaceted care path can lead to significant expenditure for the healthcare system. We aim to assess the real-world healthcare resource use (HCRU) and associated cost for children with JIA in a single center in Canada.

Methods: A single-center consecutive cohort of newly diagnosed patients with JIA attending the pediatric rheumatology clinic from 2011 to 2019 was identified using an administrative data algorithm and electronic medical charts.

Costs of Hospital-Associated Care for Patients With Juvenile Idiopathic Arthritis in the Dutch Health Care System.

Objective: The aim of this study was to quantify costs of hospital-associated care for juvenile idiopathic arthritis (JIA), provide insights in patient-level variation in costs, and investigate costs over time from the moment of JIA diagnosis. Results were reported for all JIA patients in general and by subtype.  METHODS: This study was a single-center, retrospective analysis of prospective data from electronic medical records of children with JIA, ages 0-18 years, between April 1, 2011 and March 31, 2019.

Costs of medication use among patients with juvenile idiopathic arthritis in the Dutch healthcare system.

: This study aims to quantify medication costs in juvenile idiopathic arthritis (JIA), based on subtype.: This study is a single-center, retrospective analysis of prospective data from electronic medical records of JIA patients, aged 0-18 years between 1 April 2011 and 31 March 2019. Patient characteristics (age, gender, subtype) and medication use were extracted.

Efficacy of one-stage cartilage repair using allogeneic mesenchymal stromal cells and autologous chondron transplantation (IMPACT) compared to nonsurgical treatment for focal articular cartilage lesions of the knee: study protocol for a crossover randomized controlled trial.

Background: Articular cartilage defects in the knee have poor intrinsic healing capacity and may lead to functional disability and osteoarthritis (OA). "Instant MSC Product accompanying Autologous Chondron Transplantation" (IMPACT) combines rapidly isolated recycled autologous chondrons with allogeneic MSCs in a one-stage surgery. IMPACT was successfully executed in a first-in-man investigator-driven phase I/II clinical trial in 35 patients.

Preventing overuse of laboratory diagnostics: a case study into diagnosing anaemia in Dutch general practice.

Background: More information is often thought to improve medical decision-making, which may lead to test overuse. This study assesses which out of 15 laboratory tests contribute to diagnosing the underlying cause of anaemia by general practitioners (GPs) and determines a potentially more efficient subset of tests for setting the correct diagnosis.

Methods: Logistic regression was performed to determine the impact of individual tests on the (correct) diagnosis.

Health State Utility Values in Juvenile Idiopathic Arthritis: What is the Evidence?

Objectives: The objectives of this systematic review were to identify health state utility values (HSUV) of children and adults with juvenile idiopathic arthritis in the literature and to assess whether HSUV were appropriately reported and could be used to inform parameter inputs for a model-based cost-utility analysis to inform decision making.

Methods: MEDLINE, EMBASE, PsycINFO, EconLit and CINAHL databases were searched in July 2019. Inclusion criteria were studies using preference-based instruments, targeting children or adults with juvenile idiopathic arthritis, and in the English language.

The Value of Diagnostic Information in Personalised Healthcare: A Comprehensive Concept to Facilitate Bringing This Technology into Healthcare Systems.

Health systems around the world seek to address patients' unmet health needs for a range of acute and chronic diseases. Simultaneously, governments strive to keep healthcare spending sustainable, while providing equal access to high-quality care. This has fuelled debate around what constitutes a valuable healthcare intervention in a health system and the corollary consideration of what governments are willing to pay for a certain health intervention.

Seeking the state of the art in standardized measurement of health care resource use and costs in juvenile idiopathic arthritis: a scoping review.

Background: This study aims to describe current practice in identifying and measuring health care resource use and unit costs in economic evaluations or costing studies of juvenile idiopathic arthritis (JIA).

Methods: A scoping review was conducted (in July 2018) in PubMed and Embase to identify economic evaluations, costing studies, or resource utilization studies focusing on patients with JIA. Only English language peer-reviewed articles reporting primary research were included.

Understanding the adoption and use of point-of-care tests in Dutch general practices using multi-criteria decision analysis.

Background: The increasing number of available point-of-care (POC) tests challenges clinicians regarding decisions on which tests to use, how to efficiently use them, and how to interpret the results. Although POC tests may offer benefits in terms of low turn-around-time, improved patient's satisfaction, and health outcomes, only few are actually used in clinical practice. Therefore, this study aims to identify which criteria are, in general, important in the decision to implement a POC test, and to determine their weight.

Cost-effectiveness of procalcitonin testing to guide antibiotic treatment duration in critically ill patients: results from a randomised controlled multicentre trial in the Netherlands.

Background: Procalcitonin (PCT) testing can help in safely reducing antibiotic treatment duration in intensive care patients with sepsis. However, the cost-effectiveness of such PCT guidance is not yet known.

Methods: A trial-based analysis was performed to estimate the cost-effectiveness of PCT guidance compared with standard of care (without PCT guidance).

Toward Alignment in the Reporting of Economic Evaluations of Diagnostic Tests and Biomarkers: The AGREEDT Checklist.

Objectives: General frameworks for conducting and reporting health economic evaluations are available but not specific enough to cover the intricacies of the evaluation of diagnostic tests and biomarkers. Such evaluations are typically complex and model-based because tests primarily affect health outcomes indirectly and real-world data on health outcomes are often lacking. Moreover, not all aspects relevant to the evaluation of a diagnostic test may be known and explicitly considered for inclusion in the evaluation, leading to a loss of transparency and replicability.

Early health technology assessment of future clinical decision rule aided triage of patients presenting with acute chest pain in primary care.

The objective of the paper is to estimate the number of patients presenting with chest pain suspected of acute coronary syndrome (ACS) in primary care and to calculate possible cost effects of a future clinical decision rule (CDR) incorporating a point-of-care test (PoCT) as compared with current practice. The annual incidence of chest pain, referrals and ACS in primary care was estimated based on a literature review and on a Dutch and Belgian registration study. A health economic model was developed to calculate the potential impact of a future CDR on costs and effects (ie, correct referral decisions), in several scenarios with varying correct referral decisions.

The cost-utility of point-of-care troponin testing to diagnose acute coronary syndrome in primary care.

Background: The added value of using a point-of-care (POC) troponin test in primary care to rule out acute coronary syndrome (ACS) is debated because test sensitivity is inadequate early after symptom onset. This study investigates the potential cost-utility of diagnosing ACS by a general practitioner (GP) when a POC troponin test is available versus GP assessment only.

Methods: A patient-level simulation model was developed, representing a hypothetical cohort of the Dutch population (>35 years) consulting the GP with chest complaints.