Development and validation of a Context-sensitive Positive Health Questionnaire (CPHQ): A factor analysis and multivariate regression study.

BACKGROUND: The concept of Positive Health (PH) has gained increasing attention as a way of measuring individuals' ability to adapt in the face of contextual challenges. However, a suitable measurement instrument for PH that encompasses contextual factors has not yet been developed. This paper responds to this need by developing a Context-specific Positive Health (CPH) measurement instrument that aligns with the Capability Approach (CA). METHODS: The measurement instrument was developed and tested among a representative sample of 1002 Dutch internet survey panel members with diverse sociodemographic backgrounds. The instrument was developed in two stages: a preparation phase consisting of focus groups and expert consultations, and a validation among a representative panel of Dutch citizens. The goal of the preparation phase, was to pilot test and refine previously proposed Positive Health questionnaires into an initial version of the CPHQ. The validation phase aimed to examine the initial CPHQ's factorial validity using Factor Analysis, and its concurrent validity using Multivariate Regression Analysis. RESULTS: The developed questionnaire demonstrated adequate factorial and concurrent validity. Furthermore, it explicitly includes an assessment of resilience, this being a key component of PH. CONCLUSIONS: The introduced measurement tool, the CPHQ, comprises 11 dimensions that we have labeled as follows: relaxation, autonomy, fitness, perceived environmental safety, exclusion, social support, financial resources, political representation, health literacy, resilience, and enjoyment. In this article, we present four major contributions. Firstly, we embedded the measurement in a theoretical framework. Secondly, we focused the questionnaire on a key concept of Positive Health - the "ability to adapt." Thirdly, we addressed issues of health inequality by considering contextual factors. Finally, we facilitated the development of more understandable measurement items.

The (cost-) effectiveness Of Surgical excision of Colorectal endometriosis compared to ART treatment trAjectory (TOSCA study) - a study protocol.

Currently, the optimal treatment to increase the chance of pregnancy and live birth in patients with colorectal endometriosis and subfertility is unknown. Evidence suggests that that both surgery and in vitro fertilization (IVF) or intracytoplasmic sperm injection (ICSI) are effective in improving live birth rate (LBR) among these women. However, the available evidence is of low quality, reports highly heterogeneous results, lacks direct comparison between both treatment options and does not assess whether a combination strategy results in a higher LBR compared to IVF/ICSI-only treatment. Additionally, the optimal timing of surgery within the treatment trajectory remains unclear. The primary objective of the TOSCA study is to assess the effectiveness of surgical treatment (potentially combined with IVF/ICSI) compared to IVF/ICSI-only treatment to increase the chance of an ongoing pregnancy resulting in a live birth in patients with colorectal endometriosis and subfertility, measured by cumulative LBR. Secondary objectives are to assess and compare quality of life and cost-effectiveness in both groups. Patients will be followed for 40 months after inclusion or until live birth. The TOSCA study is expected to be completed in 6 years.

Sensitivity to change in well-being and health-related quality of life in adults with eating disorder symptoms: ICECAP-A versus EQ-5D-5L.

OBJECTIVE: Economic evaluations of treatments help to inform decisions on allocating health care resources. These evaluations involve comparing costs and effectiveness in terms of quality of life. To calculate quality-adjusted life years, generic health related quality of life is often used, but is criticized for not being sensitive to change in mental health populations. Another approach, using experienced well-being measured through capabilities with the ICECAP-A, has been proposed as an alternative. The aim of this study was to investigate whether changes in individuals with eating disorder (ED) symptoms can be better captured using health related quality of life (EQ-5D-5L) or well-being (ICECAP-A). METHOD: Measurements at two time points with an interval of 1 year were used from a sample of 233 participants with self-reported ED symptoms. An analysis of variance was used to test whether the EQ-5D-5L and ICECAP-A differed in sensitivity to change over time. In order to compare the two questionnaires in terms of clinically significant outcome, the reliable change index and clinical cut-off score were calculated. RESULTS: The two questionnaires did not differ in sensitivity to change. More individuals had recovered but also more had deteriorated according to the EQ-5D-5L compared to the ICECAP. DISCUSSION: The present study revealed no differences in sensitivity to change in health-related quality of life or well-being in individuals with ED symptoms in the context of mild clinical change. Results corroborated the pervasiveness of low quality of life in this population, even after alleviation of ED symptoms. PUBLIC SIGNIFICANCE STATEMENT: Measuring treatment benefits in terms of improvements in quality of life is an integral part of economic evaluations in health care. It was expected that these treatment benefits would be better captured as changes in well-being (measured with the ICECAP-A) than as changes in health-related quality of life (measured with the EQ-5D-5L) for individuals with ED symptoms. Based on the results of this study, no preference for one of the two approaches was found.

How to Use Costs in Value-Based Healthcare: Learning from Real-life Examples.

BACKGROUND: Healthcare organizations measure costs for business operations but do not routinely incorporate costs in decision-making on the value of care. AIM: Provide guidance on how to use costs in value-based healthcare (VBHC) delivery at different levels of the healthcare system. SETTING AND PARTICIPANTS: Integrated practice units (IPUs) for diabetes mellitus (DM) and for acute myocardial infarction (AMI) at the Leiden University Medical Center and a collaboration of seven breast cancer IPUs of the Santeon group, all in the Netherlands. PROGRAM DESCRIPTION AND EVALUATION: VBHC aims to optimize care delivery to the patient by understanding how costs relate to outcomes. At the level of shared decision-making between patient and clinician, yearly check-up consultations for DM type I were analyzed for patient-relevant costs. In benchmarking among providers, quantities of cost drivers for breast cancer care were assessed in scorecards. In continuous learning, cost-effectiveness analysis was compared with radar chart analysis to assess the value of telemonitoring in outpatient follow-up. DISCUSSION: Costs vary among providers in healthcare, but also between provider and patient. The joint analysis of outcomes and costs using appropriate methods helps identify and optimize the aspects of care that drive desired outcomes and value.

Relational coordination in value-based health care.

BACKGROUND: An important element of value-based health care (VBHC) is interprofessional collaboration in integrated practice units (IPUs) for the delivery of the complete cycle of care. High levels of interprofessional collaboration between clinical and nonclinical staff in IPUs are assumed rather than proven. Factors that may stimulate interprofessional collaboration in the context of VBHC are underresearched. PURPOSE: The aim of this study was to examine relational coordination (RC) in VBHC and its antecedents. APPROACH: A questionnaire was used to examine the association of both team practices and organizational conditions with interprofessional collaboration in IPUs. Gittell's Relational Coordination Survey was drawn upon to measure interprofessional collaboration by capturing the relational dynamics in coordinated working. The questionnaire also included measures of team practices (team meetings and boundary spanning behavior) and organizational conditions (task interdependence and time constraints). RESULTS: The number of different professional groups participating in team meetings is positively associated with RC in IPUs. Boundary spanning behavior, task interdependence, and time constraints are not associated with RC. CONCLUSIONS: In IPUs, the diversity within interprofessional team meetings is important for establishing high-quality communication and relationships. PRACTICE IMPLICATIONS: Hospital managers should prioritize facilitating and encouraging shared meetings to enhance RC levels among professional groups in IPUs.

Cost effectiveness of implanting a prosthesis after anterior cervical discectomy for radiculopathy: results of the NECK randomized controlled trial.

BACKGROUND CONTEXT: In the treatment of cervical radiculopathy due to a herniated disc, potential surgical treatments include: anterior cervical discectomy (ACD), ACD and fusion using a cage (ACDF), and anterior cervical disc arthroplasty (ACDA). Previous publications yielded comparable clinical and radiological outcome data for the various implants, but research on their comparative costutility has been inconclusive. PURPOSE: To evaluate the cost utility of ACD, ACDF, and ACDA. STUDY DESIGN: Cost-utility analysis. PATIENT SAMPLE: About 109 patients with cervical radiculopathy randomized to undergo ACD, ACDF, or ACDA as part of the NEtherlands Cervical Kinetics trial. OUTCOME MEASURES: Quality-adjusted life-years (QALYs) estimated from patient-reported utilities using the EuroQol-5D questionnaire and EuroQol Visual Analogue Scale (EQ VAS), measured at baseline, 2, 4, 8, 12, 26, 52, and 104 weeks postprocedure. Societal costs including admissions to hospital (related and otherwise), GP visits, specialist visits, physical therapy, medications, home care, aids, informal care, productivity losses, and out of pocket condition-related expenses. METHODS: The cost utility of the competing strategies over 1 and 2 years was assessed following a net benefit (NB) approach, whereby the intervention with the highest NB among competing strategies is preferred. Cost effectiveness acceptability curves were produced to reflect the probability of each strategy being the most cost effective across various willingness-to-pay (WTP) thresholds. Five sensitivity analyses were conducted to assess the robustness of results. RESULTS: ACDF was more likely to be the most cost-effective strategy at WTP thresholds of €20,000 to 50,000/QALY in all but one of the analyses. The mean QALYs during the first year were 0.750, 0.817, and 0.807 for ACD, ACDF, and ACDA, respectively, with no significant differences between groups. Total healthcare costs over the first year were significantly higher for ACDA, largely due to the higher surgery and implant costs. The total societal costs of the three strategies were €12,173 for ACD, €11,195 for ACDF, and €13,746 for ACDA, with no significant differences between groups. CONCLUSION: Our findings demonstrate that ACDF is likely to be more cost-effective than ACDA or ACD at most WTP thresholds, and this conclusion is robust to most sensitivity analyses conducted. It is demonstrated that the difference in costs is mainly caused by the initial surgical costs and that there are only minimal differences in other costs during follow-up. Since clinical data are comparable between the groups, it is to the judgment of the patient and surgeon which intervention is applied.

Cost-effectiveness of Internet Interventions Compared With Treatment as Usual for People With Mental Disorders: Systematic Review and Meta-analysis of Randomized Controlled Trials.

BACKGROUND: The economic costs of mental disorders for society are huge. Internet-based interventions are often coined as cost-effective alternatives to usual care, but the evidence is mixed. OBJECTIVE: The aim was to review the literature on the cost-effectiveness of internet interventions for mental disorders compared with usual care and to provide an estimate of the monetary benefits of such interventions compared with usual care. METHODS: A systematic review and meta-analysis of randomized controlled trials was conducted, which included participants with symptoms of mental disorders; investigated a telephone- or internet-based intervention; included a control condition in the form of treatment as usual, psychological placebo, waiting list control, or bibliotherapy; reported outcomes on both quality of life and costs; and included articles published in English. Electronic databases such as PubMed (including MEDLINE), Embase, Emcare, PsycINFO, Web of Science, and the Cochrane Library were used. Data on risk of bias, quality of the economic evaluation, quality-adjusted life years, and costs were extracted from the included studies, and the incremental net benefit was calculated and pooled. RESULTS: The search yielded 6226 abstracts, and 37 studies with 14,946 participants were included. The quality of economic evaluations of the included studies was rated as moderate, and the risk of bias was high. A random-effects approach was maintained. Analyses suggested internet interventions were slightly more effective than usual care in terms of quality-adjusted life years gain (Hedges g=0.052, 95% CI 0.010-0.094; P=.02) and equally expensive (Hedges g=0.002, 95% CI -0.080 to 0.84; P=.96). The pooled incremental net benefit was US $255 (95% CI US $91 to US $419; P=.002), favoring internet interventions over usual care. The perspective of the economic evaluation and targeted mental disorder moderated the results. CONCLUSIONS: The findings indicate that the cost-effectiveness of internet interventions for mental disorders compared with a care-as-usual approach is likely, but generalizability to new studies is poor given the substantial heterogeneity. This is the first study in the field of mental health to pool cost-effectiveness outcomes in an aggregate data meta-analysis. TRIAL REGISTRATION: PROSPERO CRD42019141659; https://tinyurl.com/3cu99b34.

The assessment of psychometric properties for the subjective wellbeing-5 dimensions (SWB-5D) questionnaire in the general Dutch population.

PURPOSE: Financial resources for health care are limited, so assessment of intervention effectiveness in terms of health in relation to its costs is important. Measuring health outcomes in cost-effectiveness analyses is usually done by health-related quality of life measures, like the EQ-5D. However, over the past decade, innovations on the conceptual level of health have evolved and novel approaches are rising such as the capability approach, subjective wellbeing, and Positive Health. This study assesses the psychometric properties of the subjective wellbeing-5 dimension (SWB-5D) outcome measure. METHODS: A quantitative, cross-sectional study design was used to determine the concurrent and construct (convergent and known group) validity for the SWB-5D. Concurrent and convergent validity were estimated as correlations between the SWB-5D and the Dutch version of the EQ-5D, ICECAP-A, and PH-17. Assessment of known-groups validity was based on the variables illness, education, and the overall happiness (Cantril Ladder) and overall health scale (EQ-5D VAS). RESULTS: A representative sample of 1016 respondents of the Dutch population completed an online questionnaire. The SWB-5D showed reasonable concurrent validity and showed good convergent and known-group validity. The SWB-5D had a lower ceiling effect compared to the EQ-5D and ICECAP-A. CONCLUSION: Compared to traditional health measurement approaches, novel approaches are more focused on the mental and social pillars of health. The SWB-5D shows psychometric feasibility of comprehensive measurement of health, as indicated by a range of validity measures in a large representative sample of the Dutch population.

Cost-effectiveness of three internet-based interventions for eating disorders: A randomized controlled trial.

OBJECTIVE: The primary aim was assessing the cost-effectiveness of an internet-based self-help program, expert-patient support, and the combination of both compared to a care-as-usual condition. METHOD: An economic evaluation from a societal perspective was conducted alongside a randomized controlled trial. Participants aged 16 or older with at least mild eating disorder symptoms were randomly assigned to four conditions: (1) Featback, an online unguided self-help program, (2) chat or e-mail support from a recovered expert patient, (3) Featback with expert-patient support, and (4) care-as-usual. After a baseline assessment and intervention period of 8 weeks, five online assessments were conducted over 12 months of follow-up. The main result constituted cost-utility acceptability curves with quality-of-life adjusted life years (QALYs) and societal costs over the entire study duration. RESULTS: No significant differences between the conditions were found regarding QALYs, health care costs and societal costs. Nonsignificant differences in QALYs were in favor of the Featback conditions and the lowest societal costs per participant were observed in the Featback only condition (€16,741) while the highest costs were seen in the care-as-usual condition (€28,479). The Featback only condition had the highest probability of being efficient compared to the alternatives for all acceptable willingness-to-pay values. DISCUSSION: Featback, an internet-based unguided self-help intervention, was likely to be efficient compared to Featback with guidance from an expert patient, guidance alone and a care-as-usual condition. Results suggest that scalable interventions such as Featback may reduce health care costs and help individuals with eating disorders that are currently not reached by other forms of treatment. PUBLIC SIGNIFICANCE STATEMENT: Internet-based interventions for eating disorders might reach individuals in society who currently do not receive appropriate treatment at low costs. Featback, an online automated self-help program for eating disorders, was found to improve quality of life slightly while reducing costs for society, compared to a do-nothing approach. Consequently, implementing internet-based interventions such as Featback likely benefits both individuals suffering from an eating disorder and society as a whole.

The Box-eHealth in the Outpatient Clinic Follow-up of Patients With Acute Myocardial Infarction: Cost-Utility Analysis.

BACKGROUND: Smartphone compatible wearables have been released on the consumers market, enabling remote monitoring. Remote monitoring is often named as a tool to reduce the cost of care. OBJECTIVE: The primary purpose of this paper is to describe a cost-utility analysis of an eHealth intervention compared to regular follow-up in patients with acute myocardial infarction (AMI). METHODS: In this trial, of which clinical results have been published previously, patients with an AMI were randomized in a 1:1 fashion between an eHealth intervention and regular follow-up. The remote monitoring intervention consisted of a blood pressure monitor, weight scale, electrocardiogram device, and step counter. Furthermore, two in-office outpatient clinic visits were replaced by e-visits. The control group received regular care. The differences in mean costs and quality of life per patient between both groups during one-year follow-up were calculated. RESULTS: Mean costs per patient were €2417±2043 (US $2657±2246) for the intervention and €2888±2961 (US $3175±3255) for the control group. This yielded a cost reduction of €471 (US $518) per patient. This difference was not statistically significant (95% CI -€275 to €1217; P=.22, US $-302 to $1338). The average quality-adjusted life years in the first year of follow-up was 0.74 for the intervention group and 0.69 for the control (difference -0.05, 95% CI -0.09 to -0.01; P=.01). CONCLUSIONS: eHealth in the outpatient clinic setting for patients who suffered from AMI is likely to be cost-effective compared to regular follow-up. Further research should be done to corroborate these findings in other patient populations and different care settings. TRIAL REGISTRATION: ClinicalTrials.gov NCT02976376; https://clinicaltrials.gov/ct2/show/NCT02976376. INTERNATIONAL REGISTERED REPORT IDENTIFIER (IRRID): RR2-10.2196/resprot.8038.

The implementation of value-based healthcare: a scoping review.

BACKGROUND: The aim of this study was to identify and summarize how value-based healthcare (VBHC) is conceptualized in the literature and implemented in hospitals. Furthermore, an overview was created of the effects of both the implementation of VBHC and the implementation strategies used. METHODS: A scoping review was conducted by searching online databases for articles published between January 2006 and February 2021. Empirical as well as non-empirical articles were included. RESULTS: 1729 publications were screened and 62 were used for data extraction. The majority of the articles did not specify a conceptualization of VBHC, but only conceptualized the goals of VBHC or the concept of value. Most hospitals implemented only one or two components of VBHC, mainly the measurement of outcomes and costs or Integrated Practice Units (IPUs). Few studies examined effects. Implementation strategies were described rarely, and were evaluated even less. CONCLUSIONS: VBHC has a high level of interpretative variability and a common conceptualization of VBHC is therefore urgently needed. VBHC was proposed as a shift in healthcare management entailing six reinforcing steps, but hospitals have not implemented VBHC as an integrative strategy. VBHC implementation and effectiveness could benefit from the interdisciplinary collaboration between healthcare and management science. TRIAL REGISTRATION: This scoping review was registered on Open Science Framework https://osf.io/jt4u7/ (OSF | The implementation of Value-Based Healthcare: a Scoping Review).

The ICEpop Capability Measure for Adults Instrument for Capabilities: Development of a Tariff for the Dutch General Population.

OBJECTIVES: The ICEpop Capability Measure for Adults (ICECAP-A) assesses 5 capabilities (stability, attachment, autonomy, achievement, and enjoyment) that are important to one's quality of life and might be an important addition to generic health questionnaires currently used in economic evaluations. This study aimed to develop a Dutch tariff of the Dutch translation of the ICECAP-A. METHODS: The methods used are similar to those used in the development of the UK tariff. A profile case best-worst scaling task was presented to 1002 participants from the general Dutch population. A scale-adjusted latent class analysis was performed to test for preferences of ICECAP-A capabilities and scale heterogeneity. RESULTS: A 3-preference class 2-scale class model with worst choice as scale predictor was considered optimal and was used to calculate the resulting tariff. Results indicated that the capabilities stability, attachment, and enjoyment were considered more important aspects of quality of life than autonomy and achievement. Additionally, improving capabilities from low to moderate levels had a larger effect on quality of life than improving capabilities that were already at a higher level. CONCLUSIONS: The ICECAP-A tariffs found in this study could be used in economic evaluations of healthcare interventions in The Netherlands.

The ICECAP-A instrument for capabilities: assessment of construct validity and test-retest reliability in a general Dutch population.

PURPOSE: The ICEpop CAPability measure for Adults (ICECAP-A) assesses five capabilities that are important to one's well-being. The instrument might be an important addition to generic health questionnaires when evaluating quality of life extending beyond health. This study aimed to conduct a psychometric assessment of the Dutch translation of the ICECAP-A. METHODS: Construct validity of the instrument was assessed in two ways. First, by measuring correlations with the EQ-5D-5L questionnaire and a measure of self-efficacy and, second, by investigating the ability to distinguish between groups known to differ on the construct the ICECAP-A means to capture. Additionally, test-retest reliability was evaluated. RESULTS: In total, 1002 participants representative of the general Dutch population completed an online survey. For test-retest reliability, 252 participants completed the same questionnaire 2 weeks later. The ICECAP-A indicated moderate to strong correlations with the EQ-5D-5L and a strong correlation with self-efficacy. Furthermore, it was capable of differentiating known groups. Moreover, results indicated adequate test-retest reliability with an intraclass correlation coefficient of 0.79. CONCLUSION: In summary, results suggest adequate test-retest reliability and construct validity and indicate that the ICECAP-A might be of added value, especially when considering areas outside of the traditional health intervention model.

Cost-effectiveness of Cardiac Telerehabilitation With Relapse Prevention for the Treatment of Patients With Coronary Artery Disease in the Netherlands.

Importance: Cardiac telerehabilitation (CTR) has been found to be a safe and beneficial alternative to traditional center-based cardiac rehabilitation (CR) and might be associated with higher participation rates by reducing barriers to CR use. However, implementation of CTR interventions remains low, which may be owing to a lack of cost-effectiveness analyses of data from large-scale randomized clinical trials. Objective: To assess the cost-effectiveness of CTR with relapse prevention compared with center-based CR among patients with coronary artery disease. Design, Setting, and Participants: This economic evaluation performed a cost-utility analysis of data from the SmartCare-CAD (Effects of Cardiac Telerehabilitation in Patients With Coronary Artery Disease Using a Personalized Patient-Centred ICT Platform) randomized clinical trial. The cost-effectiveness and utility of 3 months of cardiac telerehabilitation followed by 9 months of relapse prevention were compared with the cost-effectiveness of traditional center-based cardiac rehabilitation. The analysis included 300 patients with stable coronary artery disease who received care at a CR center serving 2 general hospitals in the Netherlands between May 23, 2016, and July 26, 2018. All patients were entering phase 2 of outpatient CR and were followed up for 1 year (until August 14, 2019). Data were analyzed from September 21, 2020, to September 24, 2021. Intervention: After baseline measurements were obtained, participants were randomly assigned on a 1:1 ratio to receive CTR (intervention group) or center-based CR (control group) using computerized block randomization. After 6 supervised center-based training sessions, patients in the intervention group continued training at home using a heart rate monitor and accelerometer. Patients uploaded heart rate and physical activity data and discussed their progress during a weekly video consultation with their physical therapist. After 3 months, weekly coaching was concluded, and on-demand coaching was initiated for relapse prevention; patients were instructed to continue using their wearable sensors and were contacted in cases of nonadherence to the intervention or reduced exercise or physical activity volumes. Main Outcomes and Measures: Quality-adjusted life-years were assessed using the EuroQol 5-Dimension 5-Level survey (EQ-5D-5L) and the EuroQol Visual Analogue Scale (EQ-VAS), and cardiac-associated health care costs and non-health care costs were measured by health care consumption, productivity, and informal care questionnaires (the Medical Consumption Questionnaire, the Productivity Cost Questionnaire, and the Valuation of Informal Care Questionnaire) designed by the Institute for Medical Technology Assessment. Costs were converted to 2020 price levels (in euros) using the Dutch consumer price index (to convert to US dollars, euro values were multiplied by 1.142, which was the mean exchange rate in 2020). Results: Among 300 patients (266 men [88.7%]), the mean (SD) age was 60.7 (9.5) years. The quality of life among patients receiving CTR vs center-based CR was comparable during the study according to the results of both utility measures (mean difference on EQ-5D-5L: -0.004; P = .82; mean difference on EQ-VAS: -0.001; P = .92). Intervention costs were significantly higher for CTR (mean [SE], €224 [€4] [$256 ($4)]) compared with center-based CR (mean [SE], €156 [€5] [$178 ($6)]; P < .001); however, no difference in overall cardiac health care costs was observed between CTR (mean [SE], €4787 [€503] [$5467 ($574)] and center-based CR (mean [SE], €5507 [€659] [$6289 ($753)]; P = .36). From a societal perspective, CTR was associated with lower costs compared with center-based CR (mean [SE], €20 495 [€ 2751] [$23 405 ($3142)] vs €24 381 [€3613] [$27 843 ($4126)], respectively), although this difference was not statistically significant (-€3887 [-$4439]; P = .34). Conclusions and Relevance: In this economic evaluation, a CTR intervention with relapse prevention was likely to be cost-effective compared with center-based CR, suggesting that CTR maybe used as an alternative intervention for the treatment of patients with coronary artery disease. These results add to the evidence base in favor of CTR and may increase the implementation of CTR interventions in clinical practice.

Economic Evaluation of Different Screening Strategies for Severe Combined Immunodeficiency Based on Real-Life Data.

Although several countries have adopted severe combined immunodeficiency (SCID) into their newborn screening (NBS) program, other countries are still in the decision process of adding this disorder in their program and finding the appropriate screening strategy. This decision may be influenced by the cost(-effectiveness) of these screening strategies. In this study, the cost(-effectiveness) of different NBS strategies for SCID was estimated based on real-life data from a prospective implementation study in the Netherlands. The cost of testing per child for SCID was estimated at EUR 6.36. The cost of diagnostics after screen-positive results was assessed to vary between EUR 985 and 8561 per child dependent on final diagnosis. Cost-effectiveness ratios varied from EUR 41,300 per QALY for the screening strategy with T-cell receptor excision circle (TREC) ≤ 6 copies/punch to EUR 44,100 for the screening strategy with a cut-off value of TREC ≤ 10 copies/punch. The analysis based on real-life data resulted in higher costs, and consequently in less favorable cost-effectiveness estimates than analyses based on hypothetical data, indicating the need for verifying model assumptions with real-life data. The comparison of different screening strategies suggest that strategies with a lower number of referrals, e.g., by distinguishing between urgent and less urgent referrals, are favorable from an economic perspective.

Costs and effects of conventional vision screening and photoscreening in the Dutch preventive child health care system.

BACKGROUND: Little is known about costs and effects of vision screening strategies to detect amblyopia. Aim of this study was to compare costs and effects of conventional (optotype) vision screening, photoscreening or a combination in children aged 3-6 years. METHODS: Population-based, cross-sectional study in preventive child health care in The Hague. Children aged 3 years (3y), 3 years and 9 months (3y9m) or 5-6 years (5/6y) received the conventional chart vision screening and a test with a photoscreener (Plusoptix S12C). Costs were based on test duration and additional costs for devices and diagnostic work-up. RESULTS: Two thousand, one hundred and forty-four children were included. The estimated costs per child screened were €17.44, €20.37 and €6.90 for conventional vision screening at 3y, 3y9m and 5/6y, respectively. For photoscreening, these estimates were €6.61, €7.52 and €9.40 and for photoscreening followed by vision screening if the result was unclear (combination) €9.32 (3y) and €9.33 (3y9m). The number of children detected with amblyopia by age were 9, 14 and 5 (conventional screening), 6, 13 and 3 (photoscreening) and 10 (3y) and 15 (3y9m) (combination), respectively. The estimated costs per child diagnosed with amblyopia were €1500, €1050 and €860 for conventional vision screening, €860, €420 and €1940 for photoscreening and €730 (3y) and €450 (3y9m) for the combination. CONCLUSIONS: Combining photoscreening with vision screening seems promising to detect amblyopia in children aged 3y/3y9m, whereas conventional screening seems preferable at 5/6y. As the number of study children with amblyopia is small, further research on the effects of these screening alternatives in detecting children with amblyopia is recommended.

The Methodological Quality and Challenges in Conducting Economic Evaluations of Newborn Screening: A Scoping Review.

INTRODUCTION: Cost-effectiveness (CEA) and cost-utility analyses (CUA) have become popular types of economic evaluations (EE) used for evidence-based decision-making in healthcare resource allocation. Newborn screening programs (NBS) can have significant clinical benefits for society, and cost-effectiveness analysis may help to select the optimal strategy among different screening programs, including the no-screening option, on different conditions. These economic analyses of NBS, however, are hindered by several methodological challenges. This study explored the methodological quality in recent NBS economic evaluations and analyzed the main challenges and strategies adopted by researchers to deal with them. METHODS: A scoping review was conducted according to PRISMA methodology to identify CEAs and CUAs of NBS. The methodological quality of the retrieved studies was assessed quantitatively using a specific guideline for the quality assessment of NBS economic evaluations, by calculating a general score for each EE. Challenges in the studies were then explored using thematic analysis as a qualitative synthesis approach. RESULTS: Thirty-five studies met the inclusion criteria. The quantitative analysis showed that the methodological quality of NBS economic evaluations was heterogeneous. Lack of clear description of items related to results, discussion, and discounting were the most frequent flaws. Methodological challenges in performing EEs of neonatal screenings include the adoption of a long time horizon, the use of quality-adjusted life years as health outcome measure, and the assessment of costs beyond the screening interventions. CONCLUSIONS: The results of this review can support future economic evaluation research, aiding researchers to develop a methodological guidance to perform EEs aimed at producing solid results to inform decisions for resource allocation in neonatal screening.

An assessment of symptom burden in inflammatory bowel diseases to develop a patient preference-weighted symptom score.

PURPOSE: Inflammatory bowel disease (IBD) patients experience diverse symptoms and the impact of these different symptoms varies substantially. Current disease activity measures do not account for the relative importance of the different symptoms and severity levels. In this study, we aimed to quantify the relative importance of different symptoms for IBD patients and to develop a patient preference-weighted symptom (PWS) score to assess symptom burden in IBD. METHODS: We performed a choice-based conjoint analysis (CBCA) survey with 129 IBD patients to estimate the relative importance of four common IBD symptoms: stool frequency, abdominal pain, blood in stools, and urgency. We then developed the PWS score using the preferences obtained from the CBCA, which we validated against existing measures. RESULTS: CBCA revealed that urgency was the most important symptom to patients, followed by abdominal pain and blood in stools. Urgency associated with incontinence received particularly high scores and was perceived to be more than 3 times as important as urgency without incontinence. Our results confirmed that different symptoms are not equally bothersome, and we showed that the relation between symptom-level and importance is not linear. The PWS score, which we developed using these estimates was highly correlated with existing disease activity measures. CONCLUSIONS: We quantified the relative importance of four common IBD symptoms and developed the PWS score for IBD, which takes the relative importance of different symptoms and symptom-levels into account. The PWS score can be used to obtain a patient-centered assessment of symptom burden.

Assessing quality of life in psychosocial and mental health disorders in children: a comprehensive overview and appraisal of generic health related quality of life measures.

BACKGROUND: Mental health problems often arise in childhood and adolescence and can have detrimental effects on people's quality of life (QoL). Therefore, it is of great importance for clinicians, policymakers and researchers to adequately measure QoL in children. With this review, we aim to provide an overview of existing generic measures of QoL suitable for economic evaluations in children with mental health problems. METHODS: First, we undertook a meta-review of QoL instruments in which we identified all relevant instruments. Next, we performed a systematic review of the psychometric properties of the identified instruments. Lastly, the results were summarized in a decision tree. RESULTS: This review provides an overview of these 22 generic instruments available to measure QoL in children with psychosocial and or mental health problems and their psychometric properties. A systematic search into the psychometric quality of these instruments found 195 suitable papers, of which 30 assessed psychometric quality in child and adolescent mental health. CONCLUSIONS: We found that none of the instruments was perfect for use in economic evaluation of child and adolescent mental health care as all instruments had disadvantages, ranging from lack of psychometric research, no proxy version, not being suitable for young children, no age-specific value set for children under 18, to insufficient focus on relevant domains (e.g. social and emotional domains).