Reliability, costs, and radiation dose of dual-energy X-ray absorptiometry in diagnosis of radiologic sarcopenia in surgically menopausal women.

OBJECTIVE: The aim of this study was to evaluate and compare reliability, costs, and radiation dose of dual-energy X-ray absorptiometry (DXA) to MRI and CT in measuring muscle mass for the diagnosis of sarcopenia. METHODS: Thirty-four consecutive DXA scans performed in surgically menopausal women from November 2019 until March 2020 were analyzed by two observers. Observers analyzed muscle mass of the lower limbs in every scan twice. Reliability was assessed by calculating inter- and intra-observer variability. Reliability from CT and MRI as well as radiation dose from CT and DXA were collected from literature. Costs for each type of scan were calculated according to the guidelines for economic evaluation of the Dutch National Health Care Institute. RESULTS: The 34 participants had a median age of 58 years (IQR 53-65) and a median body mass index of 24.6 (IQR 21.7-29.7). Inter-observer variability had an intraclass correlation coefficient (ICC) of 0.997 (95% CI 0.994-0.998) with a relative variability of 0.037 ± 0.022%. Regarding intra-observer variability, observer 1 had an ICC of 0.998 (95% CI 0.996-0.999) with a relative variability of 0.019 ± 0.016% and observer 2 had an ICC of 0.997 (95% CI 0.993-0.998) with a relative variability of 0.016 ± 0.011%. DXA costs were €62, CT €77, and MRI €195. The estimated radiation dose of CT was 2.5-3.0 mSv, for DXA this was 2-4 µSv. CONCLUSIONS: DXA has lower costs and a lower radiation dose, with low inter- and intra-observer variability, compared to CT and MRI for assessing lower limb muscle mass. TRIAL REGISTRATION: Netherlands Trial Register; NL8068. CRITICAL RELEVANCE STATEMENT: DXA is a good alternative for CT and MRI in assessing lower limb muscle mass, with lower costs and lower radiation dose, while inter-observer and intra-observer variability are low. KEY POINTS: • Screening for sarcopenia should be optimized as the population ages. • DXA outperformed CT and MRI in the measured metrics. • DXA validity should be further evaluated as an alternative to CT and MRI for sarcopenia evaluation.

A care substitution service in the Netherlands: impact on referral, cost, and patient satisfaction.

BACKGROUND: In care substitution services, medical specialists offer brief consultations to provide general practitioners (GPs) with advice on diagnosis, treatment, or hospital referral. When GPs serve as gatekeepers to secondary care, these regional services could reduce pressures on healthcare systems. The aim is to determine the impact of implementing a care substitution service for dermatology, orthopaedics, and cardiology on the hospital referral rate, health care costs, and patient satisfaction. METHODS: A before-after study was used to evaluate hospital referral rates and health care costs during a follow-up period of 1 year. The study population comprised patients with eligible International Classification of Primary Care codes for referral to the care substitution service (only dermatology, orthopaedic, cardiology indications), as pre-defined by GPs and medical specialists. We compared referral rates before and after implementation by χ2 tests and evaluated patient preference by qualitative analysis. RESULTS: In total, 4,930 patients were included, 2,408 before and 2,522 after implementation. The care substitution service decreased hospital referrals during the follow-up period from 15 to 11%. The referral rate decreased most for dermatology (from 15 to 9%), resulting in a cost reduction of €10.59 per patient, while the other two specialisms experienced smaller reductions in referral rates. Patients reported being satisfied, mainly because of the null cost, improved organisation, improved care, and positive experience of the consultation. CONCLUSIONS: The care substitution service showed promise for specialisms that require fewer hospital facilities, as exemplified by dermatology.

Effectiveness of rehabilitation for working-age patients after a total hip arthroplasty: a comparison of usual care between the Netherlands and Germany.

BACKGROUND: Postoperative rehabilitation after primary total hip arthroplasty (p-THA) differs between the Netherlands and Germany. Aim is to compare clinical effectiveness and to get a first impression of cost effectiveness of Dutch versus German usual care after p-THA. METHODS: A transnational prospective controlled observational trial. Clinical effectiveness was assessed with self-reported questionnaires and functional tests. Measurements were taken preoperatively and 4 weeks, 12 weeks, and 6 months postoperatively. For cost effectiveness, long-term economic aspects were assessed from a societal perspective. RESULTS: 124 working-age patients finished the measurements. German usual care leads to a significantly larger proportion (65.6% versus 47.5%) of satisfied patients 12 weeks postoperatively and significantly better self-reported function and Five Times Sit-to-Stand Test (FTSST) results. German usual care is generally 45% more expensive than Dutch usual care, and 20% more expensive for working-age patients. A scenario analysis assumed that German patients work the same number of hours as the Dutch, and that productivity costs are the same. This analysis revealed German care is still more expensive but the difference decreased to 8%. CONCLUSIONS: German rehabilitation is clinically advantageous yet more expensive, although comparisons are less straightforward as the socioeconomic context differs between the two countries. TRIAL REGISTRATION: The study is registered in the German Registry of Clinical Trials (DRKS00011345, 18/11/2016).

Health-related quality of life, one-year costs and economic evaluation in extracorporeal membrane oxygenation in critically ill adults.

PURPOSE: This study reports on survival and health related quality of life (HRQOL) after extracorporeal membrane oxygenation (ECMO) treatment and the associated costs in the first year. MATERIALS AND METHODS: Prospective observational cohort study patients receiving ECMO in the intensive care unit during August 2017 and July 2019. We analyzed all healthcare costs in the first year after index admission. Follow-up included a HRQOL analysis using the EQ-5D-5L at 6 and 12 months. RESULTS: The study enrolled 428 patients with an ECMO run during their critical care admission. The one-year mortality was 50%. Follow up was available for 124 patients at 12 months. Survivors reported a favorable mean HRQOL (utility) of 0.71 (scale 0-1) at 12 months of 0.77. The overall health status (VAS, scale 0-100) was reported as 73.6 at 12 months. Mean total costs during the first year were $204,513 ± 211,590 with hospital costs as the major factor contributing to the total costs. Follow up costs were $53,752 ± 65,051 and costs of absenteeism were $7317 ± 17,036. CONCLUSIONS: At one year after hospital admission requiring ECMO the health-related quality of life is favorable with substantial costs but considering the survival might be acceptable. However, our results are limited by loss of follow up. So it may be possible that only the best-recovered patients returned their questionnaires. This potential bias might lead to higher costs and worse HRQOL in a real-life scenario.

Effectiveness and costs of a new framework for selecting absorbent urinary incontinence products compared with current practice: a cohort study.

OBJECTIVES: To improve continence care in the Netherlands, a new framework has been developed in which a shift has been made from incontinence severity as the sole criterion for selecting incontinence products to a focus on patient need for daily life activities. The impact of the framework on daily care has not been assessed. We aimed to compare treatment effectiveness and costs between participants who did and did not undergo re-evaluation according to the new framework. DESIGN: Cohort study SETTING: Twelve pharmacies in the Netherlands PARTICIPANTS: Existing users of absorbent incontinence materials for urinary incontinence. INTERVENTIONS: Participants were offered the option to have their incontinence products re-evaluated within the new framework at their pharmacy. PRIMARY AND SECONDARY OUTCOME MEASURES: Effectiveness and cost outcomes were assessed at 3 and 6 months through questionnaires. These questionnaires included the International Consultation on Incontinence Questionnaire (ICIQ) Lower Urinary Tract Symptoms Quality of Life Module, ICIQ Urinary Incontinence Short Form, ICIQ Absorbent Pads, questions about satisfaction and the iPCQ and iMCQ. RESULTS: 303 users consented and 279 completed the baseline questionnaire. Of these participants, 72 agreed to a re-evaluation of their incontinence materials. There was a small improvement at 3 months in the re-evaluation group compared with the other group on most outcomes. However, these improvements were not clinically relevant. Moreover, these differences did not change much from 3 to 6 months. Small differences were also observed in the changes in costs, but with very wide CIs on both sides of zero (€99.38; 95% CI -633.48 to 832.23). CONCLUSIONS: The current study showed no clinically relevant effect of a newly implemented framework for selecting incontinence materials in pharmacies when compared with an existing method. Given that the study also showed no differences in effectiveness and costs, introducing the new framework in pharmacies may not lead to better incontinence care.

Cost-effectiveness of an app-based treatment for urinary incontinence in comparison with care-as-usual in Dutch general practice: a pragmatic randomised controlled trial over 12 months.

OBJECTIVE: To assess the cost-effectiveness of app-based treatment for female stress, urgency or mixed urinary incontinence (UI) compared with care-as-usual in Dutch primary care. DESIGN: A pragmatic, randomised controlled, superiority trial. SETTING: Primary care in the Netherlands from 2015 to 2018, follow-up at 12 months. POPULATION: Women with ≥2 UI-episodes per week, access to mobile apps, wanting treatment. METHODS: The standalone app included conservative management for UI with motivation aids (e.g. reminders). Care-as-usual delivered according to the Dutch GP guideline for UI. MAIN OUTCOME MEASURES: Costs and cost-effectiveness and -utility were assessed from a societal perspective, based on incontinence impact adjusted life years (IIALYs), quality adjusted life years (QALYs) and medical, non-medical and productivity costs. Information on costs was obtained with the iMCQ and iPCQ questionnaires (medical consumption and productivity cost questionnaires). RESULTS: In all, 262 women were andomised equally to app or care-as-usual; 89 (68%) and 83 (63%) attended follow-up, respectively. Costs were lower for app-based treatment with € -161 (95% confidence interval [CI -180 to -151) per year. Cost-effectiveness showed small mean differences in effect for IIALY (0.04) and QALY (-0.03) and thus larger incremental cost-effectiveness ratios (ICER: -€3696) and incremental cost-utility ratios (ICUR: €6379). CONCLUSION: App-based treatment is a cost-effective alternative to care-as-usual for women with UI in Dutch primary care. TWEETABLE ABSTRACT: App-treatment for female urinary incontinence cost-effective compared to care-as-usual in general practice after 12 months.

Cost-effectiveness of physiotherapy in childhood functional constipation: a randomized controlled trial in primary care.

OBJECTIVE: Health care expenditures for children with functional constipation (FC) are high, while conservative management is successful in only 50% of the children. The aim is to evaluate whether adding physiotherapy to conventional treatment (CT) is a cost-effective strategy in the management of children with FC aged 4-18 years in primary care. METHODS: A cost-effectiveness analysis was performed alongside a randomized controlled trial (RCT) with 8-month follow-up. Costs were assessed from a societal perspective, effectiveness included both the primary outcome (treatment success defined as the absence of FC and no laxative use) and the secondary outcome (absence of FC irrespective of laxative use). Uncertainty was assessed by bootstrapping and cost-effectiveness acceptability curves (CEACs) were displayed. RESULTS: One hundred and thirty-four children were randomized. The incremental cost-effectiveness ratio (ICER) for one additional successfully treated child in the physiotherapy group compared with the CT group was €24,060 (95% confidence interval [CI] €-16,275 to €31,390) and for the secondary outcome €1,221 (95% CI €-12,905 to €10,956). Subgroup analyses showed that for children with chronic laxative use the ICER was €2,134 (95% CI -24,975 to 17,192) and €571 (95% CI 11 to 3,566), respectively. At a value of €1,000, the CEAC showed a probability of 0.53 of cost-effectiveness for the primary outcome, and 0.90 for the secondary outcome. CONCLUSIONS: Physiotherapy added to CT as first-line treatment for all children with FC is not cost-effective compared with CT alone. Future studies should consider the cost-effectiveness of physiotherapy added to CT in children with chronic laxative use. TRIAL REGISTRATION: The RCT is registered in the Netherlands Trial Register (NTR4797), on the 8th of September 2014. The first child was enrolled on the 2nd of December 2014. https://www.trialregister.nl/trial/4654.

Cost-effectiveness of abbreviated-protocol MRI screening for women with mammographically dense breasts in a national breast cancer screening program.

INTRODUCTION: Magnetic resonance imaging (MRI) has shown the potential to improve the screening effectiveness among women with dense breasts. The introduction of fast abbreviated protocols (AP) makes MRI more feasible to be used in a general population. We aimed to investigate the cost-effectiveness of AP-MRI in women with dense breasts (heterogeneously/extremely dense) in a population-based screening program. METHODS: A previously validated model (SiMRiSc) was applied, with parameters updated for women with dense breasts. Breast density was assumed to decrease with increased age. The base scenarios included six biennial AP-MRI strategies, with biennial mammography from age 50-74 as reference. Fourteen alternative scenarios were performed by varying screening interval (triennial and quadrennial) and by applying a combined strategy of mammography and AP-MRI. A 3% discount rate for both costs and life years gained (LYG) was applied. Model robustness was evaluated using univariate and probabilistic sensitivity analyses. RESULTS: The six biennial AP-MRI strategies ranged from 132 to 562 LYG per 10,000 women, where more frequent application of AP-MRI was related to higher LYG. The optimal strategy was biennial AP-MRI screening from age 50-65 for only women with extremely dense breasts, producing an incremental cost-effectiveness ratio of € 18,201/LYG. At a threshold of € 20,000/LYG, the probability that the optimal strategy was cost-effective was 79%. CONCLUSION: Population-based biennial breast cancer screening with AP-MRI from age 50-65 for women with extremely dense breasts might be a cost-effective alternative to mammography, but is not an option for women with heterogeneously dense breasts.

Assessment of the Benefits and Cost-Effectiveness of Population-Based Breast Cancer Screening in Urban China: A Model-Based Analysis.

BACKGROUND: To decrease the burden of breast cancer (BC), the Chinese government recently introduced biennial mammography screening for women aged 45-70 years. In this study, we assess the effectiveness and cost-effectiveness of implementing this programme in urban China using a micro-simulation model. METHODS: The 'Simulation Model on radiation Risk and breast cancer Screening' (SiMRiSc) was applied, with parameters updated based on available data for the Chinese population. The base scenario was biennial mammography screening for women aged 45-70 years, and this was compared to a reference population with no screening. Seven alternative scenarios were then simulated by varying the screening intervals and participant ages. This analysis was conducted from a societal perspective. The discounted incremental cost-effectiveness ratio (ICER) was compared to a threshold of triple the gross domestic product (GDP) per life years gained (LYG), which was 30 785 USD/LYG. Univariate sensitivity analyses were conducted to evaluate model robustness. In addition, a budget impact analysis was performed by comparing biennial screening with no screening at a time horizon of 10 years. RESULTS: Compared with no screening, the base scenario was cost-effective in urban China, giving a discounted average cost-effectiveness ratio (ACER) of 17 309 USD/LYG. The model was most sensitive to the cost of mammography per screen, followed by mean size of self-detected tumours, mammographic breast density and the cumulative lifetime risk of BC. The efficient frontier showed that at a threshold of 30 785 USD/LYG, the base scenario was the optimal scenario with a discounted ICER of 25 261 USD/LYG. Over 10 years, screening would incur a net cost of almost 38.1 million USD for a city with 1 million citizens. CONCLUSION: Compared to no screening, biennial mammography screening for women aged from 45-70 is cost-effective in urban China.

Hospital Costs of Extracorporeal Membrane Oxygenation in Adults: A Systematic Review.

BACKGROUND: Costs associated with extracorporeal membrane oxygenation (ECMO) are an important factor in establishing cost effectiveness. In this systematic review, we aimed to determine the total hospital costs of ECMO for adults. METHODS: The literature was retrieved from the PubMed/MEDLINE, EMBASE, and Web of Science databases from inception to 4 March 2020 using the search terms 'extracorporeal membrane oxygenation' combined with 'costs'; similar terms or phrases were then added to the search, i.e. 'Extracorporeal Life Support' or 'ECMO' or 'ECLS' combined with 'costs'. We included any type of study (e.g. randomized trial or observational cohort) evaluating hospital costs of ECMO in adults (age ≥18 years). RESULTS: A total of 1768 unique articles were retrieved during our search. We assessed 74 full-text articles for eligibility, of which 14 articles were selected for inclusion in this review; six papers were from the US, five were from Europe, and one each from Japan, Australia, and Taiwan. The sample sizes ranged from 16 to 18,684 patients. One paper exclusively used prospective cost data collection, while all other papers used retrospective data collection. Five papers reported charges instead of costs. There was large variation in hospital costs, ranging from US$22,305 to US$334,608 (2019 values), largely depending on the indication for ECMO support and location. The highest reported costs were for lung transplant recipients who were receiving ECMO support in the US, and the lowest reported costs were for extracorporeal cardiopulmonary resuscitation patients presenting with non-shockable rhythm in Japan. The additional costs of ECMO patients compared with non-ECMO patients varied between US$2518 and US$200,658. Personnel costs varied between 11 and 52% of the total amount. CONCLUSIONS: ECMO therapy is an advanced and expensive technology, although reported costs differ considerably depending on ECMO indication and whether charges or costs are measured. Combined with the ongoing gathering of outcome data, cost effectiveness per ECMO indication could be determined in the future.

Cost-effectiveness of oral ondansetron for children with acute gastroenteritis in primary care: a randomised controlled trial.

BACKGROUND: Acute gastroenteritis is a common childhood condition with substantial medical and indirect costs, mostly because of referral, hospitalisation, and parental absence from work. AIM: To determine the cost-effectiveness of adding oral ondansetron to care as usual (CAU) for children with acute gastroenteritis presenting to out-of-hours primary care (OOH-PC). DESIGN AND SETTING: A pragmatic randomised controlled trial from December 2015 to January 2018, at three OOHPC centres in the north of the Netherlands (Groningen, Zwolle, and Assen) with a follow-up of 7 days. METHOD: Children were recruited at the OOH-PC and parents kept a parental diary. Inclusion criteria were: aged 6 months-6 years; diagnosis of acute gastroenteritis; at least four reported episodes of vomiting 24 hours before presentation, at least one of which was in the 4 hours before presentation; and written informed consent from both parents. Children were randomly allocated at a 1:1 ratio to either CAU (oral rehydration therapy) or CAU plus one dose of 0.1 mg/kg oral ondansetron. RESULTS: In total, 194 children were included for randomisation. One dose of oral ondansetron decreased the proportion of children who continued vomiting within the first 4 hours from 42.9% to 19.5%, (a decrease of 54.5%), with an odds ratio of 0.4 (95% confidence interval [CI] = 0.2 to 0.7; number needed to treat: four). Total mean costs in the ondansetron group were 31.2% lower (€488 [£420] versus €709 [£610]), and the total incremental mean costs for an additional child free of vomiting in the first 4 hours was -€9 (£8) (95% CI = -€41 [£35] to €3 [£3]). CONCLUSION: A single oral dose of ondansetron for children with acute gastroenteritis, given in OOH-PC settings, is both clinically beneficial and cost-effective.

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).

A Randomized Trial of Initiation of Chronic Noninvasive Mechanical Ventilation at Home vs In-Hospital in Patients With Neuromuscular Disease and Thoracic Cage Disorder: The Dutch Homerun Trial.

BACKGROUND: There is an increasing demand for home mechanical ventilation (HMV) in patients with chronic respiratory insufficiency. At present, noninvasive ventilation is exclusively initiated in a clinical setting at all four centers for HMV in the Netherlands. In addition to its high societal costs and patient discomfort, commencing HMV is often delayed because of a lack of hospital bed capacity. RESEARCH QUESTION: Is HMV initiation at home, using a telemonitoring approach, noninferior to in-hospital initiation in a nationwide study? STUDY DESIGN AND METHODS: We conducted a nationwide, randomized controlled noninferiority trial, in which every HMV center recruited 24 patients (home [n = 12] vs hospital [n = 12]) with a neuromuscular disease or thoracic cage disorder, all with an indication to start HMV. Change in arterial CO2 (Paco2) over a 6-month period was considered the primary outcome, and quality of life and costs were assessed as secondary outcomes. RESULTS: A total of 96 patients were randomized, most of them diagnosed with neuromuscular disease. We found a significant improvement in Paco2 within both groups (home: from 6.1 to 5.6 kPa [P < .01]; hospital: from 6.3 to 5.6 kPa [P < .01]), with no significant differences between groups. Health-related quality of life showed significant improvement on various subscales; however, no significant differences were observed between the home and hospital groups. From a societal perspective, a cost reduction of more than €3,200 ($3,793) per patient was evident in the home group. INTERPRETATION: This nationwide, multicenter study shows that HMV initiation at home is noninferior to hospital initiation, as it shows the same improvement in gas exchange and health-related quality of life. In fact, from a patient's perspective, it might even be a more attractive approach. In addition, starting at home saves over €3,200 ($3,793) per patient over a 6-month period. TRIAL REGISTRY: ClinicalTrials.gov; No.: NCT03203577; URL: www.clinicaltrials.gov.

Value judgment of new medical treatments: Societal and patient perspectives to inform priority setting in The Netherlands.

BACKGROUND: In many countries, medical interventions are reimbursed on the basis of recommendations made by advisory boards and committees that apply multiple criteria in their assessment procedures. Given the diversity of these criteria, it is difficult to find common ground to determine what information is required for setting priorities. OBJECTIVE: To investigate whether society and patients share the same interests and views concerning healthcare priorities. METHODS: We applied a framework of discrete choice models in which respondents were presented with judgmental tasks to elicit their preferences. They were asked to choose between two hypothetical scenarios of patients receiving a new treatment. The scenarios graphically presented treatment outcomes and patient characteristics. Responses were collected through an online survey administered among respondents from the general population (N = 1,253) and patients (N = 1,389) and were analyzed using conditional logit and mixed logit models. RESULTS: The respondents' preferences regarding new medical treatments revealed that they attached the most relative importance to additional survival years, age at treatment, initial health condition, and the cause of disease. Minor differences in the relative importance assigned to three criteria: age at treatment, initial health, and cause of disease were found between the general population and patient samples. Health scenarios in which patients had higher initial health-related quality of life (i.e., a lower burden of disease) were favored over those in which patients' initial health-related quality of life was lower. CONCLUSIONS: Overall, respondents within the general population expressed preferences that were similar to those of the patients. Therefore, priority-setting studies that are based on the perspectives of the general population may be useful for informing decisions on reimbursement and other types of priority-setting processes in health care. Incorporating the preferences of the general population may simultaneously increase public acceptance of these decisions.

Supplementary data for a model-based health economic evaluation on lung cancer screening with low-dose computed tomography in a high-risk population.

This supplementary data is supportive to the research article entitled 'Cost-effectiveness of lung cancer screening with low-dose computed tomography (LDCT) in heavy smokers: A micro-simulation modelling study' (Yihui Du et al. 2020). This supplementary contains a description of the model input and the related model output data that were not included in the research article. The input data used for the tumour growth model and the self-detected tumour size model are provided. The output data of this article include the data used for cost-effectiveness analysis of lung cancer LDCT screening with the Dutch and international discount rates, the data of the sensitivity analysis, and the data of the model validation.

Cost-effectiveness of lung cancer screening with low-dose computed tomography in heavy smokers: a microsimulation modelling study.

BACKGROUND: Lung cancer screening with low-dose computed tomography (LDCT) reduces lung cancer mortality. The aim of this study was to evaluate the cost-effectiveness of lung cancer screening with LDCT in a high-risk population. METHODS: The study used an adapted microsimulation model in a cohort of Dutch heavy smokers for a lifetime horizon from a health insurance perspective. The main outcomes included average cost-effectiveness ratio (ACER), incremental cost-effectiveness ratio (ICER) and lung cancer mortality reduction. The comparator was no screening. Scenarios with different screening intervals and starting and stopping ages were evaluated for 100,000 male heavy smokers and 100,000 female heavy smokers. A cost-effectiveness threshold of 60 k€ per life year gained (LYG) was assumed acceptable. RESULTS: The evaluated screening scenarios yielded ACERs ranging from 17.7 to 32.4 k€/LYG for men and from 17.8 to 32.1 k€/LYG for women. The lung cancer mortality reduction ranged from 9.3% to 16.8% for men and from 7.8% to 13.7% for women. The optimal screening scenario was annual screening from 55 to 80 years for men and biennial screening from 50 to 80 years for women, with an ICER of 51.6 and 45.8 k€ per LYG compared with its previous efficient alternative, respectively. Compared with no screening, the optimal screening scenario yielded an ICER of 27.6 k€/LYG for men and 21.1 k€/LYG for women. The mortality reduction of lung cancer was 15.9% for men and 10.6% for women. CONCLUSIONS: Lung cancer LDCT screening is cost-effective in a high-risk population. The optimal screening scenario is dependent on sex.

The cost-effectiveness of digital breast tomosynthesis in a population breast cancer screening program.

OBJECTIVES: To evaluate at which sensitivity digital breast tomosynthesis (DBT) would become cost-effective compared to digital mammography (DM) in a population breast cancer screening program, given a constant estimate of specificity. METHODS: In a microsimulation model, the cost-effectiveness of biennial screening for women aged 50-75 was simulated for three scenarios: DBT for women with dense breasts and DM for women with fatty breasts (scenario 1), DBT for the whole population (scenario 2) or maintaining DM screening (reference). For DM, sensitivity was varied depending on breast density from 65 to 87%, and for DBT from 65 to 100%. The specificity was set at 96.5% for both DM and DBT. Direct medical costs were considered, including screening, biopsy and treatment costs. Scenarios were considered to be cost-effective if the incremental cost-effectiveness ratio (ICER) was below €20,000 per life year gain (LYG). RESULTS: For both scenarios, the ICER was more favourable at increasing DBT sensitivity. Compared with DM screening, 0.8-10.2% more LYGs were found when DBT sensitivity was at least 75% for scenario 1, and 4.7-18.7% when DBT sensitivity was at least 80% for scenario 2. At €96 per DBT, scenario 1 was cost-effective at a DBT sensitivity of at least 90%, and at least 95% for scenario 2. At €80 per DBT, these values decreased to 80% and 90%, respectively. CONCLUSION: DBT is more likely to be a cost-effective alternative to mammography in women with dense breasts. Whether DBT could be cost-effective in a general population highly depends on DBT costs. KEY POINTS: • DBT could be a cost-effective screening modality for women with dense breasts when its sensitivity is at least 90% at a maximum cost per screen of €96. • DBT has the potential to be cost-effective for screening all women when sensitivity is at least 90% at a maximum cost per screen of €80. • Whether DBT could be used as an alternative to mammography for screening all women is highly dependent on the cost of DBT per screen.

Clinical- and Cost-Effectiveness of a Mandibular Advancement Device Versus Continuous Positive Airway Pressure in Moderate Obstructive Sleep Apnea.

STUDY OBJECTIVES: Limited evidence exists on the cost-effectiveness of mandibular advancement device (MAD) compared to continuous positive airway pressure (CPAP) therapy in moderate obstructive sleep apnea (OSA). Therefore, this study compares the clinical and cost-effectiveness of MAD therapy with CPAP therapy in moderate OSA. METHODS: In a multicentre randomized controlled trial, patients with an apnea-hypopnea index (AHI) of 15 to 30 events/h were randomized to either MAD or CPAP. Incremental cost-effectiveness and cost-utility ratios (ICER/ICUR, in terms of AHI reduction and quality-adjusted life-years [QALYs, based on the EuroQol Five-Dimension Quality of Life questionnaire]) were calculated after 12 months, all from a societal perspective. RESULTS: In the 85 randomized patients (n = 42 CPAP, n = 43 MAD), AHI reduction was significantly greater with CPAP (median reduction AHI 18.3 [14.8-22.6] events/h) than with MAD therapy (median reduction AHI 13.5 [8.5-18.4] events/h) after 12 months. Societal costs after 12 months were higher for MAD than for CPAP (mean difference €2.156). MAD was less cost-effective than CPAP after 12 months (ICER -€305 [-€3.003 to €1.572] per AHI point improvement). However, in terms of QALY, MAD performed better than CPAP after 12 months (€33.701 [-€191.106 to €562.271] per QALY gained). CONCLUSIONS: CPAP was more clinically effective (in terms of AHI reduction) and cost-effective than MAD. However, costs per QALY was better with MAD as compared to CPAP. Therefore, CPAP is the first-choice treatment option in moderate OSA and MAD may be a good alternative. CLINICAL TRIAL REGISTRATION: Registry: ClinicalTrials.gov; Identifier: NCT01588275.

Patient-Centered Item Selection for a New Preference-Based Generic Health Status Instrument: CS-Base.

OBJECTIVES: To develop patient-centered health content for a novel generic instrument (Château Santé Base [CS-Base]) that is suitable to generate values for health status. METHODS: Candidate items were drawn from existing health frameworks of generic health status instruments and placed in a diagram (HealthFANTM, Zeist, the Netherlands). Through an online survey, patients with a wide range of diseases were asked to select the 9 items that were most important to them. The importance of the items for the whole study group was determined by means of frequency distributions. RESULTS: After handling duplicates and overlap, the remaining set of 47 items was placed in the HealthFAN. Among the 2256 Dutch patients who started the survey, the most common diagnoses were neck and back pain, diabetes, and asthma/chronic obstructive pulmonary disease. The 5 health items mentioned most frequently as most important were pain, personal relationships, fatigue, memory, and vision. Hearing and vision, anxiety and depression, and independence and self-esteem seemed highly intertwined, so we chose to pair these items. CONCLUSIONS: A total of 12 health items were included in CS-Base. Its content is largely based on patient input and enables classification of patients' health status. CS-Base can be administered by means of an app on a mobile phone, which makes it a convenient and attractive tool for patients and researchers.

Physiotherapy plus conventional treatment versus conventional treatment only in the treatment of functional constipation in children: design of a randomized controlled trial and cost-effectiveness study in primary care.

BACKGROUND: Our aim was to design a study to evaluate the effectiveness and cost-effectiveness of adding physiotherapy to conventional treatment for children with functional constipation in primary care. Physiotherapy is focusing on improving the coordination between the pelvic floor and abdominal musculature during bowel movement, while conventional treatment is mainly focusing on symptomatic relief of symptoms, therefore, we expect the effects of physiotherapy will be more sustainable than the effects of conventional treatment. In this paper we describe the final study design and how the design was adapted, to overcome recruitment problems. METHODS: We designed a randomized controlled trial of children aged 4-17 years with functional constipation diagnosed by a general practitioner or pediatrician. Children in the intervention group received physiotherapy plus conventional treatment, and those in the control group received conventional treatment only. Follow-up measurements took place at 4 and 8 months. The primary outcome was treatment success defined according to the Rome-III criteria as the absence of functional constipation, with no laxative use. Secondary outcomes were absence of functional constipation irrespective of laxative use, quality of life, global perceived effect, and costs. Children were recruited from September 2014 to February 2017. Initially, we aimed to include children with recent symptom onset. However, in the first phase of enrollment we were confronted with an unforeseen recruitment problem: many children and their parents refused randomization because physiotherapy was considered too burdensome for the stage of disease. Therefore, we decided to also include children with a longer duration of symptoms. In total 134 children were included. DISCUSSION: The target number of participants is achieved. Therefore, the results may change thinking about the management of functional constipation in children. TRAIL REGISTRATION: Netherlands Trial Register ( NTR4797 ), registered 8 September 2014.