Beyond hazard ratios: appropriate statistical methods for quantifying the clinical effectiveness of immune-oncology therapies - the example of the Netherlands.

Background: The Dutch Committee for the Evaluation of Oncological Drugs evaluates the effectiveness of new oncological treatments. The committee compares survival endpoints to the so-called PASKWIL-2023 criteria for palliative treatments, which define if treatment effects are considered clinically relevant. A positive recommendation depends on whether the median overall survival (OS) is below or above 12 months in the comparator arm.

A cloud-based medical device for predicting cardiac risk in suspected coronary artery disease: a rapid review and conceptual economic model.

Background: The CaRi-Heart® device estimates risk of 8-year cardiac death, using a prognostic model, which includes perivascular fat attenuation index, atherosclerotic plaque burden and clinical risk factors.

Objectives: To provide an Early Value Assessment of the potential of CaRi-Heart Risk to be an effective and cost-effective adjunctive investigation for assessment of cardiac risk, in people with stable chest pain/suspected coronary artery disease, undergoing computed tomography coronary angiography. This assessment includes conceptual modelling which explores the structure and evidence about parameters required for model development, but not development of a full executable cost-effectiveness model.

The impact of different perspectives on the cost-effectiveness of remote patient monitoring for patients with heart failure in different European countries.

Background And Objective: Heart failure (HF) is a complex clinical syndrome with high mortality and hospitalization rates. Non-invasive remote patient monitoring (RPM) interventions have the potential to prevent disease worsening. However, the long-term cost-effectiveness of RPM remains unclear.

Cost-effectiveness of remote haemodynamic monitoring by an implantable pulmonary artery pressure monitoring sensor (CardioMEMS-HF system) in chronic heart failure in the Netherlands.

Aims: Remote haemodynamic monitoring with an implantable pulmonary artery (PA) sensor has been shown to reduce heart failure (HF) hospitalizations and improve quality of life. Cost-effectiveness analyses studying the value of remote haemodynamic monitoring in a European healthcare system with a contemporary standard care group are lacking.

Methods And Results: A Markov model was developed to estimate the cost-effectiveness of PA-guided therapy compared to the standard of care based upon patient-level data of the MONITOR-HF trial performed in the Netherlands in patients with chronic HF (New York Heart Association class III and at least one previous HF hospitalization).

Effectiveness of Seizure Dogs for People With Severe Refractory Epilepsy: Results From the EPISODE Study.

Background And Objectives: The aim of this study was to evaluate whether people living with severe medically refractory epilepsy (PSRE) benefit from a seizure dog.

Methods: An individual-level stepped-wedge randomized controlled trial was conducted. The study was conducted in the Netherlands among adults with daily to weekly seizures.

Doxapram versus placebo in preterm newborns: a study protocol for an international double blinded multicentre randomized controlled trial (DOXA-trial).

Background: Apnoea of prematurity (AOP) is one of the most common diagnoses among preterm infants. AOP often leads to hypoxemia and bradycardia which are associated with an increased risk of death or disability. In addition to caffeine therapy and non-invasive respiratory support, doxapram might be used to reduce hypoxemic episodes and the need for invasive mechanical ventilation in preterm infants, thereby possibly improving their long-term outcome.

Music to prevent deliriUm during neuroSurgerY (MUSYC): a single-centre, prospective randomised controlled trial.

Objectives: Delirium is a serious complication following neurosurgical procedures. We hypothesise that the beneficial effect of music on a combination of delirium-eliciting factors might reduce delirium incidence following neurosurgery and subsequently improve clinical outcomes.

Design: Prospective randomised controlled trial.

Single-use duodenoscopes compared with reusable duodenoscopes in patients carrying multidrug-resistant microorganisms: a break-even cost analysis.

 Single-use duodenoscopes can prevent transmission of microorganisms through contaminated reusable duodenoscopes. Concerns regarding their economic and environmental impact impede the transition to single-use duodenoscopes. This study investigated the costs associated with two scenarios in which single-use duodenoscopes are used in patients carrying multidrug-resistant microorganisms (MDROs).

The lifetime health and economic burden of obesity in five European countries: what is the potential impact of prevention?

Aim: Estimating the burden of obesity in five European countries (Germany, Greece, the Netherlands, Spain and the UK) and the potential health benefits and changes in health care costs associated with a reduction in body mass index (BMI).

Materials And Methods: A Markov model was used to estimate the long-term burden of obesity. Health states were based on the occurrence of diabetes, ischaemic heart disease and stroke.

The Value of Health in a Cost-Effectiveness Analysis: Theory Versus Practice.

A cost-effectiveness analysis has become an important method to inform allocation decisions and reimbursement of new technologies in healthcare. A cost-effectiveness analysis requires a threshold to which the cost effectiveness of a new intervention can be compared. In principle, the threshold ought to reflect opportunity costs of reimbursing a new technology.

In search of a 'pan-European value set'; application for EQ-5D-3L.

Objectives: Country-specific value sets for the EQ-5D are available which reflect preferences for health states elicited from the general population. This allows the transformation of responses on EQ-5D to health state utility values. Only twelve European countries possess country-specific value sets and no value set reflecting the preferences of Europe exists.

Assessment, management, and incidence of neonatal jaundice in healthy neonates cared for in primary care: a prospective cohort study.

Jaundice caused by hyperbilirubinaemia is a common phenomenon during the neonatal period. Population-based studies evaluating assessment, management, and incidence of jaundice and need for phototherapy among otherwise healthy neonates are scarce. We prospectively explored these aspects in a primary care setting via assessing care as usual during the control phase of a stepped wedge cluster randomised controlled trial.

Future medical and non-medical costs and their impact on the cost-effectiveness of life-prolonging interventions: a comparison of five European countries.

When healthcare interventions prolong life, people consume medical and non-medical goods during the years of life they gain. It has been argued that the costs for medical consumption should be included in cost-effectiveness analyses from both a healthcare and societal perspective, and the costs for non-medical consumption should additionally be included when a societal perspective is applied. Standardized estimates of these so-called future costs are available in only a few countries and the impact of inclusion of these costs is likely to differ between countries.

Performance of the EQ-5D-5L Plus Respiratory Bolt-On in the Birmingham Chronic Obstructive Pulmonary Disease Cohort Study.

Objectives: A respiratory bolt-on dimension for the EQ-5D-5L has recently been developed and valued by the general public. This study aimed to validate the EQ-5D-5L plus respiratory dimension (EQ-5D-5L+R) in a large group of patients with chronic obstructive pulmonary disease (COPD).

Methods: Validation was undertaken with data from the Birmingham COPD Cohort Study, a longitudinal UK study of COPD primary care patients.

The Potential Impact of Digital Biomarkers in Multiple Sclerosis in The Netherlands: An Early Health Technology Assessment of MS Sherpa.

(1) : Monitoring of Multiple Sclerosis (MS) with eHealth interventions or digital biomarkers provides added value to the current care path. Evidence in the literature is currently scarce. MS sherpa is an eHealth intervention with digital biomarkers, aimed at monitoring symptom progression and disease activity.

Patient, family and productivity costs of end-stage renal disease in the Netherlands; exposing non-healthcare related costs.

Background: Healthcare costs related to ESRD are well-described, but broader societal costs of ESRD are less known. This study aimed to estimate patient and family costs, including informal care costs and out-of-pocket costs, and costs due to productivity loss related to ESRD, for patients receiving dialysis and living with a kidney transplant, using a bottom-up approach.

Methods: A total of 655 patients were asked to complete a digital questionnaire consisting of two standardised instruments (iMCQ and iPCQ) from November 2016 through January 2017.

Quality of life among people living with HIV in England and the Netherlands: a population-based study.

Background: HIV is now considered a chronic condition, and people living with HIV, when treated, have a similar life expectancy as compared to the general population. Consequently, improving and ensuring a good health-related quality of life (HrQoL) among people living with HIV (people living with HIV) is increasingly important and has risen on the global agenda in recent years. A 'fourth 90' as 90% of people with viral load suppression have a good HrQoL should therefore be adopted alongside the other 90-90-90 targets.

Productivity of Working at Home and Time Allocation Between Paid Work, Unpaid Work and Leisure Activities During a Pandemic.

Background And Objective: The COVID-19 pandemic and the measures taken by governments to contain it have affected many aspects of the daily lives of citizens. This study aimed to describe changes in the productivity of paid work and time allocation to paid and unpaid work and leisure resulting from working at home during the pandemic.

Methods: A sample of 851 people from the Netherlands who had paid work (≥ 24 h/week) and worked at least 4 hours per week extra at home because of lockdown measures completed a questionnaire during the first COVID-19 lockdown (April 2020).

Cost-effectiveness of the fixed-dose combination tiotropium/olodaterol versus tiotropium monotherapy or a fixed-dose combination of long-acting β2-agonist/inhaled corticosteroid for COPD in Finland, Sweden and the Netherlands: a model-based study.

Objectives: Chronic obstructive pulmonary disease (COPD) guidelines advocate treatment with combinations of long-acting bronchodilators for patients with COPD who have persistent symptoms or continue to have exacerbations while using a single bronchodilator. This study assessed the cost-utility of the fixed dose combination of the bronchodilators tiotropium and olodaterol versus two comparators, tiotropium monotherapy and long-acting β2 agonist/inhaled corticosteroid (LABA/ICS) combinations, in three European countries: Finland, Sweden and the Netherlands.

Methods: A previously published COPD patient-level discrete event simulation model was updated with most recent evidence to estimate lifetime quality-adjusted life years (QALYs) and costs for COPD patients receiving either tiotropium/olodaterol, tiotropium monotherapy or LABA/ICS.

Evaluating Complex Health and Social Care Program Using Multi-Criteria Decision Analysis: A Case Study of "Better Together in Amsterdam North".

Objectives: Multi-criteria decision analysis (MCDA) has been recommended to support policy making in healthcare. However, practical applications of MCDA are sparse. One potential use for MCDA is for the evaluation of programs for complex and vulnerable patients.

ACL reconstruction for all is not cost-effective after acute ACL rupture.

Objectives: To conduct a cost-utility analysis for two commonly used treatment strategies for patients after ACL rupture; early ACL reconstruction (index) versus rehabilitation plus an optional reconstruction in case of persistent instability (comparator).

Methods: Patients aged between 18 and 65 years of age with a recent ACL rupture (<2 months) were randomised between either an early ACL reconstruction (index) or a rehabilitation plus an optional reconstruction in case of persistent instability (comparator) after 3 months of rehabilitation. A cost-utility analysis was performed to compare both treatments over a 2-year follow-up.

Methodological Challenges in the Economic Evaluation of a Gene Therapy for RPE65-Mediated Inherited Retinal Disease: The Value of Vision.

The emergence of gene therapies challenge health economists to evaluate interventions that are often provided to a small patient population with a specific gene mutation in a single dose with high upfront costs and uncertain long-term benefits. The objective of this study was to illustrate the methodological challenges of evaluating gene therapies and their implications by discussing four economic evaluations of voretigene neparvovec (VN) for the treatment of RPE65-mediated inherited retinal disease. The checklist for economic evaluations of gene therapies of Drummond et al.

Cost-utility analysis of four common surgical treatment pathways for breast cancer.

Background: The aim was to evaluate the cost-utility of four common surgical treatment pathways for breast cancer: mastectomy, breast-conserving therapy (BCT), implant breast reconstruction (BR) and autologous-BR.

Methods: Patient-level healthcare consumption data and results of a large quality of life (QoL) study from five Dutch hospitals were combined. The cost-effectiveness was assessed in terms of incremental costs and quality adjusted life years (QALYs) over a 10-year follow-up period.