Cost-Utility Analysis of Treatment Sequences for Moderate-to-Severe Crohn's Disease.

Objectives: The clinical pathway for patients with moderate-to-severe Crohn's disease (CD) typically includes sequential pharmacologic treatment as well as surgery, but positioning of different therapies within these sequences remains challenging. Cost-utility analysis rarely captures these sequences and does not incorporate registry data on long-term effectiveness. In this study, we aim to overcome these limitations.

Exit strategies in patients with stable MS: Cost-effectiveness of extended interval dosing of ocrelizumab and natalizumab versus de-escalating to cladribine.

Background: Long-term treatment with standard interval dosing (SID) exposes MS patients to risks, but stopping treatment risks progression. Alternatives to stopping are 'exit strategies', such as extended interval dosing (EID) or de-escalating infection risks by switching to other DMTs.

Objective: To compare the cost-effectiveness of exit strategies for MS patients with stable disease on second line ocrelizumab or natalizumab.

Estimating the Prognostic Value of the NTRK Fusion Biomarker for Comparative Effectiveness Research in The Netherlands.

Objectives: We evaluated the prognostic value of the neurotrophic tyrosine receptor kinase (NTRK) gene fusions by comparing the survival of patients with NTRK+ tumours with patients without NTRK+ tumours.

Methods: We used genomic and clinical registry data from the Center for Personalized Cancer Treatment (CPCT-02) study containing a cohort of cancer patients who were treated in Dutch clinical practice between 2012 and 2020. We performed a propensity score matching analysis, where NTRK+ patients were matched to NTRK- patients in a 1:4 ratio.

Benefits of sphingosine-1-phosphate receptor modulators in relapsing MS estimated with a treatment sequence model.

Background: Three sphingosine-1-phosphate receptor (S1PR) modulators are currently available as disease-modifying therapies (DMTs) for relapsing MS in the Netherlands (i.e. fingolimod, ozanimod and ponesimod).

Societal Utilities for Cognitive Impairment in Schizophrenia: Developing a Preference-Based Scoring Algorithm Based on the Schizophrenia Cognition Rating Scale.

Introduction: Loss of cognitive function is a common feature in schizophrenia. However, generic measures of health-related quality of life favored by decision-makers, such as the EQ-5D, are not designed to detect changes in cognitive function. We report the valuation of the Schizophrenia Cognition Rating Scale (SCoRS), a schizophrenia-specific measure of cognitive impairment.

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.

Effectiveness and Cost-Effectiveness of 360 Disease-Modifying Treatment Escalation Sequences in Multiple Sclerosis.

Objectives: The rapid expansion in treatment options for relapsing-remitting multiple sclerosis (RRMS) of the past decade requires clinical decision making on the sequential prescription of these treatments. Here, we compare 360 treatment escalation sequences for patients with RRMS in terms of health outcomes and societal costs in The Netherlands.

Methods: We use a microsimulation model with a societal perspective, developed in collaboration with MS neurologists, to estimate the effectiveness and cost-effectiveness of 360 treatment sequences starting with first-line therapies in RRMS.

Correcting for discounting and loss aversion in composite time trade-off.

Time trade-off utilities have been suggested to be biased upwards. This bias is a result of the method being applied assuming linear utility of life duration, which is violated when individuals discount future life years or are loss averse for health. Applying a "corrective approach", that is, measuring individuals' discount function and loss aversion and correcting time trade-off utilities for these individual characteristics, may reduce this bias in utilities.

Incremental benefits of novel pharmaceuticals in the UK: a cross-sectional analysis of NICE technology appraisals from 2010 to 2020.

Objectives: To evaluate the incremental value of new drugs across disease areas receiving favourable coverage decisions by the UK's National Institute for Health and Care Excellence (NICE) over the past decade.

Design, Setting, And Participants: This cross-sectional study assessed favourable appraisal decisions of drugs between 1 January 2010 and 31 December 2020. Estimates of incremental benefit were extracted from NICE's evidence review groups reports.

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.

Burden of Providing Informal Care for Patients with Atrial Fibrillation.

Objectives: Patients with atrial fibrillation (AF) have rapid and irregular heart rates, increasing the risk of comorbidities and mortality. Next to formal medical care, many patients receive informal care from their social environment. The objective of this study was to examine the well-being and economic burden of providing informal care to patients with AF in the UK, Italy, and Germany.

Evaluation of the performance of algorithms mapping EORTC QLQ-C30 onto the EQ-5D index in a metastatic colorectal cancer cost-effectiveness model.

Background: Cost-effectiveness models require quality of life utilities calculated from generic preference-based questionnaires, such as EQ-5D. We evaluated the performance of available algorithms for QLQ-C30 conversion into EQ-5D-3L based utilities in a metastatic colorectal cancer (mCRC) patient population and subsequently developed a mCRC specific algorithm. Influence of mapping on cost-effectiveness was evaluated.

Summarizing Patient Preferences for the Competitive Landscape of Multiple Sclerosis Treatment Options.

Quantitatively summarize patient preferences for European licensed relapsing-remitting multiple sclerosis (RRMS) disease-modifying treatment (DMT) options. To identify and summarize the most important RRMS DMT characteristics, a literature review, exploratory physician interviews, patient focus groups, and confirmatory physician interviews were conducted in Germany, the United Kingdom, and the Netherlands. A discrete choice experiment (DCE) was developed and executed to measure patient preferences for the most important DMT characteristics.

Comparing the effectiveness and cost-effectiveness of self-management interventions in four high-priority chronic conditions in Europe (COMPAR-EU): a research protocol.

Introduction: Population ageing and increasing chronic illness burden have sparked interest in innovative care models. While self-management interventions (SMIs) are drawing increasing attention, evidence of their efficacy is mostly based on pairwise meta-analysis, generally derived from randomised controlled trials comparing interventions versus a control or no intervention. As such, relevant efficacy data for comparisons among different SMIs that can be applied to specific chronic conditions are missing.

[Loss is gain? Discarding TNF-α inhibitors].

According to a recent study, stopping the prescription of TNF inhibitors is a cost-effective decision at various willingness-to-accept thresholds. Discontinuing the prescription of the drug may lead to a minor loss in health - expressed in quality adjusted life years - but results in significant societal savings. In our commentary, we stress that willingness-to-pay thresholds should not be completely replaced by the willingness-to-accept threshold, also when it concerns health losses.

Estimating Informal Caregiving Time from Patient EQ-5D Data: The Informal CARE Effect (iCARE) Tool.

Background: Families and friends provide a considerable proportion of care for patients and elderly people. Caregiving can have substantial effects on caregivers' lives, health, and well-being. However, because clinical trials rarely assess these effects, no information on caregiver burden is available when evaluating the cost effectiveness of treatments.

Cost Effectiveness of the Angiotensin Receptor Neprilysin Inhibitor Sacubitril/Valsartan for Patients with Chronic Heart Failure and Reduced Ejection Fraction in the Netherlands: A Country Adaptation Analysis Under the Former and Current Dutch Pharmacoeconomic Guidelines.

Objectives: To describe the adaptation of a global health economic model to determine whether treatment with the angiotensin receptor neprilysin inhibitor LCZ696 is cost effective compared with the angiotensin-converting enzyme inhibitor enalapril in adult patients with chronic heart failure with reduced left ventricular ejection fraction in the Netherlands; and to explore the effect of performing the cost-effectiveness analyses according to the new pharmacoeconomic Dutch guidelines (updated during the submission process of LCZ696), which require a value-of-information analysis and the inclusion of indirect medical costs of life-years gained.

Methods: We adapted a UK model to reflect the societal perspective in the Netherlands by including travel expenses, productivity loss, informal care costs, and indirect medical costs during the life-years gained and performed a preliminary value-of-information analysis.

Results: The incremental cost-effectiveness ratio obtained was €17,600 per quality-adjusted life-year (QALY) gained.

Are Health State Valuations from the General Public Biased? A Test of Health State Reference Dependency Using Self-assessed Health and an Efficient Discrete Choice Experiment.

Health state valuations of patients and non-patients are not the same, whereas health state values obtained from general population samples are a weighted average of both. The latter constitutes an often-overlooked source of bias. This study investigates the resulting bias and tests for the impact of reference dependency on health state valuations using an efficient discrete choice experiment administered to a Dutch nationally representative sample of 788 respondents.

Dutch Tariff for the Five-Level Version of EQ-5D.

Background: In 2009, a new version of the EuroQol five-dimensional questionnaire (EQ-5D) was introduced with five rather than three answer levels per dimension. This instrument is known as the EQ-5D-5L. To make the EQ-5D-5L suitable for use in economic evaluations, societal values need to be attached to all 3125 health states.

Which factors may determine the necessary and feasible type of effectiveness evidence? A mixed methods approach to develop an instrument to help coverage decision-makers.

Objectives: Reimbursement decisions require evidence of effectiveness and, in general, a blinded randomised controlled trial (RCT) is the preferred study design to provide it. However, there are situations where a cohort study, or even patient series, can be deemed acceptable. The aim of this study was to develop an instrument that first examines which study characteristics of a blinded RCT are necessary, and then, if particular characteristics are considered necessary, examines whether these characteristics are feasible.

Testing mapping algorithms of the cancer-specific EORTC QLQ-C30 onto EQ-5D in malignant mesothelioma.

Background: In order to estimate utilities for cancer studies where the EQ-5D was not used, the EORTC QLQ-C30 can be used to estimate EQ-5D using existing mapping algorithms. Several mapping algorithms exist for this transformation, however, algorithms tend to lose accuracy in patients in poor health states. The aim of this study was to test all existing mapping algorithms of QLQ-C30 onto EQ-5D, in a dataset of patients with malignant pleural mesothelioma, an invariably fatal malignancy where no previous mapping estimation has been published.

Time trade-off: one methodology, different methods.

There is no scientific consensus on the optimal specification of the time trade-off (TTO) task. As a consequence, studies using TTO to value health states may share the core element of trading length of life for quality of life, but can differ considerably on many other elements. While this pluriformity in specifications advances the understanding of TTO from a methodological point of view, it also results in incomparable health state values.

Time to tweak the TTO: results from a comparison of alternative specifications of the TTO.

This article examines the effect that different specifications of the time trade-off (TTO) valuation task may have on values for EQ-5D-5L health states. The new variants of the TTO, namely lead-time TTO and lag-time TTO, along with the classic approach to TTO were compared using two durations for the health states (15 and 20 years). The study tested whether these methods yield comparable health-state values.