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.

How microsimulation translates outcome estimates to patient lifetime event occurrence in the setting of heart valve disease.

Treatment decisions in healthcare often carry lifelong consequences that can be challenging to foresee. As such, tools that visualize and estimate outcome after different lifetime treatment strategies are lacking and urgently needed to support clinical decision-making in the setting of rapidly evolving healthcare systems, with increasingly numerous potential treatments. In this regard, microsimulation models may prove to be valuable additions to current risk-prediction models.

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).

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.

Being Transparent About Brilliant Failures: An Attempt to Use Real-World Data in a Disease Model for Patients with Castration-Resistant Prostate Cancer.

Background: Real-world disease models spanning multiple treatment lines can provide insight into the (cost) effectiveness of treatment sequences in clinical practice.

Objective: Our objective was to explore whether a disease model based solely on real-world data (RWD) could be used to estimate the effectiveness of treatments for patients with castration-resistant prostate cancer (CRPC) that could then be suitably used in a cost-effectiveness analysis.

Methods: We developed a patient-level simulation model using patient-level data from the Dutch CAPRI registry as input parameters.

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.

Early cost-utility analysis of tissue-engineered heart valves compared to bioprostheses in the aortic position in elderly patients.

Objectives: Aortic valve disease is the most frequent indication for heart valve replacement with the highest prevalence in elderly. Tissue-engineered heart valves (TEHV) are foreseen to have important advantages over currently used bioprosthetic heart valve substitutes, most importantly reducing valve degeneration with subsequent reduction of re-intervention. We performed early Health Technology Assessment of hypothetical TEHV in elderly patients (≥ 70 years) requiring surgical (SAVR) or transcatheter aortic valve implantation (TAVI) to assess the potential of TEHV and to inform future development decisions.

Outcomes after surgery for functional tricuspid regurgitation: a systematic review and meta-analysis.

Aims: This study aims to provide a contemporary overview of outcomes after tricuspid valve (TV) surgery for functional tricuspid regurgitation (TR).

Methods And Results: The literature was systematically searched for papers published between January 2005 and December 2017 reporting on clinical/echocardiographic outcomes after TV surgery for functional TR. A random effects meta-analysis was conducted for outcome variables, and late outcomes are visualized by pooled Kaplan-Meier curves.

Bioprosthetic Aortic Valve Replacement in Nonelderly Adults: A Systematic Review, Meta-Analysis, Microsimulation.

Background To support decision-making in aortic valve replacement in nonelderly adults, we aim to provide a comprehensive overview of reported outcome after bioprosthetic aortic valve replacement and to translate this to age-specific patient outcome estimates. Methods and Results A systematic review was conducted for papers reporting clinical outcome after aortic valve replacement with currently available bioprostheses in patients with a mean age <55 years, published between January 1, 2000, and January 9, 2016. Pooled reported event rates and time-to-event data were pooled and entered into a microsimulation model to calculate life expectancy and lifetime event risk for the ages of 25, 35, 45, and 55 years at surgery.

What Is the Potential of Tissue-Engineered Pulmonary Valves in Children?

Background: As a living heart valve substitute with growth potential and improved durability, tissue-engineered heart valves (TEHVs) may prevent reinterventions that are currently often needed in children with congenital heart disease. We performed early health technology assessment to assess the potential cost-effectiveness of TEHVs in children requiring right ventricular outflow tract reconstruction (RVOTR).

Methods: A systematic review and meta-analysis was conducted of studies reporting clinical outcome after RVOTR with existing heart valve substitutes in children (mean age ≤12 years or maximum age ≤21 years) published between January 1, 2000, and May 2, 2018.

The Ross Procedure: A Systematic Review, Meta-Analysis, and Microsimulation.

Background: To support decision-making in aortic valve replacement in children and adults, we provide a comprehensive overview of outcome after the Ross procedure.

Methods And Results: A systematic search was conducted for publications reporting clinical outcome after the Ross procedure, published between January 1, 2000, and November 22, 2017. Reported event rates and time-to-event data were pooled and entered into a microsimulation model to calculate life expectancy and lifetime event risk.

Bioprosthetic aortic valve replacement in elderly patients: Meta-analysis and microsimulation.

Objective: To support decision-making in aortic valve replacement (AVR) in elderly patients, we provide a comprehensive overview of outcome after AVR with bioprostheses.

Methods: A systematic review was conducted of studies reporting clinical outcome after AVR with bioprostheses in elderly patients (mean age ≥70 years; minimum age ≥65 years) published between January 1, 2000, to September 1, 2016. Reported event rates and time-to-event data were pooled and entered into a microsimulation model to calculate life expectancy and lifetime event risks.

Beyond the clinical impact of aortic and pulmonary valve implantation: health-related quality of life, informal care and productivity†.

Objectives: Our aim was to provide estimates of patient-reported health-related quality of life (HRQoL), use of informal care and productivity in patients after surgical aortic and pulmonary valve replacement and transcatheter aortic valve implantation.

Methods: Consecutive cohorts of 1239 adult patients who had surgical aortic valve replacement or surgical pulmonary valve replacement and 433 patients who had transcatheter aortic valve implantation at 2 Dutch heart centres were cross-sectionally surveyed at a median time of 2.9 and 3.

How much does a heart valve implantation cost and what are the health care costs afterwards?

Objective: In the era of limited healthcare budgets, healthcare costs of heart valve implantations need to be considered to inform cost-effectiveness analyses. We aimed to provide age group-specific costs estimates of heart valve implantations, related complications and other healthcare utilisation following the intervention.

Methods: We performed retrospective analyses of healthcare costs of patients who had undergone heart valve implantations in 2010-2013 and controls using claims data from Dutch health insurers.

Systematic review of model-based economic evaluations of heart valve implantations.

Objective: To review the evidence on the cost-effectiveness of heart valve implantations generated by decision analytic models and to assess their methodological quality.

Methods: A systematic review was performed including model-based cost-effectiveness analyses of heart valve implantations. Study and model characteristics and cost-effectiveness results were extracted and the methodological quality was assessed using the Philips checklist.

Contemporary outcomes after surgical aortic valve replacement with bioprostheses and allografts: a systematic review and meta-analysis.

Many observational studies have reported outcomes after surgical aortic valve replacement (AVR), but there are no recent systematic reviews and meta-analyses including all available bioprostheses and allografts. The objective of this study is to provide a comprehensive and up-to-date overview of the outcomes after AVR with bioprostheses and allografts reported in the last 15 years. We conducted a systematic literature review (PROSPERO register: CRD42015017041) of studies published between 2000-15.

Exploring the variation in implementation of a COPD disease management programme and its impact on health outcomes: a post hoc analysis of the RECODE cluster randomised trial.

This study aims to (1) examine the variation in implementation of a 2-year chronic obstructive pulmonary disease (COPD) management programme called RECODE, (2) analyse the facilitators and barriers to implementation and (3) investigate the influence of this variation on health outcomes. Implementation variation among the 20 primary-care teams was measured directly using a self-developed scale and indirectly through the level of care integration as measured with the Patient Assessment of Chronic Illness Care (PACIC) and the Assessment of Chronic Illness Care (ACIC). Interviews were held to obtain detailed information regarding the facilitators and barriers to implementation.