Systematic review of cost-effectiveness modelling studies for haemophilia.

Aims: Haemophilia is a rare genetic disease that hinders blood clotting. We aimed to review model-based cost-effectiveness analyses (CEAs) of haemophilia treatments, describe the sources of clinical evidence used by these CEAs, summarize the reported cost-effectiveness of different treatment strategies, and assess the quality and risk of bias.

Methods: We conducted a systematic literature review of model-based CEAs of haemophilia treatments by searching databases, the Tufts Medical Center CEA registry, and grey literature.

Correction: Olaparib not cost-effective as maintenance therapy for platinum-sensitive, BRCA1/2 germline-mutated metastatic pancreatic cancer.

[This corrects the article DOI: 10.1371/journal.pone.

Transcatheter aortic valve implantation with SAPIEN 3 versus surgical aortic valve replacement in patients with symptomatic severe aortic stenosis at low risk of surgical mortality: a cost-utility analysis for Switzerland.

Aims Of The Study: The 2021 European Society of Cardiology Guidelines on valvular heart disease recommend transcatheter aortic valve implantation (TAVI) for patients with symptomatic severe aortic stenosis at low surgical risk and age ≥75 years who are suitable for a transfemoral approach (recommendation class IA) based on two large randomised controlled trials (PARTNER 3 and Evolut Low Risk) comparing transcatheter aortic valve implantation with surgical aortic valve replacement (SAVR). Whether such an approach is cost-effective in Switzerland remains unclear. The aim of this cost-utility analysis was to compare transcatheter aortic valve implantation with SAPIEN 3 versus surgical aortic valve replacement in symptomatic severe aortic stenosis patients at low risk of surgical mortality from the perspective of Swiss compulsory health insurance using data from the PARTNER 3 trial (reflecting specifically the safety and efficacy of the SAPIEN 3 TAVI device).

Design and rationale for an empirical investigation of the resource use and costs of investigator-initiated randomized trials in Switzerland, the UK, and Germany.

Background: Conducting high-quality randomized clinical trials (RCTs) is challenging, time consuming, and resource intense. Academic investigators usually depend on scarce financial resources; however, current literature lacks systematically collected empirical data on the detailed resource use and costs of investigator-initiated RCTs.

Methods: The aim of this study is to generate a database of detailed empirical resource use and cost data from 100 investigator-initiated RCTs in Switzerland, Germany, and the UK.

Resource use and costs of investigator-sponsored randomized clinical trials in Switzerland, Germany, and the United Kingdom: a metaresearch study.

Background And Objectives: Conducting high-quality randomized clinical trials (RCTs) is challenging and resource intensive. Funders and academic investigators depend on limited financial resources and, therefore, need empirical data for optimal budget planning. However, current literature lacks detailed empirical data on resource use and costs of investigator-sponsored RCTs.

MultiSCRIPT-Cycle 1-a pragmatic trial embedded within the Swiss Multiple Sclerosis Cohort (SMSC) on neurofilament light chain monitoring to inform personalized treatment decisions in multiple sclerosis: a study protocol for a randomized clinical trial.

Background: Treatment decisions for persons with relapsing-remitting multiple sclerosis (RRMS) rely on clinical and radiological disease activity, the benefit-harm profile of drug therapy, and preferences of patients and physicians. However, there is limited evidence to support evidence-based personalized decision-making on how to adapt disease-modifying therapy treatments targeting no evidence of disease activity, while achieving better patient-relevant outcomes, fewer adverse events, and improved care. Serum neurofilament light chain (sNfL) is a sensitive measure of disease activity that captures and prognosticates disease worsening in RRMS.

The association between medication use and health-related quality of life in multimorbid older patients with polypharmacy.

Purpose: To explore the association between medication use-related factors and health-related quality of life (HRQoL) in older hospitalised multimorbid patients with polypharmacy.

Methods: This cross-sectional study used the intervention arm data of the OPERAM trial (hospitalised patients ≥ 70 years with polypharmacy). HRQoL was assessed using the visual analogue scale (EQ-VAS) and the EQ-5D index score of the EuroQol questionnaire (EQ-5D-5L).

Herding-like behaviour in medical decision making: An experimental study investigating general practitioners' prescription behaviour.

Previous observational studies have indicated that social influences, such as arising from herding-like behaviour, can contribute to medical errors. In this study, we experimentally examined whether general practitioners (GPs) would follow incorrect prescription recommendations from fellow GP or specialists. To investigate this, we conducted an online survey with 475 GPs practicing in England that included two case vignettes.

Olaparib not cost-effective as maintenance therapy for platinum-sensitive, BRCA1/2 germline-mutated metastatic pancreatic cancer.

Objective: To assess the cost-effectiveness and budget impact of olaparib as a maintenance therapy in platinum-responsive, metastatic pancreatic cancer patients harboring a germline BRCA1/2 mutation, using the Swiss context as a model.

Methods: Based on data from the POLO trial, published literature and local cost data, we developed a partitioned survival model of olaparib maintenance including full costs for BRCA1/2 germline testing compared to FOLFIRI maintenance chemotherapy and watch-and-wait. We calculated the incremental cost-effectiveness ratio (ICER) for the base case and several scenario analyses and estimated 5-year budget impact.

Cost-Effectiveness Analysis of Etranacogene Dezaparvovec Versus Extended Half-Life Prophylaxis for Moderate-to-Severe Haemophilia B in Germany.

Background And Objective: Haemophilia B is a rare genetic disease that is caused by a deficiency of coagulation factor IX (FIX) in the blood and leads to internal and external bleeding. Under the current standard of care, haemophilia is treated either prophylactically or on-demand via intravenous infusions of FIX. These treatment strategies impose a high burden on patients and health care systems as haemophilia B requires lifelong treatment, and FIX is costly.

Patients on vitamin K treatment: is switching to direct-acting oral anticoagulation cost-effective? A target trial on a prospective cohort.

Aims: Direct-acting oral anticoagulants (DOACs) have, to a substantial degree, replaced vitamin K antagonists (VKA) as treatments for stroke prevention in atrial fibrillation (AF) patients. However, evidence on the real-world causal effects of switching patients from VKA to DOAC is lacking. We aimed to assess the empirical incremental cost-effectiveness of switching patients to DOAC compared with maintaining VKA treatment.

Prevention of psychosocial distress consequences in somatic hospital inpatients via a stepped and collaborative care model: protocol of SomPsyNet, a stepped wedge cluster randomised trial.

Introduction: Approximately 30% of somatic hospital inpatients experience psychosocial distress, contributing to increased (re-)hospitalisation rates, treatment resistance, morbidity, and direct and indirect costs. However, such distress often remains unrecognised and unaddressed. We established 'SomPsyNet', a 'stepped and collaborative care model' (SCCM) for somatic hospital inpatients, aiming at alleviating this issue through early identification of distress and provision of appropriate care, providing problem-focused pathways and strengthening collaborative care.

Longitudinal Changes in Health-Related Quality of Life in Patients With Atrial Fibrillation.

Background Optimizing health-related quality of life (HRQoL) is an important aim of atrial fibrillation (AF) treatment. Little is known about patients' long-term HRQoL trajectories and the impact of patient and disease characteristics. The aim of this study was to describe HRQoL trajectories in an observational AF study population and in clusters of patients with similar patient and disease characteristics.

Real-World Cost-Effectiveness of Pulmonary Vein Isolation for Atrial Fibrillation: A Target Trial Approach.

Objectives: Randomized controlled trials of pulmonary vein isolation (PVI) for treating atrial fibrillation (AF) have proven the procedure's efficacy. Studies assessing its empirical cost-effectiveness outside randomized trial settings are lacking. We aimed to evaluate the effectiveness and cost-effectiveness of PVI versus medical therapy for AF.

Estimating the cost impact of atrial fibrillation using a prospective cohort study and population-based controls.

Aims: Atrial fibrillation (AF) costs are expected to be substantial, but cost comparisons with the general population are scarce. Using data from the prospective Swiss-AF cohort study and population-based controls, we estimated the impact of AF on direct healthcare costs from the Swiss statutory health insurance perspective.

Methods: Swiss-AF patients, enrolled from 2014 to 2017, had documented, prevalent AF.

Facilitators and barriers of routine psychosocial distress assessment within a stepped and collaborative care model in a Swiss hospital setting.

Background: Stepped and Collaborative Care Models (SCCMs) have shown potential for improving mental health care. Most SCCMs have been used in primary care settings. At the core of such models are initial psychosocial distress assessments commonly in form of patient screening.

General Practitioners' Awareness and Perception of Current Pneumococcal Vaccination for Adult Patients with Known Risk Factors in Switzerland: Evidence from a Survey.

In Switzerland, the National Immunization Advisory Group (NITAG) has formulated recommendations for pneumococcal vaccination among adult risk patients. Little is known about general practitioners' (GPs') perception, knowledge, and implementation of these recommendations. Therefore, we investigated GPs' awareness and drivers of and barriers to pneumococcal vaccination using a cross-sectional web-based survey of GPs.

Determining the Relationship Between Seizure-Free Days and Other Predictors of Quality of Life in Patients with Dravet Syndrome and Their Carers from FFA Registration Studies.

Introduction: Dravet syndrome (DS) is a rare, lifelong epileptic encephalopathy characterised by frequent and severe seizures associated with premature mortality. Typically diagnosed in infancy, patients also experience progressive behavioural, motor-function and cognitive decline. Twenty percent of patients do not reach adulthood.

Optimising prescribing in older adults with multimorbidity and polypharmacy in primary care (OPTICA): cluster randomised clinical trial.

Objective: To study the effects of a primary care medication review intervention centred around an electronic clinical decision support system (eCDSS) on appropriateness of medication and the number of prescribing omissions in older adults with multimorbidity and polypharmacy compared with a discussion about medication in line with usual care.

Design: Cluster randomised clinical trial.

Setting: Swiss primary care, between December 2018 and February 2021.

Benzodiazepine Receptor Agonists Use and Cessation Among Multimorbid Older Adults with Polypharmacy: Secondary Analysis from the OPERAM Trial.

Background: Benzodiazepine receptor agonists (BZRAs) are commonly prescribed in older adults despite an unfavorable risk-benefit ratio. Hospitalizations may provide a unique opportunity to initiate BZRA cessation, yet little is known about cessation during and after hospitalization. We aimed to measure the prevalence of BZRA use before hospitalization and the rate of cessation 6 months later, and to identify factors associated with these outcomes.