Clinicians' Views on Therapeutic Outcomes of Systemic Interventions and on the Ability of the EQ-5D to Capture these Outcomes.

Background: Systemic interventions focus on improvements of interactions between clients and their environments, and are increasingly used to treat adolescents with problems of substance use and delinquency. Clients' progress may include broad and non-medical effects. When performing economic evaluations of these interventions, the common outcome of costs per quality adjusted life year (cost/QALY) may not capture all of these effects.

A conceptual disease model for adult Pompe disease.

Background: Studies in orphan diseases are, by nature, confronted with small patient populations, meaning that randomized controlled trials will have limited statistical power. In order to estimate the effectiveness of treatments in orphan diseases and extrapolate effects into the future, alternative models might be needed. The purpose of this study is to develop a conceptual disease model for Pompe disease in adults (an orphan disease).

Cost comparison of two implantable cardiac monitors in two different settings: Reveal XT in a catheterization laboratory vs. Reveal LINQ in a procedure room.

Aims: Implantable cardiac monitors (ICMs) are used for long-term heart rhythm monitoring, e.g. to diagnose unexplained syncope or for detection of suspected atrial and ventricular arrhythmias.

Value of Information Analysis Applied to the Economic Evaluation of Interventions Aimed at Reducing Juvenile Delinquency: An Illustration.

Objectives: To investigate whether a value of information analysis, commonly applied in health care evaluations, is feasible and meaningful in the field of crime prevention.

Methods: Interventions aimed at reducing juvenile delinquency are increasingly being evaluated according to their cost-effectiveness. Results of cost-effectiveness models are subject to uncertainty in their cost and effect estimates.

Access to orphan drugs in western Europe: can more systematic policymaking really help to avoid different decisions about the same drug?

When the Scottish government recently launched a £21 million fund to provide patients with rare diseases access to treatment, Alex Neill, the Scottish Health Secretary, stated that 'it was only right that Scottish patients with rare conditions had access to innovative medicines which were clinically justified, and that they were not disadvantaged due to the very high cost of these treatments'. This statement raises questions about the criteria that play a role in reimbursement decisions on orphan drugs. This editorial examines the criteria that were used in the decisions about the reimbursement of an orphan drug for Pompe disease and explores methods to improve the transparency and consistency of reimbursement decisions for orphan drugs in general.

A noticeable difference? Productivity costs related to paid and unpaid work in economic evaluations on expensive drugs.

Productivity costs can strongly impact cost-effectiveness outcomes. This study investigated the impact in the context of expensive hospital drugs. This study aimed to: (1) investigate the effect of productivity costs on cost-effectiveness outcomes, (2) determine whether economic evaluations of expensive drugs commonly include productivity costs related to paid and unpaid work, and (3) explore potential reasons for excluding productivity costs from the economic evaluation.

Orphan drugs expenditure in the Netherlands in the period 2006-2012.

Background: The relatively low budget impact of orphan drugs is often used as an argument in reimbursement decisions. However, overall, the budget impact of orphan drugs can still be substantial. In this study, we assess the uptake and budget impact of orphan drugs in the Netherlands.

Cost-effectiveness of enzyme replacement therapy with alglucosidase alfa in classic-infantile patients with Pompe disease.

Background: Infantile Pompe disease is a rare metabolic disease. Patients generally do not survive the first year of life. Enzyme replacement therapy (ERT) has proven to have substantial effects on survival in infantile Pompe disease.

How work impairments and reduced work ability are associated with health care use in workers with musculoskeletal disorders, cardiovascular disorders or mental disorders.

Purpose: The aim of this study was to explore how work impairments and work ability are associated with health care use by workers with musculoskeletal disorders (MSD), cardiovascular disorders (CVD), or mental disorders (MD).

Methods: In this cross-sectional study, subjects with MSD (n = 2,074), CVD (n = 714), and MD (n = 443) were selected among health care workers in 12 Dutch organizations. Using an online questionnaire, data were collected on individual characteristics, health behaviors, work impairments, work ability, and consultation of a general practitioner (GP), physiotherapist, specialist, or psychologist in the past year.

The impact of informal care for patients with Pompe disease: an application of the CarerQol instrument.

Background: Patients with Pompe disease, a rare progressive neuromuscular disorder, receive a considerable amount of informal care. In this study, we examined the impact of providing informal care to patients with Pompe disease.

Methods: Caregivers were administered various instruments, which measured the (impact of) informal care in the context of Pompe disease.

Systematic review of available evidence on 11 high-priced inpatient orphan drugs.

Background: Attention for Evidence Based Medicine (EBM) is growing, but evidence for orphan drugs is argued to be limited and inferior. This study systematically reviews the available evidence on clinical effectiveness, cost-effectiveness and budget impact for orphan drugs.

Methods: A systematic review was performed in PubMed, Embase, NHS EED and HTA databases for 11 inpatient orphan drugs listed on the Dutch policy rule on orphan drugs.

Framework for modelling the cost-effectiveness of systemic interventions aimed to reduce youth delinquency.

Background: Many interventions initiated within and financed from the health care sector are not necessarily primarily aimed at improving health. This poses important questions regarding the operationalisation of economic evaluations in such contexts.

Aims Of The Study: We investigated whether assessing cost-effectiveness using state-of-the-art methods commonly applied in health care evaluations is feasible and meaningful when evaluating interventions aimed at reducing youth delinquency.

Burden of illness of Pompe disease in patients only receiving supportive care.

Background: Pompe disease is an orphan disease for which enzyme replacement therapy (ERT) recently became available. This study aims to estimate all relevant aspects of burden of illness--societal costs, use of home care and informal care, productivity losses, and losses in health-related quality of life (HRQoL)--for adult Pompe patients only receiving supportive care.

Methods: We collected data on all relevant aspects of burden of illness via a questionnaire.

Cost-consequence analysis of remifentanil-based analgo-sedation vs. conventional analgesia and sedation for patients on mechanical ventilation in the Netherlands.

Introduction: Hospitals are increasingly forced to consider the economics of technology use. We estimated the incremental cost-consequences of remifentanil-based analgo-sedation (RS) vs. conventional analgesia and sedation (CS) in patients requiring mechanical ventilation (MV) in the intensive care unit (ICU), using a modelling approach.

The cost utility of solifenacin in the treatment of overactive bladder.

Objectives: Overactive bladder may cause significant discomfort to patients. The standard therapy for overactive bladder includes behavioural therapy and sometimes medication. Recently, a new medication (solifenacin 5 and 10 mg) was developed for treatment of overactive bladder.

A detailed cost analysis of in vitro fertilization and intracytoplasmic sperm injection treatment.

Objective: To provide detailed information about costs of in vitro fertilization (IVF) and intracytoplasmic sperm injection (ICSI) treatment stages and to estimate the cost per IVF and ICSI treatment cycle and ongoing pregnancy.

Design: Descriptive micro-costing study.

Setting: Four Dutch IVF centers.