Including carer health-related quality of life in NICE health technology assessments in the United Kingdom.

The impact of health technologies may extend beyond the patient and affect the health of people in their network, like their informal carers. The National Institute for Health and Care Excellence (NICE) methods guide explicitly allows the inclusion of health-related quality of life (HRQoL) effects on carers in economic evaluations when these effects are substantial, but the proportion of NICE appraisals that includes carer HRQoL remains small. This paper discusses when inclusion of carer HRQoL is justified, how inclusion can be substantiated, and how carer HRQoL can be measured and included in health economic models.

Benefits of a digital health technology for older nursing home residents. A de-novo cost-effectiveness model for digital health technologies to aid in the assessment of toileting and containment care needs.

The aim of this study was first, to introduce a comprehensive, de-novo health economic (HE) model incorporating the full range of activities involved in toileting and containment care (T&CC) for people with incontinence, capturing all the potential benefits and costs of existing and future Digital Health Technologies (DHT) aimed at improving continence care, for both residential care and home care. Second, to use this novel model to evaluate the cost-effectiveness of the DHT TENA SmartCare Identifi in the implementation of person-centred continence care (PCCC), compared with conventional continence care for Canadian nursing home residents. The de-novo HE model was designed to evaluate technologies across different care settings from the perspective of several stakeholders.

Caregiver Burden and Quality of Life Across Alzheimer's Disease Severity Stages.

Introduction: The objective is to estimate the caregiver burden in terms of informal care time, health-related quality of life (HRQoL), and societal costs by disease severity (mild, moderate, or severe) and living situation (community-dwelling or institutionalized) of persons with AD (PwAD); and PwAD HRQoL.

Methods: Caregivers were recruited through an online panel provider in the Netherlands. The survey used validated instruments, including iMTA Valuation of Informal Care Questionnaire, CarerQoL, and EQ-5D-5L.

The Disease Burden and Societal Costs of Hereditary Angioedema.

Background: According to the current treatment guidelines, the goals of treatment of patients with hereditary angioedema (HAE) are to achieve total control of the disease and to normalize patients' lives.

Objective: This study aims to establish the entire burden of HAE comprising disease control, treatment satisfaction, reductions in quality of life, and societal costs.

Methods: Adult patients with HAE under treatment at the Dutch national center of reference completed a cross-sectional survey in 2021.

Costs of Persons with Dementia Living in Nursing Homes in The Netherlands.

Background: Disease modifying treatments (DMTs) currently under development for Alzheimer's disease, have the potential to prevent or postpone institutionalization and more expensive care and might delay institutionalization of persons with dementia.

Objective: The current study estimates costs of living in a nursing home for persons with dementia in the Netherlands to help inform economic evaluations of future DMTs.

Methods: Data were collected during semi-structured interviews with healthcare professionals and from the financial administration of a healthcare organization with several nursing homes.

Cost Effectiveness of Splenic Artery Embolization versus Splenectomy after Trauma in the Netherlands.

Purpose: To demonstrate that splenic artery embolization (SAE) is more cost-effective than splenectomy from a societal perspective in the Netherlands.

Materials And Methods: Patient-level data obtained from the SPLENIQ study were used to populate a health economic model and were supplemented with expert opinion when necessary. Propensity score matching was used to correct for baseline differences in injury severity scores.

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).

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.

Assessing Costs Using the Treatment Inventory Cost in Psychiatric Patients (TIC-P), TIC-P Mini and TIC-P Midi.

Background: The Treatment Inventory Cost in Psychiatric patients (TIC-P) instrument is designed to measure societal costs in patients with psychiatric disorders and to be applied in economic evaluations. Efforts have been made to minimize respondents' burden by reducing the number of questions and meanwhile retaining the comprehensiveness of the instrument. Previously, a TIC-P Mini version and a TIC-P Midi version were developed and tested in a predominantly inpatient patient population.

Potential cost savings owing to the route of administration of oncology drugs: a microcosting study of intravenous and subcutaneous administration of trastuzumab and rituximab in the Netherlands.

There is little evidence on the costs associated with the route of administration of oncology drugs. We investigated time and resource use for hospitals and patients and compared healthcare and societal costs for intravenous (IV) and subcutaneous (SC) administration of trastuzumab and rituximab. Data for the preparation and administration of both drugs were collected at the hospital pharmacy and at the oncology day care unit.

A cost of illness study of hypoglycaemic events in insulin-treated diabetes in the Netherlands.

Objectives: Patients with diabetes mellitus are at a risk for hypoglycaemia. Besides the burden of hypoglycaemia for patients, hypoglycaemia poses an economic burden to society. The aim of this study was to calculate the per patient societal costs of hypoglycaemia among patients with type1 diabetes (T1DM) and type 2 diabetes (T2DM) on insulin therapy in the Netherlands.

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

Background: Pompe disease is a rare, progressive, metabolic disease, and the first treatable inheritable muscle disorder. Enzyme replacement therapy (ERT) with alglucosidase alfa is disease specific and the only medicinal product authorized for the treatment of Pompe disease. Costs of ERT are very high as for most orphan drugs.

Update of the Dutch manual for costing studies in health care.

Objectives: Dutch health economic guidelines include a costing manual, which describes preferred research methodology for costing studies and reference prices to ensure high quality studies and comparability between study outcomes. This paper describes the most important revisions of the costing manual compared to the previous version.

Methods: An online survey was sent out to potential users of the costing manual to identify topics for improvement.

Adoption of Biosimilar Infliximab for Rheumatoid Arthritis, Ankylosing Spondylitis, and Inflammatory Bowel Diseases in the EU5: A Budget Impact Analysis Using a Delphi Panel.

Introducing biosimilar infliximab for the treatment in rheumatology (rheumatoid arthritis and ankylosing spondylitis) and inflammatory bowel disease (Crohn's disease and ulcerative colitis) may reduce treatment costs associated with biologics. This study aimed to investigate the budget impact of adopting biosimilar infliximab in five European countries, considering that the budget impact includes the adoption of biosimilar infliximab and the availability of biologic alternatives such as vedolizumab, biosimilar etanercept, biosimilar rituximab, and other relevant factors. An existing budget impact model was adapted to forecast the budget impact in the UK, Germany, France, Spain, and Italy.

Care-related Quality of Life of informal caregivers of the elderly after a hip fracture.

Background: Reforms in the Dutch healthcare system in combination with the aging of the population will lead to a strong increase in the demand for informal care in the Netherlands. A hip fracture is one of the most important causes of hospital admissions among frail elderly and informal caregivers experience stress that may have significantly negative impact on the caregivers' Quality of Life. The purpose of the study was to determine the nature, intensity and the care-related Quality of Life (CarerQoL) of informal caregivers of elderly patients in the first six months after a hip fracture.

Quality of life and participation in daily life of adults with Pompe disease receiving enzyme replacement therapy: 10 years of international follow-up.

Background: Pompe disease is an inheritable metabolic disorder for which enzyme replacement therapy (ERT) has been available since 2006. Effects of ERT have been shown on distance walked, pulmonary function and survival. We investigated whether it also improves quality of life and participation in daily life in adult patients with the disease.

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.

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.

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.

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.