Exploring the Factors that Drive Clinical Negligence Claims: Stated Preferences of Those Who Have Experienced Unintended Harm.

Background: Better understanding of the factors that influence patients to make a financial claim for compensation is required to inform policy decisions. This study aimed to assess the relative importance of factors that influence those who have experienced a patient safety incident (PSI) to make a claim for compensation.

Method: Participants completed an online discrete choice experiment (DCE) involving 10 single profile tasks where they chose whether or not to file a claim.

Does EQ-5D Tell the Whole Story? Statistical Methods for Comparing the Thematic Coverage of Clinical and Generic Outcome Measures, With Application to Breast Cancer.

Objectives: This study aimed to develop the following: (1) methods for assessing claims in any specific application that a generic outcome measure, such as EQ-5D is deficient in its coverage of 1 or more specified domains, and (2) a simple method of judging whether any such deficiency is likely to be quantitatively important enough to call into question evaluations based on the generic instrument. Also to demonstrate the applicability of the methods in the important area of breast cancer.

Methods: The methodology requires a data set with observations from a generic instrument (eg, EQ-5D) and also a more comprehensive clinical instrument (eg, FACT-B [Functional Assessment of Cancer Therapy - Breast]).

Estimating the Relationship Between EQ-5D-5L and EQ-5D-3L: Results from a UK Population Study.

Objectives: The aim of this study was to estimate the relationship between EQ-5D-3L and EQ-5D-5L, in both directions, using a single model.

Methods: An online survey containing both variants of EQ-5D, with randomised ordering, was administered to a large UK sample in 2020. A joint statistical model of the ten EQ-5D responses (five at 5L, five at 3L), using a multi-equation ordinal regression framework was estimated.

Convergent validity of EQ-5D with core outcomes in dementia: a systematic review.

Objectives: To explore through a systematic review, the convergent validity of EQ-5D (EQ-5D-3L and EQ-5D-5L (total score and dimensions)) with core outcomes in dementia and investigate how this may be impacted by rater-type; with the aim of informing researchers when choosing measures to use in dementia trials.

Methods: To identify articles relevant to the convergent validity of EQ-5D with core dementia outcomes, three databases were electronically searched to September 2022. Studies were considered eligible for inclusion within the review if they included individual level data from people with dementia of any type, collected self and/or proxy reported EQ-5D and collected at least one core dementia outcome measure.

Assessing the psychometric performance of EQ-5D-5L in dementia: a systematic review.

Background: EQ-5D is widely used for valuing changes in quality of life for economic evaluation of interventions for people with dementia. There are concerns about EQ-5D-3L in terms of content validity, poor inter-rater agreement and reliability in the presence of cognitive impairment, but there is also evidence to support its use with this population. An evidence gap remains regarding the psychometric properties of EQ-5D-5L.

Should We Consider Including a Value for "Hope" as an Additional Benefit Within Health Technology Assessment?

Health technology assessment (HTA) typically uses average health-related quality of life gain as its main measure of benefit used in economic evaluation. Nevertheless, there have been calls to consider novel aspects of benefit including the "value of hope," defined as a patients' potential preferences for a wider distribution of treatment benefit with a positive skew, in the hope that they will be one of the lucky ones. The value of hope may also derive from feeling hopeful as a positive mental state, which may be missing from current measures of health-related quality of life.

A Discrete Choice Experiment to Elicit General Population Preferences Around the Factors Influencing the Choice to Make Clinical Negligence Claims.

Objectives: This article determines public stated preferences around different factors that influence the choice to make clinical negligence claims against a national healthcare system.

Methods: A large online survey was conducted using a discrete choice experiment (DCE) with the UK general population (N = 1013). DCE tasks involved a single profile and participants chose whether to make a claim for compensation (yes/no) after one of 3 randomly allocated patient safety incident (PSI) "scenarios" of different severities (mild, moderate, severe).

Target Trial Emulation for Transparent and Robust Estimation of Treatment Effects for Health Technology Assessment Using Real-World Data: Opportunities and Challenges.

Evidence about the relative effects of new treatments is typically collected in randomised controlled trials (RCTs). In many instances, evidence from RCTs falls short of the needs of health technology assessment (HTA). For example, RCTs may not be able to capture longer-term treatment effects, or include all relevant comparators and outcomes required for HTA purposes.

Exploring the Issues of Valuing Child and Adolescent Health States Using a Mixed Sample of Adolescents and Adults.

Preferences for child and adolescent health states used to generate health state utility values can be elicited from adults, young adults, adolescents, or combinations of these. This commentary paper provides a critical overview of issues and implications arising from valuing child and adolescent health states using a novel approach of a mixed sample of adolescents and adults. The commentary is informed by critical analysis of normative, ethical, practical and theoretical arguments in the health state valuation literature.

A Cost-Effectiveness Analysis of Remdesivir for the Treatment of Hospitalized Patients With COVID-19 in England and Wales.

Objectives: COVID-19 is associated with significant morbidity and mortality. This study aims to synthesize evidence to assess the cost-effectiveness of remdesivir (RDV) for the treatment of hospitalized patients with COVID-19 in England and Wales.

Methods: A probabilistic cost-effectiveness analysis was conducted informed by 2 large trials and uses a partitioned survival approach to assess short- and long-term clinical consequences and costs associated with COVID-19 in a hypothetical cohort of hospitalized patients requiring supplemental oxygen at the start of treatment.

Measuring quality of life of patients with axial spondyloarthritis for economic evaluation.

Objectives: To estimate the relationship between EQ5D (three levels, UK version) and the Ankylosing Spondylitis Disease Activity Score (ASDAS) for use in the economic evaluation of health technologies for people with axial spondyloarthritis (axSpA). To compare against the relationship with the Bath Ankylosing Spondylitis Disease Activity Index (BASDAI).

Methods: An electronic, prospective, Portuguese, nationwide, rheumatic disease register (Reuma.

Mapping the EORTC QLQ-C30 to EQ-5D-3L in patients with breast cancer.

Background: The types of outcomes measured collected in clinical studies and those required for cost-effectiveness analysis often differ. Decision makers routinely use quality adjusted life years (QALYs) to compare the benefits and costs of treatments across different diseases and treatments using a common metric. QALYs can be calculated using preference-based measures (PBMs) such as EQ-5D-3L, but clinical studies often focus on objective clinician or laboratory measured outcomes and non-preference-based patient outcomes, such as QLQ-C30.

An International Comparison of EQ-5D-5L and EQ-5D-3L for Use in Cost-Effectiveness Analysis.

Objectives: To estimate the impact of using EQ5D-5L (5L) compared with EQ5D-3L (3L) in cost-effectiveness analyses in 6 countries with 3L and 5L values: Germany, Japan, Korea, The Netherlands, China, and Spain.

Methods: Eight cost-effectiveness analyses based on clinical studies with 3L provided 11 pairwise comparisons. We estimated cost-effectiveness by applying the appropriate country values for 3L to observed responses.

A Review of the Psychometric Performance of Selected Child and Adolescent Preference-Based Measures Used to Produce Utilities for Child and Adolescent Health.

Objective: This review examined the psychometric performance of 4 generic child- and adolescent-specific preference-based measures that can be used to produce utilities for child and adolescent health.

Methods: A systematic search was undertaken to identify studies reporting the psychometric performance of the Child Health Utility (CHU9D), EQ-5D-Y (3L or 5L), and Health Utilities Index Mark 2 (HUI2) or Mark 3 (HUI3) in children and/or adolescents. Data were extracted to assess known-group validity, convergent validity, responsiveness, reliability, acceptability, and feasibility.

The clinical effectiveness of intensive management in moderate established rheumatoid arthritis: The titrate trial.

Objectives: Many trials have shown that intensive management is effective in patients with early active rheumatoid arthritis (RA). But its benefits are unproven for the large number of RA patients seen in routine care who have established, moderately active RA and are already taking conventional synthetic disease modifying anti-rheumatic drugs (csDMARDs). The TITRATE trial studied whether these patients also benefit from intensive management and, in particular, achieve more remissions.

A Review of the Methods Used to Generate Utility Values in NICE Technology Assessments for Children and Adolescents.

Objective: This review summarizes and critically examines methods used to generate utilities for child and adolescent health states in previous National Institute for Health and Care Excellence (NICE) technology assessments (TA) and highly specialized technology (HST) evaluations.

Methods: We identified all NICE TA and HST evaluations in which the licensed indication for the technology included people younger than 18 and included in the review all evaluations using a cost-utility analysis.

Results: The review includes 40 TA and HST evaluations.

Mapping clinical outcomes to generic preference-based outcome measures: development and comparison of methods.

Background: Cost-effectiveness analysis using quality-adjusted life-years as the measure of health benefit is commonly used to aid decision-makers. Clinical studies often do not include preference-based measures that allow the calculation of quality-adjusted life-years, or the data are insufficient. 'Mapping' can bridge this evidence gap; it entails estimating the relationship between outcomes measured in clinical studies and the required preference-based measures using a different data set.

The EQ-5D-5L Value Set for England: Findings of a Quality Assurance Program.

Objectives: Five-level EuroQol 5-dimensional questionnaire (EQ-5D-5L) values for several countries now exist. Decision makers require confidence in the underlying data and statistical analyses before advocating their use. Independent quality assurance of the published English value set is reported here.

MRI in the diagnosis of fetal developmental brain abnormalities: the MERIDIAN diagnostic accuracy study.

Background: Ultrasonography has been the mainstay of antenatal screening programmes in the UK for many years. Technical factors and physical limitations may result in suboptimal images that can lead to incorrect diagnoses and inaccurate counselling and prognostic information being given to parents. Previous studies suggest that the addition of in utero magnetic resonance imaging (iuMRI) may improve diagnostic accuracy for fetal brain abnormalities.

Identifying the Need for Good Practices in Health Technology Assessment: Summary of the ISPOR HTA Council Working Group Report on Good Practices in HTA.

The systematic use of evidence to inform healthcare decisions, particularly health technology assessment (HTA), has gained increased recognition. HTA has become a standard policy tool for informing decision makers who must manage the entry and use of pharmaceuticals, medical devices, and other technologies (including complex interventions) within health systems, for example, through reimbursement and pricing. Despite increasing attention to HTA activities, there has been no attempt to comprehensively synthesize good practices or emerging good practices to support population-based decision-making in recent years.

Mapping the FACT-B Instrument to EQ-5D-3L in Patients with Breast Cancer Using Adjusted Limited Dependent Variable Mixture Models versus Response Mapping.

Background: Preference-based measures of health, such as the three-level EuroQol five-dimensional questionnaire (EQ-5D-3L), are required to calculate quality-adjusted life-years for use in cost-effectiveness analysis, but are often not recorded in clinical studies. In these cases, mapping can be used to estimate preference-based measures.

Objectives: To model the relationship between the EQ-5D-3L and the Functional Assessment of Cancer Therapy-Breast Cancer (FACT-B) instrument, comparing indirect and direct mapping methods, and the use of FACT-B summary score versus FACT-B subscale scores.

The Impact of Moving from EQ-5D-3L to -5L in NICE Technology Appraisals.

Background: The EuroQol-5 Dimension (EQ-5D) instrument is the National Institute for Health and Care Excellence (NICE)'s preferred measure of health-related quality of life (QoL) in adults. The three-level (3L) value set is currently recommended for use, but the five-level (5L) set is increasingly being used in practice.

Objective: We aimed to explore the impact of moving from 3L to 5L in NICE appraisals.

Development of Methods for the Mapping of Utilities Using Mixture Models: Mapping the AQLQ-S to the EQ-5D-5L and the HUI3 in Patients with Asthma.

Background: Studies have shown that methods based on mixture models work well when mapping clinical to preference-based methods.

Objectives: To develop these methods in different ways and to compare performance in a case study.

Methods: Data from 856 patients with asthma allowed mapping between the Asthma Quality of Life Questionnaire and both the five-level EuroQol five-dimensional questionnaire (EQ-5D-5L) and the health utilities index mark 3 (HUI3).