The 50 most-cited articles in Value in Health, 2024

OBJECTIVES: Economic evaluations (EEs) are commonly used by decision makers to understand the value of health interventions. The Consolidated Health Economic Evaluation Reporting Standards (CHEERS 2022) provide reporting guidelines for EEs. Healthcare systems will increasingly see new interventions that use artificial intelligence (AI) to perform their function. We developed Consolidated Health Economic Evaluation Reporting Standards for Interventions that use AI (CHEERS-AI) to ensure EEs of AI-based health interventions are reported in a transparent and reproducible manner. METHODS: Potential CHEERS-AI reporting items were informed by 2 published systematic literature reviews of EEs and a contemporary update. A Delphi study was conducted using 3 survey rounds to elicit multidisciplinary expert views on 26 potential items, through a 9-point Likert rating scale and qualitative comments. An online consensus meeting was held to finalize outstanding reporting items. A digital health patient group reviewed the final checklist from a patient perspective. RESULTS: A total of 58…

OBJECTIVES: Atrial fibrillation (AF) is the most common cardiac arrhythmia, with an increasing incidence and prevalence because of progressively aging populations. Costs related to AF are both direct and indirect. This systematic review aims to identify the main cost drivers of the illness, assess the potential economic impact resulting from changes in care strategies, and propose interventions where they are most needed. METHODS: A systematic literature search of the PubMed and Scopus databases was performed to identify analytical observational studies defining the cost of illness in cases of AF. The search strategy was based on the PRISMA (Preferred Reporting Items for Systematic Reviews and Meta-Analyses) 2020 recommendations. RESULTS: Of the 944 articles retrieved, 24 met the inclusion criteria. These studies were conducted in several countries. All studies calculated the direct medical costs, whereas 8 of 24 studies assessed indirect costs. The median annual direct medical cost per patient, considering all studies, was €9409 (13 333 US dollars in purchasing power parities),…

OBJECTIVE: A key hurdle in broader next-generation sequencing (NGS) biomarker testing access in oncology is the ongoing debate on NGS's cost-effectiveness. We conducted a systematic review of existing evidence of the costs of NGS as a biomarker testing strategy in oncology and developed policy suggestions. METHODS: We searched multiple databases for studies reporting cost comparisons and cost-effectiveness of NGS across oncology indications and geographies between 2017 and 2022, inclusive. Inclusion criteria were established based on indication and type of cost-effectiveness analysis provided. We validated analyses and policy recommendations with 5 payer/policy maker interviews in the United States, Europe, and United Kingdom. RESULTS: Of the 634 identified studies, 29 met inclusion criteria, spanning 12 countries and 6 indications. Cost comparisons of NGS were evaluated using 3 methodologies: (1) comparison of direct testing costs, (2) comparison of holistic testing costs, and (3) comparison of long-term patient outcomes and costs. Targeted panel testing (2-52 genes) was considered…

OBJECTIVES: The environmental impacts of healthcare are important factors that should be considered during health technology assessments. This study aims to summarize the evidence that exists about methods to include environmental impacts in health economic evaluations and health technology assessments. METHODS: We identified records for screening using an existing scoping review and a systematic search of academic databases and gray literature up to September 2023. We screened the identified records for eligibility and extracted data using a narrative synthesis approach. The review was conducted following the JBI Manual for Evidence Synthesis and reported according to the Preferred Reporting Items for Systematic Reviews and Meta Analyses Extension for Scoping Reviews checklist. RESULTS: We identified 2898 records and assessed the full text of 114, of which 54 were included in this review. Ten methods were identified to include environmental impacts in health economic evaluations and health technology assessments. Methods included converting environmental impacts to dollars or…

OBJECTIVES: Patient-reported outcome measures (PROMs) have emerged as a promising approach to involve patients in their treatment process. Beyond serving as outcome measures, PROMs can be applied to provide feedback to healthcare providers and patients, thereby offering valuable insights that can improve health outcomes and care processes. This overview offers a comprehensive synthesis of the effects of PROM feedback, contributing to the evidence-based discussion on PROMs' potential to enhance patient care. METHODS: Following Cochrane Collaboration recommendations, this overview included literature reviews across diverse treatment areas, investigating the impact of PROM feedback on patient health outcomes (including quality of life, symptoms, or survival) and care process outcomes (including communication, symptom identification, or clinical practice). The methodological quality of the evidence was assessed with a modified version of A Measurement Tool to Assess Systematic Reviews 2, and the potential overlap of primary studies was quantified. Results were narratively synthesized.…

Healthcare decision making, including regulatory and reimbursement decisions, is based on uncertain assessments of clinical and economic value. This arises from the evidence supporting those assessments being uncertain, incomplete, or even absent. Qualitative, structured expert elicitation (SEE) is a valuable tool for extracting expert knowledge about an uncertain quantity and formulating that knowledge as a probability distribution. This creates a useful input to decision modeling and support, particularly in areas with limited evidence, such as advanced therapy products, precision medicine, rare diagnoses, and other areas with high uncertainty. Structured SEE protocols are used to improve the transparency, accuracy, and consistency of quantitative judgments from experts, limiting the effect of heuristics and biases. This task force report introduces 5 commonly used protocols for SEE (Sheffield elicitation framework; modified Delphi method; Cooke's classical method; investigate, discuss, estimate, aggregate protocol; and the Medical Research Council reference protocol). It…

OBJECTIVES: We aimed to assess the psychometric performance and added value of 9 existing bolt-ons (breathing problems, cognition, hearing, self-confidence, skin irritation, sleep, social relationships, tiredness, and vision) for the EQ-5D-5L in a general population sample. METHODS: The EQ-5D-5L, 9 bolt-ons, SF-6Dv1, Patient-Reported Outcomes Measurement Information System (PROMIS)-29+2, PROMIS Global Health, and Satisfaction with Life Scale were completed in an online cross-sectional survey among a general adult population sample in Hungary (n = 1587). The following psychometric properties were tested for the EQ-5D-5L + bolt-on(s): ceiling, divergent and convergent validity, structural validity, known-group validity, and explanatory power. RESULTS: = ǀ0.32ǀ - ǀ0.73ǀ). All bolt-ons, except cognition and self-confidence, loaded on a different factor from the EQ-5D-5L dimensions. Breathing, hearing, skin irritation, and vision significantly improved known-group validity in relevant health condition groups. The sleep bolt-on improved known-group validity in 9 of 13 chronic health…

OBJECTIVES: We aimed to provide a comprehensive summary, synthesis, and appraisal of minimally important difference (MID) estimates for EQ-5D instruments. METHODS: We conducted a systematic search using relevant terms related to "minimally/clinically, meaningful/ important difference/change" and "EQ-5D" in 6 major databases, including MEDLINE, Embase, PsycINFO, CINAHL, Scopus, and Cochrane Library (up to January 2023). We included studies that provided at least 1 original MID estimate for the EQ-5D. RESULTS: A total of 90 studies reporting 840 MID estimates were included. MID estimates for the EQ-5D-3L index score ranged from 0.075 to 0.8 using distribution-based approaches (239 estimates; 20 studies), from 0.003 to 0.72 using anchor-based approaches (189 estimates; 43 studies), and from 0.038 to 0.082 using instrument-defined approaches (4 estimates; 1 study). For the EQ-5D-5L, MID estimates ranged from 0.023 to 0.115 using distribution-based approaches (17 estimates; 12 studies), from 0.01 to 0.41 using anchor-based approaches (97 estimates; 15 studies), and from 0.037 to 0.101…

OBJECTIVES: At the Erasmus Medical Center, patient-reported outcomes measures (PROMs) are implemented on a hospital-wide scale. However, less than half of the patients and healthcare professionals (HCPs) use these PROMs. Therefore, this study aimed to investigate facilitators and barriers for adoption of PROMs to develop guidance around implementation. METHODS: A mixed-methods study with a combination of interviews and focus groups and questionnaires was conducted, involving patients, both PROM nonresponders and PROM responders, HCPs, and medicine students and nurse specialists in training (hereafter "students"). Interview transcripts were subjected to thematic content analysis. Subsequently, questionnaires were developed and presented to all stakeholders to validate the findings. Finally, identified themes and implementation recommendations were presented in a final questionnaire to the Value-Based Healthcare Erasmus Medical Center expert group to prioritize findings. RESULTS: Interviews were conducted with 15 patients, 14 HCPs and 4 students, and 2 focus groups with 5 students.…

OBJECTIVES: Several measures have been used or developed to capture the health and well-being of caregivers, including the EQ Health and Well-being (EQ-HWB) and its short form, EQ-HWB-S. This study aimed to evaluate the psychometric properties and construct validity of the EQ-HWB/EQ-HWB-S in a US caregiver population. METHODS: A cross-sectional survey was conducted involving 504 caregivers. Eligible participants were 18+ years old, provided unpaid care to a relative/friend aged 18+ in the past 6 months, and spent on average of at least 1 hour per week caregiving. Survey included the following measures: EQ-HWB, Adult Social Care Outcomes Toolkit for Carers-Carer, CarerQol, and EQ-5D-5L. Psychometric properties were assessed using response distributions, floor/ceiling effects, Spearman's correlation for convergent validity, and effect sizes (ES) for known-group validity based on caregiving situations and intensity. RESULTS: = 0.54) indices. Notably, the EQ-HWB-S index showed the largest ES among measures in differentiating caregiving scenarios with a large ES for caregiver's general…

OBJECTIVES: Controls and governance over the methodology and reporting of indirect treatment comparisons (ITCs) have been introduced to minimize bias and ensure scientific credibility and transparency in healthcare decision making. The objective of this study was to highlight ITC techniques that are key to conducting objective and analytically sound analyses and to ascertain circumstantial suitability of ITCs as a source of comparative evidence for healthcare interventions. METHODS: Ovid MEDLINE was searched from January 2010 through August 2023 to identify publicly available ITC-related documents (ie, guidelines and best practices) in the English language. This was supplemented with hand searches of websites of various international organizations, regulatory agencies, and reimbursement agencies of Europe, North America, and Asia-Pacific. The jurisdiction-specific ITC methodology and reporting recommendations were reviewed. RESULTS: Sixty-eight guidelines from 10 authorities worldwide were included for synthesis. Many of the included guidelines were updated within the last 5 years…

OBJECTIVES: This study aimed to systematically review evidence on the cost-effectiveness of chimeric antigen receptor T-cell (CAR-T) therapies for patients with cancer. METHODS: Electronic databases were searched in October 2022 and updated in September 2023. Systematic reviews, health technology assessments, and economic evaluations that compared costs and effects of CAR-T therapy in patients with cancer were included. Two reviewers independently screened studies, extracted data, synthesized results, and critically appraised studies using the Philips checklist. Cost data were presented in 2022 US dollars. RESULTS: Our search yielded 1809 records, 47 of which were included. Most of included studies were cost-utility analysis, published between 2018 and 2023, and conducted in the United States. Tisagenlecleucel, axicabtagene ciloleucel, idecabtagene vicleucel, ciltacabtagene autoleucel, lisocabtagene maraleucel, brexucabtagene autoleucel, and relmacabtagene autoleucel were compared with various standard of care chemotherapies. The incremental cost-effectiveness ratio (ICER) for CAR-T…

OBJECTIVES: The direct medical costs associated with Alzheimer's disease (AD) in the United States have been estimated to be over $360 billion USD, but this value does not reflect the substantial financial burden on unpaid caregivers and society. We estimated the economic burden of unpaid caregivers and patient productivity loss because of AD across all disease severity stages to better understand the indirect impacts of AD. METHODS: We performed a narrative literature review to identify estimates of unpaid caregiver burden and market productivity loss. In addition, we leveraged a published algorithm to estimate non-market productivity loss because of AD. Patient-level estimates were scaled to the population on the basis of AD prevalence in the United States (approximately 12.5 million), weighted by disease severity. RESULTS: The total annual indirect costs of unpaid caregiving and market and non-market productivity loss of AD increased with severity: $36 934 for mild cognitive impairment owing to AD, $65 565 for mild AD, $103 717 for moderate AD, and $145 250 for severe AD (2024…

OBJECTIVES: Digital health definitions are abundant, but often lack clarity and precision. We aimed to develop a minimum information framework to define patient-facing digital health interventions (DHIs) for outcomes research. METHODS: Definitions of digital-health-related terms (DHTs) were systematically reviewed, followed by a content analysis using frameworks, including PICOTS (population, intervention, comparator, outcome, timing, and setting), Shannon-Weaver Model of Communication, Agency for Healthcare Research and Quality Measures, and the World Health Organization's Classification of Digital Health Interventions. Subsequently, we conducted an online Delphi study to establish a minimum information framework, which was pilot tested by 5 experts using hypothetical examples. RESULTS: After screening 2610 records and 545 full-text articles, we identified 101 unique definitions of 67 secondary DHTs in 76 articles, resulting in 95 different patterns of concepts among the definitions. World Health Organization system (84.5%), message (75.7%), intervention (58.3%), and technology…

OBJECTIVES: The EQ Health and Wellbeing (EQ-HWB) is a novel measure that conceptually overlaps with the 5-level EQ-5D (EQ-5D-5L) while capturing broader dimensions of health and well-being. This study aimed to explore the extent to which the EQ-HWB and EQ-5D-5L capture overlapping or complementary constructs and to explore the discriminative ability of the EQ-HWB Short version (EQ-HWB-S) as a multiattribute utility instrument in the Australian setting. METHODS: A secondary analysis of data from a nationally representative cross-sectional survey of 2002 Australian adults was performed. The survey included socioeconomic questions and health characteristics and the EQ-HWB and EQ-5D-5L instruments. Convergent and known-group validity were evaluated through Spearman rank correlation and multivariable regression analyses, respectively. An exploratory factor analysis was also performed to explore the underlying constructs of the 2 measures. RESULTS: ≥ 0.40) between the EQ-5D-5L and the corresponding EQ-HWB dimensions (all P < .001). Based on the exploratory factor analysis, both…

OBJECTIVES: The EQ Health and Well-being, EQ-HWB (25-item) and the EQ-HWB-S (9-item), are new generic measures of health and well-being. The purpose of this study was to examine the measurement properties of the EQ-HWB and EQ-HWB-S measures in relation to the EQ-5D-5L among the Italian general population. METHODS: A cross-sectional survey was conducted from October 2020 to February 2021, followed by secondary analysis of collected data from Italian adults. This analysis included response pattern distributions, correlation strength, and known-group comparison. Known-group comparison was assessed using effect sizes (ES) across health conditions, caregiver status, and social care usage. The EQ-HWB-S index-based score was based on the UK pilot value set, whereas the Italian value set was used for scoring the EQ-5D-5L index. RESULTS: > 0.5) were found between overlapping EQ-HWB and EQ-5D-5L items. EQ-HWB-S and EQ-5D-5L index scores demonstrated similar discrimination based on symptomatic chronic conditions (ES d = 0.68 vs d = 0.71), but EQ-HWB-S had slightly higher ES for social care…

OBJECTIVES: This literature review provides an overview of meaningful change thresholds for the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (QLQ-C30) and the Functional Assessment of Cancer Therapy - General (FACT-G) used across hematological cancers and solid tumors (melanoma, lung, bladder, and prostate). METHODS: Embase, MEDLINE, and PubMed were searched to identify relevant oncology publications from 2016 to 2021. Label claims from the US Food and Drug Administration and the European Medicines Agency for 7 recently approved drugs (pembrolizumab, atezolizumab, glasdegib, gilteritinib, tisagenlecleucel, axicabtagene ciloleucel, and daratumumab plus hyaluronidase-fihj) were reviewed. RESULTS: Publications providing guidance on meaningful change thresholds for the QLQ-C30 displayed a growing trend away from broad "legacy" thresholds of 10 points for all QLQ-C30 scales), toward deriving "contemporary" thresholds (eg, subscale specific, population specific). Contemporary publications generally provide guidance on selecting…

OBJECTIVES: This review sought to identify the qualitative methods and techniques that researchers have used in the past decade to develop attributes and inform health-related discrete choice experiments (DCEs) surveys from a patient perspective. METHODS: The review followed the Preferred Reporting Items for Systematic Reviews and Meta-Analyses guidelines for reporting systematic reviews. An adapted appraisal tool following guidelines for reporting qualitative research for quantitative instruments and criteria for attribute development in DCEs was applied for quality assessment and data extraction. A narrative approach was used to synthesize data. This examination included consideration of issues pertaining to sampling, data collection, data analysis, attribute list reduction, wording, methodological adaptations to capture patient preferences, and testing the pre-experimental design decisions of the DCE survey. RESULTS: Of 8505 articles identified for abstract screening, 680 were included for full-text screening, 36 of which met the inclusion criteria. Practices to improve…

OBJECTIVES: The EQ Health and Wellbeing (EQ-HWB) is a new generic measure that captures constructs beyond health-related quality of life, with a 25-item long form and a shorter 9-item version (EQ-HWB-S). This study aimed to assess the psychometric performance of both versions in breast cancer, which is the most prevalent cancer worldwide, and compare them with other instruments. METHODS: A longitudinal survey in Indonesia (2023-2024) with 300 female patients used the EQ-HWB, 5-level EQ-5D (EQ-5D-5L), Functional Assessment of Cancer Therapy - General (from which Functional Assessment of Cancer Therapy Eight Dimension [FACT-8D] was derived), and Warwick-Edinburgh Mental Wellbeing Scale (WEMWBS, from which the Short WEMWBS was derived). Distributional characteristics, convergent validity, known-group validity (Student's t test or analysis of variance), test-retest reliability, and responsiveness were assessed. RESULTS: All patients reported problems in at least 1 EQ-HWB item. The EQ-HWB-S index (11%) had a lower ceiling than the EQ-5D-5L (35%) and the Short WEMWBS (15.3%), but not the…

OBJECTIVES: Discrete choice experiments including a duration attribute (DCEd) represent a promising candidate method for valuing health-related quality-of-life instruments. However, it has not been established that DCEd can produce similar results as composite time trade-off (cTTO) or EuroQol Valuation Technology (EQ-VT) valuations of the EQ-5D-5L instrument. This study provides a direct comparison between cTTO and EQ-VT, and DCEd valuation methods. METHODS: An EQ-VT study was conducted in Trinidad and Tobago to value the EQ-5D-5L. 1079 respondents each completed 10 cTTO tasks and 12 discrete choice experiments tasks without a duration attribute. A separate sample of 970 respondents each completed 18 split-triplet DCEd tasks. Several regression models were applied to the EQ-VT data, and the DCEd data were analyzed using mixed logit models with an exponential discount rate. The estimated values were compared using scatterplots and Bland-Altman plots. RESULTS: The ordering of dimensions was identical in level 5 for cTTO/EQ-VT and DCEd models, with pain/discomfort being the most…

OBJECTIVES: This study aimed to evaluate the impact of the National Institute for Health and Care Excellence's (NICE) new severity modifier, which has replaced the end-of-life (EoL) premium, on future NICE recommendations, considering past decision-making patterns. METHODS: NICE technology appraisals (TAs) published between January 2020 and December 2022 were reviewed. Summary statistics were generated to assess how the new severity modifier might affect hypothetical decision making in historical TAs. RESULTS: A total of 138 data points were identified from 132 TAs. Although the EoL premium was applied in 46 appraisals (33%), 57 (39%) qualify for a severity-based quality-adjusted life-year (QALY) multiplier. Only 19 appraisals (14.6%) not receiving an EoL premium met the severity criteria, the majority (17) qualifying for a 1.2× multiplier. In appraisals predicted to meet the severity criteria, 45 (79%) were in oncology, making them 4.04 times (95% CI 1.91-9.02) more likely to qualify for a severity modifier than nononcology indications. Among historically EoL indications, 42 (91%)…

OBJECTIVES: Over half of Australia's disease burden is due to morbidity, predominantly chronic conditions. Health-related quality of life instruments provide measures of morbidity and health status across different dimensions with EQ-5D being one of the most widely used. This study reports EQ-5D-5L general population norms for Queensland, Australia using the recently published Australian value set. METHODS: Population survey results from cross-sectional computer-assisted telephone interviews for Queensland adults in 2022 and 2023 were analyzed. EQ-5D-5L, as well as modifiable risk factors and sociodemographic data were collected. Using the recently published final Australian EQ-5D-5L value set, mean utility scores were calculated for Queensland, as well as by sociodemographic characteristics, including remoteness and socioeconomic area-based measures, and modifiable risk factors, such as smoking and body mass index. Results were combined with life tables to estimate quality-adjusted life expectancy (QALE) for subgroups with different lifestyles. RESULTS: The EQ-5D utility score for…

OBJECTIVES: To assess the potential number of European Union (EU) population(s), intervention, comparator(s), and outcomes (PICOs) based on European Network for Health Technology Assessment 21 (EUnetHTA 21) guidance and to explore further evidence-based opportunities to produce more predictable and workable EU PICOs. METHODS: The consolidated EU PICOs of 2 future hypothetical medicines in first-line non-small cell lung cancer (1L NSCLC) and third line multiple myeloma (3L MM) were derived using published health technology assessment reports of 2 recent medicines in similar indications based on EUnetHTA 21 proposed guidance. Sensitivity analysis assessed the impact of additional PICO requests. The number of analyses requested was estimated. RESULTS: In 1L NSCLC and 3L MM, 6 and 9 EU Member States (MS), respectively, had published health technology assessment reports. PICO consolidation resulted in 10 PICOs for 1L NSCLC and 16 PICOs for 3L MM, increasing to 14 and 18 PICOs, respectively, when England's National Institute for Health and Care Excellence scope was included to proxy…

OBJECTIVES: This study assessed the psychometric performance and construct validity of the EQ Health and Wellbeing Short (EQ-HWB-S), using a validated measure, the EQ-5D-5L, as a comparator. METHODS: The experimental version of the EQ-HWB-S was compared with the EQ-5D-5L to assess the psychometric performance of the measures. Data were drawn from the valuation stages of the Extending the Quality-Adjusted Life-Year project (UK general population, n = 429) and the EQ-5D-5L UK valuation pilot study (UK general population, n = 248). Construct validity was assessed based on convergent validity, using Spearman correlations and Pearson correlations. Known-group validity was assessed by estimating effect sizes to assess the ability of the EQ-HWB-S and EQ-5D-5L to discriminate between known groups based on "healthy" status, presence of a long-term condition, health and life satisfaction, age, and employment status. The degree of agreement in utility values across instruments was also evaluated using Bland-Altman plots. RESULTS: ≥ 0.5, P < .001) were found between conceptually overlapping…

OBJECTIVES: The primary focus of this research is the proposition of a methodological framework for the clinical application of the long COVID symptoms and severity score (LC-SSS). This tool is not just a self-reported assessment instrument developed and validated but serves as a standardized, quantifiable means to monitor the diverse and persistent symptoms frequently observed in individuals with long COVID. METHODS: A 3-stage process was used to develop, validate, and establish scoring standards for the LC-SSS. Validation measures included correlations with other patient-reported measures, confirmatory factor analysis, Cronbach's α for internal consistency, and test-retest reliability. Scoring standards were determined using K-means clustering, with comparative assessments made against hierarchical clustering and the Gaussian Mixture Model. RESULTS: = 0.516), confirming its construct validity. Structural validity was good with a comparative fit index of 0.969, with Cronbach's α of 0.93 indicating excellent internal consistency. Test-retest reliability was also satisfactory…

OBJECTIVES: Evidence about the comparative effects of new treatments is typically collected in randomized controlled trials (RCTs). In some instances, RCTs are not possible, or their value is limited by an inability to capture treatment effects over the longer term or in all relevant population subgroups. In these cases, nonrandomized studies (NRS) using real-world data (RWD) are increasingly used to complement trial evidence on treatment effects for health technology assessment (HTA). However, there have been concerns over a lack of acceptability of this evidence by HTA agencies. This article aims to identify the barriers to the acceptance of NRS and steps that may facilitate increases in the acceptability of NRS in the future. METHODS: Opinions of the authorship team based on their experience in real-world evidence research in academic, HTA, and industry settings, supported by a critical assessment of existing studies. RESULTS: Barriers were identified that are applicable to key stakeholder groups, including HTA agencies (eg, the lack of comprehensive methodological guidelines for…

OBJECTIVES: Reimbursement decisions for new Alzheimer's disease (AD) treatments are informed by economic evaluations. An open-source model with intuitive structure for model cross-validation can support the transparency and credibility of such evaluations. We describe the new International Pharmaco-Economic Collaboration on Alzheimer's Disease (IPECAD) open-source model framework (version 2) for the health-economic evaluation of early AD treatment and use it for cross-validation and addressing uncertainty. METHODS: A cohort state-transition model using a categorized composite domain (cognition and function) was developed by replicating an existing reference model and testing it for internal validity. Then, features of existing Institute for Clinical and Economic Review (ICER) and Alzheimer's Disease Archimedes Condition-Event Simulator (AD-ACE) models assessing lecanemab treatment were implemented for model cross-validation. Additional uncertainty scenarios were performed on choice of efficacy outcome from trial, natural disease progression, treatment effect waning and stopping…

OBJECTIVES: Model-based health economic evaluations of ischemic stroke are in need of cost- and utility estimates related to relevant outcome measures. This study aims to describe societal cost- and utility estimates per modified Rankin Scale (mRS)-score at different time points within 2 years post stroke. METHODS: Included patients had a stroke between 3 months and 2.5 years ago. mRS and EQ-5D-5L were scored during a telephone interview. Based on the interview date, records were categorized into a time point: 3 months (3M; 3-6 months), 1 year (Y1; 6-18 months), or 2 years (Y2; 18-30 months). Patients completed a questionnaire on healthcare utilization and productivity losses in the previous 3 months. Initial stroke hospitalization costs were assessed. Mean costs and utilities per mRS and time point were derived with multiple imputation nested in bootstrapping. Cost at 3 months post stroke were estimated separately for endovascular treatment (EVT)-/non-EVT-patients. RESULTS: 1106 patients were included from 18 Dutch centers. At each time point, higher mRS-scores were associated with…

OBJECTIVES: Health technology assessment (HTA) is increasingly crucial in medicine price negotiations in China, yet previous appraisals revealed national discrepancies on key economic evaluation issues: willingness-to-pay threshold, pricing models for multi-indication medicines, and comparator selection principles. This study aimed to collect expert opinions on these issues for future HTA evaluations. METHODS: A nationwide anonymous web-based survey encompassing experts across academia, HTA, consultancy/contract research organization/industry, service provider, and payer. In 2023, a generic invitation containing a web link to the questionnaire was disseminated via WeChat using convenience and snowball sampling. Agreement rates for questionnaire views were analyzed using descriptive statistics. The relationship between participants' responses and demographics was examined using appropriate logistic models. RESULTS: A total of 303 responses were received from experts in 34 cities. Key expert views include a suggested base willingness-to-pay threshold ranging from 0.5 to 1.5 times…

OBJECTIVES: Critics of quality-adjusted life-years argue that it discriminates against older individuals. However, little empirical evidence has been produced to inform this debate. This study aimed to compare published cost-effectiveness analyses (CEAs) on patients aged ≥65 years and those aged <65 years. METHODS: We used the Tufts Cost-Effectiveness Analysis Registry to identify CEAs published in MEDLINE between 1976 and 2021. Eligible CEAs were categorized according to age (≥65 years vs <65 years). The distributions of incremental cost-effectiveness ratios (ICERs) were compared between the age groups. We used logistic regression to assess the association between age groups and the cost-effectiveness conclusion adjusted for confounding factors. We conducted sensitivity analyses to explore the impact of mixed age and age-unknown groups and all ICERs from the same CEAs. Subgroup analyses were also conducted. RESULTS: A total of 4445 CEAs categorized according to age <65 years (n = 3784) and age ≥65 years (n = 661) were included in the primary analysis. The distributions of ICERs and…

OBJECTIVES: Head and neck cancer (HNC) is the sixth most common cancer worldwide. The condition and its treatment often lead to marked morbidities and, for some patients, premature death. Inferentially, HNC imposes a significant economic burden on society. This study aims to provide a comprehensive and detailed estimation of the cost of illness of HNC for Sweden in 2019. METHODS: This is a prevalence-based cost of illness study. Resource utilization and related costs are quantified using national registry data. A societal perspective is applied, including (1) direct costs for healthcare utilization, (2) costs for informal care from family and friends, and (3) costs for productivity loss due to morbidity and premature death. The human capital approach is used when estimating productivity losses. RESULTS: The societal cost of HNC for Sweden in 2019 was estimated at €92 million, of which the direct costs, costs for informal care, and costs for productivity loss represented 34%, 2%, and 64%, respectively. Oral cavity cancer was the costliest HNC, followed by oropharyngeal cancer,…

OBJECTIVES: When health fluctuates recurrently, estimating quality of life (QOL) is challenging, risking over-/underestimation due to measures' recall periods and timing. To inform how/when to capture QOL, we compared responses using different recall periods and assessment timings. METHODS: For one 3-week chemotherapy cycle, cancer patients were randomly assigned to complete EQ-5D-5L or SF-12v2 (daily with a daily recall, weekly with a weekly recall, and at 3 weeks with a 3-week recall); a third group completed SF-12v2 daily with a 3-week recall. EQ-5D-5L and SF-6D utilities (anchored at 1 [full health] and 0 [dead]) were generated and repeated measures analysis of variance, t tests, and effect sizes were calculated to compare recall. RESULTS: A total of 503 patients consented; all 21 daily questionnaires were completed by 84 (50%), 67 (40%), and 72 (43%) in the groups. Both measures captured fluctuations in QOL suggesting differences are due to recall effects. Mean daily scores were greater than scores for the past week on days 7, 14, and 21 (P < .0001). Utility was underestimated…

OBJECTIVES: Real-world evidence (RWE) is valuable in supporting regulatory and health technology assessment (HTA) decisions; however, the actual contribution to approvals remains elusive. This study aimed to review RWE approaches and use in oncology medicine approvals in Europe and understand cohesion and discrepancy in the acceptance of the RWE by the European Medicines Agency (EMA) and European HTA bodies. METHODS: This scoping review involved a search of the EMA database, National Institute for Health and Care Excellence (NICE), Gemeinsamer Bundesausschuss (G-BA), and Haute Autorité de Santé (HAS) websites to identify final reports and appraisals for oncology medicines with references to RWE. The selection was guided by research terms associated with RWE study designs, data sources, and outcomes. Qualitative analysis was used to systemize the data. Case studies assessed by more than one agency were selected for comparative assessment of RWE approach, use, and acceptability. RESULTS: RWE was mainly leveraged as an external control for indirect treatment comparisons or…

OBJECTIVES: To assess the accuracy and validity of the Determination of Diabetes Utilities, Costs, and Effects (DEDUCE) model, a Microsoft-Excel-based tool for evaluating diabetes interventions for type 1 and type 2 diabetes. METHODS: The DEDUCE model is a patient-level microsimulation, with complications predicted based on the Sheffield and Risk Equations for Complications Of type 2 diabetes models for type 1 and type 2 diabetes, respectively. For this tool to be useful, it must be validated to ensure that its complication predictions are accurate. Internal, external, and cross-validation was assessed by populating the DEDUCE model with the baseline characteristics and treatment effects reported in clinical trials used in the Fourth, Fifth, and Ninth Mount Hood Diabetes Challenges. Results from the DEDUCE model were evaluated against clinical results and previously validated models via mean absolute percentage error or percentage error. RESULTS: The DEDUCE model performed favorably, predicting key outcomes, including cardiovascular disease in type 1 diabetes and all-cause mortality…