Most-cited articles · 2023

The 50 most-cited articles in Value in Health, 2023

Ranked by OpenAlex citation count on 29 October 2026, out of 654 eligible articles Value in Health published in 2023. Ranks are frozen; this page will not change. Number one, with 89 citations: A Roadmap for Increasing the Usefulness and Impact of Patient-Preference Studies in Decision Making in Health: A Good Practices Report of an ISPOR Task Force.

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2026-10-29

The most-cited Value in Health articles of 2023, ranked by OpenAlex citation counts, are led by methodological and good-practices work. The top paper, an ISPOR Task Force report on patient-preference studies in decision making, has 89 citations. Other highly cited entries address health economic quality evaluation, benefit-risk assessment, and the measurement of health and wellbeing, including studies on the EQ Health and Wellbeing Short and comparisons of EQ-5D-5L with PROMIS instruments.

This list offers a view of the work that drew the most citation attention among the 654 eligible articles published in the journal that year. Readers can use it to identify recurring topics, such as preference measurement, quality assessment, and value frameworks, and to locate individual papers through their citation counts and author details.

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  1. 1
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    A Roadmap for Increasing the Usefulness and Impact of Patient-Preference Studies in Decision Making in Health: A Good Practices Report of an ISPOR Task Force

    John F. P. Bridges, Esther Wilhelmina de Bekker-Grob, Brett Hauber, Sebastian Heidenreich, Ellen M. Janssen and 6 more

    89 citationsOpen accessdoi:10.1016/j.jval.2022.12.004OpenAlex

  2. 2
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    Developing Criteria for Health Economic Quality Evaluation Tool

    David D. Kim, Lauren A. Do, Patricia G. Synnott, Tara A. Lavelle, Lisa A. Prosser and 2 more

    74 citationsOpen access (CC-BY)doi:10.1016/j.jval.2023.04.004OpenAlex

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    OBJECTIVES: Because existing publication guidelines and checklists have limitations when used to assess the quality of cost-effectiveness analysis, we developed a novel quality assessment tool for cost-effectiveness analyses, differentiating methods and reporting quality and incorporating the relative importance of different quality attributes. METHODS: We defined 15 quality domains from a scoping review and identified 72 methods and reporting quality attributes (36 each). After designing a best-worst scaling survey, we fielded an online survey to researchers and practitioners to estimate the relative importance of the attributes in February 2021. We analyzed the survey data using a sequential conditional logit model. The final tool included 48 quality attributes deemed most important for assessing methods and reporting quality (24 each), accompanied by a free and web-based scoring system. RESULTS: A total of 524 participants completed the methodology section, and 372 completed both methodology and reporting sections. Quality attributes pertaining to the "modeling" and "data inputs…

  3. 3
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    Valuing the EQ Health and Wellbeing Short Using Time Trade-Off and a Discrete Choice Experiment: A Feasibility Study

    Clara Mukuria, Tessa Peasgood, Emily McDool, Richard Norman, Donna Rowen and 1 more

    61 citationsOpen access (CC-BY)doi:10.1016/j.jval.2023.02.008OpenAlex

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    OBJECTIVES: The EQ Health and Wellbeing Short (EQ-HWB-S) is a new generic measure that covers health and wellbeing developed for use in economic evaluation in health and social care. The aim was to test the feasibility of using composite time trade-off (cTTO) and a discrete choice experiment (DCE) based on an international protocol to derive utilities for the EQ-HWB-S and to generate a pilot value set. METHODS: A representative UK general population was recruited. Online videoconference interviews were undertaken where cTTO and DCE tasks were administered using EuroQol Portable Valuation Technology. Quality control (QC) was used to assess interviewers' performance. Data were modeled using Tobit, probit, and hybrid models. Feasibility was assessed based on the distribution of data, participants, and reports of understanding from the interviewer, QC and modeling results. RESULTS: cTTO and DCE data were available for 520 participants. Demographic characteristics were broadly representative of the UK general population. Interviewers met QC requirements. cTTO values ranged between -1 to…

  4. 4
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    Quantitative Benefit-Risk Assessment in Medical Product Decision Making: A Good Practices Report of an ISPOR Task Force

    Tommi Tervonen, Jorien Veldwijk, Katherine Payne, Xinyi Ng, Bennett S. Levitan and 6 more

    45 citationsOpen accessdoi:10.1016/j.jval.2022.12.006OpenAlex

  5. 5
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    Financing and Reimbursement of Approved Advanced Therapies in Several European Countries

    Carolina Iglesias-López, Antònia Agustí, Antonio Vallano, Mercè Obach

    44 citationsOpen access (CC-BY-NC-ND)doi:10.1016/j.jval.2022.12.014OpenAlex

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    OBJECTIVES: The uncertainty in the cost-benefit of advanced therapy medicinal products (ATMPs) is a current challenge for their reimbursement in health systems. This study aimed to provide a comparative analysis of the National Health Authorities (NHAs) reimbursement recommendations issued in different European countries. METHODS: The NHA reimbursement recommendations for the approved ATMPs were compared among 8 European Union (EU) Countries (EU8: Ireland, England/Wales, Scotland, The Netherlands, France, Germany, Spain, and Italy). The search was carried out until December 31, 2021. RESULTS: A total of 19 approved ATMPs and 76 appraisal reports were analyzed. The majority of the ATMPs were reimbursed, although with uncertainty in added therapeutic value. No relationship between the type of the European Medicines Agency approval and reimbursement was found. Managed entry agreements, such as payment by results, were necessary to ensure market access. The main issue during the evaluation was to base the cost-effectiveness analyses on assumptions because of the limited long-term data.…

  6. 6
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    Multistakeholder Perceptions of Additional Value Elements for United States Value Assessment of Health Interventions

    R. Brett McQueen, András Inotai, Antal Tamás Zemplenyi, Nick Mendola, Bertalan Németh and 1 more

    42 citationsOpen access (CC-BY-NC-ND)doi:10.1016/j.jval.2023.09.2910OpenAlex

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    OBJECTIVES: Limitations in conventional cost-effectiveness methods have led to calls for incorporation of additional value elements in assessments of health technologies. However, gaps remain in how additional value elements may inform decision making. This study aimed to prioritize additional value elements from the perspective of healthy individuals without a specific condition or indicated for a specific treatment in the United States among a multistakeholder panel and compare the importance of perspective-specific value elements. METHODS: Additional value elements were prioritized in 2 phases: (1) we identified and categorized additional value elements in a targeted literature review, and (2) we convened a multistakeholder group-based preference elicitation study (N = 28) to evaluate the description of each value element and rank and generate normalized weights of each value element for its significance in value assessment. The importance of additional value elements was also weighted relative to patient-centric value elements. RESULTS: The rank and weight of contextual value…

  7. 7
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    Best Practices for the Electronic Implementation and Migration of Patient-Reported Outcome Measures

    Florence D. Mowlem, Celeste A. Elash, Kelly M. Dumais, Estelle Haenel, Paul O’Donohoe and 7 more

    41 citationsOpen access (OTHER-OA)doi:10.1016/j.jval.2023.10.007OpenAlex

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    While the use of electronic methods to collect patient-reported outcome data in clinical trials continues to increase, it remains the case that many patient-reported outcome measures (PROMs) have originally been developed and validated on paper. Careful consideration during the move from paper PROMs to electronic format is required to preserve the integrity of the measure and ensure a "faithful migration." Relevant literature has long called out the importance of following migration best practices during this process; nevertheless, such best practices are distributed across multiple documents. This article consolidates and builds upon existing electronic PROM implementation best practice recommendations to provide a comprehensive, up-to-date, single point of reference. It reflects the current consensus based on the significant advances in technology capabilities and knowledge gleaned from the growing evidence base on electronic migration and implementation, to balance the need for maintaining the integrity of the measure while optimizing respondent usability. It also specifies…

  8. 8
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    Consolidated Health Economic Evaluation Reporting Standards - Value of Information (CHEERS-VOI): Explanation and Elaboration

    Natalia Kunst, Annisa Siu, Michael Drummond, Sabine Elisabeth Grimm, Janneke P.C. Grutters and 5 more

    40 citationsOpen accessdoi:10.1016/j.jval.2023.06.014OpenAlex

  9. 10
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    Inclusion of Environmental Spillovers in Applied Economic Evaluations of Healthcare Products

    Charlotte Desterbecq, Sandy Tubeuf

    34 citationsOpen access (CC-BY-NC-ND)doi:10.1016/j.jval.2023.03.008OpenAlex

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    OBJECTIVES: Climate change and environmental factors have an impact on human health and the ecosystem. The healthcare sector is responsible for substantial environmental pollution. Most healthcare systems rely on economic evaluation to select efficient alternatives. Nevertheless, environmental spillovers of healthcare treatments are rarely considered whether it is from a cost or a health perspective. The objective of this article is to identify economic evaluations of healthcare products and guidelines that have included any environmental dimensions. METHODS: Electronic searches of 3 literature databases (PubMed, Scopus, and EMBASE) and official health agencies guidelines were conducted. Documents were considered eligible if they assessed the environmental spillovers within the economic evaluation of a healthcare product or provided any recommendations on the inclusion of environmental spillovers in the health technology assessment process. RESULTS: ) emissions, water or energy consumption, and waste disposal. The environmental spillovers were mainly assessed using the lifecycle…

  10. 11
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    A Direct Comparison of the Measurement Properties of EQ-5D-5L, PROMIS-29+2 and PROMIS Global Health Instruments and EQ-5D-5L and PROPr Utilities in a General Population Sample

    Fanni Rencz, Valentin Brodszky, Mathieu F. Janssen

    34 citationsOpen access (CC-BY-NC-ND)doi:10.1016/j.jval.2023.02.002OpenAlex

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    OBJECTIVES: We aimed to compare measurement properties of the 5-level version of EQ-5D (EQ-5D-5L) and 2 Patient-Reported Outcomes Measurement Information System (PROMIS) short forms, PROMIS-29+2 and PROMIS Global Health (PROMIS-GH-10), and of EQ-5D-5L and PROMIS-preference scoring system (PROPr) utilities. METHODS: A cross-sectional survey was conducted in a general population sample in Hungary (N = 1631). We compared the following measurement properties at the level of items, domains, and utilities, the latter using corresponding US value sets: ceiling and floor, informativity (Shannon's indices), agreement, convergent, and known-group validity. For the analyses, PROMIS items/domains were matched to EQ-5D-5L domains that cover similar concepts of health. RESULTS: The majority of PROMIS items showed enhanced distributional characteristics, including lower ceilings and higher informativity than the EQ-5D-5L. Good convergent validity was established between EQ-5D-5L and PROMIS domains capturing similar aspects of health. Mean EQ-5D-5L utilities were substantially higher than those of…

  11. 12
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    Preferences for Palliative and End-of-Life Care: A Systematic Review of Discrete Choice Experiments

    Qing Xia, Mineth Kularatna, Claudia Virdun, Elise Button, Eliana Close and 1 more

    33 citationsOpen access (CC-BY-NC-ND)doi:10.1016/j.jval.2023.07.005OpenAlex

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    OBJECTIVES: Understanding what matters most to patients and their caregivers is fundamental to delivering high-quality care. This systematic review aimed to characterize and appraise the evidence from discrete choice experiments eliciting preferences for palliative care. METHODS: A systematic literature search was undertaken for publications up until August 2022. Data were synthesized narratively. Thematic analysis was applied to categorize attributes into groups. Attribute development, frequency, and relative importance were analyzed. Subgroup analyses were conducted to compare outcomes between patient and proxy respondents. RESULTS: Seventeen studies spanning 11 countries were included; 59% of studies solely considered preferences for patients with cancer. A range of respondent groups were represented including patients (76%) and proxies (caregivers [35%], health providers [12%], and the public [18%]). A total of 117 individual attributes were extracted and thematically grouped into 8 broad categories and 21 subcategories. Clinical outcomes including quality of life, length of…

  12. 13
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    A Value Framework to Assess Patient-Facing Digital Health Technologies That Aim to Improve Chronic Disease Management: A Delphi Approach

    Madeleine Haig, Caitlin Main, Danitza Chávez, Panos G. Kanavos

    32 citationsOpen access (CC-BY)doi:10.1016/j.jval.2023.06.008OpenAlex

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    OBJECTIVES: Digital health technologies (DHTs) can optimise healthcare costs and improve quality and efficiency of care. However, the fast-paced rate of innovation and varying evidence standards can make it difficult for decision-makers to assess these technologies in an efficient and evidence-based manner. We sought to develop a comprehensive framework to assess the value of novel patient-facing DHTs used to manage chronic diseases by eliciting stakeholder value preferences. METHODS: Literature review and primary data collection from a three-round web-Delphi exercise was utilized. 79 participants from 5 stakeholder groups (patients, physicians, industry, decision makers, and influencers) and 3 countries (United States of America, United Kingdom, and Germany) took part. Likert scale data were statistically analyzed to determine intergroup differences in both country and stakeholder groups, stability of results, and overall consensus. RESULTS: The resulting co-created framework comprised 33 stable indicators with consensus from quantitative value judgments across domains: health…

  14. 16
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    Frameworks for Health Technology Assessment at an Early Stage of Product Development: A Review and Roadmap to Guide Applications

    Elisabet Rodríguez Llorian, Louloua Ashikhusein Waliji, Nick Dragojlovic, Kristina D. Michaux, Fernanda Nagase and 1 more

    30 citationsOpen accessdoi:10.1016/j.jval.2023.03.009OpenAlex

  15. 17
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    Economic Burden and Service Utilization of Children With Attention-Deficit/Hyperactivity Disorder: A Systematic Review and Meta-Analysis

    Mitchell Dodds, Sithara Wanni Arachchige Dona, Lisa H. Gold, David Coghill, Ha Le

    29 citationsOpen access (CC-BY)doi:10.1016/j.jval.2023.11.002OpenAlex

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    OBJECTIVES: Attention-deficit/hyperactivity disorder (ADHD) is one of the most common neurodevelopmental disorders in children. This study aims to systematically synthesize the literature on service utilization and costs for children with ADHD. METHODS: The search included 9 databases for peer-reviewed primary studies in English from 2007 to 2023. Two independent reviewers conducted title/abstract and full-text screenings and quality assessment. Meta-analysis was conducted on direct medical costs. RESULTS: Thirty-two studies were included. Children with ADHD have used more pharmaceuticals, mental health, and special education services than children without ADHD (counterparts). Nevertheless, one study found that children with ADHD were twice as likely to have unmet health needs than their counterparts. Annual health system costs per patient were highly varied and higher in children with ADHD ($722-$11 555) than their counterparts ($179-$3646). From a societal perspective, children with ADHD were associated with higher costs ($162-$18 340) than their counterparts ($0-2540). The…

  16. 18
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    Estimating the US Baseline Distribution of Health Inequalities Across Race, Ethnicity, and Geography for Equity-Informative Cost-Effectiveness Analysis

    Stacey L. Kowal, Carmen D. Ng, Robert F. Schuldt, Daniel M. Sheinson, Kimberly Jinnett and 1 more

    26 citationsOpen access (CC-BY-NC-ND)doi:10.1016/j.jval.2023.06.015OpenAlex

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    OBJECTIVES: Information on how life expectancy, disability-free life expectancy, and quality-adjusted life expectancy varies across equity-relevant subgroups is required to conduct distributional cost-effectiveness analysis. These summary measures are not comprehensively available in the United States, given limitations in nationally representative data across racial and ethnic groups. METHODS: Through linkage of US national survey data sets and use of Bayesian models to address missing and suppressed mortality data, we estimate health outcomes across 5 racial and ethnic subgroups (non-Hispanic American Indian or Alaska Native, non-Hispanic Asian and Pacific Islander, non-Hispanic black, non-Hispanic white, and Hispanic). Mortality, disability, and social determinant of health data were combined to estimate sex- and age-based outcomes for equity-relevant subgroups based on race and ethnicity, as well as county-level social vulnerability. RESULTS: Life expectancy, disability-free life expectancy, and quality-adjusted life expectancy at birth declined from 79.5, 69.4, and 64.3 years,…

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    Adaptive Health Technology Assessment: A Scoping Review of Methods

    Cassandra Nemzoff, Hiral Anil Shah, Lieke Fleur Heupink, Lydia Regan, Srobana Ghosh and 5 more

    26 citationsOpen access (CC-BY)doi:10.1016/j.jval.2023.05.017OpenAlex

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    OBJECTIVES: Health technology assessment (HTA) is an established mechanism for explicit priority setting to support universal health coverage. However, full HTA requires significant time, data, and capacity for each intervention, which limits the number of decisions it can inform. Another approach systematically adapts full HTA methods by leveraging HTA evidence from other settings. We call this "adaptive" HTA (aHTA), although in settings where time is the main constraint, it is also called "rapid HTA." METHODS: The objectives of this scoping review were to identify and map existing aHTA methods, and to assess their triggers, strengths, and weaknesses. This was done by searching HTA agencies' and networks' websites, and the published literature. Findings have been narratively synthesized. RESULTS: This review identified 20 countries and 1 HTA network with aHTA methods in the Americas, Europe, Africa, and South-East Asia. These methods have been characterized into 5 types: rapid reviews, rapid cost-effectiveness analyses, rapid manufacturer submissions, transfers, and de facto HTA.…

  19. 22
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    Eliciting Trade-Offs Between Equity and Efficiency: A Methodological Scoping Review

    Christopher J. Cadham, Lisa A. Prosser

    25 citationsOpen access (CC-BY)doi:10.1016/j.jval.2023.02.006OpenAlex

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    OBJECTIVES: To identify differences in the approaches and results of studies that elicit equity-efficiency trade-offs that can inform equity-informative cost-effectiveness analysis for healthcare resource allocation. METHODS: We searched Ovid (Medline), EconLit, and Scopus prior to June 25, 2021. Inclusion criteria were: (1) peer-reviewed or (2) gray literature; (3) published in English; (4) survey-based; (5) parameterized a social welfare function to quantify inequality aversion or (6) elicited a trade-off in equity and efficiency characteristics of health interventions. Exclusion criteria were: (1) studies that did not conduct a trade-off or (2) theoretical studies. We abstracted details on study methods, results, and limitations. Studies were grouped by following approach: (1) social welfare function or (2) preference ranking and distributional weighting. We described findings separately for each approach category. RESULTS: Seventy-seven papers were included, 28 parameterized social welfare functions and 49 were classified as preference ranking and distributional weighting. Study…

  20. 23
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    Cost-Effectiveness of Oral Nirmatrelvir/Ritonavir in Patients at High Risk for Progression to Severe COVID-19 in the United States

    Josh John Carlson, VOLKER FOOS, Adam Kasle, Tendai Mugwagwa, Florin Draica and 5 more

    23 citationsOpen access (CC-BY-NC-ND)doi:10.1016/j.jval.2023.11.003OpenAlex

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    OBJECTIVES: Nirmatrelvir/ritonavir (NMV/r) is an orally administered antiviral indicated for the outpatient treatment of patients with mild-to-moderate COVID-19 at high risk for disease progression to severe illness. We estimated the cost-effectiveness of NMV/r versus best supportive care for patients with mild-to-moderate COVID-19 at high risk for progression to severe illness from a US health sector perspective. METHODS: A cost-effectiveness model was developed using a short-term decision-tree (1 year) followed by a lifetime 2-state Markov model (alive and dead). The short-term decision-tree captured costs and outcomes associated with the primary infection and healthcare utilization; survivors of the short-term decision-tree were followed until death assuming US quality-adjusted life years (QALYs), adjusted in the short-term for survivors of mechanical ventilation. Baseline rate of hospitalization and NMV/r effectiveness were taken from an Omicron-era US real-world study. Remaining inputs were informed by previous COVID-19 studies and publicly available US sources. Sensitivity…

  21. 24
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    The Evolving Nature of Health Technology Assessment: A Critical Appraisal of NICE’s New Methods Manual

    Aris Angelis, Martin J. Harker, John Cairns, Mikyung Kelly Seo, Rosa Legood and 5 more

    21 citationsOpen access (CC-BY-NC-ND)doi:10.1016/j.jval.2023.05.015OpenAlex

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    OBJECTIVES: The National Institute for Health and Care Excellence (NICE) recently completed a review of its methods for health technology assessment, involving a 2-stage public consultation. We appraise proposed methodological changes and analyze key decisions. METHODS: We categorize all changes proposed in the first consultation as "critical," "moderate" or "limited" updates, considering the importance of the topic and the degree of change or the level of reinforcement. Proposals were followed through the review process, for their inclusion, exclusion, or amendment in the second consultation and the new manual. RESULTS: The end-of-life value modifier was replaced with a new "disease severity" modifier and other potential modifiers were rejected. The usefulness of a comprehensive evidence base was emphasized, clarifying when nonrandomized studies can be used, with further guidance on "real-world" evidence developed separately. A greater degree of uncertainty was accepted in circumstances when evidence generation raised challenges, in particular for children, rare diseases, and…

  22. 25
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    Data Governance for Real-World Data Management: A Proposal for a Checklist to Support Decision Making

    Oriol Solà-Morales, Katla Sigurðardóttir, Ron Akehurst, Linda A Murphy, Jorge Mestre‐Ferrandiz and 2 more

    21 citationsOpen accessdoi:10.1016/j.jval.2023.02.012OpenAlex

  23. 26
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    Systematic Review of Outcomes for Assessment of Medication Adherence Enhancing Interventions: An ISPOR Special Interest Group Report

    Tamás Ágh, Mickaël Hiligsmann, Bijan J. Borah, Charlotte Beaudart, Adina Turcu-Ştiolică and 5 more

    20 citationsOpen accessdoi:10.1016/j.jval.2023.10.016OpenAlex

  24. 27
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    Assessing the Content Validity of Preference-Based Measures in Cancer

    A Gibson, Louise Longworth, Bryan Bennett, Alan Simon Pickard, James W. Shaw

    20 citationsOpen access (CC-BY-NC-ND)doi:10.1016/j.jval.2023.10.006OpenAlex

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    OBJECTIVES: This study assessed the content validity of generic and condition-specific preference-based measures (PBMs) with patients treated for cancer, evaluated against 10 Consensus-Based Standards for the Selection of Health Measurement Instruments criteria for good content validity, to best inform measurement strategies regarding the use of PBMs in oncology development programs and real-world applications. METHODS: Individual, semistructured interviews were conducted with patients who received drug treatment for cancer in the United Kingdom (n = 47) and the United States (n = 49). During the interview, patients completed 3 generic PBMs (EQ-5D-5L, EuroQol Health and Wellbeing measure-Short Form, Château Santé Base) and 2 condition-specific PBMs (Quality of Life Utility-Core 10 Dimension, Functional Assessment of Cancer Therapy Eight Dimension [FACT-8D]). Interviews were conducted via teleconference, audio recorded, and transcribed verbatim. Transcripts were coded using thematic and content analysis methods. RESULTS: Condition-specific measures were evaluated as having better…

  25. 28
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    UK Valuation of EQ-5D-5L, a Generic Measure of Health-Related Quality of Life: A Study Protocol

    Donna Rowen, Clara Mukuria, Nathan Bray, Jill Carlton, Sophie Cooper and 4 more

    20 citationsOpen access (CC-BY)doi:10.1016/j.jval.2023.08.005OpenAlex

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    OBJECTIVES: A high-quality and widely accepted UK EQ-5D-5L value set is urgently required to enable the latest version of EQ-5D scored using recent UK public preferences to inform policy including health technology assessments submitted to the National Institute for Health and Care Excellence. This article outlines the study protocol for the generation of a new EQ-5D-5L UK value set. METHODS: Twelve hundred interviews will be undertaken using the composite time trade-off elicitation technique for 102 health states (86 from the international EQ-5D-5L valuation protocol, plus 16 with best predictive performance in an extended design used in the Native American EQ-5D-5L valuation). The sample will be UK adults (age ≥18 years) proportionately representative across England, Wales, Scotland, and Northern Ireland, representative for age, sex, ethnicity, and socioeconomic group, with inclusion of participants with/without health problems. Participants will choose to be interviewed via videoconference (by Zoom) or in-person in a central venue. Data quality will be rigorously assessed.…

  26. 29
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    Toward Best Practices for Economic Evaluations of Tumor-Agnostic Therapies: A Review of Current Barriers and Solutions

    Deirdre Weymann, Samantha Pollard, Halina Lam, Emanuel Krebs, Dean A. Regier

    20 citationsOpen access (CC-BY-NC-ND)doi:10.1016/j.jval.2023.07.004OpenAlex

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    OBJECTIVES: Cancer therapies targeting tumor-agnostic biomarkers are challenging traditional health technology assessment (HTA) frameworks. The high prevalence of nonrandomized single-arm trials, heterogeneity, and small benefiting populations are driving outcomes uncertainty, challenging healthcare decision making. We conducted a structured literature review to identify barriers and prioritize solutions to generating economic evidence for tumor-agnostic therapies. METHODS: We searched MEDLINE and Embase for English-language studies conducting economic evaluations of tumor-agnostic treatments or exploring related challenges and solutions. We included studies published by December 2022 and supplemented our review with Canadian Agency for Drugs and Technologies in Health and National Institute for Health and Care Excellence technical reports for approved tumor-agnostic therapies. Three reviewers abstracted and summarized key methodological and empirical study characteristics. Challenges and solutions were identified through authors' statements and categorized using directed content…

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    Evaluating the Value for Money of Precision Medicine from Early Cycle to Market Access: A Comprehensive Review of Approaches and Challenges

    Wenjia Chen, Yi Wang, Yaroslava Zemlyanska, Dimple Haresh Butani, Nigel Chong Boon Wong and 3 more

    20 citationsOpen accessdoi:10.1016/j.jval.2023.05.001OpenAlex

  28. 31
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    Designing Guidelines for Those Who Do Not Follow Them: The Impact of Adherence Assumptions on Optimal Screening Guidelines

    Kine Pedersen, Ivar S. Kristiansen, Stephen Sy, Jane J. Kim, Emily Annika Burger

    20 citationsOpen access (CC-BY)doi:10.1016/j.jval.2023.04.005OpenAlex

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    OBJECTIVES: Model-based cost-effectiveness analyses can inform decisions about screening guidelines by quantifying consequences of alternative algorithms. Although actual screening adherence is imperfect, incorporating nonadherence into analyses that aim to determine optimal screening may affect the policy recommendations. We evaluated the impact of nonadherence assumptions on the optimal cervical cancer screening in Norway. METHODS: We used a microsimulation model of cervical carcinogenesis to project the long-term health and economic outcomes under alternative screening algorithms and adherence patterns. We compared 18 algorithms involving primary human papillomavirus testing (5-yearly) that varied follow-up management of different human papillomavirus results. We considered 12 adherence scenarios: perfect adherence, 8 high- and low-coverage "random-complier" scenarios, and 3 "systematic-complier" scenarios that reflect conditional screening behavior over a lifetime. We calculated incremental cost-effectiveness ratios and considered a strategy with the highest incremental…

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    A Longitudinal Evaluation of the Preferences of Patients With Advanced Cancer for Quality of Life and Survival in Malaysia: A Discrete Choice Experiment

    Alene Sze Jing Yong, Ka Keat Lim, Julia A Fox-Rushby, Fuad Ismail, Ednin Hamzah and 2 more

    18 citationsOpen access (CC-BY)doi:10.1016/j.jval.2023.08.009OpenAlex

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    OBJECTIVES: This study aims to quantify the preferences of patients with advanced cancer for quality of life (QoL) outcomes versus survival extension in Malaysia. The secondary aim of this study is to explore the change in preferences over time. METHODS: A discrete choice experiment was developed to include 7 attributes valued in cancer management: physical, psychological and social functioning, pain control, survival, place of death, and cost. Patients were recruited via convenience sampling from 2 Malaysian public hospitals. The survey questionnaire was administered to patients within 6 months of their cancer diagnosis with a follow-up 3 months later. Conditional logit regression was used to estimate the preference weight, relative attribute importance, and willingness to pay. RESULTS: One hundred valid responses were collected at baseline and 45 at follow-up. Respondents placed higher values on QoL improvements from severe to moderate or mild levels and to achieve home death over survival extension from 6 to 18 months. However, additional improvements (from moderate to mild) in…

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    Opportunities and Challenges for Decentralized Clinical Trial Approaches: European Health Technology Assessment Perspective

    Amos J. de Jong, Nadi Shahid, Mira G. P. Zuidgeest, Yared Santa‐Ana‐Téllez, Milou A. Hogervorst and 4 more

    17 citationsOpen access (CC-BY)doi:10.1016/j.jval.2023.11.006OpenAlex

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    OBJECTIVES: Decentralized clinical trial (DCT) approaches are clinical trials in which some or all trial activities take place closer to participants' proximities instead of a traditional investigative site. Data from DCTs may be used for clinical and economic evaluations by health technology assessment (HTA) bodies to support reimbursement decision making. This study aimed to explore the opportunities and challenges for DCT approaches from an HTA perspective by interviewing representatives from European HTA bodies. METHODS: We conducted semistructured interviews with 25 European HTA representatives between September 2022 and February 2023, and transcripts were analyzed after thematic analysis. RESULTS: Two main themes were identified from the data relating to (1) DCT approaches in HTA and (2) trial-level acceptance and relevance. Experience with assessing DCTs was limited and a variety of knowledge about DCTs was observed. The respondents recognized the opportunity of DCTs to reduce recall bias when participant-reported outcome data can be collected more frequently and conveniently…

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    The Health Inequality Impact of Liquid Biopsy to Inform First-Line Treatment of Advanced Non–Small Cell Lung Cancer: A Distributional Cost-Effectiveness Analysis

    Jeroen Paul Jansen, Meera Vimala Ragavan, Cheng Chen, Michael P. Douglas, Kathryn A. Phillips

    17 citationsOpen access (CC-BY)doi:10.1016/j.jval.2023.08.010OpenAlex

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    OBJECTIVES: To perform a distributional cost-effectiveness analysis of liquid biopsy (LB) followed by, if needed, tissue biopsy (TB) (LB-first strategy) relative to a TB-only strategy to inform first-line treatment of advanced non-small cell lung cancer (aNSCLC) from a US payer perspective by which we quantify the impact of LB-first on population health inequality according to race and ethnicity. METHODS: With a health economic model, quality-adjusted life-years (QALYs) and costs per patient were estimated for each subgroup. Given the lifetime risk of aNSCLC, and assuming equally distributed opportunity costs, the incremental net health benefits of LB-first were calculated, which were used to estimate general population quality-adjusted life expectancy at birth (QALE) by race and ethnicity with and without LB-first. The degree of QALYs and QALE differences with the strategies was expressed with inequality indices. Their differences were defined as the inequality impact of LB-first. RESULTS: LB-first resulted in an additional 0.21 (95% uncertainty interval: 0.07-0.39) QALYs among…

  33. 38
    top 5.9%

    Economic Evaluations of Screening Programs for Chronic Kidney Disease: A Systematic Review

    M. Rifqi Rokhman, Firas Farisi Alkaff, Pim W. M. van Dorst, Jarir At Thobari, Maarten Jacobus Postma and 2 more

    17 citationsOpen access (CC-BY)doi:10.1016/j.jval.2023.08.003OpenAlex

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    OBJECTIVES: The aim of this review is to appraise and assimilate evidence from studies that have reported on the cost-effectiveness of screening programs for chronic kidney disease (CKD). METHODS: The study protocol was registered on International Prospective Register of Systematic Reviews (PROSPERO). The final search was conducted on 18 January 2023 using 7 databases. Screening of articles, data extraction, and quality assessment was performed by 2 independent reviewers. The ISPOR-AMCP-NPC checklist was used to assess the credibility of the included studies. RESULTS: From 4948 retrieved studies, a final total of 20 studies were included in the qualitative synthesis. Studies found that screening in diabetic populations was cost-effective (n = 8, 57%) or even cost-saving (n = 6, 43%). Four studies (67%) found that screening in hypertensive populations was also cost-effective. For the general population, findings were inconsistent across studies in which many found screening to be cost-effective (n = 11, 69%), some cost-saving (n = 2, 12%), and others not cost-effective (n = 3, 19%).…

  34. 40
    top 6.2%

    Across-Country Variations of Real-World Data and Evidence for Drugs: A 5-European-Country Study

    Gérard de Pouvourville, David Cunningham, Frank-Ulrich Fricke, Peter Lindgren, Lorenzo Giovanni Mantovani and 4 more

    17 citationsOpen accessdoi:10.1016/j.jval.2023.01.009OpenAlex

  35. 42
    top 6.5%

    The Healthcare Cost Burden of Asthma in Children: A Longitudinal Population-Based Study

    Khabir Ahmad, Rasheda Khanam, Enamul Kabir, Hendrik Jürges

    15 citationsOpen accessdoi:10.1016/j.jval.2023.04.003OpenAlex

  36. 43
    top 6.6%

    The Economic Cost of Thyroid Cancer in France and the Corresponding Share Associated With Treatment of Overdiagnosed Cases

    Mengmeng Li, Filip Meheus, Stéphanie Polazzi, Patricia Delafosse, Françoise Borson‐Chazot and 7 more

    15 citationsOpen accessdoi:10.1016/j.jval.2023.02.016OpenAlex

  37. 44
    top 6.8%

    A Rapid Review on the Value of Biobanks Containing Genetic Information

    Elisabet Rodríguez Llorian, Nicola Kopac, Louloua Ashikhusein Waliji, Kennedy J. Borle, Nick Dragojlovic and 2 more

    15 citationsOpen access (OTHER-OA)doi:10.1016/j.jval.2023.02.017OpenAlex

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    OBJECTIVES: Increasing access to health data through biobanks containing genetic information has the potential to expand the knowledge base and thereby improve screening, diagnosis, and treatment options for many diseases. Nevertheless, although privacy concerns and risks surrounding genetic data sharing are well documented, direct evidence in favor of the hypothesized benefits of data integration is scarce, which complicates decision making in this area. Therefore, the objective of this study is to summarize the available evidence on the research and clinical impacts of biobanks containing genetic information, so as to better understand how to quantify the value of expanding genomic data access. METHODS: Using a rapid review methodology, we performed a search of MEDLINE/PubMed and Embase databases; and websites of biobanks and genomic initiatives published from 2010 to 2022. We classified findings into 11 indicators including outputs (a direct product of the biobank activities) and outcomes (changes in scientific and clinical capacity). RESULTS: Of 8479 abstracts and 101 gray…

  38. 45
    top 6.9%

    Modeling Thyroid Cancer Epidemiology in the United States Using Papillary Thyroid Carcinoma Microsimulation Model

    Oğuzhan Alagöz, Yichi Zhang, Natalia Arroyo, Sara Fernandes‐Taylor, Dou‐Yan Yang and 5 more

    14 citationsOpen accessdoi:10.1016/j.jval.2023.12.007OpenAlex

  39. 46
    top 7.1%

    Logical Inconsistencies in the Health Years in Total and Equal Value of Life-Years Gained

    Mike Paulden, Chris Sampson, James F. O’Mahony, Eldon Spackman, Christopher J. McCabe and 2 more

    13 citationsOpen access (CC-BY)doi:10.1016/j.jval.2023.11.009OpenAlex

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    OBJECTIVES: This study aimed to assess whether recently proposed alternatives to the quality-adjusted life-year (QALY), intended to address concerns about discrimination, are suitable for informing resource allocation decisions. METHODS: We consider 2 alternatives to the QALY: the health years in total (HYT), recently proposed by Basu et al, and the equal value of life-years gained (evLYG), currently used by the Institute for Clinical and Economic Review. For completeness we also consider unweighted life-years (LYs). Using a hypothetical example comparing 3 mutually exclusive treatment options, we consider how calculations are performed under each approach and whether the resulting rankings are logically consistent. We also explore some further challenges that arise from the unique properties of the HYT approach. RESULTS: The HYT and evLYG approaches can result in logical inconsistencies that do not arise under the QALY or LY approaches. HYT can violate the independence of irrelevant alternatives axiom, whereas the evLYG can produce an unstable ranking of treatment options. HYT have…

  40. 47
    top 7.2%

    Emulating Target Trials With Real-World Data to Inform Health Technology Assessment: Findings and Lessons From an Application to Emergency Surgery

    Silvia Moler Zapata, Andrew Hutchings, Stephen O’Neill, Richard J. Silverwood, Richard Grieve

    13 citationsOpen access (CC-BY-NC-ND)doi:10.1016/j.jval.2023.04.010OpenAlex

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    OBJECTIVES: International health technology assessment (HTA) agencies recommend that real-world data (RWD) are used in some circumstances to add to the evidence base about the effectiveness and cost-effectiveness of health interventions. The target trial framework applies the design principles of randomized-controlled trials to RWD and can help alleviate inevitable concerns about bias and design flaws with nonrandomized studies. This article aimed to tackle the lack of guidance and exemplar applications on how this methodology can be applied to RWD to inform HTA decision making. METHODS: We use Hospital Episode Statistics data from England on emergency hospital admissions from 2010 to 2019 to evaluate the cost-effectiveness of emergency surgery for 2 acute gastrointestinal conditions. We draw on the case study to describe the main challenges in applying the target trial framework alongside RWD and provide recommendations for how these can be addressed in practice. RESULTS: The 4 main challenges when applying the target trial framework to RWD are (1) defining the study population,…

  41. 48
    top 7.4%

    Value of an Integrated Home Dialysis Model in the United Kingdom: A Cost-Effectiveness Analysis

    Amanda Erbe, Dana Kendzia, Ellen Busink, Suzanne Carroll, Eline Aas

    13 citationsOpen access (CC-BY)doi:10.1016/j.jval.2023.02.009OpenAlex

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    OBJECTIVES: This study aimed to determine the lifetime cost-effectiveness of increasing home hemodialysis as a treatment option for patients experiencing peritoneal dialysis technique failure compared with the current standard of care. METHODS: A Markov model was developed to assess the lifetime costs, quality-adjusted life-years, and cost-effectiveness of increasing the usage an integrated home dialysis model compared with the current patient pathways in the United Kingdom. A secondary analysis was conducted including only the cost difference in treatments, minimizing the impact of the high cost of dialysis during life-years gained. Sensitivity and scenario analyses were performed, including analyses from a societal rather than a National Health Service perspective. RESULTS: The base-case probabilistic analysis was associated with incremental costs of £3413 and a quality-adjusted life-year of 0.09, resulting in an incremental cost-effectiveness ratio of £36 341. The secondary analysis found the integrated home dialysis model to be dominant. Conclusions on cost-effectiveness did not…

  42. 49
    top 7.5%

    The Associations of Prescription Drug Insurance and Cost-Sharing With Drug Use, Health Services Use, and Health: A Systematic Review of Canadian Studies

    Godefroy Emmanuel Guindon, Erica Stone, Riya Trivedi, Sophiya Garasia, Kimia Khoee and 1 more

    13 citationsOpen access (CC-BY-NC-ND)doi:10.1016/j.jval.2023.02.010OpenAlex

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    OBJECTIVES: In Canada, public insurance for physician and hospital services, without cost-sharing, is provided to all residents. Outpatient prescription drug coverage, however, is provided through a patchwork system of public and private plans, often with substantial cost-sharing, which leaves many underinsured or uninsured. METHODS: We conducted a systematic review to examine the association of drug insurance and cost-sharing with drug use, health services use, and health in Canada. We searched 4 electronic databases, 2 grey literature databases, 5 specialty journals, and 2 working paper repositories. At least 2 reviewers independently screened articles for inclusion, extracted characteristics, and assessed risk of bias. RESULTS: The expansion of drug insurance was associated with increases in drug use, individuals who reported drug insurance generally reported higher drug use, and increases in and higher levels of drug cost-sharing were associated with lower drug use. Although a number of studies found statistically significant associations between drug insurance or cost-sharing…

  43. 50
    top 7.7%

    Stakeholder Engagement in Designing Attributes for a Discrete Choice Experiment With Policy Implications: An Example of 2 Swiss Studies on Healthcare Delivery

    Zora Föhn, Anna Nicolet, Joachim Marti, Cornel Kaufmann, Andreas Balthasar

    13 citationsOpen access (CC-BY)doi:10.1016/j.jval.2023.01.002OpenAlex

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    OBJECTIVES: The use of discrete choice experiments (DCEs) has become increasingly popular in health policy development by involving and analyzing the preferences of target groups for different aspects of the policy intervention. In this article, we aim to contribute to the standardization of the process of attribute and attribute level development for DCEs with policy relevance. To do so, we propose and empirically illustrate a framework tool for the development and reporting of attributes and attribute levels, with the systematic engagement of relevant stakeholders. METHODS: The framework tool was kept general to allow its use as a standard reporting approach on the development process of a DCE, involving relevant stakeholders at each stage. The proposed 3-stage process consists of (1) collection, (2) selection, and (3) refinement of the DCE attributes and attribute levels. The application of the framework tool is illustrated by 2 projects, Health2040 and COCONUTS-both concerning the preferences of the Swiss population for the future organization of healthcare. RESULTS: By engaging…

How to read this list

The cohort is every article, review and book chapter OpenAlex attributes to Value in Health with publication year 2023, excluding retracted works and paratext such as editorials and errata. The top 50 by citation count are listed; the percentile is the rank divided by the cohort size. 29 of the 50 entries have an abstract in OpenAlex. Counts were read on 29 October 2026 and will not be updated on this page, so a quoted rank stays verifiable. Full rules on the methodology page.

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