Value in Health最新文献

{"title":"Costs and Outcomes after Primary and Revision Hip Replacements by Implant Bearing Materials: Analysis of 550,760 linked Patient Records from English Routinely Collected Databases.","authors":"Navvuga P, Lenguerrand E, Patel R, French Jm, Blom Aw, Whitehouse Mr, Marques Emr","doi":"10.1016/j.jval.2025.04.2170","DOIUrl":"https://doi.org/10.1016/j.jval.2025.04.2170","url":null,"abstract":"<p><strong>Objective: </strong>We investigated 10-year revision risks, hospital admission costs, and quality-of-life for patients in the year after elective primary and revision total hip replacement (THR), overall and by implant bearing materials and fixation combination.</p><p><strong>Methods: </strong>We analysed linked National Joint Registry, Hospital Episode Statistics, and Patient Reported Outcome Measures databases for adults undergoing elective primary THR in England (2009-2018). Implants were classified by bearing material combinations (cobalt-chrome and stainless-steel [CC/SS], delta or alumina ceramics, and highly or non-highly crosslinked polyethylene [HCLPE]) and fixation (cemented, uncemented, and hybrid). We estimated hazard ratios with flexible parametric survival models, and generalised linear models for costs and quality-of-life. Missing quality-of-life data was imputed using multiple imputation.</p><p><strong>Results: </strong>We analysed 550,760 elective primary (mean age=69, SD=10.7) and 9,590 subsequent revisions THRs. On average, a primary THR costed, £10,365 (95% confidence interval £10,350,£10,381); quality-of-life after primary was 0.786 (0.785,0.786), and 10-year revision risk was 2.4% (2.08%,2.78%). First and second revisions costed, on average, £20,387 and £24,290; with mean quality-of-life scores of 0.609 and 0.502 , respectively. Compared with cemented CC/SS-on-HCLPE, cemented delta-on-HCLPE, uncemented delta-on-HCLPE, and hybrid delta-on-HCLPE, alumina-on-HCLPE, delta-on-delta and alumina-on-alumina were associated with lower one-year costs, improved quality-of-life, and lower 10-year revision risks.</p><p><strong>Conclusions: </strong>Revision surgery costs double that of an elective primary with drastically lower quality-of-life. Bearing materials and fixation were associated with varying one-year costs, quality-of-life scores and revision risks. Although observational data may be affected by unmeasured confounding, our findings could help guide procurement decisions given changing trends in implant choice.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":4.9,"publicationDate":"2025-05-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144151952","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"How similar is similar enough? Assessment of indirect treatment comparisons to support similarity for NICE's cost comparison route.","authors":"Dawn Lee, Alex Allen, Alan Lovell, Ahmed Abdelsabour, Edward C F Wilson, G J Melendez-Torres","doi":"10.1016/j.jval.2025.05.003","DOIUrl":"https://doi.org/10.1016/j.jval.2025.05.003","url":null,"abstract":"<p><strong>Objectives: </strong>Health technology assessment bodies are increasingly using cost comparison (cost-minimisation) analysis to manage rising demand. Such an approach requires demonstration of clinical similarity. While head-to-head comparisons from equivalence or non-inferiority studies are usually accepted as evidence of similarity, guidance is lacking on when equivalence may be assumed from indirect treatment comparison (ITC), either quantitative or qualitative.</p><p><strong>Methods: </strong>We reviewed methods papers and previous NICE appraisals to assess methods for determining equivalence when no head-to-head trial exists, identify how NICE and their committees decide if a cost-comparison route is suitable, and explore how best to present evidence of similarity to aid decision-making.</p><p><strong>Results: </strong>We identified 5 methods papers, 41 case studies, and 33 appraisals that used cost comparison based on ITC. All 33 appraisals were recommended, though none applied any of the formal methods identified from the literature. Instead, companies used narrative summaries to assert similarity, often based on a lack of significant differences, leading to committee uncertainty that was typically resolved through clinical expert input. The most promising methods we reviewed were the estimation of non-inferiority ITCs in a Bayesian framework followed by a straightforward, probabilistic comparison of the indirectly estimated treatment effect against a pre-specified non-inferiority margin.</p><p><strong>Conclusions: </strong>Formal methods to assess equivalence in ITC-based cost comparison are emerging but have not yet been applied in practice. We provide recommendations for how these methods and qualitative methods, such as evaluation of the plausibility of class effects and clinical expert input, could help address uncertainties in assuming equivalence.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":4.9,"publicationDate":"2025-05-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144143722","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Addressing subgroup-specific treatment effects to avoid inefficient care: the conflict between statistical and health economic analyses.","authors":"Sopany Saing, Gerjon Hannink, Amarens Geuzinge, Hendrik Koffijberg","doi":"10.1016/j.jval.2025.05.004","DOIUrl":"https://doi.org/10.1016/j.jval.2025.05.004","url":null,"abstract":"<p><strong>Objective: </strong>When patients are classified into subgroups based on previously identified heterogeneity, this heterogeneity may affect the cost-effectiveness of health interventions. Whether this heterogeneity is reflected or ignored in cost-effectiveness analysis (CEA) may influence reimbursement decisions. This is illustrated using a simulation study of a hypothetical treatment to prevent disease progression.</p><p><strong>Methods: </strong>With the DARTH Sick-Sicker Markov model, we analysed the cost-effectiveness of Treatment versus Standard of Care in a population comprising Group 1 (G1) and Group 2 (G2). We compared three strategies for informing reimbursement decisions; 1) ignore evidence on subgroup differences ('ignore subgroup evidence'); 2) test for subgroup differences in trial data at hand ('statistically-guided'), and 3) use all evidence on subgroup differences ('all evidence'). This simulation study varied total sample size, G2 proportion, treatment effectiveness, and baseline mortality risk. For each scenario the net health benefit (NHB) and reimbursement decision (i.e. reimburse in both subgroups, G1 only, G2 only, or no reimbursement) was determined per strategy.</p><p><strong>Results: </strong>The statistically-guided strategy led to subgroups being ignored except for the largest total sample sizes. At a willingness-to-pay threshold of €50,000/QALY gained, the statistically-guided strategy resulted in an incremental NHB of -1.00 and 0.49 when compared to the strategies of ignoring subgroup evidence and for incorporating, respectively.</p><p><strong>Conclusions: </strong>When subgroup heterogeneity is known, ignoring subgroups, or taking a statistically-guided approach will result in suboptimal reimbursement decisions and thus fail to optimise societal benefits. Therefore, subgroup-specific CEAs should be informed by all available evidence of subgroup differences.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":4.9,"publicationDate":"2025-05-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144143668","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Implementing the Generalized Risk-Adjusted Cost-Effectiveness (GRACE) Model for Sickle Cell Disease (SCD) - A Case Study.","authors":"Marlon Graf, Joris Kleintjens, Md Tahsin Hasan, Natalie Land, Jacquelyn W Chou, Karen Mulligan","doi":"10.1016/j.jval.2025.05.005","DOIUrl":"https://doi.org/10.1016/j.jval.2025.05.005","url":null,"abstract":"<p><strong>Objectives: </strong>This study aims to quantify the societal value of curing sickle cell disease (SCD) by implementing the Generalized Risk-Adjusted Cost-Effectiveness (GRACE) method, thereby adjusting for factors such as disease severity, reduction of uncertainty in treatment outcomes, and trade-offs between quality of life (QoL) and life expectancy (LE) in a cost-effectiveness analysis (CEA).</p><p><strong>Methods: </strong>Using GRACE, we recalibrated a recent health technology assessment (HTA) in SCD for two gene therapies, lovo-cel and exa-cel. The GRACE framework modified existing CEA by adjusting willingness to pay (WTP) thresholds based on untreated illness severity, incorporating treatment outcome uncertainty, and varying the substitution rates between LE and QoL across health states.</p><p><strong>Results: </strong>Implementing the GRACE framework resulted in a 6% reduction in both direct and societal incremental cost-effectiveness ratios (ICERs) for lovo-cel and exa-cel, demonstrating a decrease from $192,651 and $161,816 to $182,036 and $152,900 per quality-adjusted life year, respectively. Additionally, WTP thresholds increased by approximately 50%, reflecting a higher valuation of treatments under GRACE. GRACE-adjusted estimates suggest lovo-cel and exa-cel are cost-effective from both direct payer and societal perspectives.</p><p><strong>Conclusions: </strong>The GRACE method offers a more comprehensive and precise estimation of societal value, leading to more efficient and equitable resource allocation. This study not only highlights the limitations of traditional CEA in capturing the total societal value of treatments for severe diseases, but also provides a roadmap for incorporating GRACE model elements into HTAs, thereby facilitating broader acceptance of innovative therapies that significantly enhance patient QoL.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":4.9,"publicationDate":"2025-05-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144143724","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The increasing use of clinical outcome assessment in FDA labels and G-BA, NICE and HAS assessments from 2012-2022.","authors":"Marieke Krol, Joshua Maher, Tom Halmos, Matthew Reaney, Ali Shana'a, Benoit Arnould, Veleka Allen, Catherine Coulouvrat","doi":"10.1016/j.jval.2025.05.002","DOIUrl":"https://doi.org/10.1016/j.jval.2025.05.002","url":null,"abstract":"<p><strong>Objectives: </strong>Clinical outcome assessments (COAs) are increasingly used in clinical trials. However, knowledge gaps remain around their inclusion in market access decision-making for pharmaceuticals and the perceived added value in capturing the patient experience. This study examined trends in the inclusion of COAs in US Food and Drug Administration (FDA) labels and European health technology assessments (HTAs) beyond those essential to assess primary endpoints or determine cost-effectiveness.</p><p><strong>Methods: </strong>The inclusion of \"non-mandatory COAs\" was analysed in FDA labels and HTAs of German, French and English bodies, from 2012-2022. Changes in COA use are reported overall, by COA type, concept, and therapeutic area (TA).</p><p><strong>Results: </strong>In FDA labels the inclusion of non-mandatory COA increased from 18.6% to 28.4% from 2012-2016 to 2017-2022. The average number of unique COAs in FDA labels approximately doubled. Non-mandatory COA inclusions in HTAs increased from 45.2% to 64.9%. The average number of unique COAs per HTA also approximately doubled. Patient reported outcomes (PROs) were the most reported COAs in FDA labels and HTAs in both time periods. In FDA labels and HTAs, most TAs showed increases in non-mandatory COA inclusion. In FDA labels, COA instruments mostly measured signs and symptoms. In HTAs, they mostly measured health status and quality of life.</p><p><strong>Conclusion: </strong>The findings show increasing trends in the use of non-mandatory COA in HTAs, and to a lesser extent FDA labels, suggesting a greater awareness of COA benefits and a changing practice of inclusion towards more patient-centricity.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":4.9,"publicationDate":"2025-05-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144132081","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Synergizing Needs Assessments and Patient Preference Studies for Enhanced Patient-centred Decision-Making in Healthcare.","authors":"Elise Schoefs, Alice Vanneste, Alessandra Blonda, Zilke Claessens, Khadidja Abdallah, Fábio Cardoso Borges, Jolien Broekmans, Thomas Desmet, Teodora Lalova-Spinks, Phaedra Locquet, Janos Meszaros, Lauren Michiels, Charlotte Verbeke, Io Wens, Isabelle Huys","doi":"10.1016/j.jval.2025.04.2169","DOIUrl":"https://doi.org/10.1016/j.jval.2025.04.2169","url":null,"abstract":"<p><strong>Objective: </strong>Over the past decades, criticism has grown regarding the supply-driven approach of medicinal product development. In response, patient-centred methods have been developed to inform decision-making. This perspective paper aims to reflect on two of these methods, being needs assessments and patient preference studies (PPSs).</p><p><strong>Methods: </strong>A reflection was conducted based on existing literature and our collaborative experiences, proposing a conceptual framework that synergistically combines needs assessments and PPSs.</p><p><strong>Results: </strong>Needs assessments identify unmet health-related needs from the patient and/or societal perspective, while PPSs determine the trade-offs patients make among treatment options. Although both methods produce patient-based evidence, their differing scopes and purposes offer complementary benefits and limitations. Combining needs assessments with PPSs can result in significant advantages by providing a holistic and in-depth understanding of patients' needs and preferences. In their conceptual framework, the authors advocate for a sequential approach: conducting a needs assessment to identify a broad spectrum of unmet health-related needs, followed by a PPS to capture nuanced preferences guiding patients' priorities. While this approach enhances accuracy and relevance, practical constraints and contextual considerations may hamper its application, necessitating careful consideration of the most suitable approach based on the specific research context.</p><p><strong>Conclusions: </strong>This perspective paper provides clarity on the effective use of both methods in navigating patient-centred research in healthcare. It emphasizes the needs for well-designed, unbiased studies applying this conceptual framework to shift from a supply-driven to a needs- and preference-driven healthcare system, ensuring innovations align more closely with patients' true needs and preferences.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":4.9,"publicationDate":"2025-05-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144095226","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Evaluation of HTA Frameworks for In Vivo Diagnostics: Assessing Methodological Gaps and Implications for Market Access.","authors":"Ricardo De la Fuente, Hermilo A Cabra Gomez, Shivani K Shah, Chad Gibson, Regina C Munter-Young","doi":"10.1016/j.jval.2025.05.001","DOIUrl":"https://doi.org/10.1016/j.jval.2025.05.001","url":null,"abstract":"<p><strong>Objectives: </strong>This study investigates the evaluation of in vivo diagnostics, particularly molecular imaging (MI) tracers and contrast media (CM), within health technology assessment (HTA) frameworks across 28 countries. The aim is to identify variations in HTA methodologies and highlight gaps in the evaluation of diagnostics, focusing on market access and reimbursement.</p><p><strong>Methods: </strong>Guidance documents from Ministry of Health, national insurers, and HTA organizations were reviewed to assess roles and methodologies for evaluating In vitro diagnostics (IVDs), pharmaceuticals, and in vivo diagnostics. HTA organizations were grouped into five categories based on assessment processes and legal influence. A mapping methodology created regulatory-to-reimbursement process maps, resulting in two taxonomies. Representative countries from each group were analyzed for evidence requirements for MI tracers and CM. Five published HTA case studies were used to validate findings and evaluate the impact of HTA decisions on coverage and reimbursement.</p><p><strong>Results: </strong>The study found that IVDs were universally evaluated as medical technologies, while MI tracers and CM were often evaluated as pharmaceuticals, with diagnostic modalities considered separately. HTA frameworks in 11 countries were analyzed, revealing variation in how evidence requirements were defined. Case studies revealed discrepancies in reimbursement decisions despite similar clinical evidence, highlighting inconsistencies in HTA methodologies.</p><p><strong>Conclusion: </strong>This study identifies gaps in HTA frameworks for evaluating in vivo diagnostics, including reliance on pharmaceutical-centric models, lack of standardization, and inconsistent methodologies across markets. These gaps pose barriers to access and reimbursement for MI tracers and CM, emphasizing the need for methodologies tailored specifically to in vivo diagnostics.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":4.9,"publicationDate":"2025-05-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144095220","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Growing evidence base on condition-specific patient-reported outcomes measures for Long Covid.","authors":"Adam B Smith, Darren Greenwood, Manoj Sivan","doi":"10.1016/j.jval.2024.12.011","DOIUrl":"https://doi.org/10.1016/j.jval.2024.12.011","url":null,"abstract":"","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":4.9,"publicationDate":"2025-05-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144017652","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The psychometric performance of the EQ-HWB in caregivers of persons living with dementia.","authors":"Cate Bailey, Tessa Peasgood, Bernhard Michalowsky, Lidia Engel","doi":"10.1016/j.jval.2025.04.2168","DOIUrl":"https://doi.org/10.1016/j.jval.2025.04.2168","url":null,"abstract":"<p><strong>Introduction: </strong>The EQ Health and Wellbeing (EQ-HWB) is a generic instrument recently developed for use in economic evaluations across public health and social care, including impacts on caregivers. Providing informal care to a person living with dementia can significantly impact the health and wellbeing of carers. However, it is unclear to what extent the EQ-HWB can capture such impacts. This project aimed to investigate the psychometric properties of the EQ-HWB compared to other available instruments in caregivers of persons living with dementia.</p><p><strong>Method: </strong>An online survey included demographics, informal care-related questions, and quality-of-life measures suitable for caregivers (C-DEMQOL and CarerQol) and the 25-item EQ-HWB. We used the UK pilot value set for the index-scores for the EQ-HWB-S (9-item short form). The psychometric properties of the EQ-HWB and EQ-HWB-S were assessed in terms of acceptability (missing data), response distribution, known-groups validity analysis using t-tests and one-way ANOVAs (Cohen's d and Eta-squared for effect size), convergent validity (Spearman correlations), and an exploration of dimensionality using exploratory factor analysis.</p><p><strong>Results: </strong>In known-group validity analysis, the EQ-HWB instruments performed similarly to the C-DEMQOL in terms of effect sizes across a range of variables, and slightly better than the CarerQol on some variables. At the item level, 13 of the 25 items could discriminate respondents by caregiving time and 23 of 25 items by caregivers' general self-reported health. Most hypothesised correlations in convergent validity analysis were found to be above 0.3.</p><p><strong>Conclusion: </strong>Our results suggest that the EQ-HWB instruments performed well in this population.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":4.9,"publicationDate":"2025-05-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144017385","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Author Reply.","authors":"Yilin Chen, Peter Martin, Lurdes Y T Inoue, Anirban Basu, Josh J Carlson","doi":"10.1016/j.jval.2025.02.019","DOIUrl":"https://doi.org/10.1016/j.jval.2025.02.019","url":null,"abstract":"","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":4.9,"publicationDate":"2025-05-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144048460","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}