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The Implementation of NICE Severity Modifier in the Single Technology Appraisal of Mirvetuximab Soravtansine: External Assessment Group Perspective. NICE严重度调节剂在Mirvetuximab Soravtansine单一技术评价中的应用:外部评价组视角。
IF 6.2 2区 医学
Value in Health Pub Date : 2026-09-21 DOI: 10.1016/j.jval.2026.08.009
Ahmed Abdelsabour, Jemma Perks, Charlotte Kelman, Sophie Robinson, Caroline Farmer, Dawn Lee
{"title":"The Implementation of NICE Severity Modifier in the Single Technology Appraisal of Mirvetuximab Soravtansine: External Assessment Group Perspective.","authors":"Ahmed Abdelsabour, Jemma Perks, Charlotte Kelman, Sophie Robinson, Caroline Farmer, Dawn Lee","doi":"10.1016/j.jval.2026.08.009","DOIUrl":"https://doi.org/10.1016/j.jval.2026.08.009","url":null,"abstract":"<p><strong>Objectives: </strong>NICE's severity modifier uses absolute and proportional QALY shortfalls to determine whether higher cost-effectiveness thresholds should apply. We use the appraisal of mirvetuximab soravtansine in platinum-resistant ovarian cancer to show how modelling choices can affect the severity weight and to draw out practical recommendations for future appraisals.</p><p><strong>Methods: </strong>We reviewed the company and EAG approaches to estimating QALY shortfalls, focusing on utility values, starting age and standard-care overall survival extrapolation. We considered how these assumptions changed expected QALYs under standard care, the resulting QALY shortfalls and the severity weight applied.</p><p><strong>Results: </strong>The severity weight was sensitive to all three assumptions. The EAG's preferred utility values increased expected QALYs under standard care and reduced the severity weight from 1.7 to 1.2 in the EAG base case. Starting age was also influential, with older ages making the higher severity threshold less likely to be met. Survival extrapolation affected expected QALYs under standard care; longer-tailed curves reduced absolute and proportional QALY shortfalls, whereas shorter-tailed curves made the higher severity weight more likely.</p><p><strong>Conclusion: </strong>Severity-modifier decisions can change under plausible modelling assumptions, particularly when estimates are close to the threshold between weights. When severity weights are used, appraisals should include a standard sensitivity analysis reporting the absolute QALY shortfall, proportional QALY shortfall and resulting severity weight under alternative assumptions. Clearer reporting of these calculations would make the basis for severity-weight decisions easier to scrutinise across appraisals.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":6.2,"publicationDate":"2026-09-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149017571","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}
引用次数: 0
Generative Artificial Intelligence for Systematic Literature Reviews: A Good Practices Report of an ISPOR Special Task Force. 系统文献综述的生成人工智能:ISPOR特别工作组的良好实践报告。
IF 6.2 2区 医学
Value in Health Pub Date : 2026-09-19 DOI: 10.1016/j.jval.2026.08.007
Rachael L Fleurence, Riaz Qureshi, Rakesh Aggarwal, Jiang Bian, Dalia Dawoud, Diana Delnoij, Ruth R Faden, Sven Klijn, Raphael Sonabend, Jagpreet Chhatwal
{"title":"Generative Artificial Intelligence for Systematic Literature Reviews: A Good Practices Report of an ISPOR Special Task Force.","authors":"Rachael L Fleurence, Riaz Qureshi, Rakesh Aggarwal, Jiang Bian, Dalia Dawoud, Diana Delnoij, Ruth R Faden, Sven Klijn, Raphael Sonabend, Jagpreet Chhatwal","doi":"10.1016/j.jval.2026.08.007","DOIUrl":"https://doi.org/10.1016/j.jval.2026.08.007","url":null,"abstract":"<p><strong>Objectives: </strong>Systematic literature reviews (SLRs) are foundational to evidence-based medicine, including health technology assessment (HTA) and health economics and outcomes research (HEOR). Generative artificial intelligence (GenAI) tools are increasingly used in SLR workflows, yet no good practice guidance exists. This ISPOR Task Force report provides evidence-informed recommendations for responsible GenAI use across core SLR tasks.</p><p><strong>Methods: </strong>A PRISMA-adapted rapid evidence assessment identified 115 empirical studies evaluating GenAI in SLR tasks published between November 2022 and July 2025. Findings were synthesized qualitatively across seven tasks. A structured task-level assessment framework spanning eight domains informed good practice recommendations, derived through Task Force consensus among experts in SLR methodology, HTA, AI development, bioethics, and regulatory science.</p><p><strong>Results: </strong>Evidence supported GenAI use for high-recall title/abstract screening and structured first-pass data extraction within human-in-the-loop workflows with explicit oversight. Autonomous deployment was not supported. Evidence for full-text screening, qualitative synthesis, and report writing was more conditional, depending on workflow design and oversight, while risk of bias assessment showed the lowest readiness. End-to-end autonomous SLR generation was not recommended. Performance was most reliable within clearly defined workflows, with pre-specified rules for flagging records and explicit human review and conflict-resolution processes.</p><p><strong>Conclusions: </strong>Based on current evidence, GenAI can augment, but not replace, human expertise in SLRs. Responsible use requires evaluating GenAI suitability for each review task, retaining human accountability at all decision points, and documenting AI use as a core methodological component. Because GenAI evolves rapidly, these recommendations reflect evidence through July 2025 and warrant periodic updating.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":6.2,"publicationDate":"2026-09-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149009180","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}
引用次数: 0
Health Technology Assessment Lifecycle Concepts: A Scoping Review Outlining the Change in Policies to Manage Uncertainties Associated With Reimbursement of Medicines. 卫生技术评估生命周期概念:概述管理与药品报销相关的不确定性的政策变化的范围审查。
IF 6.2 2区 医学
Value in Health Pub Date : 2026-09-16 DOI: 10.1016/j.jval.2026.08.006
Jelle Stoelinga, Carla E Hollak, Lonneke Timmers, Wim G Goettsch, Christine Leopold
{"title":"Health Technology Assessment Lifecycle Concepts: A Scoping Review Outlining the Change in Policies to Manage Uncertainties Associated With Reimbursement of Medicines.","authors":"Jelle Stoelinga, Carla E Hollak, Lonneke Timmers, Wim G Goettsch, Christine Leopold","doi":"10.1016/j.jval.2026.08.006","DOIUrl":"https://doi.org/10.1016/j.jval.2026.08.006","url":null,"abstract":"<p><strong>Objective: </strong>Health Technology Assessment (HTA) agencies aim to evaluate the value of a health technology at specific points in its lifecycle. However, much remains unclear about how HTA lifecycle concepts are employed and which stakeholders are involved. We aimed to clarify the use of HTA lifecycle concepts and how they relate to each other to facilitate implementation and outline next steps for policy development.</p><p><strong>Methods: </strong>Following the Preferred Reporting Items for Systematic Reviews and Meta-Analyses extension for Scoping Reviews, we analyzed scientific and policy literature on HTA lifecycle concepts for medicines in the PubMed, Embase, and Overton databases. Data were extracted using standardized forms and analyzed both descriptively and using a directed qualitative content analysis.</p><p><strong>Results: </strong>We identified 60 scientific studies and 5 policy documents covering five different HTA lifecycle concepts. Health Technology Reassessment (HTR), Health Technology Management (HTM), and Lifecycle HTA (LC-HTA) were most frequently described in scientific literature (respectively, 22, 17, and 10 studies). We found that the activities of these concepts partially overlapped, but often differed in their content. In addition to activities identified in HTR and HTM, LC- HTA included pre-market entry activities that facilitate planning of risk mitigation strategies.</p><p><strong>Conclusions: </strong>The anticipatory nature and content of activities within LC-HTA led to it emerging as the most appropriate for addressing the clinical and financial risks to the initial reimbursement decisions for contemporary medicines. Both capacity building and further development of triaging for risk-based reimbursement pathway assignment can aid in the implementation of LC-HTA in practice.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":6.2,"publicationDate":"2026-09-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148983412","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}
引用次数: 0
Cost-Effectiveness of Cilta-cel in Lenalidomide-Refractory Multiple Myeloma: An Analysis of the CARTITUDE-4 Trial. cilta - cell治疗来那度胺难治性多发性骨髓瘤的成本-效果:CARTITUDE-4试验分析
IF 6.2 2区 医学
Value in Health Pub Date : 2026-09-11 DOI: 10.1016/j.jval.2026.08.004
Yanxiao Zhang, Yabei Li, Jianhua Chen, Guojun Sun, Yiman Zhang
{"title":"Cost-Effectiveness of Cilta-cel in Lenalidomide-Refractory Multiple Myeloma: An Analysis of the CARTITUDE-4 Trial.","authors":"Yanxiao Zhang, Yabei Li, Jianhua Chen, Guojun Sun, Yiman Zhang","doi":"10.1016/j.jval.2026.08.004","DOIUrl":"10.1016/j.jval.2026.08.004","url":null,"abstract":"<p><strong>Objective: </strong>Building on the survival advantages demonstrated in the Phase III CARTITUDE-4 trial, this study quantifies the cost-effectiveness of ciltacabtagene autoleucel (cilta-cel) versus standard regimens (PVd or DPd) for lenalidomide-refractory multiple myeloma from a US mixed-payer perspective.</p><p><strong>Methods: </strong>A Markov model with 3- or 4-week cycles was developed to simulate long-term clinical trajectories. Economic viability was determined by calculating incremental cost-effectiveness ratios through integrated costs and quality-adjusted life-years (QALYs), benchmarked against a willingness-to-pay threshold of $150 000/QALY. Robustness was verified by means of sensitivity and scenario analyses.</p><p><strong>Results: </strong>Under the 10-year time horizon, patients in the cilta-cel cohort accumulated an expected 3.99 QALYs, compared with 2.96 QALYs in the standard-of-care group. Concurrently, the expected total medical costs for the cilta-cel group were $574 425.45, which were lower than the $645 495.60 incurred by the standard therapy group. Compared with conventional treatment, the cilta-cel regimen demonstrated economic absolute dominance, achieving superior health outcomes alongside reduced overall costs. Scenario and sensitivity analyses confirmed the robustness of the model. The acquisition cost of cilta-cel, the subsequent treatment costs in the standard group, and the discount rate were identified as the 3 most influential factors.</p><p><strong>Conclusions: </strong>Relative to standard therapy, cilta-cel is projected to have an economic absolute cost-effectiveness advantage in the treatment of lenalidomide-refractory multiple myeloma.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":6.2,"publicationDate":"2026-09-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148945794","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}
引用次数: 0
An Application of Iterative Health Economic Evaluation: An Update on the Early Cost Effectiveness of Whole-Genome Sequencing in Advanced Non-small-Cell Lung Cancer. 迭代健康经济评估的应用:全基因组测序在晚期非小细胞肺癌早期成本效益的最新进展
IF 6.2 2区 医学
Value in Health Pub Date : 2026-09-09 DOI: 10.1016/j.jval.2026.06.029
Andrea Fernández Coves, Bram Ramaekers, Martijn Simons, Lucas F van Schaik, Sabine Grimm, Valesca P Retèl, Manuela Joore
{"title":"An Application of Iterative Health Economic Evaluation: An Update on the Early Cost Effectiveness of Whole-Genome Sequencing in Advanced Non-small-Cell Lung Cancer.","authors":"Andrea Fernández Coves, Bram Ramaekers, Martijn Simons, Lucas F van Schaik, Sabine Grimm, Valesca P Retèl, Manuela Joore","doi":"10.1016/j.jval.2026.06.029","DOIUrl":"https://doi.org/10.1016/j.jval.2026.06.029","url":null,"abstract":"<p><strong>Objective: </strong>Whole genome sequencing (WGS) can identify more druggable targets than the standard of care (SoC) panels, however, its health effects and costs are highly uncertain. Given the rapidly evolving treatment landscape and pricing, an iterative approach is crucial to continuously reassess evidence and adapt economic models. Our objective was to update a previously developed economic model for WGS.</p><p><strong>Methods: </strong>We used a structured approach to identify and report model elements requiring updates, based on established tools and methodological guidance, and applied it to the probabilistic decision model by Simons et al.(2021), which compared SoC, WGS, and SoC followed by WGS in patients with inoperable stage IIIB, C/IV NSCLC in the Dutch setting.</p><p><strong>Results: </strong>Updates included a new treatment (sotorasib), revised drug and diagnostic costs, and adherence to the latest guidelines. Drug and WGS diagnostics costs fell by 8% and 26%, respectively. SoC diagnostic prices increased by 17%. We explored the impact of the prevalence of druggable targets, effectiveness of off-label treatments, (academic-specific) diagnostic costs, and price negotiations. The ICER of WGS versus SoC decreased from €737,197 to €419,053/QALY. WGS would become cost-effective if diagnostic costs descended from €2,180 to €1,246 or if additional druggable targets were identified in ≥3.3% of patients.</p><p><strong>Conclusion: </strong>Our structured approach effectively identified items in the original analysis requiring updates and provides a foundation for further developing a checklist to guide iterative HTA. Continued monitoring and assessment of new treatment options, the dynamic diagnostics and costs throughout the life-cycle remain necessary to determine when WGS can be considered cost-effective.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":6.2,"publicationDate":"2026-09-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148921121","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}
引用次数: 0
Does Conventional Cost-Effectiveness Analysis Disadvantage Treatments for Severe Disease? Evidence From US Value Assessments. 传统的成本效益分析是否不利于重症治疗?来自美国价值评估的证据。
IF 6.2 2区 医学
Value in Health Pub Date : 2026-09-08 DOI: 10.1016/j.jval.2026.08.005
Antal T Zemplenyi, R Brett McQueen, Harry Gyimah Gyamfi, Deepika Paratane, Michael J DiStefano
{"title":"Does Conventional Cost-Effectiveness Analysis Disadvantage Treatments for Severe Disease? Evidence From US Value Assessments.","authors":"Antal T Zemplenyi, R Brett McQueen, Harry Gyimah Gyamfi, Deepika Paratane, Michael J DiStefano","doi":"10.1016/j.jval.2026.08.005","DOIUrl":"10.1016/j.jval.2026.08.005","url":null,"abstract":"<p><strong>Objectives: </strong>This study aimed to assess how frequently quality-adjusted life-year (QALY)-based and life-year (LY)-based incremental health benefits meaningfully differ and whether those differences affect cost-effectiveness conclusions; how these differences vary across disease categories and what disease severity composition underlies this variation; and whether the QALY-LY difference is associated with baseline disease severity.</p><p><strong>Methods: </strong>We analyzed 167 treatment-comparator assessment pairs from 71 Institute for Clinical and Economic Review reports (2017-2025), spanning 65 conditions across eight disease categories. We compared incremental QALYs and LYs, assessed cost-effectiveness concordance at $100 000 and $150 000 thresholds, and examined cross-category variation. In a restricted sample of 82 pairs, we assessed associations between severity measures and the QALY-LY difference using linear regression and Spearman rank correlation.</p><p><strong>Results: </strong>QALYs showed greater incremental gains than LYs in 59% of assessments; LYs were greater in 22%. Cost-effectiveness conclusions were highly concordant (92.2% at $100 000; 87.4% at $150 000), although QALYs classified 20% to 32% more interventions as cost-effective. The QALY-LY difference varied significantly across disease categories (P < .001), reflecting differences in disease burden composition. Neither LY lost nor equal-value of LYs lost was meaningfully associated with the QALY-LY difference. The only significant association emerged for baseline health-related quality of life under standard care, where greater impairment was associated with larger incremental gains in QALYs relative to LYs. Both metrics scaled proportionally with disease severity.</p><p><strong>Conclusions: </strong>QALY-based cost-effectiveness does not systematically disadvantage treatments for more severe diseases. Replacing QALYs with LYs would omit the therapeutic value most relevant to high-morbidity populations, potentially working against the interests of the patient populations that this policy debate seeks to protect.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":6.2,"publicationDate":"2026-09-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148913214","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}
引用次数: 0
Cost-Effectiveness of Sodium-Glucose Cotransporter-2 Inhibitors and Angiotensin Receptor-Neprilysin Inhibitors in Addition to Standard Treatment of Chronic Heart Failure: A Systematic Review and Meta-Analysis SGLT2i和ARNi在慢性心力衰竭标准治疗之外的成本-效果:系统回顾和荟萃分析
IF 6.2 2区 医学
Value in Health Pub Date : 2026-09-01 Epub Date: 2026-04-08 DOI: 10.1016/j.jval.2026.03.2233
Nattakrit Tongpoonsakdi MSc, Amarit Tansawet MD, PhD, Teerapat Yingchoncharoen MD, Porntep Amornritvanich MD, Rini Noviyani PhD, Montarat Thavorncharoensap PhD, Gareth J. McKay PhD, John Attia PhD, Ammarin Thakkinstian PhD
{"title":"Cost-Effectiveness of Sodium-Glucose Cotransporter-2 Inhibitors and Angiotensin Receptor-Neprilysin Inhibitors in Addition to Standard Treatment of Chronic Heart Failure: A Systematic Review and Meta-Analysis","authors":"Nattakrit Tongpoonsakdi MSc,&nbsp;Amarit Tansawet MD, PhD,&nbsp;Teerapat Yingchoncharoen MD,&nbsp;Porntep Amornritvanich MD,&nbsp;Rini Noviyani PhD,&nbsp;Montarat Thavorncharoensap PhD,&nbsp;Gareth J. McKay PhD,&nbsp;John Attia PhD,&nbsp;Ammarin Thakkinstian PhD","doi":"10.1016/j.jval.2026.03.2233","DOIUrl":"10.1016/j.jval.2026.03.2233","url":null,"abstract":"<div><h3>Background</h3><div>Guideline-directed medical therapies (GDMTs) have improved heart failure (HF) outcomes, particularly with the emergence of sodium-glucose cotransporter-2 inhibitors (SGLT2i) and angiotensin receptor-neprilysin inhibitors (ARNI). However, their cost-effectiveness remains uncertain across HF phenotypes, income levels, and analytic perspectives.</div></div><div><h3>Objectives</h3><div>The study aimed to evaluate and compare the incremental net benefit (INB) of SGLT2i and/or ARNI added to traditional GDMTs, stratified by HF phenotype, country income level, and study perspective.</div></div><div><h3>Methods</h3><div>PubMed, Scopus, and the Cost-Effectiveness Analysis Registry were searched through October 2024. Economic evaluations assessing SGLT2i or ARNI added to traditional GDMTs were included. INBs and variances were extracted or calculated; if unavailable, variances were imputed via regression or based on similar studies. Random-effects meta-analyses were performed by treatment-comparator pairs, HF phenotype, income level, and perspective. Risk of bias was assessed using the Bias in Economic Evaluation checklist.</div></div><div><h3>Results</h3><div>Seventy studies were included. Adding SGLT2i to traditional GDMTs was cost-effective in high-income countries (HICs) for HF with reduced ejection fraction (HFrEF) from healthcare perspective (pooled INB = US $13 114.52; 95% CI: 4257.40–21 971.63), whereas evidence was inconclusive for upper-middle (UMICs) or lower-income countries. Quadruple regimen (SGLT2i + ARNI) appeared cost-effective in HICs and UMICs, although evidence was limited. Replacing other renin-angiotensin-aldosterone system blockers with ARNI was only cost-effective for HFrEF from societal perspective in HICs (INB = US $14 843.66; 95% CI: 566.36–29 120.96). Evidence for other HF phenotypes remained inconclusive evidence.</div></div><div><h3>Conclusions</h3><div>Adding SGLT2i and ARNI improves GDMTs cost-effectiveness for the treatment of HFrEF, especially in HICs. More evidence is needed for other HF types and income settings.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"29 9","pages":"Pages 1725-1739"},"PeriodicalIF":6.2,"publicationDate":"2026-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147655255","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Evaluating Policy Options for New Treatments Under Uncertain External Evidence 在不确定的外部证据下评估新疗法的政策选择。
IF 6.2 2区 医学
Value in Health Pub Date : 2026-09-01 Epub Date: 2026-04-16 DOI: 10.1016/j.jval.2026.03.2240
Yizhi Liang MS, Boshen Jiao PhD, MPH
{"title":"Evaluating Policy Options for New Treatments Under Uncertain External Evidence","authors":"Yizhi Liang MS,&nbsp;Boshen Jiao PhD, MPH","doi":"10.1016/j.jval.2026.03.2240","DOIUrl":"10.1016/j.jval.2026.03.2240","url":null,"abstract":"<div><h3>Objectives</h3><div>Health policymakers in many jurisdictions must decide whether to adopt new medical treatments based on clinical evidence generated abroad, despite uncertainty about the applicability of those results to local populations. This creates a fundamental trade-off: enabling early access to promising therapies versus delaying adoption until locally relevant evidence becomes available.</div></div><div><h3>Methods</h3><div>We evaluate four policy options under uncertain external clinical evidence: delayed adoption pending a local confirmatory trial, conditional adoption with a standard trial requirement, immediate adoption without a trial, and immediate rejection. To compare these strategies, we extend a Bayesian value-of-information framework using a power prior. The power prior parameter determines the weight assigned to external evidence based on its applicability to the local population. The framework distinguishes pre- and postconfirmation periods and accounts for potential delays in trial completion when conditional approval is granted. We illustrate the approach using a lung cancer therapy trialed exclusively in China and considered for use in the United States.</div></div><div><h3>Results</h3><div>The case study shows that optimal policy depends jointly on uncertainty about external evidence applicability, trial duration, delays in trial completion, and price. High uncertainty favors delayed adoption, whereas longer trial durations make conditional adoption more attractive by increasing the cost of waiting. Longer delays in trial completion can shift the optimal policy toward delayed adoption. Price reductions shift the policy frontier toward earlier access.</div></div><div><h3>Conclusions</h3><div>Policies for treatments supported by external evidence should be tailored to the evidentiary context, accounting for applicability uncertainty, trial timelines, delays in trial completion, and pricing.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"29 9","pages":"Pages 1618-1625"},"PeriodicalIF":6.2,"publicationDate":"2026-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147718189","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}
引用次数: 0
Adoption of Managed Entry Agreements for Advanced Therapy Medicinal Products: Semistructured Interviews With Health Technology Assessment Bodies and Decision Makers in England, The Netherlands, and Spain 先进治疗药品管理准入协议的采用:对英国、荷兰和西班牙HTA机构和决策者的半结构化访谈。
IF 6.2 2区 医学
Value in Health Pub Date : 2026-09-01 Epub Date: 2026-05-08 DOI: 10.1016/j.jval.2026.04.008
Andrea Greco MSc, Geert W.J. Frederix PhD, Lotty Hooft PhD, Renske M.T. ten Ham PhD
{"title":"Adoption of Managed Entry Agreements for Advanced Therapy Medicinal Products: Semistructured Interviews With Health Technology Assessment Bodies and Decision Makers in England, The Netherlands, and Spain","authors":"Andrea Greco MSc,&nbsp;Geert W.J. Frederix PhD,&nbsp;Lotty Hooft PhD,&nbsp;Renske M.T. ten Ham PhD","doi":"10.1016/j.jval.2026.04.008","DOIUrl":"10.1016/j.jval.2026.04.008","url":null,"abstract":"<div><h3>Objectives</h3><div>This study aims to (1) identify and assess challenges experienced by reimbursement authorities adopting Managed Entry Agreements (MEAs) during Health Technology Assessment (HTA), appraisal, and reimbursement of Advanced Therapy Medicinal Products (ATMPs) in The Netherlands, Spain, and England and (2) rank the most reported adoption challenges and identify potential solutions across jurisdictions.</div></div><div><h3>Methods</h3><div>Between July 2023 and October 2024, semistructured interviews were conducted with current or former reimbursement authorities. Transcripts were analyzed using inductive thematic content analysis. Emerging themes were grouped into 3 steps: HTA, Appraisal and Decision Making, and Implementation. During the interviews, participants completed an embedded prioritization exercise developed based on adoption challenges identified in prior literature.</div></div><div><h3>Results</h3><div>From thematic analysis, 6 themes emerged: Relative Effectiveness, Cost-Effectiveness Analysis and Budget Impact, Stakeholder Involvement, Routines and Practices, MEA Design, Policy Considerations, and Moving Forward. Within HTA, participants highlighted several challenges hampering MEA adoption for ATMPs, including high upfront costs, patient response variability, long-term risks, and ethical concerns. In apprais al, ATMP negotiation dynamics were described as complex due to uncertainty, delivery, administration, and product specificities. Regarding Implementation, hybrid MEAs were seen as promising yet constrained by administrative burden, fragmented data infrastructures, and rigid accounting rules. Structural changes, early dialog, and interoperable real-world data were deemed essential. Prioritization findings confirmed qualitative insights, with administrative burden, outcome measurement, and data collection consistently ranked as key challenges in all countries.</div></div><div><h3>Conclusion</h3><div>Drawing on decision-makers’ experiences, this study identified cross-country challenges to adopting MEAs for ATMPs. Addressing these challenges will require regulatory reform, interoperable data infrastructure, collaborative evidence generation, and ethical oversight.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"29 9","pages":"Pages 1608-1617"},"PeriodicalIF":6.2,"publicationDate":"2026-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147869542","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Beyond the Base Case: Payment Perspective and Ductal Carcinoma In Situ Management Assumptions in Artificial-Intelligence-Assisted Breast Screening Models 超越基本案例:人工智能辅助乳腺筛查模型中的支付视角和DCIS管理假设。
IF 6.2 2区 医学
Value in Health Pub Date : 2026-09-01 Epub Date: 2026-06-01 DOI: 10.1016/j.jval.2026.03.2251
Runji Zhou MB, Jiayun Zou MB, Qiming Liu MB
{"title":"Beyond the Base Case: Payment Perspective and Ductal Carcinoma In Situ Management Assumptions in Artificial-Intelligence-Assisted Breast Screening Models","authors":"Runji Zhou MB,&nbsp;Jiayun Zou MB,&nbsp;Qiming Liu MB","doi":"10.1016/j.jval.2026.03.2251","DOIUrl":"10.1016/j.jval.2026.03.2251","url":null,"abstract":"","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"29 9","pages":"Pages 1745-1746"},"PeriodicalIF":6.2,"publicationDate":"2026-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148151551","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}
引用次数: 0
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