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Health Utility Values Measured by EQ-5D in Breast Cancer From Low- and Middle-Income Countries: A Systematic Review and Meta-Analysis Protocol. 用EQ-5D测量中低收入国家乳腺癌患者的健康效用价值:一项系统评价和荟萃分析方案
IF 2.4
PharmacoEconomics Open Pub Date : 2026-09-01 Epub Date: 2026-06-10 DOI: 10.1007/s41669-026-00664-9
Lynn Jia Tay, Siew Chin Ong, Lyn Xuan Tay
{"title":"Health Utility Values Measured by EQ-5D in Breast Cancer From Low- and Middle-Income Countries: A Systematic Review and Meta-Analysis Protocol.","authors":"Lynn Jia Tay, Siew Chin Ong, Lyn Xuan Tay","doi":"10.1007/s41669-026-00664-9","DOIUrl":"10.1007/s41669-026-00664-9","url":null,"abstract":"<p><strong>Background: </strong>Health utility values (HUVs) are fundamental parameters in economic evaluations used to assess the value of healthcare interventions. However, patients living with breast cancer in low- and middle-income countries (LMICs) face distinct burdens compared with those in high-income countries (HICs), driven by profound disparities in resources, infrastructure, and cultural context. High reliance on HUVs from HICs risks misrepresenting the specific disease burden in LMICs, introducing uncertainty in local healthcare resource allocation. This protocol outlines the methods for a planned systematic review and meta-analysis to synthesize available evidence from LMICs to generate pooled EuroQol-5 Dimensions HUV estimates that are contextually appropriate for local decision making.</p><p><strong>Methods: </strong>By following the Preferred Reporting Items for Systematic Review and Meta-Analysis (PRISMA) guidelines, this protocol is registered in PROSPERO (CRD420251208371). The initial literature search will be conducted in Cochrane CENTRAL, PubMed, Web of Science core collection and Scopus using keywords such as 'health-related quality of life', 'EuroQol-5 Dimensions', 'breast cancer' and 'low-middle-income countries'. Risk-of-bias assessment will be conducted using the Cochrane Risk-of-Bias Tool (RoB 2) and Newcastle-Ottawa Scale (NOS) depending on study designs. R packages (metaprop) will be used to pool the mean HUVs of people living with breast cancer using fixed-effect (inverse variance method) and random-effect models (Der Simonian-Laird method). Subgroup analysis will be conducted based on different categories of interest such as EQ-5D versions, country income level and clinical stagings.</p><p><strong>Discussion: </strong>This protocol provides a systematic guide to synthesize EQ-5D utility values specific to breast cancer in LMICs. The anticipated findings will establish a critical evidence base for decision making in resource-constrained settings. By generating contextually relevant estimates, this will minimize parameter uncertainty and support more equitable resource allocation. Hence, it will contribute to health systems that are responsive not only to the clinical burden of the disease but also to the multidimensional well-being of the patient and families it affects.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"935-939"},"PeriodicalIF":2.4,"publicationDate":"2026-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13593971/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148219356","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Budget Impact Analysis of Vacuum-Assisted Excision Versus Surgical Excision for the Assessment of Breast Lesions of Uncertain Malignant Potential in Italy. 在意大利,对不确定恶性潜能的乳腺病变评估,真空辅助切除与外科切除的预算影响分析。
IF 2.4
PharmacoEconomics Open Pub Date : 2026-09-01 Epub Date: 2026-06-09 DOI: 10.1007/s41669-026-00663-w
Carla Rognoni, Ludovico Cavallaro, Gaia Grassi, Francesca Caumo, Catherine Depretto, Secondo Folli, Salvatore Minelli, Modestino Pezzella, Nicola Rocco, Valeria Romeo, Anna Russo, Gianfranco Scaperrotta, Rosanna Tarricone
{"title":"Budget Impact Analysis of Vacuum-Assisted Excision Versus Surgical Excision for the Assessment of Breast Lesions of Uncertain Malignant Potential in Italy.","authors":"Carla Rognoni, Ludovico Cavallaro, Gaia Grassi, Francesca Caumo, Catherine Depretto, Secondo Folli, Salvatore Minelli, Modestino Pezzella, Nicola Rocco, Valeria Romeo, Anna Russo, Gianfranco Scaperrotta, Rosanna Tarricone","doi":"10.1007/s41669-026-00663-w","DOIUrl":"10.1007/s41669-026-00663-w","url":null,"abstract":"<p><strong>Background/objective: </strong>B3 breast lesions, of uncertain malignant potential, pose diagnostic challenges owing to their intermediate malignancy risk. While surgical excision (SE) is traditionally used for definitive diagnosis, vacuum-assisted excision (VAE) offers a less invasive alternative. As diagnostic practices evolve, this study assesses the financial impact of adopting VAE for B3 lesion management from the hospital perspective within the Italian healthcare system.</p><p><strong>Materials and methods: </strong>A budget impact analysis (BIA) was conducted from the hospital perspective over a 5-year time horizon. Clinical complication rates for SE and VAE were derived through a systematic literature review and meta-analysis. A micro-costing approach was used to estimate direct medical costs on the basis of detailed resource utilization data collected via structured questionnaires administered across five Italian hospitals. One-way sensitivity analyses and scenario analyses were conducted to test the robustness of the results.</p><p><strong>Results: </strong>VAE was associated with significantly lower costs than SE, mainly owing to reduced hospitalization, operating room use, and personnel time. The average per-patient cost was €820 for VAE versus €1663 for SE, yielding savings of €843. Over 5 years, with VAE adoption rising from 20% to 80%, cumulative hospital savings were estimated to be between €11.1 million and €13.7 million. These findings were further confirmed by multiple budget impact scenario analyses, which consistently demonstrated a favorable economic profile for VAE across all tested assumptions.</p><p><strong>Conclusions: </strong>In our analysis, VAE represented a cost-saving, minimally invasive alternative to SE for the management of B3 breast lesions from the hospital perspective in Italy. Its adoption can enhance hospital efficiency and support more sustainable resource allocation within the Italian healthcare system.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"919-933"},"PeriodicalIF":2.4,"publicationDate":"2026-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13594045/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148205577","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Subcutaneous Ocrelizumab for the Treatment of Relapsing Forms of Multiple Sclerosis: A Budget Impact Model. 皮下Ocrelizumab治疗复发型多发性硬化症:预算影响模型
IF 2.4
PharmacoEconomics Open Pub Date : 2026-09-01 Epub Date: 2026-08-03 DOI: 10.1007/s41669-026-00672-9
Camilla Porta, Paolo Di Procolo, Giuseppina Marrazzo, Marco Belfiore, Raffaele Palladino, Vincenzo Brescia Morra, Marcello Moccia
{"title":"Subcutaneous Ocrelizumab for the Treatment of Relapsing Forms of Multiple Sclerosis: A Budget Impact Model.","authors":"Camilla Porta, Paolo Di Procolo, Giuseppina Marrazzo, Marco Belfiore, Raffaele Palladino, Vincenzo Brescia Morra, Marcello Moccia","doi":"10.1007/s41669-026-00672-9","DOIUrl":"10.1007/s41669-026-00672-9","url":null,"abstract":"<p><strong>Objectives: </strong>The aim of this study was to evaluate the budget impact of introducing the subcutaneous (SC) form of ocrelizumab for patients with relapsing forms of multiple sclerosis (RMS) with high disease activity (HA) despite previous treatment or with rapidly evolving severe (RES) disease in Italy from the hospital perspective.</p><p><strong>Methods: </strong>A 3-year dynamic budget impact model with a Markov structure was developed de novo to simulate patient transitions across Expanded Disability Status Scale-defined health states. The current scenario (without ocrelizumab SC) was compared with an alternative scenario reflecting expected ocrelizumab SC uptake. Number of eligible patients reflected Italian population and projected market shares. Efficacy inputs were obtained from published literature, with adherence and persistence sourced from Italian real-world data. Unit costs-including drug administration, monitoring, adverse events, disease management, and relapse-were collected from Italian sources. Drug acquisition costs were considered in a scenario analysis. Parameter uncertainty was explored through deterministic sensitivity and scenario analyses.</p><p><strong>Results: </strong>Over 3 years, 22,128 patients were eligible, of whom 5389 were assumed to receive ocrelizumab SC. Ocrelizumab SC introduction would generate cumulative savings of about €2.0 million, mainly from reduced administration and monitoring costs, with additional savings from relapse and disease management expenditures. When drug acquisition costs were included, total savings rose to roughly €7.1 million. Sensitivity analyses confirmed the robustness of these findings.</p><p><strong>Conclusions: </strong>Ocrelizumab SC may be a cost-saving and resource-efficient option for HA/RES RMS management in Italy. By reducing administration time while maintaining the established clinical profile of the intravenous (IV) formulation of ocrelizumab, its adoption can enhance hospital efficiency and deliver meaningful budgetary savings.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"847-857"},"PeriodicalIF":2.4,"publicationDate":"2026-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13594067/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148664259","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
The Chronic Kidney Disease Care Model: Quantifying Management Strategies for Improving Patient Outcomes in the USA. 慢性肾脏疾病的护理模式:量化管理策略,以改善美国患者的预后。
IF 2.4
PharmacoEconomics Open Pub Date : 2026-09-01 Epub Date: 2026-08-13 DOI: 10.1007/s41669-026-00649-8
Andrew H Briggs, Ciaran N Kohli-Lynch, Adrian R Levy, Ziyi Lin, Satabdi Chatterjee, Bonnie M K Donato, Csaba P Kovesdy
{"title":"The Chronic Kidney Disease Care Model: Quantifying Management Strategies for Improving Patient Outcomes in the USA.","authors":"Andrew H Briggs, Ciaran N Kohli-Lynch, Adrian R Levy, Ziyi Lin, Satabdi Chatterjee, Bonnie M K Donato, Csaba P Kovesdy","doi":"10.1007/s41669-026-00649-8","DOIUrl":"10.1007/s41669-026-00649-8","url":null,"abstract":"<p><strong>Purpose: </strong>While chronic kidney disease (CKD) afflicts an estimated 14% of US adults, the majority remain unaware of the ailment until later stages. CKD progression can lead to reduced quality of life, cardiovascular disease (CVD), and end-stage renal disease (ESRD). Consensus guidelines highlight novel interventions for the early recognition and management of CKD. Here, we describe the rationale and structure of a policy model for CKD that provides contemporary estimates of long-term effectiveness and cost-effectiveness of medical interventions. The utility of the model is illustrated by assessing potential management strategies for CKD.</p><p><strong>Methods: </strong>A state transition model was developed to follow over their lifetime a hypothetical cohort of insured US adults with CKD. CKD progression was tracked through Kidney Disease: Improving Global Outcomes (KDIGO) categories. Patients entering the cohort could transition to ESRD, CVD, heart failure (HF), or death from other causes. Those with a nonfatal first event were followed until death. The probability of a first event was determined by cohort characteristics, including estimated glomerular filtration rate (eGFR). Survival following a first nonfatal event was modeled parametrically. Costs were estimated using linked electronic health record-linked claims that included data from more than 100 million US adults. A health sector perspective was adopted. Relevant parameters for CKD progression, treatment efficacy, and utilities were derived from peer-reviewed studies.</p><p><strong>Results: </strong>Under usual care, over a full patient lifetime, the model predicts that just 15% of patients will experience a death unrelated to CKD as their first event and that this will happen after an average of 12 years. With fully optimized treatment, and with no delay to treatment initiation, the percentage of patients dying of causes unrelated to CKD increases to 20%, and the time before that event increases to 14 years. Guideline-optimized treatment and eliminating treatment delay was estimated to result in an improvement of 0.68 QALYs over the patient lifetime at a cost of US $47,800.</p><p><strong>Conclusion: </strong>The CKD Care Model provides a flexible, transparent tool for projecting the impact of eGFR decline on clinical outcomes and evaluating management strategies for CKD. Illustrative analyses suggest that guideline-optimized treatment can improve patient outcomes at additional costs that appear favorable by conventional value-for-money standards.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"819-831"},"PeriodicalIF":2.4,"publicationDate":"2026-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13594007/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148764833","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
AMPLIFY: Estimating Clinical and Humanistic Benefits of Pembrolizumab in Portugal. AMPLIFY:评估派姆单抗在葡萄牙的临床和人文效益。
IF 2.4
PharmacoEconomics Open Pub Date : 2026-09-01 Epub Date: 2026-07-30 DOI: 10.1007/s41669-026-00662-x
Pedro Cardoso, Joana Sousa, João Ferreira, Bernardo Rodrigues
{"title":"AMPLIFY: Estimating Clinical and Humanistic Benefits of Pembrolizumab in Portugal.","authors":"Pedro Cardoso, Joana Sousa, João Ferreira, Bernardo Rodrigues","doi":"10.1007/s41669-026-00662-x","DOIUrl":"10.1007/s41669-026-00662-x","url":null,"abstract":"<p><strong>Background: </strong>Pembrolizumab has become a cornerstone in the treatment of multiple tumor types. In Portugal, it is currently reimbursed across 25 indications. However, a comprehensive holistic assessment of its cumulative clinical and humanistic impact is lacking.</p><p><strong>Objective: </strong>The aim was to estimate the population-level clinical and humanistic outcomes associated with pembrolizumab use in Portugal from 2017 to 2024, and to project its potential future impact until 2030.</p><p><strong>Methods: </strong>We developed a Microsoft Excel-based tool to aggregate outputs from validated, Portugal-adapted, cost-effectiveness models across all reimbursed indications for pembrolizumab. Using a real-world number of treated patients, clinical outcomes such as incremental life years (LYs), incremental quality-adjusted life years (QALYs), and avoided deaths were estimated. Forecasts through 2030 were developed using market and epidemiological projections. Alternative scenarios evaluated the additional benefits of accelerated reimbursement decisions and expanded patient access.</p><p><strong>Results: </strong>Between 2017 and 2024, among 13,530 treated patients, pembrolizumab was associated with estimated lifetime outcomes of 14,042 incremental QALYs, 17,914 incremental LYs, and 2021 deaths avoided. Scenario analyses showed that with accelerated reimbursement, within 180 days of market approval, and full uptake, cumulative gains could have increased by up to 14,705 QALYs (total 28,746 QALYs) and an additional 1250 deaths could have been avoided (total 3271 avoided deaths). Forecasts project continued substantial benefits for patients treated between 2025 and 2030, with lifetime outcomes estimated at approximately 31,306 QALYs, 38,274 LYs, and 4841 deaths avoided for the currently reimbursed indications.</p><p><strong>Conclusion: </strong>Pembrolizumab has delivered significant clinical benefits in Portugal. Our results suggest that more timely reimbursement decisions and broader access could have yielded measurable additional health gains. These findings highlight the potential impact of access timelines on health outcomes, while acknowledging that broader assessments of value require consideration of cost-effectiveness, budget impact, and opportunity costs. Pembrolizumab's benefits to the Portuguese health system is expected to grow through 2030.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"833-845"},"PeriodicalIF":2.4,"publicationDate":"2026-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13594025/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148631120","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Estimating the Long-Term Clinical and Economic Impact of Universal Varicella Vaccination in Portugal Using a Dynamic Transmission Model. 使用动态传播模型估计葡萄牙普遍水痘疫苗接种的长期临床和经济影响。
IF 2.4
PharmacoEconomics Open Pub Date : 2026-09-01 Epub Date: 2026-06-29 DOI: 10.1007/s41669-026-00665-8
Colleen Burgess, Stephanie A Kujawski, Miguel Amorim, John C Lang
{"title":"Estimating the Long-Term Clinical and Economic Impact of Universal Varicella Vaccination in Portugal Using a Dynamic Transmission Model.","authors":"Colleen Burgess, Stephanie A Kujawski, Miguel Amorim, John C Lang","doi":"10.1007/s41669-026-00665-8","DOIUrl":"10.1007/s41669-026-00665-8","url":null,"abstract":"<p><strong>Background: </strong>Varicella vaccination is not currently included in Portugal's childhood national immunization program. We modeled the clinical and economic impact of a two-dose universal varicella vaccination (UVV) program in Portugal.</p><p><strong>Methods: </strong>A dynamic transmission model was adapted to Portugal to assess the impact of UVV over a 50-year time horizon. Two two-dose UVV strategies (first dose at 12 months, second dose at 5 years) were compared to no vaccination. Clinical outcomes included varicella and herpes zoster incidence rates, cases, deaths, quality-adjusted life years (QALYs) lost, and varicella-related health care resource use. Costs were assessed from the payer perspective (direct costs of treatment) and societal perspective (direct costs plus indirect costs of lost workdays).</p><p><strong>Results: </strong>Both UVV strategies reduced total varicella cases by 79.4-85.3%, outpatient visits by 78.9-84.8%, and varicella hospitalizations by 69.3-71.3% compared to no varicella vaccination. Herpes zoster cases were reduced by 2.4-2.9%. These strategies resulted in the gain of 5977-6094 QALYs and cost savings of €12.0 million (M)-15.9M from the payer perspective and €84.2M-93.9M from the societal perspective.</p><p><strong>Conclusions: </strong>A two-dose UVV program is estimated to substantially reduce the burden of varicella disease in Portugal and to be cost-saving from both the payer and societal perspectives.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"887-896"},"PeriodicalIF":2.4,"publicationDate":"2026-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13594043/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148346050","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Cost-Effectiveness of an Artificial Intelligence as a Medical Device (AIaMD) for Triaging Patients Presenting to Primary Care with Concerns that They Have a Skin Cancer: A Modelling Study. 人工智能作为医疗设备(AIaMD)对患有皮肤癌的初级保健患者进行分类的成本效益:一项建模研究。
IF 2.4
PharmacoEconomics Open Pub Date : 2026-08-18 DOI: 10.1007/s41669-026-00675-6
Javad Javan, Zhivko Zhelev, Bogdan Grigore, Chris Hyde
{"title":"Cost-Effectiveness of an Artificial Intelligence as a Medical Device (AIaMD) for Triaging Patients Presenting to Primary Care with Concerns that They Have a Skin Cancer: A Modelling Study.","authors":"Javad Javan, Zhivko Zhelev, Bogdan Grigore, Chris Hyde","doi":"10.1007/s41669-026-00675-6","DOIUrl":"https://doi.org/10.1007/s41669-026-00675-6","url":null,"abstract":"<p><strong>Introduction: </strong>Health services are struggling to cope with the growing numbers of people coming with skin lesions they are worried could be cancer. Using artificial intelligence (AI) to assist in the triage process is one potential approach. Deep Ensemble for the Recognition of Melanoma (DERM) is one such AI device which has achieved Conformité Européene (CE) Class III regulatory approval in the UK and Europe. The effectiveness and cost-effectiveness of DERM have already been carefully examined post-referral. This study examines the cost-effectiveness of DERM pre-referral in the community setting.</p><p><strong>Methods: </strong>We modified a decision-analytic model developed for post-referral evaluation of DERM to the community setting. The model compared standard care (SC) with two DERM-based strategies: with second reading (2R) when discharge was recommended (DERM_2R) and without it (DERM_autonomous). The outputs were numbers of diagnostic outcomes, numbers of key events (GP appointments, DERM assessments, hospital referrals), costs and quality-adjusted life-years (QALY). The cost-effectiveness metrics were incremental cost per QALY gained. Analysis was conducted in the UK from an NHS and personal social services perspective, with a life-time horizon up to a maximum of 100 years, with uncertainty assessed mainly using deterministic sensitivity analyses.</p><p><strong>Results: </strong>We report three base-case results on the basis of different assumptions regarding GP diagnostic sensitivity and specificity. When GPs operate with maximised sensitivity as suggested by the literature, SC is the most cost-effective option. SC dominates DERM_autonomous and DERM_2R has an incremental cost-effectiveness ratio (ICER) relative to SC of £30,370. If GPs operate with maximised specificity, DERM_autonomous dominates SC and DERM_2R has an ICER of £397. If GPs diagnose cancer with a performance back calculated from NHS routine data, the cost-effectiveness of both DERM options dominate SC.</p><p><strong>Conclusions: </strong>The priority should be for better estimates of GP accuracy to be obtained to reduce uncertainty in the estimates of cost-effectiveness. The results presented do, however, show that it is plausible that DERM could be cost-effective justifying use in practice with further data collection.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":""},"PeriodicalIF":2.4,"publicationDate":"2026-08-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148796375","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Integrating Structured Expert Elicitation with External Evidence to Inform Earlier Reimbursement Decisions: A Norwegian Case Study of Selpercatinib for Non-Small Cell Lung Cancer. 整合结构化专家启发与外部证据,告知早期报销决策:挪威塞尔珀卡替尼治疗非小细胞肺癌的案例研究。
IF 2.4
PharmacoEconomics Open Pub Date : 2026-07-01 Epub Date: 2026-06-18 DOI: 10.1007/s41669-026-00656-9
Yancy S Wu, Emily A Burger, Fredrik Holmboe, Magnus Bangum, Francisco Oteiza, Christoffer Bugge, Erik Magnus Sæther, Eline Aas
{"title":"Integrating Structured Expert Elicitation with External Evidence to Inform Earlier Reimbursement Decisions: A Norwegian Case Study of Selpercatinib for Non-Small Cell Lung Cancer.","authors":"Yancy S Wu, Emily A Burger, Fredrik Holmboe, Magnus Bangum, Francisco Oteiza, Christoffer Bugge, Erik Magnus Sæther, Eline Aas","doi":"10.1007/s41669-026-00656-9","DOIUrl":"10.1007/s41669-026-00656-9","url":null,"abstract":"<p><strong>Background: </strong>The Norwegian public-private partnership, CONNECT, identified the reimbursement submission case of selpercatinib-a first-line targeted therapy for RET fusion-positive non-small cell lung cancer (NSCLC)-as a unique opportunity to assess the role of structured expert elicitation (SEE) in conjunction with phase II evidence.</p><p><strong>Objective: </strong>This study aims to compare the potential of using SEE and other sources of external evidence with phase III clinical trial results to explore the possibility of facilitating earlier (conditional) reimbursement decisions.</p><p><strong>Methods: </strong>We conducted a series of cost-effectiveness analyses, grouping different sources of evidence (i.e., published clinical evidence and SEE) to estimate the long-term health and economic consequences of selpercatinib versus pembrolizumab plus pemetrexed and platinum-based chemotherapy for patients with advanced RET fusion-positive NSCLC in Norway. We developed a probabilistic partitioned survival model to estimate and compare costs, quality-adjusted life-years (QALYs), and incremental cost-effectiveness ratios (ICERs) associated with evidence potentially available early in the product lifecycle, including SEE, with those using phase III evidence.</p><p><strong>Results: </strong>ICERs consistently ranged between $8257 and $19,819 per QALY gained when we used SEE, either alone or in combination with additional external evidence, or when we used the phase III randomized controlled trial (RCT) evidence.</p><p><strong>Conclusions: </strong>The consistent cost-effectiveness results between pre-submission and post-submission phases support the potential of SEE to complement health technology assessment (HTA) and potentially inform earlier (conditional) reimbursement decisions for this single case study. However, future research should focus on refining SEE protocols for continued methodological development and validation to improve generalizability and applicability.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"679-694"},"PeriodicalIF":2.4,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13379524/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148278322","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
A Micro-costing Study of Community-Based Primary HPV Self-Sampling and Triage Test for Cervical Cancer Screening in Rural Cambodia. 以社区为基础的原发性人乳头瘤病毒自我抽样和分类测试在柬埔寨农村宫颈癌筛查的微观成本研究。
IF 2.4
PharmacoEconomics Open Pub Date : 2026-07-01 Epub Date: 2026-05-26 DOI: 10.1007/s41669-026-00659-6
Ziyue Zhang, Hermann Bussmann, Sathiarany Vong, Hero Kol, Magnus von Knebel Doeberitz, Manuela De Allegri, Kavita Singh
{"title":"A Micro-costing Study of Community-Based Primary HPV Self-Sampling and Triage Test for Cervical Cancer Screening in Rural Cambodia.","authors":"Ziyue Zhang, Hermann Bussmann, Sathiarany Vong, Hero Kol, Magnus von Knebel Doeberitz, Manuela De Allegri, Kavita Singh","doi":"10.1007/s41669-026-00659-6","DOIUrl":"10.1007/s41669-026-00659-6","url":null,"abstract":"<p><strong>Background: </strong>Community-based human papillomavirus (HPV) self-sampling can expand cervical cancer screening coverage in rural settings, but local programmatic cost evidence is limited.</p><p><strong>Methods: </strong>We conducted an activity-based micro-costing study from the healthcare system perspective to estimate start-up (January 2022-February 2023) and implementation (March-December 2023) costs of a community-based HPV self-sampling screen-triage approach in Siem Reap Province, Cambodia. Resource use and unit costs were measured using direct observation, semi-structured interviews with program staff, and review of budgets and financial reports. All costs were converted to 2024 US dollars.</p><p><strong>Results: </strong>Between January 2022 and December 2023, 7524 women were screened; 359 (4.77%) tested HPV-positive and 312 of 359 (86.91%) completed triage during the study period. Total programmatic cost was US$204,507.14, yielding an average cost of US$27.18 per woman screened and US$569.66 per HPV-positive case identified. Laboratory testing was the largest cost component (55.01% of total programmatic cost), followed by the community-based self-sampling activity (31.12%). Estimated incremental unit costs for triage options were US$3.69 for visual inspection of cervix with acetic acid (VIA), US$16.42 for partial HPV genotyping (types 16 and 18), and US$32.92 for colposcopy per woman receiving the respective triage test.</p><p><strong>Conclusions: </strong>Community-based HPV self-sampling can be delivered at moderate programmatic cost in rural Cambodia, with overall costs driven primarily by laboratory testing and community-based screening delivery. These findings provide inputs for budgeting and planning scale-up of HPV screening from a healthcare system perspective in Cambodia.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"749-758"},"PeriodicalIF":2.4,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13379565/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148034170","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Advancing Lost Productivity Measurement in Economic Evaluations: Evidence Priorities from a Targeted Literature Review and Expert Workshop. 在经济评估中推进生产力损失测量:来自有针对性的文献综述和专家研讨会的证据优先级。
IF 2.4
PharmacoEconomics Open Pub Date : 2026-07-01 Epub Date: 2026-05-03 DOI: 10.1007/s41669-026-00654-x
R Brett McQueen, Benjamin Cohen, Caroline Kacergis, Eileen Kennedy, Katherine L Rosettie
{"title":"Advancing Lost Productivity Measurement in Economic Evaluations: Evidence Priorities from a Targeted Literature Review and Expert Workshop.","authors":"R Brett McQueen, Benjamin Cohen, Caroline Kacergis, Eileen Kennedy, Katherine L Rosettie","doi":"10.1007/s41669-026-00654-x","DOIUrl":"10.1007/s41669-026-00654-x","url":null,"abstract":"<p><strong>Background: </strong>Productivity losses from illness, treatment, and informal caregiving impose a substantial economic burden on patients, caregivers, employers, and society in the United States (US), but measurement remains inconsistent and often omits key domains, such as presenteeism and unpaid non-market work productivity. These gaps limit the integration of productivity into economic evaluations and policy and may lead to undervaluation of interventions that improve health and reduce disease burden.</p><p><strong>Objective: </strong>The aim was to identify evidence priorities and methodological improvements for measuring and valuing productivity in US economic evaluations.</p><p><strong>Methods: </strong>A targeted literature review (TLR) of US-based studies published from 2020 to 2024 was conducted to evaluate measurement of absenteeism, presenteeism, or unpaid non-market work productivity. Definitions, instruments, valuation methods, and reporting practices were extracted. Findings informed a virtual workshop with eight stakeholders, who completed prioritization exercises and participated in facilitated discussions to refine evidence priorities and methodological recommendations.</p><p><strong>Results: </strong>Thirty studies met the TLR inclusion criteria. Absenteeism was assessed in 77%, unpaid non-market work productivity in 67%, and presenteeism in 37%. The Work Productivity and Activity Impairment questionnaire was used in 40%. Only 30% assessed informal caregiving-related productivity losses, 23% included fringe benefits, and 37% used longitudinal designs. In the workshop, stakeholders prioritized four predefined domains for future evidence generation and value assessment: (1) measuring longitudinal fluctuations in productivity by disease severity; (2) valuing unpaid non-market work productivity; (3) measuring presenteeism; and (4) disaggregating productivity by job types and wfork environments. Recommendations included transparent, perspective-specific valuation; capturing total compensation in absenteeism costs; disaggregating time-based and monetary outcomes; and developing instruments suitable for remote, hybrid, and gig work.</p><p><strong>Conclusions: </strong>Productivity losses are an underrecognized component of disease burden in the US. More consistent use of validated instruments, particularly for measuring longitudinal and unpaid non-market work productivity, may improve the reliability and policy relevance of productivity estimates.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"593-608"},"PeriodicalIF":2.4,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13379543/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147819289","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
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