Applied Health Economics and Health Policy最新文献

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New Horizons? Assessing General Public Preferences for a Wellbeing Economy in the Post-COVID-19 World. 新视野号吗?评估公众对后covid -19世界福祉经济的普遍偏好。
IF 3.1 4区 医学
Applied Health Economics and Health Policy Pub Date : 2025-07-01 Epub Date: 2025-03-01 DOI: 10.1007/s40258-025-00951-2
Rachel Milte, Matthew Crocker, Gang Chen, Gordon Duff, Julie Ratcliffe
{"title":"New Horizons? Assessing General Public Preferences for a Wellbeing Economy in the Post-COVID-19 World.","authors":"Rachel Milte, Matthew Crocker, Gang Chen, Gordon Duff, Julie Ratcliffe","doi":"10.1007/s40258-025-00951-2","DOIUrl":"10.1007/s40258-025-00951-2","url":null,"abstract":"<p><strong>Objective: </strong>As societies emerge from the COVID-19 pandemic, governments are increasingly moving away from a focus on economic growth at any cost towards the principles of a wellbeing economy, focused on achieving a more equitable distribution of wealth and wellbeing. This study aimed to assess the relative importance to the Australian general public of the key principles of a wellbeing economy and to investigate heterogeneity in preferences between demographic subgroups.</p><p><strong>Methods: </strong>An online survey was developed and delivered to a general public sample of 2042 Australian adults (aged 18 years and above). Respondents were invited to rank the key principles of a wellbeing economy (dignity, nature and climate, social connection, fairness and participation) plus two additional traditional economic indicators of societal success ('economic growth' and 'economic prosperity') in order of their relative importance for informing future policy directions. Data analysis was conducted using simple summative scoring, which involved the use of a point system allocated to rankings as a dependent variable. In addition, a rank-ordered logit model was used to explore preferences for the entire sample and subgroups defined by key socio-demographic characteristics.</p><p><strong>Results: </strong>'Dignity' (people have enough to live in comfort, safety and happiness) and 'fairness' (equal opportunity for all Australians and the gap between richest and poorest greatly reduced) were ranked as the most important priorities for the total sample and for key socio-demographic subgroups differentiated by age, level of education and level of socio-economic advantage. Traditional economic indicators of societal success including 'economic prosperity' and 'economic growth' were considered important but generally ranked below the principles of 'dignity' and 'fairness'.</p><p><strong>Conclusions: </strong>The findings indicate that government movements away from traditional economic indicators and towards new broader wellbeing economy measures of societal success are likely to be supported by the general public.</p>","PeriodicalId":8065,"journal":{"name":"Applied Health Economics and Health Policy","volume":" ","pages":"679-691"},"PeriodicalIF":3.1,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12170691/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143536409","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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
Predicting Health Utilities Using Health Administrative Data: Leveraging Survey-linked Health Administrative Data from Ontario, Canada. 使用健康管理数据预测健康效用:利用来自加拿大安大略省的与调查相关的健康管理数据。
IF 3.1 4区 医学
Applied Health Economics and Health Policy Pub Date : 2025-07-01 Epub Date: 2025-02-06 DOI: 10.1007/s40258-025-00947-y
Yue Niu, Nazire Begen, Guangyong Zou, Sisira Sarma
{"title":"Predicting Health Utilities Using Health Administrative Data: Leveraging Survey-linked Health Administrative Data from Ontario, Canada.","authors":"Yue Niu, Nazire Begen, Guangyong Zou, Sisira Sarma","doi":"10.1007/s40258-025-00947-y","DOIUrl":"10.1007/s40258-025-00947-y","url":null,"abstract":"<p><strong>Background: </strong>The quality-adjusted life year (QALY) is widely used to measure health outcome that combines the length of life and health-related quality of life (HRQoL). To be a reliable QALY measure, HRQoL measurements with a preference-based scoring algorithm need to be converted into health utilities on a scale from zero (dead) to one (perfect health). However, preference-based health utility data are often not available. We address this gap by developing a predictive model for health utilities.</p><p><strong>Objectives: </strong>To develop a predictive model for health utilities using available demographic and morbidity variables in a health administrative dataset for non-institutionalised populations in Ontario, Canada.</p><p><strong>Methods: </strong>The data were obtained from the 2009 to 2010 Canadian Community Health Survey containing Health Utilities Index Mark3 (HUI3), a generic multi-attribute preference-based health utility instrument linked with Ontario health administrative (OHA) data that were collected for administrative or billing purposes for patient encounters with the health care system. We employed four regression models (linear, Tobit, single-part beta mixture, and two-part beta mixture) and a calibration technique to identify the best-fit regression model.</p><p><strong>Results: </strong>Our findings indicate that the two-part beta mixture model is the best-fit for predicting health utilities in the OHA data. The proposed predictive model reflects the original distribution of HUI3 in the population.</p><p><strong>Conclusion: </strong>Our proposed predictive model generates reasonably accurate health utility predictions from OHA data. Our model-based prediction approach is a useful strategy for real-world applications, particularly when preference-based utility data are unavailable.</p>","PeriodicalId":8065,"journal":{"name":"Applied Health Economics and Health Policy","volume":" ","pages":"661-677"},"PeriodicalIF":3.1,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143254528","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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
Compulsory Licensing as an Instrument to Tackle High Medicine Prices: A Realist Review of Industrial and Health Consequences. 强制许可作为解决高药价的工具:对工业和健康后果的现实主义审查。
IF 3.1 4区 医学
Applied Health Economics and Health Policy Pub Date : 2025-07-01 Epub Date: 2025-04-19 DOI: 10.1007/s40258-025-00956-x
Steven Simoens, Walter Van Dyck, Rosanne Janssens, Liese Barbier, Jeroen Luyten
{"title":"Compulsory Licensing as an Instrument to Tackle High Medicine Prices: A Realist Review of Industrial and Health Consequences.","authors":"Steven Simoens, Walter Van Dyck, Rosanne Janssens, Liese Barbier, Jeroen Luyten","doi":"10.1007/s40258-025-00956-x","DOIUrl":"10.1007/s40258-025-00956-x","url":null,"abstract":"<p><p>Although compulsory licensing of medicines is traditionally discussed in the context of low- and middle-income countries tackling high prices, it has recently sparked debate in several high-income countries. This study aims to examine the industrial and health consequences of compulsory licensing when applied by a high-income country. Our literature review found that the impact of compulsory licensing is challenging to predict as it can have multiple (opposing) consequences in terms of economic activity, patient outcomes and public health. Compulsory licensing can, under particular circumstances, serve as a lever of industrial policy in a country that wishes to develop its domestic generic pharmaceutical industry. However, originator pharmaceutical companies and other industries may reduce investment, which can be negative for countries with a high presence of innovators, adversely impacting long-term economic activity. Compulsory licensing may also induce state retaliation against the license-issuing country. From a health policy perspective, compulsory licensing likely increases patient access to expensive medicines and frees up resources that can be invested in other (health) programs. However, pharmaceutical companies may delay medicine launches or cancel clinical trials in the license-issuing country. Although there are benefits resulting from a credible threat to use compulsory licensing, the overall desirability of actually using it depends on the specific context and needs to be assessed on a case-by-case basis.</p>","PeriodicalId":8065,"journal":{"name":"Applied Health Economics and Health Policy","volume":" ","pages":"613-624"},"PeriodicalIF":3.1,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143957184","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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
Decision-Analytical Modelling of Medicines in the Middle East: A Systematic Review of Economic Evaluation Studies. 中东地区药物决策分析模型:经济评价研究的系统综述。
IF 3.1 4区 医学
Applied Health Economics and Health Policy Pub Date : 2025-07-01 Epub Date: 2025-04-12 DOI: 10.1007/s40258-024-00940-x
Zainab Abdali, Tuba Saygın Avşar, Sue Jowett, Muslim Syed, Khalifa Elmusharaf, Louise Jackson
{"title":"Decision-Analytical Modelling of Medicines in the Middle East: A Systematic Review of Economic Evaluation Studies.","authors":"Zainab Abdali, Tuba Saygın Avşar, Sue Jowett, Muslim Syed, Khalifa Elmusharaf, Louise Jackson","doi":"10.1007/s40258-024-00940-x","DOIUrl":"10.1007/s40258-024-00940-x","url":null,"abstract":"<p><strong>Background: </strong>Economic evaluations through decision-analytical models have played a limited role in shaping healthcare resource optimisation and reimbursement decisions in the Middle East.</p><p><strong>Objective: </strong>This review aims to systematically examine economic evaluation studies focusing on decision-analytical models of medicines in the Middle East, defining methodological characteristics and appraising the quality of the identified models.</p><p><strong>Methods: </strong>A systematic review approach was employed to identify published decision-analytical models of medicines in the Middle East. Six databases were searched (MEDLINE, EMBASE, Econlit, Web of Science, Global Health Cost-Effectiveness Analysis Registry and the Global Index Medicus) from 1998 to July 2024. Studies meeting the inclusion criteria-full economic evaluations of medicines using decision-analytical models in the Middle East-were considered. Data were extracted and tabulated to include study characteristics and methodological specifications, and data were narratively analysed. The Philips checklist was used to assess the quality of studies.</p><p><strong>Results: </strong>Sixty-three decision-analytical modelling studies of medicines were identified and reviewed, from eight Middle Eastern countries, with the majority (90%) conducted in Iran, Saudi Arabia, Qatar and Egypt. The cost-effectiveness of medications for non-communicable diseases was explored in 77% of the models. Gross domestic product-based cost-effectiveness thresholds were commonly used, and international sources provided data on intervention effectiveness and health outcomes, while national sources were mainly used for the costs of resource use. Most models incorporated an assessment of parameter uncertainty, whereas other types of uncertainty were not explored. Studies from high-income countries were generally of higher quality than those from middle-income countries.</p><p><strong>Conclusions: </strong>The number of published decision-analytical models in the Middle East was low, considering the available medicinal products and disease burden. Key elements related to the quality of decision-analytical models, including analysis of the model structure, appropriateness of model inputs and uncertainty assessment, were not consistently fulfilled. Recommendations are provided to enhance the quality of future economic evaluation studies. This includes strengthening the existing health economics capacities, establishing country-specific health technology assessment systems (where possible), and initiating collaborations to generate national cost and outcome data. PROSPERO registration: CRD42021283904.</p>","PeriodicalId":8065,"journal":{"name":"Applied Health Economics and Health Policy","volume":" ","pages":"569-612"},"PeriodicalIF":3.1,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143967422","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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
Young People's Preferences for Web-Based Mental Health Interventions for Managing Anxiety and Depression: A Discrete Choice Experiment. 年轻人对管理焦虑和抑郁的基于网络的心理健康干预的偏好:一个离散选择实验。
IF 3.1 4区 医学
Applied Health Economics and Health Policy Pub Date : 2025-07-01 Epub Date: 2025-03-28 DOI: 10.1007/s40258-025-00958-9
Thi Quynh Anh Ho, Lidia Engel, Jemimah Ride, Long Khanh-Dao Le, Glenn Melvin, Ha N D Le, Cathrine Mihalopoulos
{"title":"Young People's Preferences for Web-Based Mental Health Interventions for Managing Anxiety and Depression: A Discrete Choice Experiment.","authors":"Thi Quynh Anh Ho, Lidia Engel, Jemimah Ride, Long Khanh-Dao Le, Glenn Melvin, Ha N D Le, Cathrine Mihalopoulos","doi":"10.1007/s40258-025-00958-9","DOIUrl":"10.1007/s40258-025-00958-9","url":null,"abstract":"<p><strong>Objective: </strong>Anxiety and depression are prevalent in young people. Web-based mental health interventions (W-MHIs) have the potential to reduce anxiety and depression, yet the level of engagement remains low. This study aims to elicit young people's preferences towards W-MHIs and the relative importance of intervention attributes in influencing choice.</p><p><strong>Methods: </strong>A discrete choice experiment (DCE) was conducted online among young people aged 18-25 years who lived in Australia, self-reported experiences of anxiety and/or depression in the past 12 months and had an intention to use W-MHIs and/or previous experience with W-MHIs for managing anxiety and/or depression. Participants were recruited via social media and Deakin University notice boards. The DCE design comprised six attributes, including out-of-pocket cost, access to trained instructors (e.g., therapists, coaches) to help users stay engaged with the intervention, total time required to complete the intervention, initial screening, quizzes within the W-MHIs to check user's understanding about the intervention content, and communication with other users. The DCE design consisted of three blocks, each with eight unlabelled choice tasks, each with two alternatives. Data were analysed using a mixed logit model.</p><p><strong>Results: </strong>One hundred ninety-nine participants completed the DCE (mean age: 21.43 ± 2.29 years, 64.32% female). Lower cost, access to instructors, and moderate time required to complete the intervention (5 h) were significant facilitators. The W-MHIs including audio- or video-call access to instructors were 23 percentage points more likely to be chosen than those without and W-MHI with a moderate completion time (5 h) was 18 percentage points more likely to be chosen than one with a shorter time (2 h).</p><p><strong>Conclusion: </strong>Our results highlight that low-cost W-MHIs with access to trained instructors and moderate completion time could increase uptake. More research is required to confirm these findings and examine whether these preferences vary across different population characteristics.</p>","PeriodicalId":8065,"journal":{"name":"Applied Health Economics and Health Policy","volume":" ","pages":"737-749"},"PeriodicalIF":3.1,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12170702/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143742009","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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
Alternative Payment Models for Innovative Medicines: A Framework for Effective Implementation. 创新药物的替代支付模式:有效实施框架。
IF 3.1 4区 医学
Applied Health Economics and Health Policy Pub Date : 2025-07-01 Epub Date: 2025-04-02 DOI: 10.1007/s40258-025-00960-1
Frederick McElwee, Amanda Cole, Gomathi Kaliappan, Andrea Masters, Lotte Steuten
{"title":"Alternative Payment Models for Innovative Medicines: A Framework for Effective Implementation.","authors":"Frederick McElwee, Amanda Cole, Gomathi Kaliappan, Andrea Masters, Lotte Steuten","doi":"10.1007/s40258-025-00960-1","DOIUrl":"10.1007/s40258-025-00960-1","url":null,"abstract":"<p><p>Scientific advancements offer significant opportunities for better patient outcomes, but also present new challenges for value assessment, affordability and access. Alternative payment models (APMs) can offer solutions to the ensuing payer challenges. However, a comprehensive framework that matches the spectrum of challenges with the right solution, and places them within a framework for implementation, is currently missing. To fill this gap, we propose evidence-based steps for the effective selection and implementation of APMs. First, contracting challenges should be identified and mapped to potential APM solutions. We developed a decision guide that can serve as a starting point to articulate core problems and map these to APM solutions. The main problem categories identified are: budget impact and uncertainty, value uncertainty, and the scope of value assessment and negotiation. Sub-categories include affordability, uncertainty of effectiveness, and patient heterogeneity, which map onto APM solutions such as outcome-based agreements, instalments, and subscription models. Just as important are the subsequent identification and assessment of the feasibility of potential solutions as well as collaboration to reach agreement on the terms of the APM and lay the groundwork for effective implementation. We adduce recent examples of APM implementation as evidence of how commonly cited implementation barriers can be overcome by applying pragmatic design choices and collaboration. This step-by-step framework can aid payers and manufacturers in the process of effectively identifying, agreeing on, and implementing APMs to advance patient access to cost-effective medicines, while at the same time providing appropriate incentives to support future innovation.</p>","PeriodicalId":8065,"journal":{"name":"Applied Health Economics and Health Policy","volume":" ","pages":"535-549"},"PeriodicalIF":3.1,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12170698/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143762645","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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
A Head-On Comparison of EQ-VT- and Crosswalk-Based EQ-5D-5L Value Sets. EQ-VT和基于人行横道的EQ-5D-5L值集的正面比较
IF 3.1 4区 医学
Applied Health Economics and Health Policy Pub Date : 2025-07-01 Epub Date: 2025-03-11 DOI: 10.1007/s40258-025-00954-z
Henry Bailey, Bram Roudijk
{"title":"A Head-On Comparison of EQ-VT- and Crosswalk-Based EQ-5D-5L Value Sets.","authors":"Henry Bailey, Bram Roudijk","doi":"10.1007/s40258-025-00954-z","DOIUrl":"10.1007/s40258-025-00954-z","url":null,"abstract":"<p><strong>Background: </strong>No systematic country-level comparison has been undertaken between crosswalk- and EQ-VT-derived EQ-5D-5L value sets. Crosswalk values can differ from EQ-VT-based EQ-5D-5L value sets owing to valuation protocols, changes in societal preferences over time, and a change in the label of the highest level on mobility in moving from EQ-5D-3L to EQ-5D-5L. This study aimed to compare the five-level (5L) crosswalk and EQ-VT value sets to explore differences between them at the country level.</p><p><strong>Methods: </strong>From the countries with both time trade-off (TTO)- or discrete choice experiment (DCE) + TTO-based EQ-5D-3L value sets and EQ-VT-based EQ-5D-5L value sets, 19 pairs of EQ-5D-3L/EQ-5D-5L sets were found. For each of these EQ-5D-3L value sets, 5L crosswalk sets were developed and compared with the corresponding national EQ-5D-5L valuation set using correlation analysis, ranges, values of specific states, Bland-Altman plots, and scatter plots. Three of the countries have EQ-5D-3L and EQ-5D-5L valuation data for the same set of respondents. These three cases were analyzed separately, as they provide a \"true\" test of the differences between the two value sets.</p><p><strong>Results: </strong>Spearman correlation between the crosswalk and valuation sets ranged from 0.831 to 0.989, being below 0.9 in 11 pairs of value sets. The difference in the percentage of negative values ranged from +22.5 to -18.8%, and the difference in the ranges within each pair of value sets ranged from +42.7 to -18.4%. The average mean absolute difference of values (crosswalk versus EQ-VT) was 0.149. This was below 0.1 in only 5 of the 19 EQ-VT/crosswalk set pairs. For the states comprising one level 5 and four level 1s, no country preserved its ranking of importance of the five dimensions in moving from crosswalk to EQ-VT values. Most of the Bland-Altman plots and scatterplots revealed a pattern that placed states with the highest level on mobility as a separate band from other states.</p><p><strong>Discussion: </strong>All of the criteria showed poor agreement between the crosswalk- and EQ-VT-based value sets. The differences in labels for the most extreme response option for the mobility dimension leads to substantial differences in values between these value sets.</p><p><strong>Conclusions: </strong>Crosswalk and EQ-VT value sets should not be used interchangeably, except under circumstances where it is not possible or feasible to conduct a direct EQ-5D-5L valuation study.</p>","PeriodicalId":8065,"journal":{"name":"Applied Health Economics and Health Policy","volume":" ","pages":"725-736"},"PeriodicalIF":3.1,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12170785/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143603668","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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
Cost-Effectiveness and Value of Information Analyses of Caplacizumab for the Treatment of Thrombotic Thrombocytopenic Purpura in Brazil. 巴西卡普拉珠单抗治疗血栓性血小板减少性紫癜的成本-效果和信息价值分析
IF 3.1 4区 医学
Applied Health Economics and Health Policy Pub Date : 2025-07-01 Epub Date: 2025-04-24 DOI: 10.1007/s40258-025-00968-7
Julia Simões Correa Galendi, Hannes Rasch, Carlos Antonio Caramori, Rafael Dezen Gaiolla, Dirk Müller, Vania Santos Nunes Nogueira
{"title":"Cost-Effectiveness and Value of Information Analyses of Caplacizumab for the Treatment of Thrombotic Thrombocytopenic Purpura in Brazil.","authors":"Julia Simões Correa Galendi, Hannes Rasch, Carlos Antonio Caramori, Rafael Dezen Gaiolla, Dirk Müller, Vania Santos Nunes Nogueira","doi":"10.1007/s40258-025-00968-7","DOIUrl":"10.1007/s40258-025-00968-7","url":null,"abstract":"<p><strong>Objectives: </strong>The objective was to evaluate the cost effectiveness of caplacizumab for the treatment of patients with acquired immune thrombocytopenic thrombotic purpura (iTTP) compared to standard of care from the perspective of the Brazilian Unified Health System (SUS).</p><p><strong>Methods: </strong>A decision tree followed by a Markov model with a lifetime horizon was developed. Patients entered the model with an acute iTTP event. All patients were assumed to be admitted to hospital where they either respond to treatment or die. The model offered three health states: remission, relapse or death. Input data were obtained from literature searches with the data on efficacy of caplacizumab based on the HERCULES trial. The incremental cost-effectiveness ratio (ICER) was compared to the willingness-to-pay threshold for rare diseases of Brazilian reais (R$)120,000/quality-adjusted life years (QALYs). In addition to various sensitivity analyses, a value of information (VOI) analysis was conducted.</p><p><strong>Results: </strong>In the base case, caplacizumab resulted in 0.70 QALYs gained, and cost R$1,333,601 more, with an ICER of R$1,901,729/QALY. The cost of the caplacizumab vial was the most influential parameter. Probabilistic analysis showed that caplacizumab was not cost effective in any iterations for the threshold of the rare disease. The expected value of perfect information per year is R$0.</p><p><strong>Conclusion: </strong>Although caplacizumab results in incremental QALYs, based on the proposed cost, caplacizumab is not cost effective from the SUS perspective, and VOI results indicate that further research would not be worthwhile.</p>","PeriodicalId":8065,"journal":{"name":"Applied Health Economics and Health Policy","volume":" ","pages":"649-660"},"PeriodicalIF":3.1,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12170793/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143970304","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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
Machine Learning-Assisted Health Economics and Policy Reviews: A Comparative Assessment. 机器学习辅助的卫生经济学和政策审查:比较评估。
IF 3.1 4区 医学
Applied Health Economics and Health Policy Pub Date : 2025-07-01 Epub Date: 2025-03-28 DOI: 10.1007/s40258-025-00963-y
Ludovico Cavallaro, Vittoria Ardito, Michael Drummond, Oriana Ciani
{"title":"Machine Learning-Assisted Health Economics and Policy Reviews: A Comparative Assessment.","authors":"Ludovico Cavallaro, Vittoria Ardito, Michael Drummond, Oriana Ciani","doi":"10.1007/s40258-025-00963-y","DOIUrl":"10.1007/s40258-025-00963-y","url":null,"abstract":"<p><strong>Introduction: </strong>The growth of scientific literature in health economics and policy represents a challenge for researchers conducting literature reviews. This study explores the adoption of a machine learning (ML) tool to enhance title and abstract screening. By retrospectively assessing its performance against the manual screening of a recent scoping review, we aimed to evaluate its reliability and potential for streamlining future reviews.</p><p><strong>Methods: </strong>ASReview was utilised in 'Simulation Mode' to evaluate the percentage of relevant records found (RRF) during title/abstract screening. A dataset of 10,246 unique records from three databases was considered, with 135 relevant records labelled. Performance was assessed across three scenarios with varying levels of prior knowledge (PK) (i.e., 5, 10, or 15 records), using both sampling and heuristic stopping criteria, with 100 simulations conducted for each scenario.</p><p><strong>Results: </strong>The ML tool demonstrated strong performance in facilitating the screening process. Using the sampling criterion, median RRF values stabilised at 97% with 25% of the sample screened, saving reviewers approximately 32 working days. The heuristic criterion showed similar median values, but greater variability due to premature conclusions upon reaching the threshold. While higher PK levels improved early-stage performance, the ML tool's accuracy stabilised as screening progressed, even with minimal PK.</p><p><strong>Conclusions: </strong>This study highlights the potential of ML tools to enhance the efficiency of title and abstract screening in health economics and policy literature reviews. To fully realise this potential, it is essential for regulatory bodies to establish comprehensive guidelines that ensure ML-assisted reviews uphold rigorous evidence quality standards, thereby enhancing their integrity and reliability.</p>","PeriodicalId":8065,"journal":{"name":"Applied Health Economics and Health Policy","volume":" ","pages":"639-647"},"PeriodicalIF":3.1,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12170730/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143735586","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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
Design Patents: A Potential Threat to Drug Competition. 外观设计专利:对药品竞争的潜在威胁。
IF 3.1 4区 医学
Applied Health Economics and Health Policy Pub Date : 2025-07-01 Epub Date: 2025-04-08 DOI: 10.1007/s40258-025-00967-8
Janet Freilich, Aaron S Kesselheim
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