PharmacoEconomics Open最新文献

{"title":"Very Early Health Technology Assessment for Potential Predictive Biomarkers in the Treatment of Advanced Non-Small Cell Lung Cancer.","authors":"Leila-Sophie Otten, Alessandra I G Buma, Berber Piet, Rob Ter Heine, Michel M van den Heuvel, Valesca P Retèl","doi":"10.1007/s41669-025-00557-3","DOIUrl":"10.1007/s41669-025-00557-3","url":null,"abstract":"<p><strong>Objectives: </strong>Immune checkpoint inhibitor (ICI)-containing treatment is currently prescribed as first-line treatment for all patients with advanced non-small cell lung cancer (NSCLC) without targetable driver mutations. However, only 30-45% of patients show no progression within 12 months after treatment start. Various biomarkers are being studied to save costly and potentially harmful treatment in non-responders. We evaluated the cost-effectiveness of implementing a hypothetical predictive biomarker for ICI-containing treatment response compared with standard of care (e.g., no implemented biomarker) for pembrolizumab-containing treatment in patients with advanced NSCLC in the Netherlands.</p><p><strong>Materials and methods: </strong>Standard-of-care-based and predictive-biomarker-based strategies were compared using Markov models for three first-line pembrolizumab-containing treatments depending on a patient's tumor programmed cell death ligand-1 (PD-L1) expression and histology. A Dutch healthcare system perspective was adopted. Assuming a receiver operating characteristic-area under the curve of 1.0 in identifying responders, alternative treatments were offered for non-responders in the predictive-biomarker-based strategy. Parameters and assumptions were based on real-world data from surveys, literature using a targeted search, expert opinion, and registries. Outcomes included differences in costs, survival (life years (LYs)), and survival corrected for health-related quality of life (QoL) quality-adjusted life-years (QALYs) between the predictive-biomarker- and standard-of-care-based strategy.</p><p><strong>Results: </strong>Implementing a predictive biomarker in pembrolizumab-carboplatin-paclitaxel treatment led to a mean survival reduction of 24 days (- 0.067 LYs) (18 days corrected for QoL (- 0.049 QALYs)), with cost savings of €22,606 compared with standard of care. Pembrolizumab monotherapy and pembrolizumab-pemetrexed-platinum treatments showed survival reductions of 4.5 and 3.9 months, respectively (3.6 and 2.8 months corrected for QoL), with cost savings of €24,345 and €28,456. Sensitivity analyses confirmed consistent cost savings and survival reductions. Survival losses were mainly observed due to the lower survival rates associated with the alternative first-line treatment options available for non-responders in the predictive-biomarker-based strategy within each pembrolizumab-containing treatment regimen. Pembrolizumab-carboplatin-paclitaxel treatment also showed survival gains under certain conditions related to QoL and survival estimates.</p><p><strong>Conclusions: </strong>Our study highlights the importance of careful de-implementation of ICI-treatments in advanced NSCLC, balancing costs reductions and side effects without comprising survival. In the pembrolizumab-carboplatin-paclitaxel treatment regimen, the survival loss could be considered negligible. Future research should define acceptable tradeoffs and","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"471-485"},"PeriodicalIF":2.0,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12037958/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143060133","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}

{"title":"Pricing for Multi-Indication Drugs in the Italian Regulatory Context.","authors":"Maria Grazia Ursino, Annalisa Milano, Filippo Viti De Angelis, Eva Alessi, Francesco Trotta","doi":"10.1007/s41669-024-00555-x","DOIUrl":"10.1007/s41669-024-00555-x","url":null,"abstract":"<p><strong>Background: </strong>The authorization of new therapeutic indications for drugs already reimbursed by the Italian National Health Service (NHS) represents a matter of importance. This study aims to estimate the additional discount attributed to the extension of indications (EoIs) to explore the potential correlation between spending and negotiated discounts and to find specific factors (determinants) that impact on discount.</p><p><strong>Methods: </strong>The study focused on drugs approved in Italy between 2003 and 2017 with at least four therapeutic indications, including the first approved and EoIs, with follow-up extended until 2021 to acquire all the information on the negotiation process that has been completed. Data were obtained from reimbursement and pricing dossiers, and negotiation assessments. Trends in the number of EoIs submitted and the additional discounts negotiated were analyzed, along with the relationship between the negotiated discount and subsequent drug expenditure. Determinants influencing the extent of the negotiated discount were assessed, including drug type, orphan drug designation, innovativeness status, number of EoIs, disease incidence and prevalence, estimated number of patients, revenue projections, availability of therapeutic alternatives, and efficacy outcomes. A Wilcoxon nonparametric test was used to evaluate the associations between determinants and the negotiated additional discount, with a significance level of 0.05.</p><p><strong>Results: </strong>The study identified nine medicines: five of these were used in onco-hematologic therapeutic areas, while the remaining four were immunosuppressants for dermatologic and/or rheumatologic conditions. These nine active substances accounted for 65 approved therapeutic indications, of which 50 were reimbursed by the Italian NHS, including the first indication; the analysis focused only on 40 reimbursed EoIs. The additional discount obtained for EoIs averages approximately 12.5% (95% CI 9.4-16.6%), with a median value of approximately 11%. This latter value was used as the threshold in the analysis of the determinants potentially impacting the negotiated discount amount. Discounts greater than 11% were significantly associated with EoI beyond the fifth and oncology drugs. The additional discount seemed small when compared with the increased spending.</p><p><strong>Conclusion: </strong>The study provides valuable insights into the negotiation outcomes for medicines with multiple therapeutic indications, particularly in onco-hematologic and immunosuppressive areas. The analysis revealed that additional discounts for EoIs averaged 12.5%, with a median of 11%, a value used to assess the impact of specific determinants. A discount higher than 11% was statistically correlated with drugs having more than five indications and oncology treatments, showing their influence in negotiations. However, the savings from discounts were modest relative to the increased drug","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"415-422"},"PeriodicalIF":2.0,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12037441/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143024275","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}

{"title":"So Many Choices, So Little Value: Potential Savings from Selecting Cost-Effective Proton Pump Inhibitors.","authors":"Hye-Young Kwon","doi":"10.1007/s41669-025-00563-5","DOIUrl":"10.1007/s41669-025-00563-5","url":null,"abstract":"<p><strong>Background: </strong>Given that Korea lacks measures to promote cost-effective prescribing, this study investigated the efficiency of prescribers' decision-making when faced with the choice of 398 products containing 15 proton pump inhibitors (PPI).</p><p><strong>Objective: </strong>This study aimed to explore PPI prescribing patterns in ambulatory care over 10 years and elucidate prescribers' practices. Further analysis was conducted to estimate the achievable potential savings, assuming that the recommendation of first-choice drugs is based on the rational use of medicines.</p><p><strong>Methods: </strong>Retrospective PPI prescribing data from the ambulatory sector pertaining to medical services provided to the entire South Korean population from 2013 to 2022 were extracted from the Korean National Health Information Database; the data were analyzed to identify annual trends in PPI spending and utilization volume according to defined daily doses (DDDs). Unit price per DDD was calculated, and a preferred cost-effective choice with identical efficacy and safety profile was selected among PPIs. The potential savings were then simulated using Dirichlet distribution.</p><p><strong>Results: </strong>In ambulatory care, PPI drug costs increased by 13.0% per annum over 10 years. The preferred substances were esomeprazole (31.6%), rabeprazole (23.2%), and tegoprazan (16.5%), which are among the most expensive PPIs. However, the most cost-effective substance was dexlansoprazole (South Korean won [KRW] 522.7; standard deviation [SD] = 72.2; and median = 583). If dexlansoprazole is recommended as the first-choice PPI in the Korean context, the estimated cost savings would be 404.24 billion KRW, equivalent to 47.0% of the current PPI spending in ambulatory care.</p><p><strong>Conclusions: </strong>This study identified opportunities for substantial cost savings related to PPI prescribing. Guided decision-making toward cost-effective PPI prescribing would increase the efficiency of prescribing and the sustainability of the healthcare system.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"461-469"},"PeriodicalIF":2.0,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12037450/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143414767","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}

{"title":"COVID-19 Vaccine Preferences in China: A Comparison of Discrete Choice Experiment and Profile Case Best-Worst Scaling.","authors":"Enxue Chang, Yanni Jia, Xiaoying Zhu, Lunan Wang, Ying Yan, Kejun Liu, Weidong Huang","doi":"10.1007/s41669-025-00559-1","DOIUrl":"10.1007/s41669-025-00559-1","url":null,"abstract":"<p><strong>Objectives: </strong>Little is known about the diversity of residents' preferences for COVID-19 vaccines during the time when COVID-19 management was downgraded in China. This study aims to investigate these preferences using discrete choice experiment (DCE) and profile case best-worst scaling (BWS-2), and to assess the concordance between these two methods.</p><p><strong>Methods: </strong>Chinese residents recruited for the online survey were asked to evaluate COVID-19 vaccine profiles through both DCE and BWS-2 from April to July 2023. Attributes included effectiveness, duration of protection, risk of severe adverse events (degree), the total out-of-pocket (OOP) cost, brand, and the vaccination method. We utilized conditional regression and mixed logit regression models to estimate the preference levels for potential attributes. To assess preference concordance between the two methods, re-scaling and the Spearman correlation test were used. Additionally, subgroup analysis was conducted to determine the most suitable method for different population groups, categorized by vaccine hesitancy and risk level.</p><p><strong>Results: </strong>A total of 438 (71.22%) respondents were included. A similar pattern was found in the DCE and BWS-2 methods, with the respondents having a strong preference for 90% vaccine effectiveness. However, the methods diverged in other preferences; DCE favored domestic brands and low severe adverse event risk, while BWS-2 preferred moderate risk and three years of protection. Concordance assessment, including Spearman's correlation and linear regression, showed no significant correlation and poor concordance between the methods, underscoring these differences. Preference heterogeneity is revealed among different groups; however, effectiveness remained the most important attribute for all subgroups of the population. Oral vaccination was the preferred option for both the vaccine-hesitant and high-risk groups.</p><p><strong>Conclusion: </strong>This study offers new insights into the varying preferences for COVID-19 vaccines among Chinese residents following the downgrading of pandemic management measures. The findings underscore the need for diverse strategies in vaccine policy design. Special emphasis should be placed on vaccine attributes that align with public priorities, such as high effectiveness and low risk levels, to enhance vaccine uptake.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"399-413"},"PeriodicalIF":2.0,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12037921/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143075287","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}

{"title":"Do We Understand Unmet Need? A Proposal to Use Length-Of-Life Equivalent (LOLE) as a Patient-Centric Measure of Unmet Need.","authors":"Kevin Marsh, Robert F Reynolds, Linda Nelsen, Stephen Watt, Omar A Escontrías, Brett Hauber","doi":"10.1007/s41669-025-00560-8","DOIUrl":"10.1007/s41669-025-00560-8","url":null,"abstract":"<p><p>Many decision-makers have emphasized the importance of leveraging patient experience data to measure unmet need. However, there is no standardized, patient-centric unmet need measure that formalizes how the value judgements inherent in such a measure should be made. Several initiatives have identified measuring unmet need as one of the primary uses of patient preference data. After reviewing how decision-makers define unmet need, this paper proposes that a thresholding method could be used to generate a standardized, patient-centric, disease-agnostic, quantitative unmet need estimate, length of life equivalent (LOLE). LOLE would address some of the limitations of current methods, including facilitating capture of the impact of disease beyond health-related quality of life, and being more sensitive to the impact of a disease on patients. However, the acceptability of LOLE raises questions for decision-makers, including: Is length of life equivalence the best common metric in which to express unmet need? Is it appropriate to rate a disease as having no unmet need if patients are unwilling to trade off life expectancy for improvements in their quality of life? Can LOLE be estimated for more complex disease profiles? Is thresholding the appropriate method to use to estimate LOLE? How should LOLE be integrated into decision-making, including the level of LOLE that defines different levels of unmet need? Further work could usefully address these questions with decision-makers.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"341-350"},"PeriodicalIF":2.0,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12037453/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143441786","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}

{"title":"Using Generative Artificial Intelligence in Health Economics and Outcomes Research: A Primer on Techniques and Breakthroughs.","authors":"Tim Reason, Sven Klijn, Will Rawlinson, Emma Benbow, Julia Langham, Siguroli Teitsson, Kasper Johannesen, Bill Malcolm","doi":"10.1007/s41669-025-00580-4","DOIUrl":"https://doi.org/10.1007/s41669-025-00580-4","url":null,"abstract":"&lt;p&gt;&lt;p&gt;The emergence of generative artificial intelligence (GenAI) offers the potential to enhance health economics and outcomes research (HEOR) by streamlining traditionally time-consuming and labour-intensive tasks, such as literature reviews, data extraction, and economic modelling. To effectively navigate this evolving landscape, health economists need a foundational understanding of how GenAI can complement their work. This primer aims to introduce health economists to the essentials of using GenAI tools, particularly large language models (LLMs), in HEOR projects. For health economists new to GenAI technologies, chatbot interfaces like ChatGPT offer an accessible way to explore the potential of LLMs. For more complex projects, knowledge of application programming interfaces (APIs), which provide scalability and integration capabilities, and prompt engineering strategies, such as few-shot and chain-of-thought prompting, is necessary to ensure accurate and efficient data analysis, enhance model performance, and tailor outputs to specific HEOR needs. Retrieval-augmented generation (RAG) can further improve LLM performance by incorporating current external information. LLMs have significant potential in many common HEOR tasks, such as summarising medical literature, extracting structured data, drafting report sections, generating statistical code, answering specific questions, and reviewing materials to enhance quality. However, health economists must also be aware of ongoing limitations and challenges, such as the propensity of LLMs to produce inaccurate information ('hallucinate'), security concerns, issues with reproducibility, and the risk of bias. Implementing LLMs in HEOR requires robust security protocols to handle sensitive data in compliance with the European Union's General Data Protection Regulation (GDPR) and the United States' Health Insurance Portability and Accountability Act (HIPAA). Deployment options such as local hosting, secure API use, or cloud-hosted open-source models offer varying levels of control and cost, each with unique trade-offs in security, accessibility, and technical demands. Reproducibility and transparency also pose unique challenges. To ensure the credibility of LLM-generated content, explicit declarations of the model version, prompting techniques, and benchmarks against established standards are recommended. Given the 'black box' nature of LLMs, a clear reporting structure is essential to maintain transparency and validate outputs, enabling stakeholders to assess the reliability and accuracy of LLM-generated HEOR analyses. The ethical implications of using artificial intelligence (AI) in HEOR, including LLMs, are complex and multifaceted, requiring careful assessment of each use case to determine the necessary level of ethical scrutiny and transparency. Health economists must balance the potential benefits of AI adoption against the risks of maintaining current practices, while also considering issues such ","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":""},"PeriodicalIF":2.0,"publicationDate":"2025-04-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144022846","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}

{"title":"Assessing the Value for Money of Enzyme Replacement Therapy in Gaucher Disease Types 1 and 3b: Can Expanded Coverage Be Justified?","authors":"Waranya Rattanavipapong, Thunyarat Anothaisintawee, Wanrudee Isaranuwatchai, Duangrurdee Wattanasirichaigoon, Thipwimol Tim-Aroon, Khunton Wichajarn, Achara Sathienkijkanchai, Pimlak Charoenkwan, Kanya Suphapeetiporn, Chanchai Traivaree, Chulaluck Kuptanon, Yot Teerawattananon","doi":"10.1007/s41669-025-00579-x","DOIUrl":"https://doi.org/10.1007/s41669-025-00579-x","url":null,"abstract":"<p><strong>Background and objectives: </strong>The Health Intervention and Technology Assessment Program was commissioned to conduct a cost-utility and budget impact analysis of enzyme replacement therapy (ERT) for Gaucher disease types 1 and 3b. The findings from this assessment are to support the decision-making process regarding the potential expansion of ERT coverage within Thailand's public health system.</p><p><strong>Methods: </strong>The analysis compared the current policy, which provides treatment with imiglucerase only for patients with Gaucher disease type 1, as listed in the National List of Essential Medicine, with a proposed policy that extends coverage to include Gaucher disease types 1 and 3b with either imiglucerase or velaglucerase. Cost-utility analysis of these policy options was performed using decision tree and Markov models over a lifetime horizon from a societal perspective. The financial implications for the relevant budgetary authority over 5 years were estimated. The research methodology adheres rigorously to Thailand's health technology assessment guidelines.</p><p><strong>Results: </strong>The study found that the incremental cost-effectiveness ratios for treating both Gaucher disease types 1 and 3b are 6,769,000 and 9,359,000 baht per quality-adjusted life year (QALY) for imiglucerase and velaglucerase, respectively, which is well beyond Thailand's cost-effectiveness threshold of 160,000 baht per QALY. Such an expansion would incur an additional budgetary burden of approximately 81 million baht for imiglucerase and 138 million baht for velaglucerase. Increasing the rate of hematopoietic stem cell transplantation (HSCT) can improve the cost-effectiveness of the expansion.</p><p><strong>Conclusions: </strong>The study concludes that expanding ERT with either imiglucerase or velaglucerase to treat both Gaucher disease types 1 and 3b is not cost-effective at current prices in Thailand; however, it could become cost-effective with a reduction of approximately 60% in drug prices or if all eligible patients undergo HSCT.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":""},"PeriodicalIF":2.0,"publicationDate":"2025-04-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144037695","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}

{"title":"Cost-Effectiveness Analysis of Molnupiravir Versus Best Supportive Care for the Treatment of Outpatient COVID-19 in High-Risk Older Adults in Japan.","authors":"Hardik Goswami, Atsushi Tajima, Taizo Matsuki, Amy Puenpatom","doi":"10.1007/s41669-025-00578-y","DOIUrl":"https://doi.org/10.1007/s41669-025-00578-y","url":null,"abstract":"<p><strong>Objectives: </strong>This analysis aimed to evaluate the cost effectiveness of molnupiravir versus best supportive care for the treatment of older adult patients (aged ≥ 65 years) in Japan with mild to moderate COVID-19 who are at risk of disease progression leading to hospitalization, predominantly using input data derived from the Omicron era of the SARS-CoV2 pandemic.</p><p><strong>Methods: </strong>A decision-analytic model was developed, comprising a decision-tree model for the acute COVID-19 phase (30 days), followed by a lifetime Markov model. Inputs used to parametrize the model were derived from a database study conducted in Japan and a published systematic literature review of real-world studies, and from ad-hoc literature searches and other research (for disease progression, cost, and utility estimates). This analysis modelled death averted due to COVID-19 hospitalization as an indirect effect of molnupiravir (through preventing hospitalization). Costs were expressed in 2022 Japanese yen (¥; JPY), from the perspective of payers (the base case) or society (in a scenario analysis). Costs and QALYs were discounted at 2% per year. Cost effectiveness of molnupiravir versus best supportive care was primarily compared to a willingness-to-pay (WTP) threshold of ¥5,000,000 per quality-adjusted life year (QALY) gained.</p><p><strong>Results: </strong>Treatment with molnupiravir is associated with a QALY gain of 0.018 and an incremental cost of ¥81,472 over best supportive care and is cost effective (with an incremental cost-effectiveness ratio [ICER] of ¥4,638,477) versus best supportive care based on the predefined WTP threshold of ¥5,000,000 per QALY gained. Molnupiravir leads to a reduction in the proportion of patients who die due to COVID-19 hospitalization (0.09% with molnupiravir vs 0.29% with best supportive care). Molnupiravir is also associated with lower costs associated with COVID-19 hospitalizations compared with best supportive care (¥22,527 vs ¥27,472). In a deterministic sensitivity analysis, the top five most sensitive parameters were baseline hospitalization rate, mortality benefit of molnupiravir, mortality rate in general ward, discount rate, and mortality rate in intensive care unit. In a probabilistic sensitivity analysis, at the predefined WTP threshold of ¥5,000,000 per QALY gained, molnupiravir had an 80% probability of being cost effective versus best supportive care.</p><p><strong>Conclusions: </strong>Molnupiravir is a cost-effective treatment option for the treatment of older adult outpatients (age ≥ 65 years) with symptomatic COVID-19 in Japan, relative to best supportive care.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":""},"PeriodicalIF":2.0,"publicationDate":"2025-04-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144005178","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}

{"title":"A Systematic Literature Review of the Economic and Healthcare Resource Utilization Burden of Relapsed/Refractory Follicular Lymphoma.","authors":"Bijal Shah, Mei Xue, Wesley Furnback, Erlene K Seymour, Jin Kim, Po-Ya Chuang, Madeline Dec, Keri Yang","doi":"10.1007/s41669-025-00577-z","DOIUrl":"https://doi.org/10.1007/s41669-025-00577-z","url":null,"abstract":"<p><strong>Objective: </strong>To quantify the economic or healthcare resource utilization (HCRU) burden and examine the value of interventions for relapsed or refractory (R/R) follicular lymphoma (FL).</p><p><strong>Methods: </strong>The PubMed and Embase databases were searched for full-text studies and conference abstracts published between 1 January 2019 and 31 December 2023 that reported either the economic or HCRU burden of R/R FL or reported the results of health economic models assessing interventions for R/R FL. A supplemental manual search was also undertaken to identify conference abstracts that may not have been indexed in the primary databases. A data extraction sheet was used to develop evidence tables.</p><p><strong>Results: </strong>A total of 30 records were included spanning 11 retrospective or prospective studies, 11 cost-effectiveness evaluations, and 8 other economic models. Costs and HCRU generally tended to increase as the line of therapy increased, reaching over US$400,000 annually in later lines. Costs associated with recently approved chimeric antigen receptor T-cell therapy (CAR-T) ranged from US$450,000 to over US$700,000 per patient. Economic models evaluating novel therapies, such as CAR-T, tazemetostat, and mosunetuzumab, estimated they would generally be cost-effective and have minimal budget impact or cost-savings. However, these models noted considerable assumptions regarding treatment duration and discontinuation. Real-world costs and resource use for newly approved therapies including CAR-Ts and bispecifics were limited.</p><p><strong>Conclusions: </strong>The burden of R/R FL is substantial and increases as patients progress. Considerable gaps exist for the real-world impact of novel therapies, including CAR-Ts and bispecifics, on the economic burden and will need to be studied to properly assess their value.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":""},"PeriodicalIF":2.0,"publicationDate":"2025-04-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144034466","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}

{"title":"Indirect Treatment Comparisons in EUnetHTA Relative Effectiveness Assessments: Learnings and Recommendations for the Implementation of EU Joint Clinical Assessments.","authors":"Sophie van Beekhuizen, Menglu Che, Loraine Monfort, Mahmoud Hashim, Ali Azough, Nicole Kubitz, Adrian Griffin, Martin Price","doi":"10.1007/s41669-025-00575-1","DOIUrl":"https://doi.org/10.1007/s41669-025-00575-1","url":null,"abstract":"<p><strong>Background: </strong>Beginning in January 2025, all new active substances must undergo evaluation of relative clinical effectiveness through European Union (EU) joint clinical assessments (JCAs). In the absence of head-to-head data, indirect treatment comparisons (ITCs) become indispensable in meeting the numerous population, intervention, comparators and outcomes (PICO) criteria to support decision-making.</p><p><strong>Objective: </strong>This study examined ITCs in European Network for Health Technology Assessment (EUnetHTA) relative effectiveness assessments (REAs) to obtain valuable insights into their potential implications for future JCAs.</p><p><strong>Methods: </strong>The EUnetHTA website was hand-searched for REAs of pharmaceutical products between 2010 and 2021. Information on PICO, ITC methods, ITC limitations/critiques, and relative effectiveness conclusions were systematically extracted. On the basis of the final EUnetHTA critiques, suitability of ITC evidence was categorised by the current study authors as appropriate, unsuitable or unclear.</p><p><strong>Results: </strong>Twenty-three REAs were identified. Twelve REAs included an ITC, of which six were in oncology indications. Across the REAs, 64 comparisons were required, with a median of four comparators per REA (range 1-18). In total, 25 comparisons were informed by indirect evidence; the suitability of ITCs was categorised as unclear in all but one of the 25 comparisons.</p><p><strong>Conclusion: </strong>Multiple analyses and ITCs were necessary to address multiple PICOs. Although most ITCs were categorised as unclear within the REAs, they were still considered appropriate to inform decision-making. The EU JCA process will most likely require health technology developers to use various ITC approaches to address the multiple PICOs requested, recognising the inherent limitations of these methodologies. Efforts to address potential challenges for EU JCA should focus on supporting JCA assessors/co-assessors and national HTA agencies in the evaluation and interpretation of ITCs to enable decision-making.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":""},"PeriodicalIF":2.0,"publicationDate":"2025-04-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144009731","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}