Naren Kumar Surendra, Jiamin Ong, Xin Yi Wong, Michelle Poon, Lydia Loke, Liang Lin, Mohamed Ismail Abdul Aziz, Benjamin Shao Kiat Ong, Kwong Ng
{"title":"Cost-effectiveness of ruxolitinib in Singapore for patients with chronic graft-versus-host disease.","authors":"Naren Kumar Surendra, Jiamin Ong, Xin Yi Wong, Michelle Poon, Lydia Loke, Liang Lin, Mohamed Ismail Abdul Aziz, Benjamin Shao Kiat Ong, Kwong Ng","doi":"10.1080/14737167.2025.2529366","DOIUrl":"10.1080/14737167.2025.2529366","url":null,"abstract":"<p><strong>Objective: </strong>To evaluate the cost-effectiveness of ruxolitinib in Singapore for patient with chronic graft-versus-host disease (GVHD) and inadequate response to corticosteroids.</p><p><strong>Methods: </strong>A three-state partitioned survival model was developed to evaluate the cost-effectiveness of ruxolitinib from the Singapore healthcare system perspective over a five-year time horizon. Clinical data were sourced from the REACH3 trial, health state utilities were retrieved from literature, and direct costs were obtained from public healthcare institutions in Singapore. Sensitivity and scenario analyses were conducted to explore the impact of uncertainties and assumptions on the cost-effectiveness results.</p><p><strong>Results: </strong>Compared to best available therapy, ruxolitinib yielded a base-case incremental cost-effectiveness ratio (ICER) of S$776,653 (US$574,724) per quality-adjusted life-years gained. Sensitivity analyses revealed that the ICER was particularly sensitive to utilities in failure-free and progressed disease states. Scenario analyses confirmed that the ICERs remained high even under favorable assumptions, and a substantial price reduction was necessary to lower the ICER.</p><p><strong>Conclusion: </strong>At its current price, ruxolitinib is not cost-effective for treating chronic GVHD in Singapore. This finding helps to inform funding decision-making, which also considers other factors such as clinical effectiveness, safety, and budget impact, in addition to cost-effectiveness.</p>","PeriodicalId":12244,"journal":{"name":"Expert Review of Pharmacoeconomics & Outcomes Research","volume":" ","pages":"1-8"},"PeriodicalIF":1.8,"publicationDate":"2025-07-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144539753","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}
{"title":"Transdisciplinary health economics for 2050: the challenge of preventing the adverse health effects of obesity, inequalities, and climate change.","authors":"Rhiannon Tudor Edwards","doi":"10.1080/14737167.2025.2498658","DOIUrl":"10.1080/14737167.2025.2498658","url":null,"abstract":"","PeriodicalId":12244,"journal":{"name":"Expert Review of Pharmacoeconomics & Outcomes Research","volume":" ","pages":"845-848"},"PeriodicalIF":1.8,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143976793","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}
Sophia Aguiar Monteiro Borges, Natacha Regina de Moraes Cerchiari, Erick Ohanesian Polli, Ana Carolina Nonato, Felipe Lima Barreto, Alexandre de Oliveira Esteves, Maarten Jacobus Postma, Ana Marli Christovam Sartori, Patrícia Coelho de Soárez
{"title":"Disease burden estimates in economic evaluation studies of respiratory syncytial virus (RSV) maternal immunization: a systematic review.","authors":"Sophia Aguiar Monteiro Borges, Natacha Regina de Moraes Cerchiari, Erick Ohanesian Polli, Ana Carolina Nonato, Felipe Lima Barreto, Alexandre de Oliveira Esteves, Maarten Jacobus Postma, Ana Marli Christovam Sartori, Patrícia Coelho de Soárez","doi":"10.1080/14737167.2025.2498663","DOIUrl":"10.1080/14737167.2025.2498663","url":null,"abstract":"<p><strong>Introduction: </strong>Recently, new technologies have emerged for the prevention of respiratory syncytial virus (RSV) infections. Reliable epidemiological data are essential for accurately assessing the disease burden and informing health economic evaluations (HEE). This review evaluates how HEE of RSV maternal vaccination (MV) estimated the RSV disease burden.</p><p><strong>Methods: </strong>A systematic search was conducted in MEDLINE, SCOPUS, EMBASE, NHS EED, HTA, Tufts CEA Registry, LILACS, and Web of Science for full HEE of RSV MV. Reporting quality was assessed with the CHEERS 2022 checklist, costs were converted to 2024 U.S. dollars, and a descriptive, interpretive synthesis of the data was performed.</p><p><strong>Results: </strong>All 21 included studies were cost-utility analyses, 10 conducted in high-income countries. The HEE utilized a wide range of data sources to build epidemiological estimates, frequently relying on non-local data, particularly for outpatient rates. National data on RSV hospitalization rates were the most commonly available. No study included equity assessments. The maternal vaccine dose price was identified as a critical factor in the strategy's cost-effectiveness.</p><p><strong>Conclusion: </strong>Enhancing local data availability for RSV, by strengthening the respiratory virus surveillance, is crucial to improve the reliability of HEE of RSV prevention strategies and enable more informed and effective policy decisions.</p><p><strong>Registration: </strong>PROSPERO: CRD42024549989.</p>","PeriodicalId":12244,"journal":{"name":"Expert Review of Pharmacoeconomics & Outcomes Research","volume":" ","pages":"863-896"},"PeriodicalIF":1.8,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143960070","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}
Won Chan Lee, Chris Blanchette, Shibani Pokras, Javed Shaikh, Jared Miller
{"title":"The evolution and future of integrated evidence planning.","authors":"Won Chan Lee, Chris Blanchette, Shibani Pokras, Javed Shaikh, Jared Miller","doi":"10.1080/14737167.2025.2497876","DOIUrl":"10.1080/14737167.2025.2497876","url":null,"abstract":"<p><strong>Introduction: </strong>Integrated Evidence Planning (IEP) is a strategic approach that optimizes drug development and market access by ensuring evidence generation aligns with regulatory, clinical, and market needs. The increasing integration of advanced technologies, including artificial intelligence/machine learning (AI/ML), natural language processing (NLP), and generative AI is set to revolutionize IEP by enhancing decision-making and improving patient access.</p><p><strong>Areas covered: </strong>This article examines the role of IEP in drug development, focusing on its application across the product lifecycle, pre-clinical to post-launch. It highlights the integration of various analytical techniques, including descriptive analysis, ML, and causal inference to generate evidence. Challenges in implementing IEP, such as organizational barriers, data accessibility, and needs for specialized software tools are discussed. The evolving role of real-world evidence is emphasized, advocating for IEP as a dynamic, iterative process that adapts to market changes. Additionally, the potential of generative AI and real-time analytics to improve evidence generation and stakeholder collaboration is explored.</p><p><strong>Expert opinion: </strong>The transformative potential of generative AI in IEP facilitates on-demand insights and conversational data access. However, challenges such as organizational inertia and the need for cross-functional alignment remain. Successful IEP implementation requires strong leadership, stakeholder buy-in, and optimized resource allocation to fully capitalize on its benefits.</p>","PeriodicalId":12244,"journal":{"name":"Expert Review of Pharmacoeconomics & Outcomes Research","volume":" ","pages":"855-862"},"PeriodicalIF":1.8,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143976432","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}
Beatrice Canali, Giovanni Apolone, Paolo A Ascierto, Filippo De Braud, Francesco Grossi, Francesco Perrone, Francesca Fiorentino, Alessandra Di Costanzo, Laura Candelora, Giovanni Patanè, Giuseppina Zapparelli, Claudia Mezzanotte, Guido Didoni, Massimo Riccaboni
{"title":"Effect of immuno-oncology on clinical and economic outcomes for a selection of cancers in Italy.","authors":"Beatrice Canali, Giovanni Apolone, Paolo A Ascierto, Filippo De Braud, Francesco Grossi, Francesco Perrone, Francesca Fiorentino, Alessandra Di Costanzo, Laura Candelora, Giovanni Patanè, Giuseppina Zapparelli, Claudia Mezzanotte, Guido Didoni, Massimo Riccaboni","doi":"10.1080/14737167.2025.2493130","DOIUrl":"10.1080/14737167.2025.2493130","url":null,"abstract":"<p><strong>Objectives: </strong>This study assesses the impact of immune-oncology (IO) drugs' availability on cancer incidence-adjusted mortality rates from melanoma, lung, and renal cancers at population level in Italy between 2008 and 2019.</p><p><strong>Methods: </strong>We conducted a retrospective study on cross-sectional time-series aggregated data collected from publicly available sources and IQVIA proprietary databases. Three fixed-effects regression models were used to estimate how IO availability affects incidence-adjusted mortality for each cancer type. Estimated deaths were compared with deaths in a scenario with no IO drugs availability. Finally, the number of averted deaths was valued using the human capital approach.</p><p><strong>Results: </strong>A 1% increase in IO availability reduces incidence-adjusted mortality rates for melanoma, lung, and renal cancers by 0.125% (95% CI: 0.138-0.112; <i>p</i> < 0.01), 0.011% (95% CI: 0.013-0.009; <i>p</i> < 0.01) and 0.005% (95% CI: 0.006-0.003; <i>p</i> < 0.01) between the introduction of the drug in the therapeutic area and 2019. This reduction resulted in total savings of € 49.0 million, € 61.3 million, and € 10.9 million in indirect costs due to premature mortality, respectively.</p><p><strong>Conclusions: </strong>IO drugs introduction in Italy between 2008 and 2019 was associated with a significant decrease in deaths from each cancer and, consequently, in savings in indirect costs related to premature mortality.</p>","PeriodicalId":12244,"journal":{"name":"Expert Review of Pharmacoeconomics & Outcomes Research","volume":" ","pages":"905-915"},"PeriodicalIF":1.8,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143971404","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}
Adriënne Rotteveel, Saskia Knies, Ardine de Wit, Johan Polder, Bram Wouterse
{"title":"Valuing prevention: lessons and recommendations from a Dutch expert committee.","authors":"Adriënne Rotteveel, Saskia Knies, Ardine de Wit, Johan Polder, Bram Wouterse","doi":"10.1080/14737167.2025.2504946","DOIUrl":"10.1080/14737167.2025.2504946","url":null,"abstract":"<p><strong>Introduction: </strong>Countries have been struggling with investments in prevention. How to obtain insight in the costs, benefits, and budgetary impacts of investments in prevention? And how to weigh investments in prevention against other investments in health, such as in curative health care. In the Netherlands, in 2023, a methodological expert committee was established to answer such questions. Their advice, published in January 2024, has been impactful, receiving much attention in Dutch politics and policy and leading to the establishment of a subsequent scientific committee on appropriate evidence and the development of an investment model for prevention.</p><p><strong>Areas covered: </strong>In this perspective, we present the main recommendations from the advice and embed these recommendations in the Dutch institutional context. Furthermore, we highlight why the financing of prevention is challenging and suggest some solutions. Finally, we discuss how the advice was received and what follow-up was given, reflect on why this advice had such a large impact, and distill recommendations for other countries from this experience.</p><p><strong>Expert opinion: </strong>Economic evaluation of prevention is not intrinsically more challenging than that of other health investments. For a direct comparison of investments in health, similar methods should be used for both prevention and medical care.</p>","PeriodicalId":12244,"journal":{"name":"Expert Review of Pharmacoeconomics & Outcomes Research","volume":" ","pages":"849-854"},"PeriodicalIF":1.8,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144076924","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}
Valentina Perrone, Domenica Daniela Ancona, Marco Gnesi, Valentina Mongelli, Sara Migliaccio, Felice Renato Colanero, Emanuele Antonio Lenoci, Lidia Bonfanti, Melania Leogrande, Maria Cappuccilli, Cataldo Procacci, Luca Degli Esposti
{"title":"Clinical and economic outcomes of patients with cardiorenal and metabolic diseases: a real-world analysis in a single Italian Local Health Unit.","authors":"Valentina Perrone, Domenica Daniela Ancona, Marco Gnesi, Valentina Mongelli, Sara Migliaccio, Felice Renato Colanero, Emanuele Antonio Lenoci, Lidia Bonfanti, Melania Leogrande, Maria Cappuccilli, Cataldo Procacci, Luca Degli Esposti","doi":"10.1080/14737167.2025.2504944","DOIUrl":"10.1080/14737167.2025.2504944","url":null,"abstract":"<p><strong>Background: </strong>This single-center real-world analysis describes the epidemiology, demographic and clinical characteristics, mortality, and therapeutic-care management of patients grouped by the presence of seven cardiorenal and metabolic diseases.</p><p><strong>Research design and methods: </strong>From the administrative databases of a single Local Health Unit in Southern Italy with approximately 400,000 inhabitants, the following cohorts were identified during 2020-2021: (1) type 1 diabetes (T1D); (2) type 2 diabetes (T2D); (3) heart failure (HF); (4) chronic kidney disease (CKD); (5) hyperkalemia (HK); (6) nonalcoholic steatohepatitis (NASH); (7) dyslipidemia. The variables examined for each cohort were: prevalence, demographic and clinical data, comorbidity profile, biochemical tests, hospitalization rates, therapies, 1-year all-cause mortality.</p><p><strong>Results: </strong>From a cohorts overview, the population with cardiorenal and metabolic diseases was characterized by a high proportion of elderly subjects, a relatively high rate of all-cause hospitalizations and elevated mortality. The analysis of the comorbidity pattern in each group confirmed the interrelation of these pathologies, with diabetes, dyslipidemia, and CKD as the most common concomitant conditions.</p><p><strong>Conclusions: </strong>From these real-world data, patients with cardiorenal and metabolic diseases emerge as a fragile population, with multimorbidity, and a substantial risk of hospitalizations and death, confirming as chronicity represent a priority issue for health systems.</p>","PeriodicalId":12244,"journal":{"name":"Expert Review of Pharmacoeconomics & Outcomes Research","volume":" ","pages":"955-965"},"PeriodicalIF":1.8,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144157594","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}
{"title":"Unleashing economic potential of AI-driven technology to prevent adverse events and sub-optimal treatment.","authors":"L Maas, C Contreras-Meca, W Vos, M Hiligsmann","doi":"10.1080/14737167.2025.2499118","DOIUrl":"10.1080/14737167.2025.2499118","url":null,"abstract":"","PeriodicalId":12244,"journal":{"name":"Expert Review of Pharmacoeconomics & Outcomes Research","volume":" ","pages":"837-839"},"PeriodicalIF":1.8,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143985711","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}
{"title":"Intravenous and subcutaneous vedolizumab for moderately to severely active ulcerative colitis in Iran: a model-based cost-effectiveness evaluation.","authors":"Kimia Niksalehi, Anita Oyarhossein, Soroush Fariman, Adel Ahmadi, Behniya Azadmehr, Naeim Karimpour-Fard, Laleh Vaziri, Monireh Afzali","doi":"10.1080/14737167.2025.2499718","DOIUrl":"10.1080/14737167.2025.2499718","url":null,"abstract":"<p><strong>Objective: </strong>To evaluate the cost-effectiveness of intravenous and subcutaneous vedolizumab compared to infliximab for moderately to severely active ulcerative colitis (UC) from an Iranian societal perspective.</p><p><strong>Methods: </strong>A decision-analytic cost-utility model was developed using a decision tree and a Markov model. Direct and indirect costs were calculated. Treatment effectiveness and health state utility values were extracted from published literature. Effects were estimated using quality-adjusted life-years (QALYs). Costs and QALYs were projected over a lifetime and discounted at 3% per year. Deterministic and probabilistic sensitivity analyses were performed to evaluate uncertainties.</p><p><strong>Results: </strong>In base-case analysis, infliximab was associated with $28,888.5 costs and 15.17 QALYs. Vedolizumab was associated with 15.34 QALYs and costs of $27,916.90 for IV (induction and maintenance) and $28,200.40 for IV (induction) followed by SC (maintenance). The ICERs were estimated at -$5673.3/QALY and -$4,017.8/QALY, remaining negative across all sensitivity analyses. The cost of infliximab and vedolizumab and the probability of response to vedolizumab during maintenance were the key determinants of cost-effectiveness.</p><p><strong>Conclusions: </strong>Vedolizumab (both IV and SC) resulted in reduced costs and improved QALYs compared to infliximab for moderate to severe UC in Iran. Therefore, we recommended including vedolizumab in the Iranian national formulary as a cost-effective treatment strategy.</p>","PeriodicalId":12244,"journal":{"name":"Expert Review of Pharmacoeconomics & Outcomes Research","volume":" ","pages":"933-941"},"PeriodicalIF":1.8,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143958639","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}
Yoana Seitaridou, Dominik Grega, Sorin Paveliu, Agnes Männik, Przemysław Holko, Ivana Tadić, Ivana Stevic, Teodora Chamova, Guenka Petrova, Maria Kamusheva
{"title":"Access to medicines, care, and services for multiple sclerosis patients in Central and Eastern European countries: a comparative analysis.","authors":"Yoana Seitaridou, Dominik Grega, Sorin Paveliu, Agnes Männik, Przemysław Holko, Ivana Tadić, Ivana Stevic, Teodora Chamova, Guenka Petrova, Maria Kamusheva","doi":"10.1080/14737167.2025.2493131","DOIUrl":"10.1080/14737167.2025.2493131","url":null,"abstract":"<p><strong>Background: </strong>The study compared access to disease-modifying treatments (DMTs), care, and services for multiple sclerosis (MS) patients across six Central and Eastern European Countries (CEECs).</p><p><strong>Research design and methods: </strong>A 2-part questionnaire-based survey was conducted among experts from Bulgaria, Estonia, Poland, Romania, Serbia, and Slovakia over 4 months (1 December 2022 - 1 April 2023).</p><p><strong>Results: </strong>The study reveals notable variations in the legal requirements for pricing and reimbursement of MS medications, as well as criteria for health technology assessment (HTA) across countries. Despite these differences, DMTs were fully reimbursed in all countries. Poland exhibited the highest MS expenditures in 2021, totaling 171 million euros. Administrative procedures for prescribing and dispensing MS drugs were similar across countries, but the range of services and support offered to MS patients varied. Assessment of treatment accessibility, based on the number of authorized DMTs listed in Positive Drug Lists (PDLs), shows Bulgaria leading with 84.21% access, followed closely by Poland and Slovakia with comparable rates of 78.95%.</p><p><strong>Conclusions: </strong>The study revealed disparities in health care and services for MS patients across the countries surveyed. Addressing these disparities and the need for targeted interventions to ensure equitable access is critical. Further research is needed to find effective solutions.</p>","PeriodicalId":12244,"journal":{"name":"Expert Review of Pharmacoeconomics & Outcomes Research","volume":" ","pages":"917-926"},"PeriodicalIF":1.8,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144001312","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}