E Reith, L Maas, A Joos, I Heikkinen, M De Iaco, M Hiligsmann
{"title":"Qualitative research on the acceptance of evolving evidence for HTA body approval of innovative health technologies in selected European countries.","authors":"E Reith, L Maas, A Joos, I Heikkinen, M De Iaco, M Hiligsmann","doi":"10.1080/14737167.2025.2530622","DOIUrl":"10.1080/14737167.2025.2530622","url":null,"abstract":"<p><strong>Objective: </strong>With increasing efforts of streamlining market access of innovative drugs, the current acceptance of health technology assessment (HTA) bodies on non-traditional trial designs is still unclear. The aim of this study was to assess the perspective of drug developers on the acceptance of non-traditional trial designs by HTA bodies in Europe.</p><p><strong>Methods: </strong>Eleven semi-structured interviews were conducted with experts to gain insights on their perception of non-traditional trial design acceptance by HTA bodies from the Netherlands, Germany, United Kingdom, Sweden, and France. Future perspectives on and recommendations for facilitating the acceptance of non-traditional trials were provided.</p><p><strong>Results: </strong>All experts highlighted the HTA bodies' preference for randomized controlled trials over non-traditional trial designs. The degree of acceptance varied between countries. Experts recommended multistakeholder discussions between drug developers and HTA bodies for early scientific advice and reevaluation of evidence requirements. The EU-HTA regulation was viewed as both a potential opportunity for harmonization and limiting innovative approach.</p><p><strong>Conclusion: </strong>The results of this study highlighted significant variations in the acceptance of non-traditional trial designs by HTA bodies across Europe. Actions are needed to facilitate a more progressive view on non-traditional clinical trial designs to ensure fast patient access to highly innovative treatments.</p>","PeriodicalId":12244,"journal":{"name":"Expert Review of Pharmacoeconomics & Outcomes Research","volume":" ","pages":"1-8"},"PeriodicalIF":1.8,"publicationDate":"2025-07-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144567335","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}
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":"Collaborative economic tools for prevention strategies: case of the EQUIPT model.","authors":"Kei Long Cheung, Mickaël Hiligsmann","doi":"10.1080/14737167.2025.2529363","DOIUrl":"10.1080/14737167.2025.2529363","url":null,"abstract":"","PeriodicalId":12244,"journal":{"name":"Expert Review of Pharmacoeconomics & Outcomes Research","volume":" ","pages":"1-4"},"PeriodicalIF":1.8,"publicationDate":"2025-07-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144559643","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}
Fernando Martínez Valle, Lucía Galán Dávila, Juan Buades Reinés, Juan Gonzalez-Moreno, Inés Losada López, Teresa Sevilla, Francisco Muñoz Beamud, José Eulalio Bárcena Llona, Manuel Romero Acebal, Patricia Tarilonte, Francesca Setaro, Javier Rejas-Gutierrez, Alejandra López-Ibáñez de Aldecoa, Carmen Peral
{"title":"Economic and healthcare resource utilization for hereditary transthyretin amyloidosis with polyneuropathy in Spain: real-world insights from early-stage patients and mutation carriers.","authors":"Fernando Martínez Valle, Lucía Galán Dávila, Juan Buades Reinés, Juan Gonzalez-Moreno, Inés Losada López, Teresa Sevilla, Francisco Muñoz Beamud, José Eulalio Bárcena Llona, Manuel Romero Acebal, Patricia Tarilonte, Francesca Setaro, Javier Rejas-Gutierrez, Alejandra López-Ibáñez de Aldecoa, Carmen Peral","doi":"10.1080/14737167.2025.2527269","DOIUrl":"10.1080/14737167.2025.2527269","url":null,"abstract":"<p><strong>Objective: </strong>Variant transthyretin (ATTRv) amyloidosis is a rare genetic multisystem disease inducing progressive morbidity. Evidence about its economic burden is scarce. This study estimated annual use and costs of healthcare-resource-utilization (HRU) and indirect costs for ATTRv mutation carriers and patients with polyneuropathy from a societal perspective in Spain.</p><p><strong>Methods: </strong>A cross-sectional, retrospective non-interventional multicenter study was conducted in 2018-2020 to characterize asymptomatic carriers of mutations in the TTR gen (AC) and Coutinho stage 1 transthyretin amyloidosis polyneuropathy patients (PA). HRU, associated costs, and indirect wages were assessed during 12 months prior to enrollment.</p><p><strong>Results: </strong>One-hundred-five participants were analyzed: 86 AC and 19 PA. Average healthcare cost-per-person-per-year was significantly higher in PA: €2,323.6 vs. €953.3 (<i>p</i> < 0.001), due to higher utilization of medical specialty visits, hospitalizations, and nerve conduction studies. Indirect costs were statistically not different (€380.1 for AC and €781.3 for PA, <i>p</i> = 0.581). Days worked with fatigue/pain were significantly higher in PA: 53.9 vs 5.5 (<i>p</i> = 0.034). Total cost was significantly higher in PA than AC (€3,105.0 vs €1,333.4; <i>p</i> < 0.001).</p><p><strong>Conclusions: </strong>Both AC and early-stage ATTRv-PN patients showed a meaningful economic burden to the National Health System and society. We observed an annual incremental economic burden of €1,771.5 in PA versus AC.</p>","PeriodicalId":12244,"journal":{"name":"Expert Review of Pharmacoeconomics & Outcomes Research","volume":" ","pages":"1-9"},"PeriodicalIF":1.8,"publicationDate":"2025-07-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144559644","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}
Marzieh Nosrati, Hossein Vatankhah, Mahdi Asfia, Mohammad Hossein Sharifnia, Farhad Shahi, Mandana Hasanzad, Akbar Abdollahi Asl, Mohammad Abdollahi, Shekoufeh Nikfar
{"title":"Proposed method for economic evaluation based on basket trials: a case study of pembrolizumab.","authors":"Marzieh Nosrati, Hossein Vatankhah, Mahdi Asfia, Mohammad Hossein Sharifnia, Farhad Shahi, Mandana Hasanzad, Akbar Abdollahi Asl, Mohammad Abdollahi, Shekoufeh Nikfar","doi":"10.1080/14737167.2025.2526065","DOIUrl":"https://doi.org/10.1080/14737167.2025.2526065","url":null,"abstract":"<p><strong>Background: </strong>Precision medicine's emphasis on genetic profiles rather than diseases has reshaped oncology trials, prioritizing biomarker-specific approaches named basket trials. They involve heterogeneous populations, necessitating new economic evaluation methods, as traditional approaches fail to capture this diversity. This study introduced a methodology for economic evaluations based on basket trials, exemplified through pembrolizumab's cost-effectiveness analysis using Keynote-158 data as a case study and compared the results to disease-specific methods.</p><p><strong>Research design and methods: </strong>A regression-based approach was developed to aggregate costs and QALYs and calculate transition probabilities for economic evaluations in populations sharing a biomarker and various cancers. The proposed method involved five steps: designing a general model, selecting comparator arms, comparing Kaplan-Meier curves and conducting network analysis to evaluate treatment responses, calculating disease-specific coefficients to aggregate QALYs, and aggregating costs using weighted averages.</p><p><strong>Result: </strong>All the steps have been followed for conducting cost-effectiveness of Pembrolizumab in patients with dMMR/MSI-H biomarker. The results revealed significant differences in ICERs calculated by the proposed method versus disease-specific evaluations.</p><p><strong>Conclusion: </strong>This study introduces a biomarker-focused model for estimating ICERs across diverse diseases, enabling informed decision-making in healthcare resource allocation. Further research is recommended to broaden the model's applicability and address additional population heterogeneity factors.</p>","PeriodicalId":12244,"journal":{"name":"Expert Review of Pharmacoeconomics & Outcomes Research","volume":" ","pages":""},"PeriodicalIF":1.8,"publicationDate":"2025-07-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144567360","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}