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}
{"title":"Cost-effectiveness analysis of Risdiplam and Nusinersen for the treatment of Chinese patients with spinal muscular atrophy.","authors":"Yueyang Huang, Hongmei Yuan, Zhe Huang","doi":"10.1080/14737167.2025.2499720","DOIUrl":"10.1080/14737167.2025.2499720","url":null,"abstract":"<p><strong>Background: </strong>As a rare disease with the largest number of cases in China, spinal muscular atrophy (SMA) causes severe motor deficits as well as multi-system organ damage. Some potent drugs for SMA, such as Nusinersen (NU) and Risdiplam (RI) represented by targeted survival motor neuron gene therapy, have become a new trend in the treatment of SMA. RI has gradually attracted attention due to its ease of administration, safety, and efficacy.</p><p><strong>Objective: </strong>The study was to analyze the cost-effectiveness of RI and NU for the treatment of SMA in China from the perspective of the healthcare system.</p><p><strong>Methods: </strong>We constructed a five-state Markov model based on the different stages of SMA. The willingness-to-pay threshold was chosen to be 1-3 times the gross domestic product (GDP) per capita. The base-case analysis was used to calculate the incremental cost-effectiveness ratio (ICER), and one-way and probabilistic sensitivity analyses were performed.</p><p><strong>Results: </strong>The ICER value was ¥132,402.10/QALY, which was in the range of 1-3 times GDP per capita. Sensitivity analysis results showed the cost of RI and NU had a significant effect on ICER as well as proving the stability of results.</p><p><strong>Conclusions: </strong>RI is a cost-effective option compared to NU in SMA treatment.</p>","PeriodicalId":12244,"journal":{"name":"Expert Review of Pharmacoeconomics & Outcomes Research","volume":" ","pages":"943-953"},"PeriodicalIF":1.8,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143999774","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}
Max Lelie, Riet Parmentier, Eva de Jaegere, Steven Simoens, Lieven Annemans, Koen Putman
{"title":"A cost-effectiveness estimation of the Attempted Suicide Short Intervention Programme (ASSIP).","authors":"Max Lelie, Riet Parmentier, Eva de Jaegere, Steven Simoens, Lieven Annemans, Koen Putman","doi":"10.1080/14737167.2025.2497877","DOIUrl":"10.1080/14737167.2025.2497877","url":null,"abstract":"<p><strong>Objective: </strong>Patients who have previously attempted suicide are at a substantially increased risk of a repeated attempt. We have conducted a cost-utility analysis of the Attempted Suicide Short Intervention Programme (ASSIP) and compared it with treatment as usual in a Flemish population.</p><p><strong>Methods: </strong>A closed-cohort Markov-model was used to simulate suicide over a time-horizon of 20 years in a cohort of prior suicide attempt patients. This model is used to estimate Incremental Cost Effectiveness Ratios (ICERs) from societal and healthcare perspectives. A separate 'tipping-point' scenario was included, where the treatment effectiveness regresses over time.</p><p><strong>Results: </strong>ASSIP is shown to be a dominant strategy from the societal perspective and cost-effective from a healthcare perspective: the ICER after 10 years is € 1,133 and after 20 years € 304. In the tipping-point scenario, an regression of up to 82,7% after the intervention remains cost-effective, assuming an ICER threshold of € 44000 per QALY.</p><p><strong>Conclusion: </strong>Our study found that ASSIP is cost-effective in the Flemish region, saving both healthcare costs and societal expenses over time. Implementing ASSIP could provide significant economic and health benefits within 10 years, making it a valuable investment for improving mental health care.</p>","PeriodicalId":12244,"journal":{"name":"Expert Review of Pharmacoeconomics & Outcomes Research","volume":" ","pages":"897-904"},"PeriodicalIF":1.8,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143975713","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}
Faizur Rehman, Muhammad Wasay Shahid, Mehran Riaz, Malik Muhammad Umair, Farah Azhar, Muhammad Amer, Ali Ahmed
{"title":"Willingness to pay for a quality-adjusted life year (QALY) in Pakistan: implications for health policy.","authors":"Faizur Rehman, Muhammad Wasay Shahid, Mehran Riaz, Malik Muhammad Umair, Farah Azhar, Muhammad Amer, Ali Ahmed","doi":"10.1080/14737167.2025.2504945","DOIUrl":"10.1080/14737167.2025.2504945","url":null,"abstract":"<p><strong>Objectives: </strong>Allocating healthcare resources in developing countries like Pakistan is constrained by economic limitations and uneven distribution. Therefore, in this study, we aimed to investigate the Willingness to Pay (WTP) for one additional Quality-Adjusted Life Year (QALY) among the general population in Pakistan to establish contextually relevant thresholds for health technology assessment (HTA).</p><p><strong>Methods: </strong>We conducted a cross-sectional survey using the convenient sampling technique to estimate the WTP for one additional QALY among the general population of Pakistan. The contingent valuation method (CVM) using the payment card technique was used to assess its monetary value.</p><p><strong>Results: </strong>A total of 600 participants participated in the survey and resulted in 1200 WTP responses for further analysis. The mean WTP/QALY was 114,006.4 Pakistani rupee (PKR) (United States Dollar 410.11), equivalent to 0.29 times Pakistan's GDP per capita. The WTP/QALY for the quality-of-life improvement scenario was lower than the life-extension scenario. The two-part regression model showed that higher education and income were positively associated with WTP value.</p><p><strong>Conclusion: </strong>This study provides empirical evidence of the monetary value of one additional QALY from a sample of the Pakistani population. These findings highlight country-specific cost-effectiveness benchmarks, and incorporating WTP insights into the healthcare policy for better resource allocation, affordability, and long-term sustainability of the interventions.</p>","PeriodicalId":12244,"journal":{"name":"Expert Review of Pharmacoeconomics & Outcomes Research","volume":" ","pages":"967-975"},"PeriodicalIF":1.8,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143989824","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}