{"title":"Protecting the patient in value-based pricing schemes.","authors":"Laura Levaggi, Rosella Levaggi","doi":"10.1080/14737167.2026.2719624","DOIUrl":"10.1080/14737167.2026.2719624","url":null,"abstract":"<p><strong>Background: </strong>Heterogeneity in patients' responses across and within patient groups is redesigning research and pricing policies, since a uniform price may not be able to capture differences in health benefits across indications. However, pricing mechanisms may create strategic behaviors that may reduce drug availability for some groups of patients.</p><p><strong>Method: </strong>In this narrative review we define a common framework to compare different forms of pricing policies that are currently used in the presence of heterogeneity in indication and patients' characteristics.</p><p><strong>Results: </strong>When heterogeneity derives from verifiable differences, Indication-value-Based Prices (IBP) may increase ex-post effectiveness, but they may reduce access. Furthermore, IBPs may be difficult to implement in practice. When uncertainty depends also on unverifiable patients' characteristics, performance-based price schemes are sub-optimal because they increase/reduce access according to how generous they are. Real-world evidence shows that operational complexity, high administrative costs, and information asymmetry frequently reduce their benefits.</p><p><strong>Conclusions: </strong>Personalized medicine opens new treatment possibilities but poses new challenges as concerns price strategies. Performance-based prices, which are the natural candidates to reimburse drugs, may reduce or delay drug availability across patients' groups. Regulators should enforce transparency, and designing multi-indication pricing architectures such as blended agreements that protect patient access.</p>","PeriodicalId":12244,"journal":{"name":"Expert Review of Pharmacoeconomics & Outcomes Research","volume":" ","pages":"1-7"},"PeriodicalIF":2.2,"publicationDate":"2026-09-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148765720","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}
Lane Beckes, Elias Assaf, Ryan McDade, Kali Sassack, Megan Winebrenner
{"title":"The impact of clinical pharmacy services within an oncology center of excellence on patient outcomes and cost efficiency.","authors":"Lane Beckes, Elias Assaf, Ryan McDade, Kali Sassack, Megan Winebrenner","doi":"10.1080/14737167.2026.2715981","DOIUrl":"10.1080/14737167.2026.2715981","url":null,"abstract":"<p><strong>Background: </strong>Evidence is mounting that clinical programs for specialty pharmacies can substantively contribute to improved treatment. As cancer is currently the second leading cause of death in the United States, clinical pharmacy programs may improve outcomes for millions of cancer patients. A center of excellence in outpatient specialty pharmacies may best leverage these benefits.</p><p><strong>Research design and methods: </strong>We observed the relationship between engaging with an insurance provider's affiliated pharmacy's remotely operated Oncology Center of Excellence (COE) and health outcomes and medical costs for Medicare Part-D beneficiaries. We then tested whether engagement with COE engagement mediated outcomes during a 12-month period.</p><p><strong>Results: </strong>Both propensity score matching and regression models indicated lower hospitalizations, emergency department (ED) visits, and medical costs for those who primarily used the affiliated pharmacy. Follow‑up mediation analyses indicated these effects were mediated by engagement with the COE.</p><p><strong>Conclusion: </strong>Engagement with this COE was associated with lower medical costs and fewer ED visits and hospitalizations. The high-quality clinical pharmacy programs and services associated with COE's likely contributed to better and less expensive oncology care overall.</p>","PeriodicalId":12244,"journal":{"name":"Expert Review of Pharmacoeconomics & Outcomes Research","volume":" ","pages":"1-9"},"PeriodicalIF":2.2,"publicationDate":"2026-08-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148711929","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":"Five-year durability in cell and gene therapy: evidence review and implications for payer coverage.","authors":"Elias C Pittos, Joe DePinto","doi":"10.1080/14737167.2026.2723931","DOIUrl":"https://doi.org/10.1080/14737167.2026.2723931","url":null,"abstract":"<p><strong>Introduction: </strong>Cell and gene therapies (CGTs) offer the potential for durable clinical benefit following a single administration. Although early regulatory approvals were often based on limited follow-up, an increasing number of U.S. Food and Drug Administration (FDA)-approved CGTs now report extended durability data from clinical trials, registries, and real-world evidence (RWE) sources.</p><p><strong>Areas covered: </strong>This report synthesizes publicly available long-term follow-up (LTFU) evidence for FDA-approved CGTs in the United States, with a focus on durability of clinical benefit and the implications for payer coverage and reimbursement. Long-term efficacy and safety data for marketed CGTs were identified through a review of peer-reviewed publications and publicly available conference abstracts and presentations. As of December 2025, 21 therapies (62%) report follow-up extending to 5 years or longer. The review examines the available durability evidence and explores its relevance to ongoing discussions around durability-related uncertainty and the role of LTFU and real-world evidence in assessing CGT outcomes.</p><p><strong>Expert opinion: </strong>Growing LTFU evidence suggests that durable clinical benefit can be achieved across multiple CGT platforms and disease settings. While uncertainties remain, accumulating data may help reduce durability-related uncertainty and inform payer coverage, reimbursement, and value assessment discussions.</p>","PeriodicalId":12244,"journal":{"name":"Expert Review of Pharmacoeconomics & Outcomes Research","volume":" ","pages":""},"PeriodicalIF":2.2,"publicationDate":"2026-08-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148864462","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}
Ivo Abraham, Mickael Hiligsmann, Kenneth K C Lee, Giorgio L Colombo, Leslie Citrome, Mike Gregg
{"title":"What to expect in 2026-2027: important health economics and outcomes research topics.","authors":"Ivo Abraham, Mickael Hiligsmann, Kenneth K C Lee, Giorgio L Colombo, Leslie Citrome, Mike Gregg","doi":"10.1080/14737167.2026.2692271","DOIUrl":"https://doi.org/10.1080/14737167.2026.2692271","url":null,"abstract":"","PeriodicalId":12244,"journal":{"name":"Expert Review of Pharmacoeconomics & Outcomes Research","volume":" ","pages":"1-7"},"PeriodicalIF":2.2,"publicationDate":"2026-08-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148817268","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":"The economic and societal burden associated with Post-COVID in the Netherlands: a cost-of-illness and quality of life study protocol.","authors":"Lieke Maas, Mickael Hiligsmann, Susanne Young, Marcia Spoelder, Marielle Goossens","doi":"10.1080/14737167.2026.2717292","DOIUrl":"10.1080/14737167.2026.2717292","url":null,"abstract":"<p><p>Post-COVID poses a substantial yet under-quantified burden on the Dutch healthcare system and labor market, lacking detailed data on societal costs and health-related quality of life (HRQOL). This study outlines a cross-sectional, prevalence-based, bottom-up design aimed at mapping individual patient costs and the impact of HRQOL within the Netherlands. Patient data will be further stratified according to context-specific factors such as disease severity and symptom clusters if sufficient data is available. The study collects individual resource-use, productivity and HRQOL data by self-report. Adult (≥18 years) respondents with Post-COVID are recruited nationally via patient platforms and the Post-COVID Network Netherlands (PCNN). The target sample for the quantitative component is ~400 respondents to provide robust descriptive cost and HRQOL estimates and exploratory subgroup descriptions. Resource use is measured primarily with the adapted versions of iMTA Medical Consumption Questionnaire (iMCQ) and iMTA Productivity Cost Questionnaire (iPCQ) and valued using Dutch reference unit prices. HRQOL is measured using the EQ-5D-5L, applying the Dutch tariff. The study results will describe the economic impact of Post-COVID, focusing on cost-of-illness (€) and HRQOL (utilities) in the Netherlands. The study follows national data-protection procedures and aims to inform policy, care organization and future economic evaluations.</p>","PeriodicalId":12244,"journal":{"name":"Expert Review of Pharmacoeconomics & Outcomes Research","volume":" ","pages":"1-5"},"PeriodicalIF":2.2,"publicationDate":"2026-08-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148790176","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":"From \"cluster imitation\" to \"first generic\": the impact of market exclusivity incentives on Chinese generic drug manufacturers.","authors":"Zhixue Liu, Xiaoqin Chen, Zonghua Wang, Yongfa Chen","doi":"10.1080/14737167.2026.2715978","DOIUrl":"https://doi.org/10.1080/14737167.2026.2715978","url":null,"abstract":"<p><strong>Background: </strong>China's generic drug industry has long suffered from 'clustering' and homogeneous competition, constraining structural upgrading. In 2021, the drug patent linkage system introduced a 12-month market exclusivity period for first-to-market generic drugs, representing a key institutional change aimed at incentivizing high-quality generic development.</p><p><strong>Research design and methods: </strong>Using panel data from 1,248 firm-year observations of A-share listed pharmaceutical companies (2015-2023), this study employs a difference-in-differences model comparing chemical generic drug manufacturers (treatment group) with other pharmaceutical sub-sectors (control group) to estimate the net policy effect.</p><p><strong>Results: </strong>The market exclusivity incentive significantly increased R&D intensity (by approximately 1.2% points, p < 0.01) and patent applications (by 18.5%, p < 0.05) in the treatment group. R&D human capital positively moderates the policy effect. Non-state-owned enterprises and firms in highly competitive sub-sectors exhibit stronger responsiveness.</p><p><strong>Conclusions: </strong>The market exclusivity period is positively associated with increased innovation inputs and outputs among Chinese generic drug manufacturers. However, given the short post-policy observation period and concurrent pharmaceutical reforms, these findings should be interpreted cautiously regarding broad causal claims about industry transformation.</p>","PeriodicalId":12244,"journal":{"name":"Expert Review of Pharmacoeconomics & Outcomes Research","volume":" ","pages":""},"PeriodicalIF":2.2,"publicationDate":"2026-08-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148808491","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-neutral adoption of anti-VEGF therapies in neovascular age-related macular degeneration.","authors":"Olga Pitsillidou, Panagiotis Petrou, M J Postma","doi":"10.1080/14737167.2026.2717293","DOIUrl":"10.1080/14737167.2026.2717293","url":null,"abstract":"<p><strong>Objective: </strong>In therapeutic classes characterized by outcome equivalence, the decision problem shifts from incremental cost-effectiveness to cost-neutral pricing. This study formalizes a break-even pricing framework, translating real-world utilization and costs into cost-neutral price thresholds, using anti-VEGF therapies for neovascular age-related macular degeneration (nAMD) as a case study.</p><p><strong>Methods: </strong>A cost-minimization framework was applied from a public payer perspective in a single-payer system (Cyprus). Five anti-VEGF agents were evaluated over 3 years with 3.5% discounting. Injection frequencies were derived as weighted averages of treat-and-extend and pro re nata regimens from Phase 3/4 trials, with year 3 extrapolated from year 2. Break-even prices were defined as unit prices yielding equal discounted per-patient costs versus comparators. First-line thresholds used bevacizumab; second-line thresholds used a utilization-weighted comparator. Sensitivity analyses were conducted.</p><p><strong>Results: </strong>Bevacizumab defined the lowest-cost benchmark (€4,130). Cost-neutral first-line use of newer agents required price reductions of 84-98%. Under second-line utilization, several agents fell below break-even thresholds at list prices. Key drivers were acquisition price, injection frequency, and monitoring costs.</p><p><strong>Conclusion: </strong>Break-even pricing generates explicit cost-neutral thresholds under outcome equivalence. While jurisdiction-specific, the framework is transferable and supports consistent pricing decisions in single-payer systems.</p>","PeriodicalId":12244,"journal":{"name":"Expert Review of Pharmacoeconomics & Outcomes Research","volume":" ","pages":"1-6"},"PeriodicalIF":2.2,"publicationDate":"2026-08-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148790161","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}
Li-Wei Wu, Minh-Anh Le-Dang, Blessing Ijeoma Okoye, Chanhyun Park
{"title":"Machine learning-based prediction of cardiovascular adverse events in patients with cancer: a systematic review.","authors":"Li-Wei Wu, Minh-Anh Le-Dang, Blessing Ijeoma Okoye, Chanhyun Park","doi":"10.1080/14737167.2026.2719628","DOIUrl":"10.1080/14737167.2026.2719628","url":null,"abstract":"<p><strong>Introduction: </strong>Cardiovascular (CV) adverse events are increasingly recognized in patients with cancer. Previous reviews of AI/ML have focused on single cancer types, imaging-based data, and lacked evaluation of methodological rigor. This systematic review synthesized AI/ML models developed to predict CV adverse events from patient-level clinical data across diverse cancer populations.</p><p><strong>Methods: </strong>This review followed the PRISMA 2020 guidelines. PubMed and Web of Science were searched through 26 October 2025. Study characteristics, model development, and handling of features and missing data were extracted. Study quality was assessed using the IJMEDI checklist.</p><p><strong>Results: </strong>Of 32 included studies, 18 compared multiple algorithms and 14 used a single algorithm. Random forest and XGBoost were the most common methods (<i>n</i> = 17, respectively), and XGBoost was most often the best-performing model in multi-algorithm studies, although substantial study heterogeneity precludes concluding general algorithmic superiority. Common limitations were unreported missing data handling (<i>n</i> = 17), limited external validation (<i>n</i> = 8), and rare calibration assessment (<i>n</i> = 4). Most studies were rated medium quality (<i>n</i> = 28).</p><p><strong>Conclusions: </strong>AI/ML models show promise for predicting CV adverse events in patients with cancer; however, clinical applicability is constrained by insufficient preprocessing transparency, limited external validation, and inadequate calibration reporting.</p>","PeriodicalId":12244,"journal":{"name":"Expert Review of Pharmacoeconomics & Outcomes Research","volume":" ","pages":"1-18"},"PeriodicalIF":2.2,"publicationDate":"2026-08-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148758861","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}
Chi Chun Steve Tsang, Junling Wang, Yuan Zhang, Ashley Ellis
{"title":"Impact of the inflation reduction act on Medicare MTM eligibility among beneficiaries with diabetes: evidence from 2022-2023.","authors":"Chi Chun Steve Tsang, Junling Wang, Yuan Zhang, Ashley Ellis","doi":"10.1080/14737167.2026.2717290","DOIUrl":"https://doi.org/10.1080/14737167.2026.2717290","url":null,"abstract":"<p><strong>Background: </strong>The Inflation Reduction Act (IRA) introduced an insulin cost cap in 2023 to reduce patient out-of-pocket costs, but these changes may have unintended implications for drug-cost-based eligibility for the Medicare Medication Therapy Management (MTM) program. MTM services, intended to improve pharmacotherapy outcomes, had lower enrollment among racial and ethnic minorities. This study evaluated whether the IRA was associated with differential changes in MTM eligibility across racial and ethnic groups.</p><p><strong>Research design and methods: </strong>Data from the 2022-2023 Medical Expenditure Panel Survey were analyzed. Medicare beneficiaries with diabetes were compared with privately insured near-elderly adults using a difference-in-differences framework. Changes in rates of exceeding the drug cost threshold for MTM eligibility were compared by race and ethnicity.</p><p><strong>Results: </strong>The temporal increases in these rates were significantly smaller among Medicare beneficiaries than in the comparison group for non-Hispanic White (White) individuals. Hispanic and non-Hispanic Asian beneficiaries experienced greater increases in these rates than their White counterparts. No significant differential change was observed between non-Hispanic Black and White beneficiaries.</p><p><strong>Conclusions: </strong>Initial IRA implementation was associated with narrowing Hispanic-White and Asian-White differences in meeting the drug-cost threshold for MTM eligibility. Part D affordability reforms may influence cost-based program eligibility, with equity implications.</p>","PeriodicalId":12244,"journal":{"name":"Expert Review of Pharmacoeconomics & Outcomes Research","volume":" ","pages":""},"PeriodicalIF":2.2,"publicationDate":"2026-08-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148790159","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}
Igor Rubinić, Robert Likić, Marina Paladin, Nataša Skočibušić, Ines Mujčin, Ivana Mikolašević, Vera Vlahović-Palčevski
{"title":"Institutional and national budget impact of alternative pembrolizumab dosing in non-small cell lung cancer: a real-world pharmacoeconomic analysis.","authors":"Igor Rubinić, Robert Likić, Marina Paladin, Nataša Skočibušić, Ines Mujčin, Ivana Mikolašević, Vera Vlahović-Palčevski","doi":"10.1080/14737167.2026.2717291","DOIUrl":"https://doi.org/10.1080/14737167.2026.2717291","url":null,"abstract":"<p><strong>Background: </strong>Pembrolizumab is among the costliest anticancer therapies; fixed dosing may cause drug wastage, particularly in lower-weight patients, adding to health-system budgetary pressure.</p><p><strong>Research design and methods: </strong>This retrospective, single-center pharmacoeconomic analysis compared standard flat-dose pembrolizumab (200 mg every three weeks [Q3W] or 400 mg every six weeks [Q6W]) with a simulated weight-banded strategy (<65 kg, 65-90 kg, ≥90 kg) in adults with advanced non-oncogene-addicted non-small cell lung cancer treated at the Clinical Hospital Centre Rijeka, Croatia, during 2024. Institutional and national public-payer budget impact was modeled using list drug-acquisition prices, with one-way and probabilistic sensitivity analyses.</p><p><strong>Results: </strong>Ninety-six patients received pembrolizumab during the study period. Weight-banded dosing reduced institutional drug consumption by 25%, yielding annual savings exceeding €1.1 million. National extrapolation, assuming Rijeka accounts for 10-30% of national pembrolizumab use, projected savings of €3.7-11.2 million annually.</p><p><strong>Conclusions: </strong>Weight-banded pembrolizumab dosing may reduce drug expenditure without evidence of compromised outcomes in this economic model; prospective clinical validation and consideration of pharmacodynamic, safety, and regulatory implications are warranted before implementation.</p>","PeriodicalId":12244,"journal":{"name":"Expert Review of Pharmacoeconomics & Outcomes Research","volume":" ","pages":"1-9"},"PeriodicalIF":2.2,"publicationDate":"2026-08-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148790194","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}