PharmacoEconomics Open最新文献

{"title":"Retractions of Health Economic Evaluations: A Systematic Review.","authors":"Eric Julian Manalastas, Aditi Hombali, Juliette C Thompson, David Alexander Scott","doi":"10.1007/s41669-025-00624-9","DOIUrl":"10.1007/s41669-025-00624-9","url":null,"abstract":"<p><strong>Objectives: </strong>Retractions of scientific articles have increased over time, across different health fields, disease indications, and study designs. However, it is currently unknown whether, and to what extent, retractions have impacted the literature of health economic evaluations. This research aimed to identify retracted health economic evaluations, describe the characteristics of such studies, and analyse reasons for retraction.</p><p><strong>Methods: </strong>We conducted a systematic review of health economic evaluations published in peer-reviewed journals and subsequently retracted, identified using MEDLINE, Embase, and the Retraction Watch database from inception to May 2025. Retraction notices were examined and publication details, including reasons for retraction, were extracted.</p><p><strong>Results: </strong>We identified 17 retracted economic evaluations published from 2006 to 2024. Studies evaluated a range of interventions in a broad array of disease indications. Retracted economic evaluations were published in 17 unique journals, 11 of which were top-tier outlets. Errors were the most common reason for retraction (11/17 studies, 64%), including errors in inputs, analysis, and/or model structure. Evidence of misconduct (plagiarism, duplicate publication, and peer-review manipulation) was found in 4/17 studies (24%). Retractions were rapid, with 12/17 studies (71%) retracted within the same year. Mean citation count was 4.8; the highest was 33. Despite retraction, studies were included in subsequent evidence reviews (6/17 studies, 35%) and one was used to inform clinical practice recommendations (1/17 studies, 6%).</p><p><strong>Conclusion: </strong>Evidence for retractions of health economic evaluations was found, spanning a variety of disease indications, interventions, and journal sources, due to errors or scientific misconduct. In some cases, papers produced subsequent citations in the literature despite retraction.</p><p><strong>Trial registration: </strong>OSF https://osf.io/8c6jb/?view_only=0ed118b9830e45058e56fc333b93a62f .</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"215-227"},"PeriodicalIF":2.4,"publicationDate":"2026-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13000055/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145844123","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Potential Economic Value of Multi-cancer Early Detection Testing Under Differential Cost Trends for Cancer Screening and Management.","authors":"Anuraag R Kansal, Ali Tafazzoli, Weicheng Ye, Walter Morris, Denise Zou, Bruce Quinn, A Mark Fendrick","doi":"10.1007/s41669-025-00626-7","DOIUrl":"10.1007/s41669-025-00626-7","url":null,"abstract":"<p><strong>Objectives: </strong>Multi-cancer early detection (MCED) tests aim to reduce the clinical and economic burden of cancer by detecting cases at earlier, more treatable stages. Recent trends indicate that the costs of cancer management are escalating, while screening costs have remained relatively stable over the past decade. This study evaluated the impact of these differing cost trends on the cost-effectiveness and net cost of MCED testing in the United States (US).</p><p><strong>Methods: </strong>A hybrid state-transition and decision-tree model was adapted for a lifetime analysis from commercial insurance and Medicare perspectives. Base-case treatment costs were derived from a Surveillance, Epidemiology, and End Results (SEER)-Medicare database study, and MCED test prices were set at $949 or $712 (25% reduction). Historical annual growth rates ranging from - 5 to 10% were then applied to management and screening costs. Contour plots were used to depict the results from these cost growth scenarios. MCED test performance was informed based on results from a case-control study.</p><p><strong>Results: </strong>Across perspectives, the incremental cost-effectiveness ratio (ICER) of MCED was between $50,000 and $100,000/quality-adjusted life year (QALY) with no cost growth. In the commercial insurance perspective, MCED was found to gain QALYs and lower spending when the growth rate of cancer management costs exceeded 2-4% with no MCED cost growth. The ICER exceeded $150,000/QALY if the growth rate of MCED costs exceeded 4-6% with no cancer management cost growth. In Medicare, the same thresholds were reached with management growth rates of 7-8% and screening growth rates > 7%, respectively. Cost neutrality was achieved within 5-10 years when cancer management growth rates significantly exceeded MCED cost growth, but was not reached within 30 years if MCED cost growth rates exceed those of cancer management. Results were robust across conducted scenario and sensitivity analyses.</p><p><strong>Conclusions: </strong>Early detection with MCED may help mitigate rising cancer management costs, yielding improved cost-effectiveness and, in some scenarios, a path to cost neutrality.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"275-287"},"PeriodicalIF":2.4,"publicationDate":"2026-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13000096/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145820240","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Global Assessment of Health Utilities Associated with Pneumococcal Disease in Adults: Targeted Literature Reviews.","authors":"Min Huang, Jipan Xie, Hela Romdhani, Yan Song, Sun Lee, Daisy Liu, Elamin Elbasha, Salini Mohanty, Donna Rowen, Matthew S Kelly","doi":"10.1007/s41669-025-00608-9","DOIUrl":"10.1007/s41669-025-00608-9","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Background: &lt;/strong&gt;Pneumococcal disease can significantly reduce quality of life (QoL). Health utilities are commonly used to assess QoL burden and calculate quality-adjusted life years (QALYs), which are critical inputs in cost-utility analyses (CUAs) to evaluate the economic value of pneumococcal vaccines.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Objectives: &lt;/strong&gt;This study aimed to synthesize utility values used in CUAs of pneumococcal vaccines in adult populations in North America and Europe and to summarize global utility studies on pneumococcal disease and post-meningitis sequelae (PMS) in adults.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Methods: &lt;/strong&gt;Two targeted literature reviews were conducted in June 2024 using MEDLINE to identify CUAs of pneumococcal vaccines and original studies on health utilities for pneumococcal disease and PMS in adults. References from existing literature reviews on QoL associated with pneumococcal disease and CUAs of pneumococcal vaccines were also screened to identify additional original utility studies. Health utility input values applied in the CUAs were summarized, and the original source studies were reviewed. Utility and disutility inputs were converted to QALY decrements using disease durations reported in the CUAs. Methods and utility estimates from original utility studies were summarized for each health state.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;Sixty-five CUAs focusing on adults in North America and Europe were identified. These CUAs frequently reported utility values and QALY decrements. The ranges of reported and derived QALY decrements were 0.006-0.8 for unspecified invasive pneumococcal disease (IPD), 0.0013-0.1759 for meningitis, 0.0010-0.1759 for non-meningitis IPD, 0.0002-0.13 for inpatient pneumonia, and 0.0001-0.0115 for outpatient pneumonia. The ranges of reported utility values for PMS were 0.319-0.83 for neurological deficits and 0.635-0.91 for hearing loss. Twenty-five original utility studies in adults were identified, published between 1993 and 2022. Utility estimates from these studies showed considerable variability, with ranges of - 0.330 to 0.39 for meningitis, - 0.331 to 0.93 for non-meningitis IPD, - 0.054 to 0.71 for inpatient pneumonia, 0.35-0.825 for outpatient pneumonia, 0.217-0.89 for neurological deficits, and 0.433-0.89 for hearing loss. This variability was primarily attributable to differences in case definitions, preference weights, and geographical locations.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Conclusions: &lt;/strong&gt;This review identified substantial QoL burden associated with pneumococcal disease and PMS in adults; however, considerable variability in utility estimates exists. Utility inputs in CUAs of pneumococcal vaccines in adults often rely upon assumptions, pediatric studies, or utilities from other diseases, and there is relatively little data on the health utilities of IPD and neurological deficits in adults. Given the importance of health utilities in economic evaluations, CUAs should carefully select utility values b","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"5-33"},"PeriodicalIF":2.1,"publicationDate":"2026-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12796033/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145715075","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Cost of Chronic Heart Failure Among Adult Europeans-A Systematic Literature Review.","authors":"Alexandra Mincă, Claudiu C Popescu, Dragoş I Mincă, Amalia L Călinoiu, Adina Rusu, Ana Ciobanu, Valeriu Gheorghiţă, Dana G Mincă","doi":"10.1007/s41669-025-00613-y","DOIUrl":"10.1007/s41669-025-00613-y","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Background: &lt;/strong&gt;Chronic heart failure (CHF) imposes a substantial and growing burden on healthcare systems. While individual studies have evaluated the cost of CHF, variability in reporting standards and regional data gaps limit the comparability of findings.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Objective: &lt;/strong&gt;This systematic literature review and exploratory meta-analysis aimed to synthesize current evidence on the total annual per-patient cost of CHF in Europe.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Methods: &lt;/strong&gt;Eligibility Criteria. The review included original studies, published in English, on human adult patients with a diagnosis of CHF from Europe, evaluating its economic burden. Studies were excluded if costs were not expressed in monetary currency, if costs represented fractions of the total cost, or if cost analysis focused either on a specific element of CHF management or intervention or patient subgroup.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Information sources: &lt;/strong&gt;The review was conducted across PubMed, Embase, Scopus, Web of Science, and the Cochrane Library from January 2008 to March 2025. Risk of Bias. Newcastle-Ottawa scale (NOS) grading was used to assess risk of bias.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Synthesis of results: &lt;/strong&gt;Costs were adjusted to 2024 euro (€) values using the Harmonised Index of Consumer Prices. An exploratory random-effects meta-analysis was performed to pool mean annual costs, and risk of bias was assessed using validated tools.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;Included Studies. In total, 19 studies met inclusion criteria.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Synthesis of results: &lt;/strong&gt;Across the included studies, the adjusted mean annual total cost of CHF per patient ranged from 3452 € to 23,335 €, reflecting considerable variation in cost estimates across different healthcare systems and study designs. Most studies reported hospitalization as the main cost driver, typically accounting for over half of total direct medical expenditures; 13 studies reported the proportion of total annual cost explained by CHF hospitalization, with a mean of 64.9% ± 18.3%. NOS grading did not exclude any study on the basis of risk of bias alone; from the 19 included studies, only 1 was rated as low risk of bias (NOS = 8), while the majority (16 studies) were evaluated to have moderate risk of bias.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Discussion: &lt;/strong&gt;Limitations of Evidence. Substantial heterogeneity (I&lt;sup&gt;2&lt;/sup&gt; &gt; 99%) was present across included studies, reflecting differences in healthcare settings, costing methods, and population characteristics. Evidence of potential publication bias was identified, suggesting that lower-cost studies may be underrepresented. Variability in cost components, perspectives (healthcare system versus societal) and geographical representation further limited the precision of the pooled estimate. Future research should prioritize standardized cost reporting and include underrepresented regions to support more accurate economic evaluations and inform health policy decisions.&lt;/p&gt;&lt;p&gt;&lt;s","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"53-69"},"PeriodicalIF":2.1,"publicationDate":"2026-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12796061/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145506208","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Health Utility Decrement of Injection Treatment-Related Attributes Using Time Trade-Off Among Type 2 Diabetes Patients: A Vignette-Based Study.","authors":"Xinran Liu, Chang Luo, Shitong Xie, Jing Wu","doi":"10.1007/s41669-025-00615-w","DOIUrl":"10.1007/s41669-025-00615-w","url":null,"abstract":"<p><strong>Objectives: </strong>To identify health utility decrements of injection treatment-related attributes among patients with type 2 diabetes mellitus (T2DM) in China.</p><p><strong>Methods: </strong>Health states of four attributes (hypoglycemia, dose frequency, flexibility and injection site reaction) were generated using a vignette-based method. Patients with T2DM were recruited from eight cities in China. The sample was broadly consistent with Chinese T2DM population with regard to age and sex distribution. Respondents completed seven time trade-off (TTO) tasks during face-to-face interviews. The ordinary least square (OLS), fixed effects (FE) and random effects (RE) models were used for TTO data. In subgroup analysis, groups were categorized based on whether injection treatment was currently used, number of medications, needle phobia, duration of injectable treatment and travel frequency.</p><p><strong>Results: </strong>A total of 400 patients (52.75% male, mean [SD] age 50.30 [12.05] years) were included in this study. Severe hypoglycemia had the largest disutility value of all attributes (-0.023, P < 0.001). Three times daily, twice daily and once daily injection (needed to be carried with the patient on short trips) were associated with -0.023 (P < 0.001), -0.018 (P < 0.001) and -0.011 (P = 0.022) disutility values compared with once weekly injection (not required to be carried with the patient on short trips), respectively. The disutility value associated with injection site reaction attribute was -0.013 (P < 0.001). In subgroup analysis, the relative importance of treatment-related attributes was found to depend on patient characteristics.</p><p><strong>Conclusions: </strong>This study provides disutility values associated with several injection treatment-related attributes for Chinese patients with T2DM. Hypoglycemia appears to be the most important attribute, followed by dose frequency, flexibility and injection site reaction.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"167-178"},"PeriodicalIF":2.1,"publicationDate":"2026-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12796039/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145471611","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Estimation of Health Utility Values for Eosinophilic Granulomatosis With Polyangiitis.","authors":"Bhovina Buguth, Jacqui Bernarde, Daniel Aggio, Arya Pimprikar, Michael Watt, Lotte Westerink, Josefine Persson, Andrew Lloyd","doi":"10.1007/s41669-025-00620-z","DOIUrl":"10.1007/s41669-025-00620-z","url":null,"abstract":"<p><strong>Background: </strong>Eosinophilic granulomatosis with polyangiitis (EGPA) is a rare autoimmune disease, characterised by inflammation of small- to medium-sized blood vessels, however, it remains poorly described in the literature. The purpose of this study was to characterise EGPA health states and to estimate utility weights for each to inform economic evaluations. To achieve this, we aimed to characterise and develop health-state vignettes representing EGPA and to estimate utility values from the United Kingdom (UK) public and individuals from the UK with EGPA.</p><p><strong>Methods: </strong>Vignettes were developed using evidence from a literature review and patient-reported outcomes (PRO) data. Qualitative interviews with patients (n = 7), clinicians (n = 4), and one health-related quality of life (HRQoL) expert were conducted to refine the content of the vignettes. To derive utility values, vignettes were assessed using an online survey by members of the UK public (N = 316) and patients with EGPA (N = 20). Participants evaluated each vignette by imagining themselves in the described vignettes and completed the EQ-5D-5L for each vignette.</p><p><strong>Results: </strong>Based on the literature review and PRO data from a clinical trial and real-world study, four health-state vignettes for EGPA were defined: remission, relapse, non-refractory, and refractory, which clinicians recognised as clinically meaningful. These vignettes captured the physical, psychological, and functional impacts of EGPA across health states. The subsequent survey was completed by 316 members of the UK public, and 20 patients with EGPA. Utility scores for remission (public: 0.675; patients: 0.690) scored highest, followed by non-refractory (public: 0.619; patients: 0.621), relapse (public: 0.453; patients: 0.410), and refractory (public: 0.151; patients: 0.059). EGPA patients' scores were largely consistent with those of the public, except for the refractory state, where patients reported lower scores.</p><p><strong>Conclusions: </strong>The findings highlight the substantial HRQoL impact of EGPA across all health states, with a higher perceived impact for the relapse and refractory health states. The public and EGPA patient samples produced consistent ratings. This study addresses an evidence gap by characterising health states in EGPA and defining the HRQoL of patients with EGPA across these health states. Multiple evidence sources were integrated to develop these values, which we believe represents a robust approach to estimating health state utilities in a rare disease.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"197-207"},"PeriodicalIF":2.1,"publicationDate":"2026-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12796046/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145541737","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Individual Preference for Fecal Immunochemical Test Options among Younger Adults: A Discrete Choice Experiment.","authors":"Kailu Wang, Ho-Man Shum, Carrie Ho-Kwan Yam, Shangfeng Tang, Chunyan Li, Yushan Wu, Eliza Lai-Yi Wong, Eng-Kiong Yeoh","doi":"10.1007/s41669-025-00598-8","DOIUrl":"10.1007/s41669-025-00598-8","url":null,"abstract":"<p><strong>Objectives: </strong>This study aims to quantify the preference of adults below 50 years of age for fecal immunochemical test (FIT) options as screening for colorectal cancer (CRC) and its disparities across different subgroups.</p><p><strong>Methods: </strong>A discrete choice experiment (DCE) was conducted among adults aged < 50 years old in Hong Kong. A mixed logit model and latent class model were used to estimate their preference, taking into account their preference heterogeneity. Marginal willingness-to-pay for changes in the attribute levels was also estimated.</p><p><strong>Results: </strong>A total of 408 participants with valid responses were included for analysis. The outcomes showed 5.2% of participants would consistently reject all the 20 FIT alternatives presented to them in the DCE survey. Mortality reduction in FIT programs was deemed as the most important attribute, followed by testing frequency, early detection likelihood, familiar physician, and reduction of false positive rate. The FIT acceptance was sensitive to the change of out-of-pocket payment amount. The latent class model showed participants with different ages, educational level, and self-reported health status would have different preferences for FIT attribute levels.</p><p><strong>Conclusions: </strong>The study showed that most adults below 50 years old would be willing to consider receiving FIT if suitable testing options are available. They are more sensitive to changes in mortality reduction than changes in CRC early detection likelihood, suggesting the former is more important in information dissemination. The impact of out-of-pocket payments for FIT on its acceptance suggests that the introduction of financial subsidies can be considered to improve screening acceptance. The preference heterogeneity highlights the importance of healthcare professionals' understanding of people's values and preferences for decision-making.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"109-120"},"PeriodicalIF":2.1,"publicationDate":"2026-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12796079/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144992902","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Estimating the Value of Adding 30 Days to the 180-Day Market Exclusivity of the First-to-File Generic Drug Manufacturer.","authors":"Aylin Sertkaya, Zeid El-Kilani, Sean Klein, Andreas Lord, Ruben Jacobo-Rubio, Sonal Parasrampuria","doi":"10.1007/s41669-025-00607-w","DOIUrl":"10.1007/s41669-025-00607-w","url":null,"abstract":"<p><strong>Objectives: </strong>As an additional incentive to bringing more generic drugs to more markets sooner, our aim was to estimate the value of adding 30 days to the 180 days of market exclusivity currently granted to a qualifying first-to-file (FTF) generic drug entrant after its manufacturer successfully challenges a brand drug's patents.</p><p><strong>Methods: </strong>Using IQVIA's monthly data on drug sales in the U.S. from January 2014 through June 2021, we identified 37 generic drug markets in which one or more generic companies successfully challenged the relevant brand drug patents and began commercial marketing within 75 days of receiving final approval of their Abbreviated New Drug Application (ANDA). We compared each generic drug's sales during the last month of its 180-day exclusive market access (Month 6) with its sales during the first month after their exclusivity expired (Month 7). Our calculation of this change in sales accounted for confounding factors such as the presence of an authorized generic in the market during the exclusivity period and the number of new generic entrants in Month 7. This calculated change represents the value to the FTF manufacturer of extending the 180-day market exclusivity to 210 days.</p><p><strong>Results: </strong>Our analysis shows that an FTF generic experiences a 13.0% reduction in sales from Month 6 to Month 7, on average. Extending the 180-day exclusivity by 30 days could lower the magnitude of this reduction to 3.1%, resulting in a 9.8% gain in sales by the FTF generic. This gain represents about $870,000 in additional sales during Month 7, on average.</p><p><strong>Conclusions: </strong>The value of an additional month of exclusivity to a generic firm is sizable and could entice more generic companies on the margin to take on the risk of patent challenges, especially in large markets.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"143-152"},"PeriodicalIF":2.1,"publicationDate":"2026-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12796020/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145150472","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Model-Based Economic Analyses of Haemophilia from a Societal Perspective: A Scoping Review.","authors":"Amr A El-Sayed, Nancy S Bolous","doi":"10.1007/s41669-025-00618-7","DOIUrl":"10.1007/s41669-025-00618-7","url":null,"abstract":"<p><strong>Introduction: </strong>Haemophilia imposes substantial lifelong humanistic and economic burdens, necessitating economic evaluations that adopt a societal perspective and comprehensively capture all relevant costs. Given the rarity of haemophilia and the limitations in data availability, modelling is crucial for integrating data from diverse sources to construct hypothetical cohorts. This review aims to identify and summarise model-based economic studies conducted in haemophilia from a societal perspective.</p><p><strong>Methods: </strong>A systematic search was conducted across eight databases, grey literature, and manual searches up to January 5th, 2024. Eligible studies included English-language full-text publications of economic studies employing modelling techniques from a societal perspective with no date limits. The reported cost values from the included studies were adjusted to the international dollar (I$) and the United States dollar (US$) in 2022.</p><p><strong>Results: </strong>Out of 3340 unique records, 26 studies from 20 countries met the inclusion criteria. Among these, 17 (65.4%) were full economic evaluations, while the remaining nine (34.6%) comprised partial economic evaluations and cost analyses. Most studies (n = 20) focused on haemophilia A, with or without inhibitors. Nine studies compared prophylactic to episodic treatment. Reporting of model characteristics was frequently incomplete. Specifically, 17.6% (n = 3) and 35.3% (n = 6) of the 17 full economic evaluation studies did not report the number of health states and the cycle length, respectively. Additionally, among all studies, four (15.4%) did not perform sensitivity analysis. Key input parameters were also underreported by relevant studies, including the dose, frequency, and consumption of haemostatic therapies (19/24), unit cost of the haemostatic therapy (16/24), average body weight of the study population (10/25), the number and unit cost of days off (14/26), and the methodology for estimating indirect costs (10/26). All studies reported direct medical costs; however, only 46% reported direct formal non-medical costs, 15% reported direct informal non-medical costs, and one study included intangible costs. Indirect costs were reported in all studies. The results generally supported the cost-effectiveness or dominance of prophylaxis over episodic treatment, emicizumab over recombinant activated factor VII (rFVIIa), and gene therapy over factor VIII (FVIII) prophylaxis and emicizumab.</p><p><strong>Conclusion: </strong>There were notable gaps, including inadequate reporting of model characteristics and input parameters, along with omission and mislabelling of cost categories. The findings underscore the urgent need for a disease-specific, standardised framework for societal cost categories and globally accessible costing datasets to enhance the consistency and transparency of economic studies in haemophilia.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"71-95"},"PeriodicalIF":2.1,"publicationDate":"2026-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12796068/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145564869","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The Consistency and Transferability of Economic Evaluations of Disease-Modifying Treatments for Multiple Sclerosis: Analysis of Cost-Effectiveness Assessments from Australia, England, Canada and Scotland.","authors":"Xiang Wang, Feili Zhao, David Newby, Lan Gao, Shuchuen Li","doi":"10.1007/s41669-025-00606-x","DOIUrl":"10.1007/s41669-025-00606-x","url":null,"abstract":"<p><strong>Objective: </strong>Our study examines the consistency and transferability of cost-effectiveness values, specifically the incremental cost-effectiveness ratio (ICER), focusing on several disease-modifying therapies (DMTs) for relapsing-remitting multiple sclerosis (RRMS) approved by four of the most established Health Technology Assessment (HTA) bodies.</p><p><strong>Methods: </strong>Data of economic evaluation results for DMTs were extracted from the HTA reports published by four agencies. Descriptive statistics were employed to identify correlations between the accepted ICERs within and across agencies. Two different currency conversion approaches were employed to investigate the feasibility and efficiency for transferring accepted ICERs across jurisdictions.</p><p><strong>Results: </strong>The study analysed ten DMTs that received positive recommendations from four agencies: the National Institute for Health and Care Excellence (NICE), Scottish Medicines Consortium (SMC), Pharmaceutical Benefits Advisory Committee (PBAC) and Canada's Drug Agency (CDA). The consistency of accepted ICER values for the class of DMTs was observed within the four agencies; however, the commonality of accepted ICER values was only observed between some agencies, such as the CDA and NICE, as well as the PBAC and SMC. Purchasing power parity-adjusted ICER values were found to provide better transference of ICER outcomes as compared to the exchange-rate method.</p><p><strong>Conclusion: </strong>This study highlighted the consistency of the economic evaluation for DMTs within the four agencies. We also identified a particularly strong alignment between NICE and the CDA, as well as the PBAC and SMC. Additionally, the examination of two ICER value conversion methods pinpointed key factors that could impact the transference of ICER values.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"97-108"},"PeriodicalIF":2.1,"publicationDate":"2026-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12796072/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145355669","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}