Journal of comparative effectiveness research最新文献

{"title":"Healthcare cost comparison between first-line ibrutinib and acalabrutinib in chronic lymphocytic leukemia patients in the Veterans Affairs.","authors":"Lindsey Fitzgerald, Sabyasachi Ghosh, Alex Bokun, Angela Lax, Fan Mu, Eric Wu, Yilu Lin, Lizheng Shi, Zaina P Qureshi, Solomon A Graf","doi":"10.57264/cer-2025-0084","DOIUrl":"https://doi.org/10.57264/cer-2025-0084","url":null,"abstract":"<p><p><b>Aim:</b> Bruton's tyrosine kinase inhibitors (BTKis), including ibrutinib and acalabrutinib, transformed the treatment landscape of chronic lymphocytic leukemia (CLL) and small lymphocytic lymphoma (SLL) by improving outcomes compared with chemoimmunotherapy. Real-world economic comparisons between BTKis are needed in diverse populations. This study aimed to compare healthcare costs in the Veterans Health Administration (VHA) among patients with CLL/SLL treated with, and remaining persistent on, first-line (1L) ibrutinib versus acalabrutinib monotherapy for 12 months. <b>Materials & methods:</b> This retrospective study used VHA electronic medical record data from January 2006 to July 2024. Eligible patients initiated 1L ibrutinib or acalabrutinib monotherapy on or after November 2019 and remained on continuous treatment for ≥12 months. All-cause and CLL/SLL-related costs were assessed over 12 months of follow-up. Generalized linear models were used to estimate adjusted costs and compare differences between treatment cohorts. <b>Results:</b> A total of 1059 patients were included (ibrutinib: n = 732; acalabrutinib: n = 327). During the 12-month follow-up of continuous 1L treatment, the annual adjusted all-cause total healthcare cost difference between ibrutinib and acalabrutinib was -$2422 (p = 0.46) (adjusted medical cost difference: $5259, p = 0.03; adjusted pharmacy cost difference: -$5886, p = 0.02). The annual adjusted CLL/SLL-related total healthcare cost difference between ibrutinib and acalabrutinib was -$3793 (p = 0.15) (adjusted medical cost difference: $2085, p = 0.05; adjusted pharmacy cost difference: -$5860, p = 0.02). <b>Conclusion:</b> Among VHA patients with CLL/SLL who initiated and remained on treatment with 1L BTKi monotherapy for 12 months, annual all-cause and CLL/SLL-related total healthcare costs were similar between ibrutinib and acalabrutinib. Pharmacy costs were lower for ibrutinib, while medical costs were lower for acalabrutinib, resulting in overall comparable total costs.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e250084"},"PeriodicalIF":2.5,"publicationDate":"2025-10-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145286174","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":"Blood neurofilament light chain as a predictive biomarker for functional outcome of acute ischemic stroke: a systematic review and meta-analysis.","authors":"Hongyu Xu, Hongyu Lin, Rongxing Qin, Lingduo Shao, Wei Xu, Qingchun Qin, Xiaojun Liang, Xinyu Lai, Li Chen","doi":"10.57264/cer-2025-0130","DOIUrl":"https://doi.org/10.57264/cer-2025-0130","url":null,"abstract":"<p><p><b>Aim:</b> Ischemic stroke continues to be a significant contributor to mortality and disability on a global scale. The blood neurofilament light chain (bNfL) as a prognostic indicator for stroke functional outcomes is a topic of ongoing debate. Thus, the objective of this systematic review is to assess the efficacy of bNfL as a predictor of stroke functional outcomes. <b>Materials & methods:</b> A systematic search was conducted in Pubmed, Cochrane and Embase databases from their inception to 21 October 2023. Two reviewers independently screened the search results to identify studies reporting on the association between bNfL and acute ischemic stroke outcomes. The quality of the studies was assessed using the Newcastle-Ottawa scale. Meta-analysis was conducted using the Comprehensive Meta-Analysis software Stata 12.0, utilizing a random effects model to estimate the pooled effect. <b>Results:</b> Nine studies involving 2302 patients were included in the analysis. A pooled analysis of adjusted odds ratios (ORs) from multivariate regression models in the meta-analysis revealed a pooled adjusted OR of 1.929 [95% CI:1.459, 2.550], suggesting that the patients with higher bNfL levels are at a greater risk of experiencing unfavorable functional outcomes compared with those with lower bNfL levels. Subgroup analysis indicated that factors such as sampling time, study region, participant age, blood specimen and sample size, may contributed to high heterogeneity in the results. After conducting a thorough analysis using funnel plot and Egger's test, no significant evidence of publication bias was found in our study. <b>Conclusion:</b> In summary, bNfL demonstrates potential as a predictive biomarker for functional outcomes in acute ischemic stroke patients, albeit subject to influence from confounding variables. Additional rigorously designed and meticulously executed prospective studies on a larger scale are warranted to validate these findings.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e250130"},"PeriodicalIF":2.5,"publicationDate":"2025-10-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145238645","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":"Estimating per-protocol effects in external comparator analyses using real-world data.","authors":"Alind Gupta, Evie Merinopoulou, Stephen J Duffield, Manuel Gomes, Seamus Kent, Nicolas Scheuer, Gerardo Machnicki, Thibaut Sanglier","doi":"10.57264/cer-2025-0029","DOIUrl":"https://doi.org/10.57264/cer-2025-0029","url":null,"abstract":"<p><p>Analysis of single arm trials complemented with external comparator arms (ECAs) may be used to support evidence of effectiveness of novel therapies in oncology research when a randomized trial is unavailable or unfeasible. However, the intention-to-treat effect, which is a common target of estimation in ECA studies, is difficult to interpret when there are differences in adherence between the trial and ECA. This paper describes an approach to estimation of per-protocol effects in ECA studies using the target trial emulation framework for study design and analysis based on the results from an exploratory case study (TBASEL). We highlight challenges, potential solutions and future opportunities from the perspectives of protocol specification, data suitability and analysis, to help guide future implementations of per-protocol effects in ECAs.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e250029"},"PeriodicalIF":2.5,"publicationDate":"2025-10-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145232749","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":"Healthcare costs and treatment patterns associated with glucagon-like peptide 1 receptor agonist use among patients with metabolic dysfunction-associated steatohepatitis.","authors":"Elliot B Tapper, Taylor Ryan, Ni Zeng, Renee Carter, Jessamine P Winer-Jones, Machaon Bonafede, Yestle Kim","doi":"10.57264/cer-2024-0223","DOIUrl":"https://doi.org/10.57264/cer-2024-0223","url":null,"abstract":"<p><p><b>Aim:</b> While only recently approved for the treatment of metabolic dysfunction-associated steatohepatitis (MASH), many patients with MASH have taken glucagon-like peptide-1 receptor agonists (GLP-1 RAs) for the treatment of comorbid Type 2 diabetes (T2D) or obesity. This real-world study evaluated treatment patterns, weight loss, healthcare resource utilization, and costs among patients with MASH who initiated GLP-1 RAs. <b>Materials & methods:</b> In a linked electronic health records (Veradigm Network EHR) and claims (Komodo Health) dataset, we identified adults (≥18 years old) with a MASH diagnosis who initiated GLP-1 RA treatment (1 July 2018 to 30 April 2023; index date = date of the first GLP-1 RA claim). Patients with other causes of liver disease or severe complications from MASH were excluded. We required ≥12 months of continuous enrollment pre- (baseline) and post-index (follow-up). Patients were stratified into high and low-dose subgroups. We also identified a comparator cohort of patients who initiated a different class of T2D medication (DPP4, SGLT2, or sulfonylurea) during the same time period. We captured patient characteristics, change in BMI, GLP-1 RA treatment patterns, liver-related events, healthcare utilization, and costs. <b>Results:</b> We identified 10,316 patients with MASH who initiated a GLP-1 RA (high dose: 2043 [19.8%]; low dose: 8273 [80.2%]) and 2915 who initiated a non-GLP-1 RA T2D medication. GLP-1 RA users were 52.7 years old and 64.3% female. A 5.8% decrease in the percentage of patients with class III obesity was observed among GLP-1 RA users (10.7% among high-dose users; 0.8% among non-users). Overall, 56.1% of GLP-1 RA users discontinued during the 12-month follow-up. Total costs among GLP-1 RA users and non users were $20,912 and $19,019 in the baseline period and $27,586 and $24,917 in the follow-up period, respectively. Medical costs among GLP-1 RA users were $16,293 (baseline) and $16,886 (follow-up). Results were similar for high and low-dose subgroups. <b>Conclusion:</b> Although some patients with MASH on GLP-1 RAs, particularly those taking higher dosages, may achieve weight loss, outcomes remain suboptimal with frequent discontinuation and high healthcare costs. Real-world GLP-1 RA utilization may be insufficient for resolving chronic metabolic issues, including MASH.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240223"},"PeriodicalIF":2.5,"publicationDate":"2025-10-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145232761","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":"R WE ready for reimbursement? A round-up of developments in real-world evidence relating to health technology assessment: part 21.","authors":"Paul Arora, Sreeram V Ramagopalan","doi":"10.57264/cer-2025-0148","DOIUrl":"https://doi.org/10.57264/cer-2025-0148","url":null,"abstract":"<p><p>In this update, we explore the vocal adoption of the target trial emulation framework by the US FDA, the FRAME methodology for systematically evaluating the use and impact of RWE in health technology assessment and regulatory submissions, and the Canadian CanREValue framework for incorporating RWE into cancer medicine reassessment decisions.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e250148"},"PeriodicalIF":2.5,"publicationDate":"2025-10-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145206432","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":"Access in all areas? A round up of developments in market access and health technology assessment: part 9.","authors":"Sreeram V Ramagopalan, Annie Jullien Pannelay","doi":"10.57264/cer-2025-0120","DOIUrl":"10.57264/cer-2025-0120","url":null,"abstract":"<p><p>In this update, we examine Spain's comprehensive pharmaceutical legislation reform; France's refined health economic evaluation approach; and the US' proposed Most Favored Nation pricing mechanism.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e250120"},"PeriodicalIF":2.5,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12465938/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144789289","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Matching-adjusted indirect comparison of ribociclib + nonsteroidal aromatase inhibitor versus abemaciclib + endocrine therapy in hormone receptor-positive/HER2-negative early breast cancer.","authors":"Stephen Chia, Jie Li, Fei Ma, Daniel Stellato, Andriy Danyliv, Ilia Ferrusi, Huilin Hu, Murat Akdere, Andreas Makris","doi":"10.57264/cer-2025-0082","DOIUrl":"10.57264/cer-2025-0082","url":null,"abstract":"<p><p><b>Aim:</b> Ribociclib + nonsteroidal aromatase inhibitor (NSAI) and abemaciclib + endocrine therapy (ET) are approved for high-risk hormone receptor-positive/human epidermal growth factor receptor 2-negative (HR+/HER2-) early breast cancer based on data from the NATALEE and monarchE trials, respectively. No trials have directly compared efficacy and safety of adjuvant ribociclib and abemaciclib. This study compared relative efficacy and safety of adjuvant ribociclib + NSAI versus abemaciclib + ET using matching-adjusted indirect comparison (MAIC). <b>Materials & methods:</b> Individual patient data from NATALEE and aggregate data from monarchE were analyzed, with patients from NATALEE selected to match the key eligibility criteria of monarchE cohort 1. Unanchored MAIC was used to compare invasive disease-free survival, distant relapse-free survival, and grade ≥3 treatment-emergent adverse events between treatment arms in NATALEE versus monarchE. Cox regression was used to estimate hazard ratios pre- and post-MAIC weighting. Logistic regression was used to estimate odds ratios (ORs). <b>Results:</b> After weighting, the effective sample size for the ribociclib + NSAI arm of NATALEE was 448. Cox regression yielded similar invasive disease-free survival with weighted ribociclib + NSAI versus abemaciclib + ET (hazard ratio, 0.901; 95% CI: 0.678-1.197). Unweighted efficacy comparisons were consistent with the weighted approach. Lower odds (OR <1) for diarrhea, leukopenia and lymphopenia and higher odds (OR >1) for increased alanine aminotransferase and neutropenia with ribociclib + NSAI versus abemaciclib + ET were noted before and after weighting. Sensitivity analyses were consistent with the primary analysis. <b>Conclusion:</b> This MAIC suggests similar efficacy between ribociclib + NSAI and abemaciclib + ET but different safety profiles in the HR+/HER2- early breast cancer patient population corresponding to the monarchE cohort 1, which has implications for treatment decisions. This analysis has limitations inherent to unanchored MAIC and should be interpreted in context of the trials and with clinical judgement.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e250082"},"PeriodicalIF":2.5,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12465954/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144956464","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Composite endpoints in health technology assessment: Part 1 - an illustration of best modeling practice.","authors":"Andrew Briggs, Aris Angelis, Jieling Chen, David Booth, Jason A Davis, Muthiah Vaduganathan, Pardeep S Jhund","doi":"10.57264/cer-2024-0117","DOIUrl":"10.57264/cer-2024-0117","url":null,"abstract":"<p><p>Composite endpoints amalgamate multiple clinical outcomes into a single measure, offering efficiency gains in clinical trials through increased event rates and reduced sample sizes, thus accelerating clinical development and regulatory approval. However, employing composite endpoints introduces complexities into health technology assessments (HTAs), particularly in economic modeling, due to the varying clinical significance and cost implications of the components. In this paper, we explore best modeling practice for HTAs that are based on clinical trials that employ composite endpoints. We examine regulatory guidance and discuss statistical solutions for differential component impacts, before presenting a case study based on a recent dapagliflozin submission for reimbursement in heart failure. Our investigation reveals that while composite endpoints can streamline trial analyses and hasten regulatory approval, they also pose a risk of bias in HTA if treatment effects for the components are inappropriately pooled. The paper discusses HTA principles in the context of composite endpoint trials and proposes strategies to develop modeling scenarios and interpret results, especially concerning whether to combine or split out estimates of component treatment effects. A particular focus is the accurate capture of uncertainty, both in terms of the parameter inputs to the model and over the ultimate decision to reimburse. This paper serves as a potential resource for researchers, practitioners and decision-makers, offering insights into best modeling practices that can unlock the full potential of composite endpoints in the pursuit of evidence-based healthcare decision-making.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240117"},"PeriodicalIF":2.5,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12465925/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145029836","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Cost-per-response of Acthar Gel versus standard of care for the treatment of noninfectious keratitis: a United States healthcare perspective.","authors":"Jas Bindra, Ishveen Chopra, Kyle Hayes, John Niewoehner, Mary Panaccio, George J Wan","doi":"10.57264/cer-2025-0088","DOIUrl":"10.57264/cer-2025-0088","url":null,"abstract":"<p><p><b>Aim:</b> Persistently active noninfectious keratitis can lead to permanent corneal damage and loss of vision. Given the limited treatment options, Acthar Gel, a US FDA-approved treatment for ocular inflammatory conditions, can be a potential therapy. This study evaluated the cost-per-response of Acthar^® Gel compared with standard of care ([SoC]; adalimumab or infliximab) for noninfectious keratitis from a US healthcare payer perspective over 1-3 years. <b>Materials & methods:</b> A probabilistic, cohort-level state-transition decision-analytic model was developed to assess the economic value of Acthar Gel. The model simulated the treatment pathway over 3 years, divided into 3-month cycles. Patients started with active keratitis and received Acthar Gel or SoC. Based on treatment success, patients transitioned between relapse, response and no-response states, with no response potentially leading to complications. Clinical parameters for Acthar Gel were derived from a phase IV open-label study and for SoC from observational studies. Healthcare resource utilization and costs were derived from administrative claims data or published literature. <b>Results:</b> Over 1 year, Acthar Gel demonstrated a lower cost per response ($167,928) than SoC, adalimumab ($228,450) and infliximab ($195,083). This benefit for Acthar Gel over SoC was sustained for 2 and 3 years. Acthar Gel, despite having a higher acquisition cost than SoC, had lower disease management costs and incurred no treatment-related (administration, monitoring or adverse event) costs over 1 year. Acthar Gel also demonstrated a lower overall cost of care than SoC at 2 and 3 years. Further, Acthar Gel had higher response rates versus SoC over 1-3 years. Acthar Gel was a dominant treatment option versus SoC at 2 and 3 years. <b>Conclusion:</b> From a US healthcare payer perspective, Acthar Gel may be a cost-effective, value-based treatment option for appropriate patients with noninfectious keratitis.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e250088"},"PeriodicalIF":2.5,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12465900/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144649538","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"How much do ex-US revenues make a difference for pharmaceutical investment returns?","authors":"Sreeram V Ramagopalan, Harshal Thaker, Mel Walker, Om Narasimhan","doi":"10.57264/cer-2025-0121","DOIUrl":"10.57264/cer-2025-0121","url":null,"abstract":"","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e250121"},"PeriodicalIF":2.5,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12465917/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144956388","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}