Journal of comparative effectiveness research最新文献

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Development and validation of a method to compare outcomes between switchers and continuers in routine clinical practice. 开发和验证一种方法来比较常规临床实践中切换者和继续者之间的结果。
IF 2.5 4区 医学
Journal of comparative effectiveness research Pub Date : 2026-05-08 DOI: 10.57264/cer-2024-0099
Chuan Gao, Jenny Jiang, Nipun Atreja, Amiee Kang, Xiaoyan Li, Xuemei Luo
{"title":"Development and validation of a method to compare outcomes between switchers and continuers in routine clinical practice.","authors":"Chuan Gao, Jenny Jiang, Nipun Atreja, Amiee Kang, Xiaoyan Li, Xuemei Luo","doi":"10.57264/cer-2024-0099","DOIUrl":"https://doi.org/10.57264/cer-2024-0099","url":null,"abstract":"<p><p><b>Aim:</b> In clinical practice, patients may initiate and continue with a treatment (continuers) or switch to another treatment (switchers). Comparison of clinical outcomes between these cohorts can be challenging owing to several factors including differences in risk profiles, particularly when switching is related to the occurrence of a clinical event of interest and the time-varying nature of the risk of an event. Analyses may be biased if these factors are not considered, including determining the appropriate start point to evaluate outcomes. We developed and validated a method to address these issues. <b>Materials & methods:</b> The proposed method (SMARTS) assigned random pseudo-switching times to continuers, matching the distribution of actual switching times among switchers. Baseline characteristics at (pseudo-) switching time were balanced using propensity score matching, inverse probability of treatment weighting (IPTW) or standardized morbidity ratio weighting. For validation, we conducted a factorial simulation with two clinical scenarios: sicker switchers where switching helps (true post-switching HR = 0.7) and healthier switchers where it harms (true post-switching HR = 1.5), crossed with three hazard trends (constant, increasing, decreasing). A time-varying confounder followed different trajectories for switchers and continuers, with five analysis methods evaluated each condition. The utility of the developed method was assessed in a real-world study. <b>Results:</b> Under increasing hazard, the conventional approach (evaluating continuers from treatment initiation) showed substantial timing bias. For the sicker-switchers scenario (post-switching HR = 0.7), conventional IPTW HR was 1.06 (bias = +0.36); SMARTS IPTW reduced this to 0.78 (bias = +0.08). For the healthier-switchers scenario (post-switching HR = 1.5), conventional IPTW HR was 1.80 (bias = + 0.30); SMARTS IPTW reduced this to 1.41 (bias = -0.09). Under decreasing hazard, timing bias reversed direction: notably, for the healthier-switchers scenario (post-switching HR = 1.5) appeared protective using the conventional approach (IPTW HR = 0.61); SMARTS correctly identified the harmful effect (IPTW HR = 1.46). Under constant hazard, both approaches performed well, confirming that SMARTS specifically addresses timing bias. Application of the method improved results in a real-world study comparing outcomes between continuers and switchers. <b>Conclusion:</b> When hazard varies over time, evaluating continuers from treatment initiation introduces timing bias that can reverse the apparent direction of treatment effect. SMARTS, combined with confounder adjustment via propensity score matching, IPTW or standardized morbidity ratio weighting, substantially reduced this bias across diverse clinical scenarios and hazard trends.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240099"},"PeriodicalIF":2.5,"publicationDate":"2026-05-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147838589","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}
引用次数: 0
Evaluation of steroid toxicity using the Glucocorticoid Toxicity Index-Metabolic Domains instrument in adults with myasthenia gravis: a real-world study in the USA. 使用糖皮质激素毒性指数-代谢域仪器评估成人重症肌无力的类固醇毒性:美国的一项真实世界研究。
IF 2.5 4区 医学
Journal of comparative effectiveness research Pub Date : 2026-05-08 DOI: 10.57264/cer-2025-0141
Glenn Phillips, Cynthia Qi, Deborah Gelinas, Martha Stone, Anthony Chamberas, Dakshinamoorthy Amirthaganesan, Rucha Kulkarni, Albert Whangbo, John H Stone
{"title":"Evaluation of steroid toxicity using the Glucocorticoid Toxicity Index-Metabolic Domains instrument in adults with myasthenia gravis: a real-world study in the USA.","authors":"Glenn Phillips, Cynthia Qi, Deborah Gelinas, Martha Stone, Anthony Chamberas, Dakshinamoorthy Amirthaganesan, Rucha Kulkarni, Albert Whangbo, John H Stone","doi":"10.57264/cer-2025-0141","DOIUrl":"https://doi.org/10.57264/cer-2025-0141","url":null,"abstract":"<p><p><b>Aim:</b> The Glucocorticoid Toxicity Index-Metabolic Domains (GTI-MD) has been utilized to assess steroid toxicity in clinical trials and practice but has not been applied previously to large datasets. The GTI-MD was used to investigate steroid toxicity in patients with myasthenia gravis (MG) using a large real-world dataset. <b>Materials & methods:</b> Steroid toxicity was assessed in adults with MG (≥2 diagnoses occurring between ≥30 and ≤730 days apart) using Optum<sup>®</sup> electronic health records. Patients were categorized into steroid-initiator (MG-SI) or steroid-naive (MG-SN) cohorts. The GTI-MD algorithm assessed changes in steroid toxicity during follow-up using vital signs and simple laboratory tests pertaining to toxicity in the body mass index, blood pressure, glucose tolerance and lipid metabolism domains. Changes in steroid toxicity were reported as cumulative worsening scores (CWS) and aggregate improvement scores (AIS). <b>Results:</b> A total of 682 (MG-SI:377; MG-SN: 305) patients with MG were included. The potential for steroid toxicity at index date, as assessed by the GT-SNAPSHOT scores, was comparable between the cohorts at baseline (92.0 ± 31.1 vs 88.8 ± 32.8; p = 0.117). At follow-up, the MG-SI cohort had higher CWS scores than the MG-SN cohort (22.6 ± 22.8 vs 18.7 ± 21.2; p = 0.023) and were more likely to exceed the 10-point minimum clinically important difference (68% vs 59%; p = 0.016) and the 20-point threshold (44% vs 36%; p = 0.030). Patients with multiple steroid exposures had higher CWS (25.7 ± 24.8 vs 19.2 ± 19.9; p = 0.006) and AIS (9.6 ± 34.7 vs -0.3 ± 33.7; p = 0.005) values than one-time steroid users. Patients who received ≥20 mg/day of steroids (prednisone-equivalent) at index had higher CWS values (25.0 ± 23.0 vs 19.9 ± 22.3; p = 0.033) and AIS values (8.3 ± 32.9 vs 1.1 ± 35.9; p = 0.044) compared with those who received <20 mg/day of steroids. <b>Conclusion:</b> The GTI-MD is a valuable and sensitive method for unmasking steroid toxicity in MG using large datasets to gather real-world evidence.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e250141"},"PeriodicalIF":2.5,"publicationDate":"2026-05-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147838642","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}
引用次数: 0
Transportability of the comparative effect of finerenone for the treatment of symptomatic chronic heart failure with left ventricular ejection fraction of ≥40%: insights from the FINEARTS-HF trial. 芬尼酮治疗左心室射血分数≥40%的症状性慢性心力衰竭的转运性:finhearts - hf试验的见解
IF 2.5 4区 医学
Journal of comparative effectiveness research Pub Date : 2026-05-06 DOI: 10.57264/cer-2026-0003
Alex J Turner, Claire Leboucher, Cécile Remuzat, Yik Ming Fung, Elena Pessina, Kerstin Folkerts
{"title":"Transportability of the comparative effect of finerenone for the treatment of symptomatic chronic heart failure with left ventricular ejection fraction of ≥40%: insights from the FINEARTS-HF trial.","authors":"Alex J Turner, Claire Leboucher, Cécile Remuzat, Yik Ming Fung, Elena Pessina, Kerstin Folkerts","doi":"10.57264/cer-2026-0003","DOIUrl":"https://doi.org/10.57264/cer-2026-0003","url":null,"abstract":"<p><p><b>Aim:</b> Global randomized controlled trials (RCTs) are used to inform reimbursement decisions in multiple markets, meaning the transportability of findings from these RCTs to multiple country-specific populations is critical for evidence-based decision making. This study evaluated the transportability of the FINEARTS-HF trial, which assessed finerenone in patients with heart failure (HF) and left ventricular ejection fraction ≥40%, to a real-world US population. <b>Materials & methods:</b> A three-phase transportability assessment was conducted. First, potential effect modifiers were identified through systematic literature review and confirmed through interaction analyses using data from FINEARTS-HF. Second, representativeness was assessed by comparing the distribution of effect modifiers in the trial and a population derived from a US electronic health record dataset. Third, direct proxy tests explored heterogeneity of treatment effects in FINEARTS-HF between US and non-US patients. <b>Results:</b> Effect modifier analysis identified that treatment effects of finerenone were homogeneous across subgroups, with limited evidence of effect modification. Comparisons with the US target population indicated overall good alignment across key characteristics, with only modest imbalances, suggesting that the trial results may underestimate positive treatment effects in the US target population (i.e., effect on the primary outcome over 0.84 [0.74; 0.95]). Direct proxy tests found no statistically significant regional heterogeneity in treatment effects. <b>Conclusion:</b> This study provides a structured assessment of finerenone trial transportability. Findings support the robustness of FINEARTS-HF results for US clinical practice and are not impacted by the prevalence of SGLT2-is use. The risk of transportability bias is likely to be low.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e260003"},"PeriodicalIF":2.5,"publicationDate":"2026-05-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147838570","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}
引用次数: 0
R WE ready for reimbursement? A round-up of developments in real-world evidence relating to health technology assessment: part 26. 我们准备好报销了吗?与卫生技术评估有关的现实证据发展综述:第26部分。
IF 2.5 4区 医学
Journal of comparative effectiveness research Pub Date : 2026-05-05 DOI: 10.57264/cer-2026-0074
Paul Arora, Sreeram V Ramagopalan
{"title":"R WE ready for reimbursement? A round-up of developments in real-world evidence relating to health technology assessment: part 26.","authors":"Paul Arora, Sreeram V Ramagopalan","doi":"10.57264/cer-2026-0074","DOIUrl":"https://doi.org/10.57264/cer-2026-0074","url":null,"abstract":"<p><p>In this update, we review a framework for identifying and mitigating information bias in electronic health records and administrative claims data, highlighting practical recommendations for study design, variable definition, and statistical analysis. We also discuss a perspective on emerging privacy-preserving technologies - synthetic data and federated networks - that enable secure cross-border data access while maintaining patient privacy.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e260074"},"PeriodicalIF":2.5,"publicationDate":"2026-05-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147838612","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}
引用次数: 0
The inaugural HEMA report: a missed opportunity for comprehensive value assessment. 首份盒马报告:错失全面价值评估的良机。
IF 2.5 4区 医学
Journal of comparative effectiveness research Pub Date : 2026-05-01 Epub Date: 2026-03-18 DOI: 10.57264/cer-2026-0049
Kimberly Westrich, Tyler D Wagner, Jonathan D Campbell
{"title":"The inaugural HEMA report: a missed opportunity for comprehensive value assessment.","authors":"Kimberly Westrich, Tyler D Wagner, Jonathan D Campbell","doi":"10.57264/cer-2026-0049","DOIUrl":"10.57264/cer-2026-0049","url":null,"abstract":"","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e260049"},"PeriodicalIF":2.5,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147473868","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}
引用次数: 0
Maximizing the impact of real-world evidence on cancer treatment: what do we need to achieve it? 将真实世界证据对癌症治疗的影响最大化:我们需要做些什么来实现它?
IF 2.5 4区 医学
Journal of comparative effectiveness research Pub Date : 2026-05-01 Epub Date: 2026-04-24 DOI: 10.57264/cer-2025-0186
Begoña Bermejo, Rodrigo Dienstmann, Meritxell Bellet, Susana de la Cruz, Elena Galve-Calvo, Verónica Obadia, Ana Santaballa, Rocío Ramos-Medina, Mónica Cuenca, Fernando Moreno
{"title":"Maximizing the impact of real-world evidence on cancer treatment: what do we need to achieve it?","authors":"Begoña Bermejo, Rodrigo Dienstmann, Meritxell Bellet, Susana de la Cruz, Elena Galve-Calvo, Verónica Obadia, Ana Santaballa, Rocío Ramos-Medina, Mónica Cuenca, Fernando Moreno","doi":"10.57264/cer-2025-0186","DOIUrl":"10.57264/cer-2025-0186","url":null,"abstract":"<p><p>There is an increasing number of studies using real-world data to examine outcomes of drugs in clinical practice. Therefore, it is crucial to carefully evaluate their quality and findings, extract valuable insights, acknowledge their limitations and identify gaps to advance further their use in decision-making. Here we provide an overview of the opportunities offered throughout drug lifecycle, and challenges presented by real-world evidence (RWE) studies. We also highlight the importance of the culture of transparency, along with the development of good practices for reporting RWE studies. In addition, we review common biases and other methodological aspects that can undermine the potential insights a real-world oncology study can provide. We briefly illustrate some questions that RWE studies address that can help us guide clinical decisions. Several possible future directions expected to contribute for RWE improving cancer patients' outcomes emerge, from prospective data collection initiatives to normalization of biomarker data integration or collaborative data sharing platforms.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e250186"},"PeriodicalIF":2.5,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147772886","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}
引用次数: 0
Clinical outcomes of pressure-enabled drug delivery for trans-arterial chemoembolization and radioembolization. 加压给药经动脉化疗栓塞和放射栓塞的临床效果。
IF 2.5 4区 医学
Journal of comparative effectiveness research Pub Date : 2026-05-01 Epub Date: 2026-04-09 DOI: 10.57264/cer-2025-0144
Deepshekhar Gupta, Yunjuan Liu, Chris Miller-Rosales, Fangzhou Wei, Edward Tuttle, Ekaterina Byers, Richard Marshak, Richard Marshall
{"title":"Clinical outcomes of pressure-enabled drug delivery for trans-arterial chemoembolization and radioembolization.","authors":"Deepshekhar Gupta, Yunjuan Liu, Chris Miller-Rosales, Fangzhou Wei, Edward Tuttle, Ekaterina Byers, Richard Marshak, Richard Marshall","doi":"10.57264/cer-2025-0144","DOIUrl":"10.57264/cer-2025-0144","url":null,"abstract":"<p><p><b>Aim:</b> Prior research observed that pressure-enabled drug delivery (PEDD) counteracts intra-tumoral pressure during trans-arterial chemoembolization (TACE) and radioembolization (TARE), improving drug penetration, yet real-world evidence remains limited. This study examined outcomes associated with PEDD use, including in high-adopter facilities, and assessed potential cost avoidance associated with PEDD. <b>Materials & methods:</b> We used the Clarivate Real World Data repository to identify adults with hepatocellular carcinoma or secondary liver metastases who received TACE or TARE procedures (January 2020-March 2024). We compared baseline characteristics between PEDD and non-PEDD cohorts, then used a two-stage matching approach to develop a matched sample for comparative analyses. Facilities in the top fifth percentile of PEDD volume were examined separately. <b>Results:</b> In total, 603 PEDD and 16,210 non-PEDD patients were identified. PEDD patients had higher baseline disease burden on average. In matched analyses, PEDD was associated with reduced rates of fatigue overall (20.9% vs 26.4%, p < 0.05) and 30-day inpatient visits among patients receiving TACE procedures (8.0% vs 20.5%, p < 0.05). In high-adopter facilities, PEDD use was associated with reduced lymphopenia among all patients (0.6% vs 5.2%, p < 0.05) and reduced fatigue (19.2% vs 39.7%, p < 0.05) and lymphopenia (0.0% vs 8.2%, p < 0.05) among patients with secondary liver metastases. PEDD use was linked to a per-patient charge avoidance of $7734 through fewer complications ($4599) and inpatient stays ($3135). <b>Conclusion:</b> Despite greater patient comorbidity, PEDD use was associated with fewer post-procedure complications and lower healthcare resource utilization. Greater institutional experience may enhance patient outcomes. Reduced complications and hospitalizations can translate into cost avoidance, reinforcing the value of PEDD technology in clinical practice.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e250144"},"PeriodicalIF":2.5,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147638958","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}
引用次数: 0
Readmissions and retreatment among stent-assisted endovascular coiling patients with unruptured intracranial aneurysm using ENTERPRISE® 2 stent versus LVIS™ stent. ENTERPRISE®2支架与LVIS™支架在支架辅助血管内盘绕未破裂颅内动脉瘤患者中的再入院和再治疗
IF 2.5 4区 医学
Journal of comparative effectiveness research Pub Date : 2026-05-01 Epub Date: 2026-04-13 DOI: 10.57264/cer-2025-0166
Reade De Leacy, Rahul Khanna, Emilie Kottenmeier, Yiran Rong
{"title":"Readmissions and retreatment among stent-assisted endovascular coiling patients with unruptured intracranial aneurysm using ENTERPRISE<sup>®</sup> 2 stent versus LVIS™ stent.","authors":"Reade De Leacy, Rahul Khanna, Emilie Kottenmeier, Yiran Rong","doi":"10.57264/cer-2025-0166","DOIUrl":"10.57264/cer-2025-0166","url":null,"abstract":"<p><p><b>Aim:</b> The treatment of wide-necked unruptured intracranial aneurysms (UIA) remains clinically challenging. Stent-assisted coiling (SAC) is a commonly used treatment modality for UIAs. The objective of this study was to examine the differences in 180-day all-cause inpatient readmissions, UIA-related inpatient readmission, and retreatment among UIA patients treated with the laser cut nitinol ENTERPRISE<sup>®</sup> 2 stent versus the braided nitinol LVIS™ stent during SAC procedures. <b>Materials & methods:</b> Using Premier Healthcare Database (PHD), a US nationwide hospital database, UIA patients aged ≥18 years old undergoing SAC were identified. Patients were then classified into ENTERPRISE 2 and LVIS cohorts based on the stent used. Study outcomes, including 180-day all-cause and UIA-related inpatient readmissions, and UIA-related retreatment, were compared between the two cohorts. Inverse probability of treatment weighting of propensity score approach was used to balance covariates (i.e., patient demographic, clinical characteristics and hospital characteristics) between the two study groups. Chi-square test and weighted generalized estimating equation (GEE) model was used to assess outcomes among the weighted ENTERPRISE 2 and LVIS cohorts. <b>Results:</b> A total of 249 patients were included after applying study inclusion and exclusion criteria (with 130 in the ENTERPRISE 2 cohort and 119 in the LVIS cohort). Patient characteristics were well balanced after weighting. Bivariate analysis revealed that patients undergoing stent-assisted endovascular coiling using ENTERPRISE 2 stent had a significantly lower rate of 180-day all-cause inpatient readmissions (9.1% vs 24.3%, chi-square p = 0.016) and a significantly lower rate of 180-day UIA-related inpatient readmission (2.6% vs 12.4%, chi-square p = 0.036) compared with those treated with LVIS stent. GEE regression model indicated that patients in the ENTERPRISE2 stent cohort were 69% less likely to have 180-day all-cause inpatient readmissions (odds ratio: 0.31, 95% CI: 0.12-0.82, GEE p = 0.018) versus the LVIS cohort. However, no significant difference in 180-day UIA-related inpatient readmission and 180-day UIA-related retreatment was observed in GEE analysis. <b>Conclusion:</b> Patients who were treated with the laser-cut ENTERPRISE 2 stent during endovascular coiling were observed to have significantly lower risk of all-cause inpatient readmissions compared with those treated with the braided LVIS stent. No significant differences were observed for 180-day UIA-related readmission and retreatment among the study cohorts.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e250166"},"PeriodicalIF":2.5,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147673990","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}
引用次数: 0
Access in all areas? A round-up of developments in market access and health technology assessment: part 14. 所有地区都能通行吗?市场准入和卫生技术评估方面的发展综述:第14部分。
IF 2.5 4区 医学
Journal of comparative effectiveness research Pub Date : 2026-05-01 Epub Date: 2026-03-26 DOI: 10.57264/cer-2026-0056
Sreeram V Ramagopalan, Annie Jullien Pannelay
{"title":"Access in all areas? A round-up of developments in market access and health technology assessment: part 14.","authors":"Sreeram V Ramagopalan, Annie Jullien Pannelay","doi":"10.57264/cer-2026-0056","DOIUrl":"10.57264/cer-2026-0056","url":null,"abstract":"<p><p>In this update we examine the selection of 15 drugs for the third cycle of Medicare price negotiations under the Inflation Reduction Act and analyze emerging evidence of the Most-Favored-Nation impact on global pharmaceutical access, including launch delays and market withdrawals. We also review recent research on the tension between US patient values and foreign health technology assessment frameworks and discuss the review of the first year of implementation of the Joint Clinical Assessment procedure in Europe.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e260056"},"PeriodicalIF":2.5,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147512493","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}
引用次数: 0
Comparative safety of direct oral anticoagulants and low-molecular-weight heparin in patients with venous thromboembolism and cancer in Europe. 欧洲静脉血栓栓塞和癌症患者直接口服抗凝剂和低分子肝素的安全性比较
IF 2.5 4区 医学
Journal of comparative effectiveness research Pub Date : 2026-05-01 DOI: 10.57264/cer-2025-0072
Ellen Brodin, Annie Young, Florian Langer, Raza Alikhan, Lauri Timo, Dimitra Lambrelli, Beth Nordstrom, Alison Booth, Sophie Graham, Ruth Mokgokong, Aaron Jenkins, Jose Chaves, Feng Dai, Rupesh Subash, Moritz Lehne, Marco Ghiani, Antje Mevius, Daniela Fliegner, Anders Gottsäter
{"title":"Comparative safety of direct oral anticoagulants and low-molecular-weight heparin in patients with venous thromboembolism and cancer in Europe.","authors":"Ellen Brodin, Annie Young, Florian Langer, Raza Alikhan, Lauri Timo, Dimitra Lambrelli, Beth Nordstrom, Alison Booth, Sophie Graham, Ruth Mokgokong, Aaron Jenkins, Jose Chaves, Feng Dai, Rupesh Subash, Moritz Lehne, Marco Ghiani, Antje Mevius, Daniela Fliegner, Anders Gottsäter","doi":"10.57264/cer-2025-0072","DOIUrl":"10.57264/cer-2025-0072","url":null,"abstract":"<p><p><b>Aim:</b> This study aimed to compare the safety and effectiveness of direct oral anticoagulants (DOACs) with low-molecular-weight heparin (LMWH) in patients with venous thromboembolism (VTE) and active cancer in a real-world setting in Sweden, Norway, Finland, the UK and Germany. <b>Materials & methods:</b> This observational cohort study used datasets from Sweden, Norway, Finland (National Patient and Prescription Registers) the UK (Clinical Practice Research Datalink and Hospital Episode Statistics) and Germany (AOK Plus and GWQ) from 2013 to 2020. We identified treatment-naive adult patients with cancer-related VTE treated with a DOAC or LMWH. We employed inverse probability of treatment weighting for each DOAC-LMWH comparison and assessed recurrent VTE and bleeding risk (overall and by site: gastrointestinal [GI], intracranial hemorrhage [ICH] or other) within 6 months of treatment initiation. Fine-Gray models were fitted to estimate adjusted hazard ratios and country level estimates were meta-analyzed. <b>Results:</b> After inverse probability of treatment weighting, 30,002 and 2892 patients were included in the LMWH-apixaban comparison, and 29,976 and 4321 in the LMWH-rivaroxaban comparison, respectively. Recurrent VTE could not be assessed comparatively because of low numbers. At 6 months, apixaban was associated with a lower risk of overall and other (HR: 0.67 [95% CI: 0.53,0.86]; 0.64 [0.41,0.998]) bleeding compared with LMWH, with similar risks for GI and ICH bleeding (0.89 [0.61,1.29]; 0.67 [0.31,1.44], respectively). Rivaroxaban had a similar risk of overall, GI, and other bleeding (0.98 [0.83, 1.15]; 0.86 [0.47,1.58]; 0.89 [0.74,1.08]) compared with LMWH, but a lower risk of ICH (0.32 [0.13, 0.82]). <b>Conclusion:</b> These findings suggest that apixaban may offer a safer bleeding profile than LMWH for patients with cancer-associated VTE, particularly in reducing overall and other types of bleeding. Rivaroxaban also appears to be a viable alternative to LMWH, with a notably lower risk of ICH. These results provide valuable real-world insights in an area where evidence remains very limited and support the role of DOACs as a safe alternative to LMWH in patients with VTE and cancer.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e250072"},"PeriodicalIF":2.5,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147816013","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}
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