Journal of comparative effectiveness research最新文献

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The maze of real-world evidence frameworks: from a desert to a jungle! An environmental scan and comparison across regulatory and health technology assessment agencies. 真实世界证据框架的迷宫:从沙漠到丛林!环境扫描以及监管机构和卫生技术评估机构之间的比较。
IF 1.9 4区 医学
Journal of comparative effectiveness research Pub Date : 2024-09-01 Epub Date: 2024-08-12 DOI: 10.57264/cer-2024-0061
Grammati Sarri, Luis G Hernandez
{"title":"The maze of real-world evidence frameworks: from a desert to a jungle! An environmental scan and comparison across regulatory and health technology assessment agencies.","authors":"Grammati Sarri, Luis G Hernandez","doi":"10.57264/cer-2024-0061","DOIUrl":"10.57264/cer-2024-0061","url":null,"abstract":"<p><p><b>Aim:</b> Regulatory and health technology assessment (HTA) agencies have increasingly published frameworks, guidelines, and recommendations for the use of real-world evidence (RWE) in healthcare decision-making. Variations in the scope and content of these documents, with updates running in parallel, may create challenges for their implementation especially during the market authorization and reimbursement phases of a medicine's life cycle. This environmental scan aimed to comprehensively identify and summarize the guidance documents for RWE developed by most well-established regulatory and reimbursement agencies, as well as other organizations focused on healthcare decision-making, and present their similarities and differences. <b>Methods:</b> RWE guidance documents, including white papers from regulatory and HTA agencies, were reviewed in March 2024. Data on scope and recommendations from each body were extracted by two reviewers and similarities and differences were summarized across four topics: study planning, choosing fit-for-purpose data, study conduct, and reporting. Post-authorization or non-pharmacological guidance was excluded. <b>Results:</b> Forty-six documents were identified across multiple agencies; US FDA produced the most RWE-related guidance. All agencies addressed specific and often similar methodological issues related to study design, data fitness-for-purpose, reliability, and reproducibility, although inconsistency in terminologies on these topics was noted. Two HTA bodies (National Institute for Health and Care Excellence [NICE] and Canada's Drug Agency) each centralized all related RWE guidance under a unified framework. RWE quality tools and checklists were not consistently named and some differences in preferences were noted. European Medicines Agency, NICE, Haute Autorité de Santé, and the Institute for Quality and Efficiency in Health Care included specific recommendations on the use of analytical approaches to address RWE complexities and increase trust in its findings. <b>Conclusion:</b> Similarities in agencies' expectations on RWE studies design, quality elements, and reporting will facilitate evidence generation strategy and activities for manufacturers facing multiple, including global, regulatory and reimbursement submissions and re-submissions. A strong preference by decision-making bodies for local real-world data generation may hinder opportunities for data sharing and outputs from international federated data networks. Closer collaboration between decision-making agencies towards a harmonized RWE roadmap, which can be centrally preserved in a living mode, will provide manufacturers and researchers clarity on minimum acceptance requirements and expectations, especially as novel methodologies for RWE generation are rapidly emerging.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240061"},"PeriodicalIF":1.9,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11367564/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141916823","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}
引用次数: 0
Lessons on the use of real-world data in medical device research: findings from the National Evaluation System for Health Technology Test-Cases. 在医疗设备研究中使用真实世界数据的经验教训:国家卫生技术测试案例评估系统的发现。
IF 1.9 4区 医学
Journal of comparative effectiveness research Pub Date : 2024-09-01 Epub Date: 2024-08-16 DOI: 10.57264/cer-2024-0078
Justin W Timbie, Alice Y Kim, Lawrence Baker, Rosemary Li, Thomas W Concannon
{"title":"Lessons on the use of real-world data in medical device research: findings from the National Evaluation System for Health Technology Test-Cases.","authors":"Justin W Timbie, Alice Y Kim, Lawrence Baker, Rosemary Li, Thomas W Concannon","doi":"10.57264/cer-2024-0078","DOIUrl":"10.57264/cer-2024-0078","url":null,"abstract":"<p><p><b>Aim:</b> Although the US FDA encourages manufacturers of medical devices to submit real-world evidence (RWE) to support regulatory decisions, the ability of real-world data (RWD) to generate evidence suitable for decision making remains unclear. The 2017 Medical Device User Fee Amendments (MDUFA IV), authorized the National Evaluation System for health Technology Coordinating Center (NESTcc) to conduct pilot projects, or 'Test-Cases', to assess whether current RWD captures the information needed to answer research questions proposed by industry stakeholders. We synthesized key lessons about the challenges conducting research with RWD and the strategies used by research teams to enhance their ability to generate evidence from RWD based on 18 Test-Cases conducted between 2020 and 2022. <b>Materials & methods:</b> We reviewed study protocols and reports from each Test-Case team and conducted 49 semi-structured interviews with representatives of participating organizations. Interview transcripts were coded and thematically analyzed. <b>Results:</b> Challenges that stakeholders encountered in working with RWD included the lack of unique device identifiers, capturing key data elements and their appropriate meaning in structured data, limited reliability of diagnosis and procedure codes in structured data, extracting information from unstructured electronic health record (EHR) data, limited capture of long-term study end points, missing data and data sharing. Successful strategies included using manufacturer and supply chain data, leveraging clinical registries and registry reporting processes to collect and aggregate data, querying standardized EHR data, implementing natural language processing algorithms and using multidisciplinary research teams. <b>Conclusion:</b> The Test-Cases identified numerous challenges working with RWD but also opportunities to address these challenges and improve researchers' ability to use RWD to generate evidence on medical devices.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240078"},"PeriodicalIF":1.9,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11367563/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141988044","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}
引用次数: 0
Val (EU) xit: do we need an international ISPOR value flower? Val (EU) xit:我们是否需要一朵国际 ISPOR 价值之花?
IF 1.9 4区 医学
Journal of comparative effectiveness research Pub Date : 2024-09-01 Epub Date: 2024-07-19 DOI: 10.57264/cer-2024-0083
Sreeram V Ramagopalan, Manuel Gomes, Isabelle Durand-Zaleski, Bill Malcolm, Jose Diaz, Grace Mitchell, Jonathan Pearson-Stuttard, Louis P Garrison
{"title":"Val (EU) xit: do we need an international ISPOR value flower?","authors":"Sreeram V Ramagopalan, Manuel Gomes, Isabelle Durand-Zaleski, Bill Malcolm, Jose Diaz, Grace Mitchell, Jonathan Pearson-Stuttard, Louis P Garrison","doi":"10.57264/cer-2024-0083","DOIUrl":"10.57264/cer-2024-0083","url":null,"abstract":"","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240083"},"PeriodicalIF":1.9,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11363197/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141723722","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}
引用次数: 0
Improvements in daytime sleepiness and disrupted nighttime sleep with once-nightly sodium oxybate in people with narcolepsy type 1 and type 2: a plain language summary. 1型和2型嗜睡症患者每晚服用一次羟苯甲酸钠可改善白天嗜睡和夜间睡眠紊乱:通俗易懂的摘要。
IF 1.9 4区 医学
Journal of comparative effectiveness research Pub Date : 2024-09-01 Epub Date: 2024-08-01 DOI: 10.57264/cer-2024-0031
Yves Dauvilliers, Thomas Roth, Richard Bogan, Michael J Thorpy, Anne Marie Morse, Fèri Ascencion, Jennifer Gudeman
{"title":"Improvements in daytime sleepiness and disrupted nighttime sleep with once-nightly sodium oxybate in people with narcolepsy type 1 and type 2: a plain language summary.","authors":"Yves Dauvilliers, Thomas Roth, Richard Bogan, Michael J Thorpy, Anne Marie Morse, Fèri Ascencion, Jennifer Gudeman","doi":"10.57264/cer-2024-0031","DOIUrl":"10.57264/cer-2024-0031","url":null,"abstract":"<p><strong>What is this summary about?: </strong>This is a plain language summary of a published article in the journal <i>Sleep</i>. Narcolepsy is a sleep condition that has 2 different subtypes: narcolepsy type 1 and narcolepsy type 2. These are called NT1 and NT2 for short. Sodium oxybate (SXB) is approved to treat excessive daytime sleepiness (EDS) and cataplexy. People with NT1 and NT2 both have EDS, but cataplexy is only present in people with NT1. Limited information is available about how SXB works in people with NT2. This is because previous trials have included only people with NT1 or people with unspecified narcolepsy. For more than 20 years, the only available formulation of this medicine had to be given twice during the night. Many people with narcolepsy find that chronically waking up in the middle of the night for a second dose of SXB is disruptive to themselves or others in their household. People have also reported sleeping through alarm clocks, missing their second dose, and feeling worse the next day. Some people have accidentally taken the second dose too early, putting them at risk for serious adverse effects. These adverse effects may include slow breathing, low blood pressure, or sedation. The US Food and Drug Administration (FDA) approved a medicine called LUMRYZ<sup>™</sup> (sodium oxybate) for extended-release oral suspension in May 2023. LUMRYZ is a once-nightly formulation of SXB (ON-SXB for short) and is taken as a single dose before bedtime. This medicine treats EDS and muscle weakness (also known as cataplexy) in people with narcolepsy. A clinical trial called REST-ON studied ON-SXB to find out if it was better at treating narcolepsy symptoms than a medicine with no active ingredients (placebo). This summary describes a study that tested whether ON-SXB was better than placebo at treating narcolepsy symptoms in people with NT1 or NT2.</p><p><strong>What were the results?: </strong>This study showed that compared to people who took placebo, people who took ON-SXB were able to stay awake longer during the day, felt less sleepy during the daytime, had less cataplexy, and had more improvements in their symptoms overall than people who took placebo.</p><p><strong>What do the results mean?: </strong>ON-SXB has been proven effective for people with NT1 or NT2. Unlike prior formulations of SXB, ON-SXB is taken once at bedtime, without requiring waking up in the middle of the night for a second dose.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240031"},"PeriodicalIF":1.9,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11363175/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141859944","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}
引用次数: 0
Patient-reported preferences for subcutaneous or intravenous administration of parenteral drug treatments in adults with immune disorders: a systematic review and meta-analysis. 成人免疫性疾病患者对皮下或静脉注射肠外药物治疗的偏好:系统回顾和荟萃分析。
IF 1.9 4区 医学
Journal of comparative effectiveness research Pub Date : 2024-09-01 Epub Date: 2024-08-08 DOI: 10.57264/cer-2023-0171
Vera Bril, Johannes Lampe, Nichola Cooper, Peter Kiessling, Ann Gardulf
{"title":"Patient-reported preferences for subcutaneous or intravenous administration of parenteral drug treatments in adults with immune disorders: a systematic review and meta-analysis.","authors":"Vera Bril, Johannes Lampe, Nichola Cooper, Peter Kiessling, Ann Gardulf","doi":"10.57264/cer-2023-0171","DOIUrl":"10.57264/cer-2023-0171","url":null,"abstract":"<p><p><b>Aim:</b> Several studies have found subcutaneous (SC) and intravenous (IV) administration of similar drugs for long-lasting immunological and autoimmune diseases to have similar clinical effectiveness, meaning that what patients report they prefer is, or should be, a major factor in treatment choices. Therefore, it is important to systematically compile evidence regarding patient preferences, treatment satisfaction and health-related quality of life (HRQL) using SC or IV administration of the same drug. <b>Materials & methods:</b> PubMed database searches were run on 15 October 2021. Studies involving patients with experience of both home-based SC and hospital-based IV administration of immunoglobulins or biological therapies for the treatment of any autoimmune disease or primary immunodeficiencies (PIDs) were included. The outcomes assessed were patient preferences, treatment satisfaction and HRQL. Preference data were meta-analyzed using a random-effects model. <b>Results:</b> In total, 3504 citations were screened, and 46 publications describing 37 studies were included in the review. There was a strong overall preference for SC over IV administration, with similar results seen for PIDs and autoimmune diseases: PID, 80% (95% confidence interval [CI], 64-94%) preferred SC; autoimmune diseases, 83% (95% CI: 73-92%); overall, 82% (95% CI: 75-89%). The meta-analysis also found that 84% (95% CI: 75-92%) of patients preferred administration at home to treatment in hospital. Analysis of treatment satisfaction using the life quality index found consistently better treatment interference and treatment setting scores with SC administration than with IV administration. <b>Conclusion:</b> Compared with IV infusions in hospital, patients tend to prefer, to be more satisfied with and to report better HRQL with SC administration of the same drug at home, primarily due to the greater convenience. This study contributes to evidence-based care of patients with autoimmune diseases or PIDs.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e230171"},"PeriodicalIF":1.9,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11363182/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141901987","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}
引用次数: 0
Efficacy of CARVYKTI in CARTITUDE-4 versus other conventional treatment regimens for lenalidomide-refractory multiple myeloma using inverse probability of treatment weighting. CARTITUDE-4中的CARVYKTI治疗来那度胺难治性多发性骨髓瘤的疗效与其他常规治疗方案的疗效对比,采用反治疗概率加权法。
IF 1.9 4区 医学
Journal of comparative effectiveness research Pub Date : 2024-09-01 Epub Date: 2024-08-20 DOI: 10.57264/cer-2024-0080
Winfried Alsdorf, Joris Diels, Francesca Ghilotti, João Mendes, Teresa Hernando, Patricia Cost, Jordan M Schecter, Nikoletta Lendvai, Nitin Patel, Ana Triguero, Margherita Ursi
{"title":"Efficacy of CARVYKTI in CARTITUDE-4 versus other conventional treatment regimens for lenalidomide-refractory multiple myeloma using inverse probability of treatment weighting.","authors":"Winfried Alsdorf, Joris Diels, Francesca Ghilotti, João Mendes, Teresa Hernando, Patricia Cost, Jordan M Schecter, Nikoletta Lendvai, Nitin Patel, Ana Triguero, Margherita Ursi","doi":"10.57264/cer-2024-0080","DOIUrl":"10.57264/cer-2024-0080","url":null,"abstract":"<p><p><b>Aim:</b> The phase III randomized controlled trial (RCT) CARTITUDE-4 (NCT04181827) demonstrated superiority of CARVYKTI (ciltacabtagene autoleucel; cilta-cel) over daratumumab, pomalidomide and dexamethasone (DPd) and pomalidomide, bortezomib and dexamethasone (PVd) for relapsed/refractory multiple myeloma (RRMM) patients who have received one to three prior line(s) of therapy (LOT[s]) including an immunomodulatory agent and a proteasome inhibitor, and are refractory to lenalidomide. These analyses estimate the relative efficacy between cilta-cel and other common treatment regimens, for which no direct comparative evidence is available. <b>Materials & methods:</b> Patient data were available from the CARTITUDE-4, CASTOR, CANDOR and APOLLO RCTs. Imbalances between cohorts on key patient characteristics were adjusted for using inverse probability of treatment weighting (IPTW). Relative efficacies were estimated with response rate ratios (RRs) and 95% confidence intervals (CIs) for overall response rate (ORR), very good partial response or better rate (≥VGPR) and complete response or better rate (≥CR), and with hazard ratios (HRs) and 95% CIs for progression-free survival (PFS). Sensitivity analyses using different analytical methods and additional covariates were explored. <b>Results:</b> Key characteristics were well balanced across cohorts after IPTW. Cilta-cel showed statistically significant benefit in PFS (HRs: 0.11-0.51), ≥VGPR (RRs: 1.51-5.13) and ≥CR (RRs: 2.90-35.24) versus all comparators, and statistically significant improvements in ORR over most comparator regimens (RRs: 1.22-1.90). Results were consistent across sensitivity analyses. <b>Conclusion:</b> Cilta-cel demonstrated benefit over other common treatment regimens, highlighting its potential to become a new standard of care option for lenalidomide-refractory RRMM patients with one to three prior LOT(s). These comparisons help to demonstrate the improved efficacy of cilta-cel in countries where the standard of care may differ from DPd/PVd.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240080"},"PeriodicalIF":1.9,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11363176/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142004379","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}
引用次数: 0
Why is target trial emulation not being used in health technology assessment real-world data submissions? 为什么健康技术评估真实世界数据提交中没有使用目标试验模拟?
IF 1.9 4区 医学
Journal of comparative effectiveness research Pub Date : 2024-08-01 Epub Date: 2024-06-08 DOI: 10.57264/cer-2024-0091
Alejandra Castanon, Stephen Duffield, Sreeram Ramagopalan, Robert Reynolds
{"title":"Why is target trial emulation not being used in health technology assessment real-world data submissions?","authors":"Alejandra Castanon, Stephen Duffield, Sreeram Ramagopalan, Robert Reynolds","doi":"10.57264/cer-2024-0091","DOIUrl":"10.57264/cer-2024-0091","url":null,"abstract":"","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240091"},"PeriodicalIF":1.9,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11284816/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141288183","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}
引用次数: 0
Adverse event costs of systemic therapies for metastatic colorectal cancer previously treated with fluoropyrimidine-, oxaliplatin- and irinotecan-based chemotherapy and biologics in the US. 美国既往接受过氟嘧啶、奥沙利铂和伊立替康化疗和生物制剂治疗的转移性结直肠癌患者接受系统疗法的不良事件成本。
IF 1.9 4区 医学
Journal of comparative effectiveness research Pub Date : 2024-08-01 Epub Date: 2024-07-08 DOI: 10.57264/cer-2024-0084
Victoria Federico Paly, Arvind Dasari, Joleen Hubbard, Tanios Bekaii-Saab, Thihan Padukkavidana, Luis Hernandez
{"title":"Adverse event costs of systemic therapies for metastatic colorectal cancer previously treated with fluoropyrimidine-, oxaliplatin- and irinotecan-based chemotherapy and biologics in the US.","authors":"Victoria Federico Paly, Arvind Dasari, Joleen Hubbard, Tanios Bekaii-Saab, Thihan Padukkavidana, Luis Hernandez","doi":"10.57264/cer-2024-0084","DOIUrl":"10.57264/cer-2024-0084","url":null,"abstract":"<p><p><b>Aim:</b> The objective of this study was to compare adverse event (AE) management costs for fruquintinib, regorafenib, trifluridine/tipiracil (T/T) and trifluridine/tipiracil+bevacizumab (T/T+bev) for patients with metastatic colorectal cancer (mCRC) previously treated with at least two prior lines of therapy from the US commercial and Medicare payer perspectives. <b>Materials & methods:</b> A cost-consequence model was developed to calculate the per-patient and per-patient-per-month (PPPM) AE costs using rates of grade 3/4 AEs with incidence ≥5% in clinical trials, event-specific management costs and duration treatment. Anchored comparisons of AE costs were calculated using a difference-in-differences approach with best supportive care (BSC) as a common reference. AE rates and treatment duration were obtained from clinical trials: FRESCO and FRESCO-2 (fruquintinib), RECOURSE (T/T), CORRECT (regorafenib) and SUNLIGHT (T/T, T/T+bev). AE management costs for the commercial and Medicare perspectives were obtained from publicly available sources. <b>Results:</b> From the commercial perspective, the AE costs (presented as per-patient, PPPM) were: $4015, $1091 for fruquintinib (FRESCO); $4253, $1390 for fruquintinib (FRESCO-2); $17,110, $11,104 for T/T (RECOURSE); $9851, $4691 for T/T (SUNLIGHT); $8199, $4823 for regorafenib; and $11,620, $2324 for T/T+bev. These results were consistent in anchored comparisons: the difference-in-difference for fruquintinib based on FRESCO was -$1929 versus regorafenib and -$11,427 versus T/T; for fruquintinib based on FRESCO-2 was -$2257 versus regorafenib and -$11,756 versus T/T. Across all analyses, results were consistent from the Medicare perspective. <b>Conclusion:</b> Fruquintinib was associated with lower AE management costs compared with regorafenib, T/T and T/T+bev for patients with previously treated mCRC. This evidence has direct implications for treatment, formulary and pathways decision-making in this patient population.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240084"},"PeriodicalIF":1.9,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11284812/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141558821","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}
引用次数: 0
Serious treatment-emergent adverse events in chronic low back pain patients treated with buprenorphine or oral opioids: a retrospective commercial claims analysis. 使用丁丙诺啡或口服阿片类药物治疗慢性腰背痛患者的严重治疗突发不良事件:一项回顾性商业索赔分析。
IF 1.9 4区 医学
Journal of comparative effectiveness research Pub Date : 2024-08-01 Epub Date: 2024-07-16 DOI: 10.57264/cer-2023-0183
Filip Stanicic, Dimitrije Grbic, Djurdja Vukicevic, Vladimir Zah
{"title":"Serious treatment-emergent adverse events in chronic low back pain patients treated with buprenorphine or oral opioids: a retrospective commercial claims analysis.","authors":"Filip Stanicic, Dimitrije Grbic, Djurdja Vukicevic, Vladimir Zah","doi":"10.57264/cer-2023-0183","DOIUrl":"10.57264/cer-2023-0183","url":null,"abstract":"<p><p><b>Aim:</b> Explore the safety of Belbuca® (buprenorphine buccal film), buprenorphine transdermal patches and oral opioids for chronic low back pain (cLBP) treatment. <b>Methods:</b> The retrospective analysis of the MarketScan Commercial database (2018-2021) included treatment-naive cLBP adults. The first date of buprenorphine (Belbuca and transdermal patch) or opioid prescription was index date. Cohorts were defined based on the index medication. Observation included a 6-month pre-index period, while post-index lasted until the end of continuous insurance coverage. There were 44 relevant treatment-emergent adverse events (TEAEs) identified in the literature. Incidence rate ratio (IRR) and incidence rate difference (IRD) were used to compare serious TEAE rates (in 1000 person-years) between cohorts. Propensity-score matching minimized the selection bias. <b>Results:</b> Buprenorphine had lower rates of 15 serious TEAEs than oral opioids (all p ≤ 0.037), while higher rates only for serious dizziness (IRR 2.44, p = 0.011; driven by Belbuca), opioid abuse/dependence (IRR 3.13, p = 0.004; driven by patches) and cholecystitis (IRD 20.25, p = 0.044; an outlier). Additionally, a comparison between Belbuca and oral opioids showed lower rates of 13 serious TEAEs (all p ≤ 0.024) and a higher serious dizziness rate (IRR 3.17, p = 0.024). Although the rates of serious opioid abuse/dependence were similar (24.60 vs 26.93, p = 0.921), all Belbuca patients and none of the opioid patients had a positive history of these events. Belbuca also had lower rates of five serious TEAEs than transdermal patches (all p ≤ 0.018), including a serious opioid abuse/dependence (IRR 0.04, p < 0.001), but higher rates of serious cholecystitis (IRD 52.17, p = 0.035; an outlier) and suicidal ideation (IRD 156.50, p < 0.001; an outlier). <b>Conclusion:</b> Buprenorphine had a better safety profile than oral opioids in cLBP treatment. Belbuca showed a more favorable TEAE profile than buprenorphine transdermal patches and oral opioids.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e230183"},"PeriodicalIF":1.9,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11284811/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141620047","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}
引用次数: 0
The value of evidence and its role in driving product strategy. 证据的价值及其在推动产品战略中的作用。
IF 1.9 4区 医学
Journal of comparative effectiveness research Pub Date : 2024-08-01 Epub Date: 2024-07-01 DOI: 10.57264/cer-2024-0074
Melvin Skip Olson, Gorana Capkun
{"title":"The value of evidence and its role in driving product strategy.","authors":"Melvin Skip Olson, Gorana Capkun","doi":"10.57264/cer-2024-0074","DOIUrl":"10.57264/cer-2024-0074","url":null,"abstract":"","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240074"},"PeriodicalIF":1.9,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11284809/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141468468","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}
引用次数: 0
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