Journal of comparative effectiveness research最新文献

{"title":"Evaluation of reporting in time-driven activity-based costing studies on cardiovascular diseases: a scoping review.","authors":"Nayê Balzan Schneider, Erica Caetano Roos, Miriam Allein Zago Marcolino, Fabio Caldana, Filipe Rodrigues Vargas do Nascimento, Sérgio Renato da Rosa Decker, Ana Paula Beck da Silva Etges, Carisi Anne Polanczyk","doi":"10.57264/cer-2024-0013","DOIUrl":"https://doi.org/10.57264/cer-2024-0013","url":null,"abstract":"<p><p><b>Aim:</b> This scoping review evaluates the application of the time-driven activity-based costing (TDABC) methodology in cardiovascular disease (CVD) studies. <b>Materials & methods:</b> The evaluation was conducted using the 32-item TDABC Healthcare Consortium Consensus Statement Checklist. A systematic search was performed in Medline, Embase and Scopus in September 2023, including only full-text, peer-reviewed studies reporting the application of TDABC in CVD research. <b>Results:</b> Twenty studies were included in the review. The positive response rate for individual studies ranged from 31 to 81%. The most frequently addressed checklist item was the clear definition of study objectives, while presenting costs per patient included in the analysis was the least reported item. Although 70% of the studies achieved a positive response rate above 50%, adherence to the TDABC checklist remains inconsistent. <b>Conclusion:</b> There is significant room for improvement in the reporting of TDABC methodology in CVD studies. Providing a more comprehensive and standardized description of the methodology would enhance the utility, reproducibility and accuracy of the information generated, supporting the development of evidence-based health policies and improving accountability in healthcare cost assessments.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240013"},"PeriodicalIF":1.9,"publicationDate":"2025-02-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143501842","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":"Matching-adjusted indirect comparisons of efficacy outcomes between etrasimod and ozanimod for moderately to severely active ulcerative colitis.","authors":"Vipul Jairath, Tim Raine, Thomas P Leahy, Ravi Potluri, Karolina Wosik, David Gruben, Joseph C Cappelleri, Peter Hur, Lauren Bartolome","doi":"10.57264/cer-2024-0193","DOIUrl":"https://doi.org/10.57264/cer-2024-0193","url":null,"abstract":"<p><p><b>Aim:</b> Etrasimod and ozanimod are selective sphingosine 1-phosphate receptor modulators targeting the S1P_1,4,5, and S1P_1,5 receptors, respectively, for the treatment of patients with moderately to severely active ulcerative colitis (UC). No head-to-head trial data exist between the two treatments. We compared these treatments indirectly using key efficacy outcomes from pivotal trials with induction and maintenance phase data adjusting for differences in clinical trial design and populations. <b>Materials & methods:</b> Individual patient data for etrasimod were matched to published aggregate data of ozanimod by key baseline characteristics. An anchored matching-adjusted indirect comparison (MAIC) was conducted for the induction period. An unanchored MAIC was utilized during the maintenance period due to differences in placebo arms between trials as a result of differing trial designs. Matching characteristics measured at baseline were age, sex, corticosteroid use, duration of UC, biologic exposure, modified Mayo score, and presence of left-sided colitis. Outcomes were clinical response and clinical remission for the induction period, and clinical response and clinical remission among induction phase responders for the maintenance period. Two sensitivity analyses were conducted. The first matched on prior TNFi exposure rather than biologic exposure, the second sensitivity analysis included an induction only etrasimod trial (ELEVATE UC 12). <b>Results:</b> There were no significant differences between etrasimod and ozanimod at the end of the induction period for clinical response and clinical remission, respectively (relative risk [RR] 0.98 [95% confidence interval (CI): 0.76-1.33], RR: 1.25 [95% CI: 0.71-2.92]). At the end of maintenance, etrasimod demonstrated improved outcomes compared with ozanimod for both clinical response (RR: 1.18 [95% CI: 1.05-1.30]) and clinical remission among induction phase responders (RR: 1.33 [95% CI: 1.12-1.55]). In the sensitivity analysis that matched on prior TNFi exposure rather than biologic exposure, there were no notable differences compared with the primary analyses. In the sensitivity analysis pooling ELEVATE UC 12 and ELEVATE UC 52 data, results were similar for clinical response (RR: 0.90 [95% CI: 0.75-1.10]) but etrasimod showed reduced efficacy for clinical remission (RR: 0.72 [95% CI: 0.50-1.12]) compared with the primary analysis, though overall remained not significantly different from ozanimod. <b>Conclusion:</b> MAIC results suggest that patients receiving etrasimod have similar induction results but are more likely to have clinical response and clinical remission at the end of the maintenance phase compared with patients receiving ozanimod. Despite the approach to ensure similarity between the trials by weighting, residual imbalance is possible, and results should be interpreted in the context of the assumptions.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240193"},"PeriodicalIF":1.9,"publicationDate":"2025-02-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143483330","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":"Comparison of real-world healthcare resource utilization and costs among patients with hereditary angioedema on lanadelumab or berotralstat long-term prophylaxis.","authors":"Nicole Princic, Kristin A Evans, Chintal H Shah, Krystal Sing, Salomé Juethner, Bob G Schultz","doi":"10.57264/cer-2024-0205","DOIUrl":"https://doi.org/10.57264/cer-2024-0205","url":null,"abstract":"<p><p><b>Aim:</b> Hereditary angioedema (HAE) is a rare and chronic genetic condition. Lanadelumab and berotralstat, two plasma kallikrein inhibitors, have both been approved for long-term prophylaxis in patients with HAE; however, real-world data comparing costs and healthcare resource utilization (HCRU) are lacking. <b>Materials & methods:</b> This retrospective study used administrative healthcare insurance claims data (Merative™ MarketScan^® Commercial, Medicare and Early View Research Databases; 1 July 2017-31 July 2023) to identify patients with HAE who initiated lanadelumab or berotralstat and were persistent for ≥18 months or 6 months, respectively. Sex, baseline healthcare costs and baseline number of on-demand treatment/short-term prophylaxis medication claims were used to calculate covariate balancing propensity scores for inverse probability of treatment weighting. Following weighting, outcomes during the 6-month follow-up period in patients receiving berotralstat were compared with those during months 0-6, 7-12 and 13-18 in lanadelumab-treated patients. <b>Results:</b> Fifty-seven lanadelumab- and 32 berotralstat-treated patients were included. After weighting, more berotralstat-treated patients had an all-cause inpatient admission (berotralstat, 9.4%; lanadelumab, months 0-6, 4.0%, 7-12, 1.8%, months 13-18, 2.0%) and emergency room visit (berotralstat, 21.9%; lanadelumab, months 0-6, 14.0%, 7-12, 8.0%, months 13-18, 17.9%). Total HAE treatment costs were similar during months 0-6 (lanadelumab, $377,326 vs berotralstat, $373,010), but decreased in months 7-12 ($319,967) and 13-18 ($283,241) of lanadelumab. On-demand treatment/short-term prophylaxis costs were lower for lanadelumab across the three follow-up periods than for berotralstat during months 0-6 (berotralstat, $60,451; lanadelumab, months 0-6, $46,336, months 7-12, $37,578, months 13-18, $23,968). The proportion of lanadelumab-treated patients who reduced dosing frequency was 24.8% during months 7-12 and 21.6% during months 13-18. <b>Conclusion:</b> Patients with HAE initiating lanadelumab versus berotralstat may require less on-demand and supportive HAE treatments and incur lower treatment-related and total healthcare costs. The ability to reduce lanadelumab dosing frequency after an attack-free period may be key in treatment selection, given the combination of cost savings and lower healthcare resource utilization.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240205"},"PeriodicalIF":1.9,"publicationDate":"2025-02-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143458260","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The role of health economic evidence in clinical practice guidelines for colorectal cancer: a comparative analysis across countries.","authors":"Xiaoyu Yan, Yue Wang, Aixia Ma, Hongchao Li","doi":"10.57264/cer-2024-0226","DOIUrl":"https://doi.org/10.57264/cer-2024-0226","url":null,"abstract":"<p><p><b>Aim:</b> Colorectal cancer (CRC) is among the most prevalent malignancies globally and causes massive resource consumption and economic burden. Health economic evidence (HEE) has been used in clinical practice guidelines (CPGs) for cancer to facilitate the rational allocation of health resources. However, in certain guideline development organizations, HEE is not yet utilized as a formal decision-making criterion. This study aimed to compare the discrepancies in the utilization of health economics as evidence in CRC CPGs across different countries and review specific features of economic evidence concerning the guidelines' applicability. <b>Materials & methods:</b> A systematic review was conducted using databases including Medline, Embase, CNKI, WanFang, and other guidelines databases to identify CPGs for CRC published in English or Chinese from January 2017 to September 2023. Data on the incorporation and application of HEE were extracted, and the method and quality of cost-effectiveness analysis (CEA) studies were evaluated. Descriptive analyses were used to summarize the results. <b>Results:</b> Out of 53 CPGs from 14 countries, most originated from the USA (n = 17 of 53 [32%]) and Canada (n = 9 of 53 [17%]). Sixty-eight percent (36/53) considered cost justification, and 57% (30/53) incorporated health economics studies as evidence. The included HEE cited in CPGs ranged from 1990 to 2021 and were not aligned with the countries in which the guidelines were issued. Among these CEA studies, 52% (26/50) were related to screening strategies, and 32% (16/50) pertained to treatment measures. The Markov model was the most frequently used (n = 27 of 50 [54%]). Based on the CHEQUE tool, the methodological quality of these CEA studies was inadequate in areas such as multiple data sources, approaches to select data sources, assessing the quality of data, and relevant equity or distribution. <b>Conclusion:</b> In summary, 57% of guidelines incorporated health economics studies as evidence, with a variation between different countries. The included HEE still had deficiencies in methodology and reporting quality. In the future, it is suggested that health economics research should use a standardized methodology and reporting approach to assist in clinical decision making.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240226"},"PeriodicalIF":1.9,"publicationDate":"2025-02-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143449213","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 18.","authors":"Paul Arora, Sreeram V Ramagopalan","doi":"10.57264/cer-2025-0014","DOIUrl":"https://doi.org/10.57264/cer-2025-0014","url":null,"abstract":"<p><p>In this update, we discuss recent publications examining the use of real-world data as a measure of treatment effectiveness in submissions to the National Institute of Health and Care Excellence, the results of a pilot study from the Coalition to Advance Real-World Evidence initiative and a validation study of synthetic data.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e250014"},"PeriodicalIF":1.9,"publicationDate":"2025-02-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143441107","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":"Eculizumab or ravulizumab treatment effect in people with neuromyelitis optica spectrum disorder: a plain language summary of three studies.","authors":"Alfredo Damasceno, Mariano Marrodan","doi":"10.57264/cer-2024-0177","DOIUrl":"https://doi.org/10.57264/cer-2024-0177","url":null,"abstract":"<p><strong>What is this summary about?: </strong>Neuromyelitis optica spectrum disorder (NMOSD for short) is a rare autoimmune health condition, meaning that the body's natural defense system (the immune system) attacks the body's own tissues. This summary describes NMOSD and the results of three studies of the effects of treatment with two medicines called eculizumab and ravulizumab. Eculizumab and ravulizumab are approved to treat people with a type of NMOSD called AQP4-Ab+ NMOSD. The three studies included in this summary are the PREVENT and CHAMPION-NMOSD clinical studies and a study done in everyday clinical practice in Japan.</p><p><strong>What are the key conclusions from these studies?: </strong>The PREVENT study was a phase 3 study (a large study testing safety and effectiveness of a treatment before it is approved) with three parts. The PREVENT main study compared eculizumab to a placebo (a treatment with no active ingredients, used to test how well a new treatment works). A long-term follow-up study assessed the safety and effectiveness of eculizumab over time, without a placebo group. Another long-term follow-up focused on people taking eculizumab alone, without other immunosuppressive treatments. The CHAMPION-NMOSD study was another phase 3 study testing ravulizumab, a treatment based on eculizumab but given less often, using the placebo group from the PREVENT study for comparison. A daily clinical practice study with eculizumab in Japan looked at how eculizumab works in everyday medical practice (outside of controlled clinical trials) after the medication was already approved. The studies found eculizumab and ravulizumab to be safe and effective for preventing relapses in AQP4-Ab+ NMOSD. More than 95% of people treated with these medicines stayed relapse-free during the months or years of the studies follow-up periods. Most people reported side effects that were mild or moderate. The most common side effects were headache, runny nose or sore throat (nasopharyngitis), and infections in the upper respiratory system. The daily practice study confirmed that eculizumab works well in everyday medical practice.</p><p><strong>What do the findings of the study mean?: </strong>These studies suggest that eculizumab and ravulizumab are safe and effective treatments for people with AQP4-Ab+ NMOSD. Eculizumab may help reduce or stop the need for other treatments that weaken the immune system (immunosuppressive therapies). These treatments help people maintain their ability to carry out daily activities and their quality of life.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240177"},"PeriodicalIF":1.9,"publicationDate":"2025-02-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143399377","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":"Real-world studies of crizotinib in patients with ROS1-positive non-small-cell lung cancer: experience from China.","authors":"Hua Zhong, Jun Lu, Mengzhao Wang, Baohui Han","doi":"10.57264/cer-2024-0043","DOIUrl":"10.57264/cer-2024-0043","url":null,"abstract":"<p><p>The treatment of non-small-cell lung cancer (NSCLC) has progressed from histology-oriented cytotoxic therapy to the era of molecular biology-oriented targeted therapy and immunotherapy. As the first tyrosine kinase inhibitor (TKI) targeting the <i>ROS1</i> pathway, crizotinib is widely used as a first-line regimen for <i>ROS1</i>-rearranged NSCLC. However, due to the paucity of solid data from randomized, controlled phase III clinical studies, clinicians often require more systematic, real-world data-based guidance for its optimal clinical use. As one of the leading countries of real-world research on crizotinib, China has contributed significantly to data on standardization of the therapeutic use of crizotinib, including its clinical treatment patterns, the timing and duration of treatment and drug resistance monitoring and management.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240043"},"PeriodicalIF":1.9,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11773893/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142837125","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-effectiveness of cetuximab combined with radiotherapy versus radiotherapy alone in locally advanced head and neck cancer in Spain.","authors":"Ruth Álvarez Cabellos, Jon Cacicedo, Susana Redondo Capafons, Heidi De Los Santos Real, Miriam Brines Julián, Darío Rubio-Rodríguez, Carlos Rubio-Terrés","doi":"10.57264/cer-2024-0116","DOIUrl":"10.57264/cer-2024-0116","url":null,"abstract":"<p><p><b>Aim:</b> To estimate the cost-effectiveness of cetuximab in combination with radiotherapy compared with radiotherapy alone, for the treatment of locally advanced head and neck cancer patients in Spain. <b>Methods:</b> A probabilistic Markov model (second-order Monte Carlo simulation) with a five-year time horizon and quarterly Markov cycles was performed from the perspective of the Spanish National Health System (NHS). <b>Results:</b> The additional cost and quality-adjusted life-year (QALY) gain per patient receiving radiotherapy in combination with cetuximab compared with radiotherapy alone was €4356 (95% CI: €4350-4362) and 0.2380 (95% CI: 0.2370-0.2391) QALY, respectively. The incremental cost per QALY gain was €18,303 (95% CI: €18,243-18,354) with a probability of cost-effectiveness of 65.4% for a willingness to pay of €30,000 per QALY gained. <b>Conclusion:</b> According to the results of this analysis, the addition of cetuximab to radiotherapy would be a cost-effective alternative to radiotherapy alone in the treatment of locally advanced head and neck cancer in Spain.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240116"},"PeriodicalIF":1.9,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11773860/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142949710","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":"The burden of Alagille syndrome: uncovering the potential of emerging therapeutics - a comprehensive systematic literature review.","authors":"Philip Bufler, Robin Howard, Lucia Quadrado, Guy Lacey, Jolan Terner-Rosenthal, Andrea Goldstein, Pamela Vig, Deirdre Kelly","doi":"10.57264/cer-2024-0188","DOIUrl":"10.57264/cer-2024-0188","url":null,"abstract":"<p><p><b>Aim:</b> Alagille syndrome (ALGS) is a rare, cholestatic multiorgan disease associated with bile duct paucity, leading to cholestasis. Clinical symptoms of cholestasis include debilitating pruritus, xanthomas, fat-soluble vitamin deficiencies, growth failure, renal disease and impaired health-related quality of life (HRQoL). The main objective was to review the current literature on the epidemiological, clinical, psychosocial and economic burden of ALGS in view of the development of ileal bile acid transporter (IBAT) inhibitors. <b>Methods:</b> Electronic literature databases were searched in accordance with the Preferred Reporting Items for Systematic Reviews and Meta-Analyses checklist. <b>Results:</b> 330 publications were screened, 119 were relevant: 11 randomized controlled trials (RCTs), 21 non-RCTs, 10 HRQoL studies, two studies assessing cost/resource use and 77 epidemiological studies across several databases through 31 July 2024. Studies confirm that patients with ALGS experience cardiac anomalies, impaired growth, renal disease, poor HRQoL, fat-soluble vitamin deficiencies and debilitating pruritus; until the approval of IBAT inhibitors for the treatment of cholestatic pruritus in patients with ALGS, supportive management was the standard of care. <b>Conclusion:</b> This review confirms the substantial clinical, economic and HRQoL burden associated with ALGS and consolidates current treatment evidence. Data from recent trials in ALGS demonstrate the potential impact of IBAT inhibitors to transform lives by improving cholestatic pruritus symptoms, HRQoL and native liver survival.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240188"},"PeriodicalIF":1.9,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11773862/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142978814","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":"Real-world ePRO use and clinical outcomes using electronic patient-reported symptom monitoring for patients with advanced non-small-cell lung cancer receiving first-line pembrolizumab.","authors":"L Johnetta Blakely, Sabine Oskar, Ian Kudel, Ashley Roush, Zoya Shamsi, Toni Perry, Annette Christianson, Brittni Smith, Thomas Burke","doi":"10.57264/cer-2024-0122","DOIUrl":"10.57264/cer-2024-0122","url":null,"abstract":"<p><p><b>Aim:</b> This ambispective observational study assessed the impact of Noona, an electronic patient-reported outcomes (ePRO) platform, for patients with non-small cell lung cancer (NSCLC) treated in a community oncology setting. <b>Methods:</b> Adults with advanced NSCLC, ECOG performance status of 0-2, who received first-line (1L) pembrolizumab (monotherapy or with chemotherapy) were eligible. Those initiating pembrolizumab from 1 July 2017 to 30 June 2019, identified retrospectively (historical cohort), were compared with those initiating pembrolizumab from 1 October 2019 to 30 September 2021 who were prospectively offered Noona (standard of care [SoC] cohort). The Kaplan-Meier method and Cox proportional hazards models were used to compare pembrolizumab real-world time on treatment (rwToT; primary outcome measure) and rw time to next treatment or death (rwTTNTD) between historical and SoC cohorts. Healthcare resource use (HCRU) was compared using generalized linear models with Poisson distribution. Analyses were repeated to compare outcomes in the SoC cohort between Noona users (created a profile and used any function ≥one-time during 1L therapy) and nonusers with >42 days on 1L pembrolizumab. Data cutoff was 30 June 2020 and 30 September 2022 for historical and SoC cohorts, respectively. <b>Results:</b> Median pembrolizumab rwToT was 4.4 months (95% CI: 3.9-5.1) in the historical cohort (n = 448) versus 4.1 months (95% CI: 3.3-4.8) in the SoC cohort (n = 462; adjusted hazard ratio [aHR], 0.9; 95% CI: 0.8-1.0; p = 0.14 vs historical cohort). In the SoC cohort, 147 of 341 eligible patients (43%) established a Noona profile; 122/341 (36%) were Noona users. Median rwToT was 6.4 months (95% CI: 5.1-7.4) and 6.9 months (95% CI: 5.6-7.6) among Noona users and Noona nonusers (n = 219), respectively (aHR, 1.1; 95% CI: 0.8-1.4; p = 0.95 vs Noona users). The rwTTNTD and HCRU were comparable in historical versus SoC cohorts and for Noona users versus nonusers. During the first year after establishing a Noona profile, 92 of 147 patients (63%) used the platform; monthly use was 32-42%, and checking laboratory results was the most used function overall (by 52% of the 147). <b>Conclusion:</b> Notwithstanding the null findings of this study, positive results of ePRO use in clinical trials and observational studies support the treatment-related symptom monitoring and survival benefits of ePRO utilization.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240122"},"PeriodicalIF":1.9,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11773859/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143006395","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}