Journal of comparative effectiveness research最新文献

{"title":"Economic impact of reduced postoperative visits after inflatable penile prosthesis implantation.","authors":"Bradley Gill, Young Eun Shin, Kathryn Durand, Andrew Sun, Paurush Babbar, Sirikan Rojanasarot","doi":"10.57264/cer-2024-0204","DOIUrl":"10.57264/cer-2024-0204","url":null,"abstract":"<p><p><b>Aim:</b> This study assessed the economic impact of reducing one postoperative visit following inflatable penile prosthesis (IPP) implantation. <b>Methods:</b> Scenario analyses were used to model the effects of eliminating one 30-min IPP postoperative visit from the expected 2.5 visits accounted for by the American Medical Association resource-based relative value scale data. The reduction was attributed to simplified teaching with a modified device. The recaptured time was applied to: the most frequent in-office CPT codes utilized by IPP implanters; evaluation and management of new ED patients pursuing/receiving IPPs; and in-office vasectomy. Physician work time and reimbursement were conservatively estimated using the 2024 Medicare Physician Fee Schedule and an alternative scenario where Advanced Practice Providers conducted IPP teaching was also modeled. <b>Results:</b> Annually, reducing one 30-min IPP postoperative visit for practices performing 25/50/100 IPP implants recaptured 750/1500/3000 min, respectively. This recaptured time translates into as much as $18,325 additional annual Medicare reimbursement. At 25 implants yearly, urologists could help an additional 13-25 patients with office visits and observe an additional $2049-$2270 reimbursement. At 50 implants yearly, office evaluation and counseling for 7 ED patients who progress to IPP implantation results in an additional $4125 reimbursement, excluding any diagnostic procedures and/or downstream surgical cases. At 100 implants yearly, recaptured schedule capacity can facilitate 37 in-office vasectomies, which translates to a $12,563 reimbursement. <b>Conclusion:</b> Achieving fewer IPP postoperative visits can optimize postoperative care and open schedule capacity that improves access to care for patients with urological needs.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240204"},"PeriodicalIF":1.9,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11864081/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142983805","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":"Corrigendum.","authors":"","doi":"10.57264/cer-2024-0241","DOIUrl":"10.57264/cer-2024-0241","url":null,"abstract":"","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":"14 3","pages":"e240241"},"PeriodicalIF":1.9,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11866415/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143449216","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 impact of a patient advisory board on a clinical comparative effectiveness trial: a comparison of patient and researcher perspectives.","authors":"Laura M Kernan, Monica Baczko Pearl, Adina Harri, Carol A Lambourne, Robert Schlegel, C McCollister Evarts, Mary Beth Crummer, Conrad Persels, Nancy Mullen, Vincent D Pellegrini","doi":"10.57264/cer-2024-0050","DOIUrl":"10.57264/cer-2024-0050","url":null,"abstract":"<p><p><b>Aim:</b> To examine contributions of a patient advisory board (PAB) to the design and conduct of The Pulmonary Embolism Prevention after Hip and Knee Replacement (PEPPER) Trial (NCT02810704) and compare perceptions of PAB members and researchers on the Trial. <b>Materials & methods</b> This evaluation of the PAB was conducted by Clinical Coordinating Center (CCC) members who first discussed PAB contributions, leading to the design of a semi-structured WebEx interview individually querying PAB members on their experience. Two study team members analyzed transcriptions of the interviews for common themes, which were discussed and affirmed at an in-person meeting with PAB members. <b>Results:</b> The contribution most frequently cited as meaningful by PAB members was the creation of a recruitment video. In contrast, the research team considered the most impactful PAB recommendation to be omission of pneumatic compression boots as a study variable. PAB members spoke highly of their involvement in the trial and emphasized shared decision-making in the patient-physician relationship. <b>Conclusion:</b> Researchers and PAB members had different opinions about which PAB contributions were most impactful to the study. This likely derives from differences in perspective; PAB members focused on patient experience and the patient-surgeon relationship while researchers focused primarily on trial outcomes. PAB contributions led to two major protocol changes that had a substantial positive effect on trial design, recruitment and enrollment. This evaluation adds to the engagement literature, which contains little on what patients think of their involvement in the design and conduct of clinical research studies and will aid in encouraging treatment preference discussions between patient and surgeon, thereby supporting the goal of improved patient outcomes.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240050"},"PeriodicalIF":1.9,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11864086/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143066003","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":"Clinical characteristics and treatment outcomes in multiple sclerosis patients treated with anti-CD20s who switched to fumarates: a retrospective analysis of a US healthcare claims database.","authors":"Aliza B Ben-Zacharia, Jenny J Feng, Brandon P Moss, Nicholas Belviso, Yu Zhang, Filipe Branco, Jason P Mendoza, James B Lewin, Sarah M England","doi":"10.57264/cer-2024-0071","DOIUrl":"10.57264/cer-2024-0071","url":null,"abstract":"<p><p><b>Aim:</b> Anti-CD20 monoclonal antibodies and fumarates are common multiple sclerosis (MS) disease-modifying therapies (DMTs). Data on switching from anti-CD20s to other DMTs are limited. This retrospective, observational study of the US Komodo Health Sentinel claims database aimed to evaluate a de-escalation strategy in a real-world cohort, comparing clinical characteristics, relapses, healthcare encounters (HCEs) and healthcare costs (HCCs) between patients aged ≥18 years with stable MS who switched from anti-CD20s to fumarates ('Switchers') versus patients who stayed on anti-CD20s ('Stayers'). <b>Materials & methods:</b> Patients with MS (diagnosed 1 January 2015-31 August 2022) were propensity score matched 5:1 (Stayers:Switchers) and followed from index to end of study; end of insurance eligibility; >45-day gap in index DMT; or DMT switch. Primary outcomes were clinical characteristics and claims-based annualized relapse rate (ARR). Rates of HCEs and HCCs were estimated. <b>Results:</b> Baseline characteristics were well balanced between cohorts (Stayers, n = 540; Switchers, n = 108). Mean (SD) duration of post-index follow-up was 341.4 (250.0) days for both cohorts. Mean (SD) ARR was 0.08 (0.41; Stayers) versus 0.14 (0.5; Switchers; p = 0.3). Twenty-one Stayers (3.9%) and 1 Switcher (0.9%) were hospitalized for infections, with mean stays of 9.9 and 1 day, respectively. Mean annualized all-cause HCEs were similar between cohorts; annualized inpatient infection-related HCEs were higher for Stayers versus Switchers (mean difference: -0.05; p = 0.005). Annualized all-cause HCCs were similar between cohorts; Switchers had lower annualized infection-related HCCs overall (mean difference: -$2412; p = 0.002) and in the inpatient setting (mean difference: -$2325; p = 0.002). <b>Conclusion:</b> After 1 year, no significant differences in ARR emerged between cohorts. Switchers experienced lower inpatient infection-related HCEs, shorter inpatient infection-related hospital stays and lower overall infection-related HCCs.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240071"},"PeriodicalIF":1.9,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11864085/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143399374","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":"Efficacy of treatment options for complicated urinary tract infections including acute pyelonephritis: a systematic literature review and network meta-analysis.","authors":"Florian Wagenlehner, Verónica Rico Caballero, Vikalp Maheshwari, Ayantika Biswas, Priyanka Saini, Juan Quevedo, Juergen Polifka, Leonardo Ruiz, Sandrine Cure","doi":"10.57264/cer-2024-0214","DOIUrl":"10.57264/cer-2024-0214","url":null,"abstract":"<p><p><b>Aim:</b> Compared with uncomplicated urinary tract infections (UTIs), complicated UTIs (cUTIs) including acute pyelonephritis (AP) present with significant morbidity, a higher risk of treatment failure and typically require longer courses of treatment, or alternative antibiotics. The emergence of drug-resistant organisms represents a considerable challenge in the treatment of patients with cUTIs/AP and has limited antibiotic options. Carbapenems are considered the current last line of therapy, however, carbapenem resistance represents a growing problem. Although several established and novel treatment options are available, direct comparative evidence is lacking. <b>Methods:</b> Randomized controlled trials (RCTs) were identified by systematic literature review of Embase^®, MEDLINE^® and Cochrane databases (database inception to 15th June 2022). Relevant conference proceedings (2020-2022) were also reviewed. Following feasibility assessment to verify network connectivity at an overall level, outcome specific networks were prepared. Bayesian network meta-analysis (NMA) was performed (using R version 4.2.1) to determine the relative efficacy of various treatments for cUTI/AP, including cefepime + enmetazobactam. Convergence was assessed by visual inspection of trace plots. The accuracy of the posterior estimates was assessed using the Monte Carlo error for each parameter. Published study results were included in the synthesis of the relative risk (RR) of efficacy end points, using a logit link with binomial likelihood distribution. <b>Results:</b> Feasibility assessment was conducted for 40 RCTs identified, to assess the viability of constructing a network of interlinked RCTs. Of those, 28 studies were included in the master NMA network. A fixed effects model (FEM) was selected due to low statistical heterogeneity, according to I² values. For composite outcome at test of cure (TOC), ceftolozane + tazobactam, cefepime + enmetazobactam, cefiderocol, levofloxacin and plazomicin demonstrated significantly higher RRs versus carbapenems. For microbiological eradication at TOC, cefepime + enmetazobactam, plazomicin, cefiderocol, fosfomycin, meropenem + vaborbactam and ceftazidime + avibactam demonstrated significantly higher RRs versus carbapenems. RRs for cefepime + enmetazobactam were also significantly higher versus several established and novel treatment options for composite outcome, microbiological eradication and clinical cure. <b>Conclusion:</b> Against the backdrop of increasing bacterial resistance, these findings suggest that cefepime + enmetazobactam may represent an effective carbapenem-sparing treatment option in patients with cUTI including AP.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240214"},"PeriodicalIF":1.9,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11864080/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143006384","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":"Mapping methods gaps between EU joint clinical assessments and local health technology assessment decision-making: an environmental scan of guidance in select EU markets and harmonization challenges.","authors":"Grammati Sarri, Lydia Vinals, Lilia Leisle, Ingrid Claverie Chau, David Smalbrugge, Kai Lucassen, Yannis Jemiai","doi":"10.57264/cer-2024-0240","DOIUrl":"https://doi.org/10.57264/cer-2024-0240","url":null,"abstract":"<p><p><b>Aim:</b> Under the newly instituted health technology assessment (HTA) regulation (HTAR), health technology developers must build evidence packages that meet the needs for both the upcoming EU joint clinical assessment (JCA) and national decision-making. In-depth knowledge of local methodological requirements as well as preparedness for effective strategic development is crucial. This study aimed to review methodological guidance documents to map similarities/misalignments between the EU HTAR and select HTA agencies. <b>Materials & methods:</b> An environmental scan was performed in March 2024 and updated in December 2024 of the websites for European Network for HTA, the European Commission and HTA agencies in France, Germany, The Netherlands and Spain. The search aimed to systematically identify and summarize methodological guidance documents from the respective organizations on scoping considerations, evidence identification and synthesis. <b>Results:</b> Overall, published EU HTAR methods guidelines are detailed, prescriptive and make reference to a preference (or lack thereof) for specific analytical methods. There was consensus among EU JCA and local HTA guidelines that clinical comparative assessments should be based on a systematically identified, unbiased selected evidence base derived from various sources. However, agencies differed on guidance related to evidence derived from indirect treatment comparisons. <b>Conclusion:</b> An environmental scan of methods documents revealed that it will likely be challenging for health technology developers to build strong evidence packages that can support both EU JCA and local reimbursement decision-making. A greater understanding of the similarities and differences between EU and local HTA requirements will be needed, including a greater capacity to demonstrate value through advanced analytics.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240240"},"PeriodicalIF":1.9,"publicationDate":"2025-02-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143501861","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}