Journal of comparative effectiveness research最新文献

{"title":"Corrigendum.","authors":"","doi":"10.57264/cer-2025-0066","DOIUrl":"https://doi.org/10.57264/cer-2025-0066","url":null,"abstract":"","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e250066"},"PeriodicalIF":1.9,"publicationDate":"2025-05-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144021711","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":"Elranatamab versus physician's choice of treatment in patients with triple-class exposed/refractory multiple myeloma: an updated matching-adjusted indirect comparison.","authors":"Isha Mol, Yannan Hu, Thomas W LeBlanc, Joseph C Cappelleri, Haitao Chu, Guido Nador, Didem Aydin, Isabel Perez Cruz, Patrick Hlavacek","doi":"10.57264/cer-2024-0236","DOIUrl":"10.57264/cer-2024-0236","url":null,"abstract":"<p><p><b>Aim:</b> Despite the availability of novel treatment options for patients with triple-class exposed/refractory multiple myeloma , there is a lack of consensus on the optimal regimen. A previous unanchored matching-adjusted indirect comparison (MAIC) of the MagnetisMM-3 (NCT04649359) and LocoMMotion (NCT04035226) study reported significant improvements in progression-free survival (PFS) and overall survival (OS) with elranatamab versus physician's choice of treatment. <b>Materials & methods:</b> We conducted an updated MAIC based on more recent data (28.4 months for MagnetisMM-3). Following reweighting of MagnetisMM-3 individual patient data to match the LocoMMotion population, the effective sample size was 64 for PFS and 63 for OS in the base-case analysis. <b>Results:</b> Consistent with the original MAIC, significantly improved PFS (hazard ratio [HR] [95% confidence interval (CI)]: 0.32 [0.21, 0.50], p < 0.01) and OS (HR [95% CI]: 0.50 [0.33, 0.78], p < 0.01) were observed with elranatamab versus physician's choice of treatment. The robustness of the results was demonstrated in sensitivity analyses in which missing baseline characteristics data for elranatamab were imputed. <b>Conclusion:</b> Overall, the findings of this study align with and add confidence to the conclusions of the previous MAIC that elranatamab is associated with significantly improved outcomes versus standard treatment, supporting the value to elranatamab for the treatment of patients with triple-class exposed/refractory multiple myeloma.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240236"},"PeriodicalIF":1.9,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12007480/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143795541","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":"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":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12007483/pdf/","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":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Effectiveness and safety of asfotase alfa for people with hypophosphatasia: a plain language summary of three studies.","authors":"Fatma Al Jasmi, Zhanna Belaya","doi":"10.57264/cer-2024-0227","DOIUrl":"10.57264/cer-2024-0227","url":null,"abstract":"<p><strong>What is this summary about?: </strong>Hypophosphatasia (HPP for short) is a rare inherited condition that can present at any stage of life, with symptoms typically being more severe in cases that manifest earlier, such as during infancy or childhood. This article summarizes three published studies that looked at the safety and effectiveness of asfotase alfa for people with HPP. The aim of this article is to present the findings of three studies clearly and engagingly to make them accessible to readers without a scientific background. The first study (called study 1) discussed in this article looked at how safe and effective asfotase alfa was for children with life-threatening HPP. The second study (called study 2) looked at how safe and effective asfotase alfa was for children or adolescents who had showed the first signs of HPP at birth or in childhood. They received treatment in clinical practice in Japan, rather than in a clinical research study. The third study (called study 3) looked at how safe and effective asfotase alfa was for adults with HPP who had showed the first signs of the condition in childhood. They received treatment in clinical practice, rather than in a clinical research study.</p><p><strong>What were the results?: </strong>In study 1, asfotase alfa improved bone health, the ability to breathe, and growth in children with life-threatening HPP. The researchers saw these benefits over 6 years of follow-up by HPP care teams. The treatment was generally well-tolerated. In study 2, asfotase alfa relieved symptoms such as pain, improved growth, and improved the ability to breathe in children and adolescents with HPP in Japan. The treatment was generally well-tolerated, with no serious side effects. Some participants had mild reactions at injection sites. In study 3, asfotase alfa improved physical function and quality of life for adults with HPP. People were able to walk further and faster and had improved grip strength. The researchers saw these benefits as early as 3 months, and they continued over 2 years. The treatment was welltolerated. Some people had mild reactions at injection sites.</p><p><strong>What do the results of the studies mean?: </strong>The combined results from these studies indicate that asfotase alfa is effective and safe to treat people with HPP of different ages and levels of symptom severity. Asfotase alfa improves bone health, mobility, growth, and breathing ability, and relieves pain. Treatment can improve quality of life for people with HPP. The findings from these studies support the continued use and further development of asfotase alfa as a treatment for people with HPP.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240227"},"PeriodicalIF":1.9,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12007477/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143663545","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":"Economic burden of sickle cell disease in the United States: a retrospective analysis of a commercial insurance database.","authors":"Giovanna Tedesco Barcelos, Telma Peixoto, Jose Alvir, Jay Lin, Christine L Baker","doi":"10.57264/cer-2025-0006","DOIUrl":"10.57264/cer-2025-0006","url":null,"abstract":"<p><p><b>Aim:</b> To evaluate healthcare resource utilization (HCRU) and costs for US commercially insured adult and pediatric patients with sickle cell disease (SCD) and matched non-SCD cohorts. <b>Materials & methods:</b> Patients with ≥3 SCD diagnosis codes (D57.0-D57.219; D57.4-D57.819) from July 2016 to December 2020 were identified from the IBM^® MarketScan^® Commercial database. The earliest SCD diagnosis was defined as the index date. Non-SCD control patients were matched 1:1 on age, gender and region. Continuous 6-month baseline and ≥12-month follow-up coverage was required. Follow-up HCRU and costs (2020 USD) were calculated per patient per year. Pediatric (<18 years) and adult (≥18 years) patients were analyzed separately. <b>Results:</b> For 1299 pediatric patients with SCD and matched controls, mean (SD) age was 10.0 (4.8) years and 51% were female; mean (SD) follow-up was 34.3 (14.4) months. In the first 12 months, pediatric patients with SCD had higher HCRU (hospitalizations: 0.6 vs 0.01; hospital length of stay: 2.4 vs 0.05 days; outpatient visits: 13.4 vs 6.0; office visits: 6.9 vs 4.7; prescriptions: 12.8 vs 3.8) and mean total costs ($31,445 vs $2844), mainly due to hospitalizations ($15,195 vs $477) and outpatient visits ($12,746 vs $1758), versus controls (all p < 0.0001). For 2792 adults with SCD and matched controls, mean (SD) age was 38.0 (13.2) years and 62% were female; mean (SD) follow-up was 31.8 (13.7) months. Adults with SCD had higher per-patient per-year HCRU (hospitalizations: 0.8 vs 0.06; hospital length of stay: 4.3 vs 0.2 days; outpatient visits: 20.9 vs 9.3; office visits: 10.4 vs 6.9; prescriptions: 20.5 vs 11.7) and mean total costs ($42,550 vs $7522), also due to hospitalizations ($20,056 vs $1326) and outpatient visits ($17,508 vs $4301), versus controls (all p < 0.0001). <b>Conclusion:</b> The economic burden of SCD among pediatric and adult patients is substantial with increased HCRU and costs compared with matched controls. Better treatments for SCD could reduce the economic burden for patients, as well as payers.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e250006"},"PeriodicalIF":1.9,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12007473/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143764157","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":"A process to validate prognostic factors for unanchored matching-adjusted indirect comparison of single-arm trials in oncology: a proof-of-concept study.","authors":"Yao Yi, Yawen Jiang","doi":"10.57264/cer-2024-0235","DOIUrl":"10.57264/cer-2024-0235","url":null,"abstract":"<p><p><b>Aim:</b> The choice of covariates in unanchored matching-adjusted indirect comparisons (MAICs) of single-arm cancer trials with time-to-event outcomes remains a challenge. Currently, there is a lack of a systematic approach for validating the selection of covariates for bias reduction in unanchored MAIC. <b>Materials & methods:</b> This study proposes a validation framework to evaluate the appropriateness of selected prognostic factors before their use in unanchored MAIC. The process involves identifying potential prognostic factors from individual patient data and calculating risk scores using the prognostic factors with regression; artificially creating two groups that are unbalanced in risk such that a predetermined hazard ratio (HR) between the two groups is achieved; creating weights based on the prognostic factors; running a re-weighted Cox regression to assess the HR, the value of which should suggest balanced risks across groups to indicate the sufficiency of prognostic factors being included. We also conducted a proof-of-concept analysis using a simulated dataset to showcase this process. <b>Results:</b> The process successfully stratified the sample into two risk groups with a pre-determined HR of 1.8. When all covariates were included in the weighting, the HR was 0.9157 (95% CI: 0.5629-2.493), which was close to one. When one of the critical prognostic factors was omitted from the covariates, the HR became 1.671 (95% CI: 1.194-2.340), which was significantly different from one. <b>Conclusion:</b> Filling a gap in the existing evidence synthesis literature, the study introduces a structured data-driven approach for covariate prioritization in unanchored MAIC. The process may be a useful tool for quantitative covariate selection.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240235"},"PeriodicalIF":1.9,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12007475/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143795538","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":"Impact analysis of expanding antibiotic use for treatment of uncomplicated appendicitis without appendicolith.","authors":"Diana Poehler, Sydney Kirsch, Matthew Dempsey, Kristen Giombi, Olga Khavjou","doi":"10.57264/cer-2024-0234","DOIUrl":"https://doi.org/10.57264/cer-2024-0234","url":null,"abstract":"<p><p><b>Aim:</b> Approximately 20% of patients with uncomplicated appendicitis receive antibiotics as a first-line treatment; this study explores the impacts of expanding patient uptake of first-line antibiotics for appendicitis treatment. <b>Materials</b> <b>&</b> <b>methods:</b> We model the impacts on the patient, caregiver, payer and total societal costs associated with expanding antibiotics use from 20 to 50% for patients with appendicitis through use of a decision-tree model. <b>Results:</b> Increasing antibiotics uptake to 50% of eligible appendicitis patients is expected to decrease overall societal economic costs by $192 million, and these savings are driven by a $493 million reduction in initial treatment costs. For patients and their caregivers, out-of-pocket costs are expected to decrease by $18 million, appendectomies by 27,410 and missed work hours by 639,682. <b>Conclusion:</b> Increasing national uptake of antibiotics for the treatment of appendicitis may reduce patient, payer and total societal costs with minimal impact to patient health outcomes. An expansion may also decrease the total number of appendectomies, appendectomy-related medical complications, and lost wages for patients and caregivers, with minimal decreases in the number of appendiceal cancer cases treated early.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":"14 5","pages":"e240234"},"PeriodicalIF":1.9,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12007479/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143995018","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 need to consider market access for pharmaceutical investment decisions: a primer.","authors":"Sreeram V Ramagopalan, Catherine Bacon, Mel Walker, Michael L Ryan","doi":"10.57264/cer-2025-0036","DOIUrl":"10.57264/cer-2025-0036","url":null,"abstract":"<p><p>Biotech/Pharma investors employ valuation methods to support capital deployment that consider the costs of drug development, projected sales and risks of failure. Often, the major focus for valuation is placed on the likely success rates of taking a compound from phase I to regulatory approval, with the notion being that just by obtaining regulatory approval sales will follow. However, as exemplified by recent cases with hemophilia gene therapies, achieving forecasted sales depends not only on regulatory success but crucially on market access outcomes. This primer examines how pharmaceutical investment decisions must consider market access factors, particularly in light of recent regulatory changes such as the US Inflation Reduction Act and European Union Joint Clinical Assessment. Effective market access strategies can enhance commercial success through better pricing, broader reimbursement, and/or faster uptake, and having a clear market access plan should encourage investment by providing a clearer path to commercial success. As health technology assessment processes become more sophisticated globally, treating market access as an essential strategic capability rather than a tactical exercise will be important for attracting investment and ultimately, successful drug development and commercialization.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e250036"},"PeriodicalIF":1.9,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12007478/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143780153","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"How once-nightly sodium oxybate is processed in the body in healthy volunteers: a plain language summary.","authors":"Richard Bogan, Michael J Thorpy, Sarah Berkowitz, Jennifer Gudeman","doi":"10.57264/cer-2024-0243","DOIUrl":"10.57264/cer-2024-0243","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;What is this summary about?: &lt;/strong&gt;This is a plain language summary of an article originally published in the journal Sleep Medicine. Narcolepsy is a sleep condition in which people have periods of extreme sleepiness during the day. People with narcolepsy may also have symptoms of muscle weakness (cataplexy); seeing, hearing, smelling, tasting, or feeling something that seems real but isn't actually there before falling asleep or while waking up (hallucinations); an inability to move before falling asleep or while waking up (sleep paralysis); and poor sleep at night. Sodium oxybate (SXB for short) has been used to treat narcolepsy for over 20 years. For more than 20 years, the only available form of SXB needed to be taken twice each night. Twice-nightly SXB (TN-SXB) requires people to take the first dose at bedtime. Most people fall asleep within 5 to 15 minutes after the first dose. Patients then need to wake up and take the second dose of TN-SXB 2.5 to 4 hours later, which is when most of the medicine has left the body. The United States Food and Drug Administration (also called FDA) approved a once-nightly form of SXB called LUMRYZ&lt;sup&gt;™&lt;/sup&gt; (sodium oxybate for extended-release oral suspension; ON-SXB for short) in May 2023. ON-SXB treats excessive daytime sleepiness and muscle weakness, also known as cataplexy. People with narcolepsy who take ON-SXB only need to take 1 dose at bedtime. This summary describes a study that looked at how ON-SXB enters, travels through, and exits the body of healthy volunteers (people without narcolepsy). The study measured the amount of SXB in the blood after taking SXB in 2 different ways: as a once-nightly version and as a twice-nightly version. Looking at the amount of ON-SXB and TN-SXB in the blood at different time points helps researchers see if people who take the 2 medicines, with different dosing, have the same amount of drug in the body overnight.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;What were the results?: &lt;/strong&gt;Overall, the study found that healthy volunteers had the same amount of SXB in their bodies after taking ON-SXB as they did after taking TN-SXB. When they took the same amounts of ON-SXB and TN-SXB, the SXB stayed in their bodies for similar amounts of time. The highest amount and the total amount of SXB in the participants' blood were similar with ON-SXB and TN-SXB. The amount of SXB in the participants' blood 8 hours after taking the medicine was significantly lower with ON-SXB than with TN-SXB. This means that people taking ON-SXB will have less medication in their blood when they wake up the next morning and may be less groggy.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;What do the results mean?: &lt;/strong&gt;The results mean that if people with narcolepsy take the same dose amount of ON-SXB or TN-SXB, their bodies will receive the same amount of SXB in their blood. ON-SXB provides an option for people with narcolepsy to take their medicine once at bedtime without the need for a middle-of-the-night dose, which can improve qua","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240243"},"PeriodicalIF":1.9,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12007474/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143730226","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 and safety of advanced therapies for moderately to severely active ulcerative colitis in induction and maintenance: systematic literature review and Bayesian network meta-analysis.","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-2024-0225","DOIUrl":"10.57264/cer-2024-0225","url":null,"abstract":"<p><p><b>Aim:</b> Several therapies have recently been licensed for the treatment of patients with moderately to severely active ulcerative colitis (UC). To provide comparative evidence of newly available treatments, Bayesian network meta-analyses were conducted to compare their relative efficacy and safety profiles in both the induction and maintenance phases. <b>Materials & methods:</b> A systematic literature review was conducted to identify the available literature on randomized controlled trials for advanced treatments (AT) of moderately to severely active UC. Bayesian network meta-analyses were used to synthesize evidence on prespecified efficacy and safety outcomes. Primary efficacy end points clinical response and clinical remission were measured at the end of induction and clinical response and clinical remission among induction phase responders were assessed at the end of the maintenance period. Efficacy outcomes were analyzed separately for AT-naive and -experienced populations. Safety outcomes included serious infections over the induction period, and serious infections among others over the maintenance period. Treat-through trial outcomes were adjusted to align with responder rerandomized trial outcomes. <b>Results:</b> The systematic review identified 58 relevant trials of which 28 met criteria for inclusion in the main analysis networks. At the end of the induction period, all treatments were efficacious against placebo for both AT-naive and AT-experienced populations. Upadacitinib 45 mg demonstrated a higher likelihood of clinical response and remission compared with other treatments. Adalimumab had less favorable performance over the induction period. Among induction phase responders, most treatments demonstrated similar efficacy at the end of the maintenance period. Tofacitinib 10 mg was more likely to achieve clinical response and remission than several other treatments in the AT-naive population. In the AT-experienced population, upadacitinib 30 mg demonstrated a higher likelihood of clinical response and remission compared with other treatments. The safety outcomes among treatments were similar. <b>Conclusion:</b> This study provides an updated comparison of treatments for moderately to severely active UC. Most treatments demonstrated comparable efficacy at the end of maintenance. The findings from this study can inform decision making in treatment choice for patients with moderately to severely active UC.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240225"},"PeriodicalIF":1.9,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12007476/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143649259","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}