Journal of comparative effectiveness research最新文献

{"title":"Real-world clinical outcomes and rationale for initiating abatacept as a first-line biologic for patients with anticitrullinated protein antibody- and rheumatoid factor-positive rheumatoid arthritis.","authors":"Alexandrina Balanean, Cherrishe Brown-Bickerstaff, Andrew Klink, Vardhaman Patel, Hanke Zheng, Laetitia N'Dri, Keith Wittstock, Bruce Feinberg, Mark Chaballa, Vadim Khaychuk, Jill Kaufman, Prathamesh Pathak, Gordon Lam","doi":"10.57264/cer-2023-0144","DOIUrl":"10.57264/cer-2023-0144","url":null,"abstract":"<p><p><b>Aim:</b> In rheumatoid arthritis (RA), seropositivity for both anticitrullinated protein antibody (ACPA) and rheumatoid factor (RF) is associated with disease severity and therapeutic response. Biologic (b) disease-modifying antirheumatic drugs (DMARDs) such as abatacept are recommended after inadequate response or contraindication to conventional synthetic DMARDs. This retrospective cohort study aimed to describe changes in Clinical Disease Activity Index (CDAI) measures over 12 months among patients with ACPA+ and RF+ RA with an inadequate response to methotrexate treated with abatacept as a first-line bDMARD. <b>Patients & methods:</b> Patient data were abstracted from medical records by treating rheumatologists. Analyses included McNemar tests for paired proportions or paired <i>t</i>-tests to assess longitudinal changes in CDAI scores, and Kaplan-Meier methods for time-to-event outcomes. Serious AEs and rationale for initiating treatment were recorded. <b>Results:</b> Overall, 296 patients were included. Mean CDAI scores improved (decreased) by 34.0, 61.0 and 74.0% (all p < 0.001) from baseline to 3-6 months, 6-12 months and ≥12 months after abatacept initiation, respectively. Of 279 patients not in CDAI low disease activity (LDA) or remission at baseline, 24.7% of patients achieved it within 6 months, 56.3% within 12 months and 71.0% at any point during follow-up after abatacept initiation. Median time to CDAI LDA/remission was 10.2 months. Serious AEs were reported in 2.4% of patients. Common reasons reported by rheumatologists for initiating abatacept were effectiveness/efficacy (52.7%), safety (31.4%) and patient preference (25.3%). <b>Conclusion:</b> In this analysis of patients with ACPA+ and RF+ RA treated with abatacept as a first-line bDMARD in a clinical practice setting, clinical outcomes and remission rates were improved at all time points, providing real-world evidence to further support the use of abatacept in this patient population.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e230144"},"PeriodicalIF":1.9,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11609992/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142647999","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 lung cancer screening with volume computed tomography in Portugal.","authors":"Hilde Ten Berge, Katerina Togka, Xuanqi Pan, Marina Borges, Fernando Palma Martelo, Fernando Guedes, Daniel Cabral, Encarnação Teixeira, Gabriela Fernandes, Lurdes Ferreira, Sara Figueiredo, Rita Sousa, Lourdes Barradas, Fernanda Estevinho, António Araújo, Venceslau Hespanhol, Rui Medeiros","doi":"10.57264/cer-2024-0102","DOIUrl":"10.57264/cer-2024-0102","url":null,"abstract":"<p><p><b>Aim:</b> Lung cancer is the most common cause of cancer death in Portugal. The Dutch-Belgian lung cancer screening (LCS) study (NELSON), the biggest European LCS study, showed a lung cancer mortality reduction in a high-risk population when being screened. In this study, the cost-effectiveness of LCS, based on the NELSON study protocol and outcomes, was evaluated compared with no screening in Portugal. <b>Methods:</b> The present study modified an established decision tree by incorporating a state-transition Markov model to evaluate the health-related advantages and economic implications of low-dose computed tomography (LDCT) LCS from the healthcare standpoint in Portugal. The analysis compared screening versus no screening for a high-risk population aged 50-75 with a smoking history. Various metrics, including clinical outcomes, costs, quality-adjusted life years (QALYs), life-years (LYs) and the incremental cost-effectiveness ratio (ICER), were calculated to measure the impact of LDCT LCS. Furthermore, scenario and sensitivity analyses were executed to assess the robustness of the obtained results. <b>Results:</b> Annual LCS with volume-based LDCT resulted in €558 million additional costs and 86,678 additional QALYs resulting in an ICER of €6440 per QALY for one screening group and a lifetime horizon. In total, 13,217 premature lung cancer deaths could be averted, leading to 1.41 additional QALYs gained per individual diagnosed with lung cancer. Results are robust based on the sensitivity analyses. <b>Conclusion:</b> This study showed that annual LDCT LCS for a high-risk population could be cost-effective in Portugal based on a willingness to pay a threshold of one-time the GDP (€19,290 per QALY gained).</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240102"},"PeriodicalIF":1.9,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11542083/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142347889","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":"Ravulizumab in adults and children with atypical hemolytic uremic syndrome: a plain language summary of three studies.","authors":"Michal Nowicki, Nikoleta Printza","doi":"10.57264/cer-2024-0103","DOIUrl":"10.57264/cer-2024-0103","url":null,"abstract":"<p><strong>What is this summary about?: </strong>This summary gives an overview of three published articles that report the results of research studies of ravulizumab, an approved treatment for people with atypical hemolytic uremic syndrome (often shortened to aHUS). This is a rare and serious condition where blood clots form in small blood vessels. Blood vessels are structures that transport blood around the body. Blood clots are the body's way of stopping someone from bleeding too much. However, if they form when they are not needed, they can cause harm. In atypical hemolytic uremic syndrome, the blood clots can cause injury to organs like the kidney. In the three studies, the researchers wanted to know if ravulizumab could decrease the formation of these clots and improve kidney function. Children who had never received ravulizumab or a similar treatment took part in the first study. Adults who had never received ravulizumab or a similar treatment took part in the second study. In the third study, children whose disease was already controlled by a medication called eculizumab switched to ravulizumab. Ravulizumab is dosed less frequently than eculizumab. The researchers looked at kidney function and the levels of different blood components to see how well the treatment was working. They also monitored the adverse effects that participants experienced.</p><p><strong>What were the results?: </strong>Across the three studies, ravulizumab improved indicators of blood clotting in small vessels and improved kidney function in both children and adults. In addition, ravulizumab was similarly effective to eculizumab for children who were already receiving eculizumab and switched to ravulizumab. Overall, the adverse effects that people experienced with ravulizumab were manageable.</p><p><strong>What do the results mean?: </strong>These studies showed that ravulizumab is a treatment option for children and adults with aHUS. In addition, a switch to ravulizumab can be considered for children who are already responding well to eculizumab and would benefit from less frequent dosing.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240103"},"PeriodicalIF":1.9,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11542079/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142467107","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":"Access in all areas? A round up of developments in market access and HTA: part 5.","authors":"Alice Beattie, Francisco Olivença, Catrin Treharne, Sreeram V Ramagopalan","doi":"10.57264/cer-2024-0179","DOIUrl":"10.57264/cer-2024-0179","url":null,"abstract":"<p><p>In this latest update, we explore the recent announcement by Canada's Drug Agency (CDA-AMC, formerly CADTH) on their pilot to include the societal perspective in the evaluation of certain new medicines; a recent Office of Health Economics (OHE) report on the evaluation of HTA agency methods over time; and publications examining the impact of Project Orbis on patient access to oncology treatments.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240179"},"PeriodicalIF":1.9,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11542086/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142347888","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 economic impact associated with stent retriever selection for the treatment of acute ischemic stroke: a cost-effectiveness analysis of MASTRO I data from a Chinese healthcare system perspective.","authors":"Osama O Zaidat, Xinguang Yang, Waleed Brinjikji, Emilie Kottenmeier, Hendramoorthy Maheswaran, Thibaut Galvain, Patrick A Brouwer, Mahmood Mirza, Tommy Andersson","doi":"10.57264/cer-2024-0160","DOIUrl":"10.57264/cer-2024-0160","url":null,"abstract":"<p><p><b>Aim:</b> The aim of this analysis was to assess the cost-effectiveness of the EmboTrap^® Revascularization Device compared with the Solitaire™ Revascularization Device and Trevo^® Retriever for the treatment of acute ischemic stroke (AIS) from the perspective of the Chinese healthcare system. <b>Methods:</b> According to MASTRO I, a recent living systematic literature review and meta-analysis, mechanical thrombectomy (MT) with EmboTrap in the treatment of AIS resulted in better functional outcomes compared with the use of Solitaire or Trevo. Based on the proportion of patients that achieved 90-day modified Rankin Scale (mRS) scores of 0-2, 3-5 and 6 reported in MASTRO I, a combined 90-day short-term decision tree and Markov model with a 10-year time horizon was used to compare the cost-effectiveness of the three devices. The primary outcome was the incremental cost-effectiveness ratio (ICER), representing the incremental cost (in 2022 Chinese Yuan [CNY]) per incremental quality-adjusted life-year (QALY). The ICERs were compared against willingness-to-pay (WTP) thresholds of 1, 1.5 and 3-times the 2022 national gross domestic product (GDP) per capita in China. <b>Results:</b> Treatment with EmboTrap resulted in total QALYs of 3.28 and total costs of 110,058 CNY per patient. Treatment with Trevo resulted in total QALYs of 3.05 and total costs of 116,941 CNY per patient. Treatment with Solitaire resulted in total QALYs of 2.81 and total costs of 99,090 CNY per patient. Trevo was dominated by EmboTrap as it was a more costly and less effective intervention. As such, Trevo was not cost-effective at any WTP threshold. Compared with Solitaire, EmboTrap was more effective and more costly, with an ICER of 23,615 CNY per QALY. This result suggests that EmboTrap is cost-effective when compared with Solitaire since the ICER was lower than all WTP thresholds assessed. <b>Conclusion:</b> EmboTrap dominated Trevo and is cost-effective for the treatment of patients with AIS compared with Solitaire when assessed from the perspective of the Chinese healthcare system and based on the device-level meta-analysis MASTRO I. Selecting a stent retriever (SR) that optimizes 90-day mRS score is an important consideration from both a clinical and healthcare payer perspective in China as it is associated with reduced long-term costs and increased quality of life.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":"13 11","pages":"e240160"},"PeriodicalIF":1.9,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11542088/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142576238","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":"Perceptions of indirect treatment comparisons as an evidence base in oncology decision-making: results of an international survey of health technology assessment and payer decision-makers.","authors":"Ioannis Katsoulis, Alex Graham, Allison Thompson, Norbek Gharibian, Vivek Pawar, Vivek Khurana, Rui Ferreira, Abhishek Panikar, Mairead Kearney","doi":"10.57264/cer-2024-0040","DOIUrl":"10.57264/cer-2024-0040","url":null,"abstract":"<p><p><b>Aim:</b> Health technology assessment (HTA) and payer organizations are often faced with early decision-making in oncology. To design and conduct robust indirect treatment comparisons (ITCs), it is important to better understand HTA and payer decision-maker perceptions of ITCs. Here we aim to describe what individuals with HTA and payer experience see as the acceptability of ITCs for HTA and payer organization coverage and reimbursement decision-making. <b>Materials & methods:</b> This survey included 30 current and former HTA and payer decision-makers from five countries: Australia, France, Germany, the UK (n = 5 each) and the US (n = 10). Main outcomes included the ratings of acceptance of ITCs and the presence of well-defined methodological guidance for ITCs. <b>Results:</b> ITCs are generally accepted by participants in Australia and the UK but are more likely evaluated on a case-by-case basis in France, Germany and the US. Four of five participants in Germany and the UK, two of five in Australia and one of five in France reported that well-defined and prescribed criteria regarding the use of ITCs were in place. <b>Conclusion:</b> There is a need for harmonization of methods used to assess ITCs by HTA and payers, especially in the rapidly evolving treatment landscape in oncology.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":"13 11","pages":"e240040"},"PeriodicalIF":1.9,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11542087/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142576236","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":"Use of transportability methods for real-world evidence generation: a review of current applications.","authors":"Natalie S Levy, Patrick J Arena, Thomas Jemielita, Shahrul Mt-Isa, Shane McElwee, David Lenis, Ulka B Campbell, Ashley Jaksa, Gleicy M Hair","doi":"10.57264/cer-2024-0064","DOIUrl":"10.57264/cer-2024-0064","url":null,"abstract":"<p><p><b>Aim:</b> To evaluate how transportability methods are currently used for real-world evidence (RWE) generation to inform good practices and support adoption and acceptance of these methods in the RWE context. <b>Methods:</b> We conducted a targeted literature review to identify studies that transported an effect estimate of the clinical effectiveness or safety of a biomedical exposure to a target real-world population. Records were identified from PubMed-indexed articles published any time before 25 July 2023 (inclusive). Two reviewers screened abstracts/titles and reviewed the full text of candidate studies to identify the final set of articles. Data on the therapeutic area, exposure(s), outcome(s), original and target populations and details of the transportability analysis (e.g., analytic method used, estimate transported, stated assumptions) were abstracted from each article. <b>Results:</b> Of 458 unique records identified, six were retained in the final review. Articles were published during 2021-2023, focused on the US/Canada context, and covered a range of therapeutic areas. Four studies transported an RCT effect estimate, while two transported effect estimates derived from real-world data. Almost all articles used weighting methods to transport estimates. Two studies discussed all transportability assumptions, and one evaluated the likelihood of meeting all assumptions and the impact of potential violations. <b>Conclusion:</b> The use of transportability methods for RWE generation is an emerging and promising area of research to address evidence gaps in settings with limited data and infrastructure. More transparent and rigorous reporting of methods, assumptions and limitations may increase the use and acceptability of transportability for producing robust evidence on treatment effectiveness and safety.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240064"},"PeriodicalIF":1.9,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11542082/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142371982","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":"Ravulizumab for adults with generalized myasthenia gravis: a plain language summary of three studies.","authors":"Florencia Aguirre, Renata Andrade","doi":"10.57264/cer-2024-0109","DOIUrl":"10.57264/cer-2024-0109","url":null,"abstract":"<p><strong>What is this summary about?: </strong>Generalized myasthenia gravis (often shortened to gMG) is a rare health condition that causes muscular weakness. This summary gives an overview of three published articles that report the results of research studies of a medicine called ravulizumab, a treatment approved for adults with gMG. These studies are: The CHAMPION MG study. The CHAMPION MG extension study. A study of how the body processes and responds to ravulizumab (known as pharmacokinetics and pharmacodynamics). These studies looked at how effective and safe ravulizumab is for people with gMG.</p><p><strong>What were the results?: </strong>Overall, participants with gMG who received ravulizumab showed a significant and rapid improvement in their muscle strength and ability to do daily activities. These improvements were sustained for up to 60 weeks of treatment. Ravulizumab was well-tolerated overall, and no-one in the study had a meningococcal infection (a type of bacterial infection preventable with vaccination). Results from the pharmacokinetic and pharmacodynamic study support the use of ravulizumab every 8 weeks to maintain improvements in gMG.</p><p><strong>What do the results of the study mean?: </strong>Ravulizumab can be considered as a treatment option for adults with gMG who are appropriately protected against meningococcal infection before starting treatment. The drug, administered every 8 weeks, improves muscle strength and daily performance. These positive effects have been observed to persist over a long period of time.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240109"},"PeriodicalIF":1.9,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11542078/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142467106","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":"One-year budget impact of InTandem™: a novel neurorehabilitation system for individuals with chronic stroke walking impairment.","authors":"Kirsten E Smayda, Jennifer Lavanture, Megan Bourque, Nathashi Jayawardena, Sarah Kane, Holly Roberts, Barbara Heikens","doi":"10.57264/cer-2024-0010","DOIUrl":"10.57264/cer-2024-0010","url":null,"abstract":"<p><p><b>Aim:</b> Chronic stroke walking impairment is associated with high healthcare resource utilization (HCRU) costs. InTandem™ is a neurorehabilitation system that autonomously delivers a rhythmic auditory stimulation (RAS)-based intervention for the at-home rehabilitation of walking impairment in adults in the chronic phase of stroke recovery. This study was conducted to estimate the budget impact of InTandem in comparison with currently available intervention strategies for improvement of gait/ambulation in individuals with chronic stroke walking impairment. <b>Methods & materials:</b> A budget impact analysis (BIA) for InTandem was conducted based on a 1-million-member US third-party payer perspective over a 1-year time horizon. Key inputs for the budget impact model were: costs for each intervention strategy (InTandem, physical therapy, self-directed walking and no treatment), HCRU costs for persons with chronic stroke and anticipated HCRU cost offsets due to improvements in gait/ambulatory status as measured by self-selected comfortable walking speed (based on functional ability). In addition to the reference case analysis, a sensitivity analysis was conducted. <b>Results:</b> Based on the reference case, introduction of InTandem was projected to result in overall cost savings of $439,954 in one year. Reduction of HCRU costs (-$2,411,778) resulting from improved walking speeds with InTandem offset an increase in intervention costs (+$1,971,824). Demonstrations of cost savings associated with InTandem were robust and were consistently evident in nearly all scenarios evaluated in the sensitivity analysis (e.g., with increased/decreased patient shares, increased HCRU cost or increased InTandem rental duration). <b>Conclusion:</b> The InTandem system is demonstrated to improve walking and ambulation in adults in the chronic phase of stroke recovery after a five-week intervention period. The BIA predicts that introduction of InTandem will be associated with overall cost savings to the payer.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240010"},"PeriodicalIF":1.9,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11426287/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142119974","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 clinical decision support tool for metabolic dysfunction-associated steatohepatitis in real-world clinical settings: a mixed-method implementation research study protocol.","authors":"Jesse Fishman, Theresa Alexander, Yestle Kim, Iris Kindt, Patricia Mendez","doi":"10.57264/cer-2024-0085","DOIUrl":"10.57264/cer-2024-0085","url":null,"abstract":"<p><p><b>Aim:</b> A clinical decision support (CDS) tool for metabolic dysfunction-associated steatohepatitis (MASH) was developed to align health systems with clinical guidelines detailed in the MASH Clinical Care Pathway and improve patients' proactive self-management of their disease. The tool includes a provider-facing web-based application and a mobile application (app) for patients. This protocol outlines a pilot study that will systematically evaluate the implementation of the tool in real-world clinical practice settings. <b>Materials & methods:</b> This implementation research study will use a simultaneous mixed-methods design and is guided by the Consolidated Framework for Implementation Research. The CDS tool for MASH will be piloted for ≥3 months at multiple US-based sites with eligible gastroenterologists and hepatologists (n = 5-10 per site) and their patients (n = 50-100 per site) with MASH or suspected MASH. Each pilot site may choose one or all focus areas within the tool (i.e., risk stratification, screening and referral, or patient care management), based on on-site capabilities. Prior to and at the end of the pilot period, providers and patients will complete quantitative surveys and partake in semi-structured interviews. Outcomes will include understanding the feasibility of implementing the tool in real-world clinical settings, its effectiveness in increasing patient screenings and risk stratification for MASH, its ability to improve provider and patient knowledge of MASH, barriers to adoption of the tool and the tool's capacity to enhance patient engagement and satisfaction with their care. <b>Conclusion:</b> Findings will inform the scalable implementation of the tool to ensure patients at risk for MASH are identified early, referred to specialty care when necessary and managed appropriately. Successful integration of the patient app can contribute to better health outcomes for patients by facilitating their active participation in the management of their condition.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240085"},"PeriodicalIF":1.9,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11426282/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142288745","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}