Journal of comparative effectiveness research最新文献

{"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}

{"title":"An international observational study assessing conservative management in hemorrhoidal disease: results of CHORALIS (aCute HemORrhoidal disease evALuation International Study).","authors":"Philippe Godeberge, Zoltan Csiki, Mykhailo Zakharash, Elly Nyaim Opot, Yuri A Shelygin, Trung Tin Nguyen, Ashraf Amir, Ibrahima Konaté, Moses Momoh, Joanna Chirol, Vanessa Blanc-Guillemaud, Ren Donglin","doi":"10.57264/cer-2024-0070","DOIUrl":"10.57264/cer-2024-0070","url":null,"abstract":"<p><p><b>Aim:</b> Real-world evidence on the management of hemorrhoidal disease (HD) is limited. This international study collected clinical practice data on the effectiveness of conservative treatments for acute HD on symptoms and quality of life (QoL), providing perspectives of treatment modalities from different continents. <b>Patients & methods:</b> The 4-week observational prospective CHORALIS study involved adult outpatients consulting for spontaneous complaints of hemorrhoids (graded using Goligher classification) and prescribed conservative treatments according to usual clinical practice. Assessments were: anal pain/discomfort (visual analog scale [VAS]), other signs/symptoms (patient questionnaire), Patient Global Impression of Change (PGI-C) questionnaire and disease-specific QoL (HEMO-FISS-QoL questionnaire). <b>Results:</b> Of 3592 participants, 3505 were analyzed (58.4% male; age 40.5 ± 13.7 years; history of HD in 48.4%; 72.1% Goligher grade I and II). Pain and discomfort were the most common symptoms. Most treatments were venoactive drugs (VADs; 90.9%), particularly micronized purified flavonoid fraction (MPFF; 73.7%) and diosmin (14.6%). All VAD-based therapies improved signs/symptoms (number/intensity/frequency of pain, discomfort, bleeding, swelling, itching and soiling) and QoL. MPFF was associated with a significantly greater proportion of patients with no symptoms (48.8 vs diosmin 34.4%, p < 0.001), pain disappearance (69.7 vs diosmin 52.8%, p < 0.001), treatment impact at 1 week rated on PGI-C as 'very much better' (30.5 vs diosmin 17.9%, p < 0.001) and shorter times to improvement (mean ± SD 3.9 ± 1.5 days vs diosmin 4.2 ± 1.7 days). <b>Conclusion:</b> In this prospective real-world study of patients with acute HD, conservative therapies consisting mainly of VADs, including MPFF, improved the clinical signs and symptoms of disease, as well as QoL. This study evidence supports clinical advantages associated with VADs, mostly MPFF, for effectively managing acute HD.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240070"},"PeriodicalIF":1.9,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11426285/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141916822","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":"Why is the market design for innovative pharmaceuticals not well understood?","authors":"Francisco Olivença, Jose Diaz, Sreeram V Ramagopalan, Louis P Garrison","doi":"10.57264/cer-2024-0105","DOIUrl":"10.57264/cer-2024-0105","url":null,"abstract":"","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240105"},"PeriodicalIF":1.9,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11426280/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142288758","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":"Comparing the efficacy of cipaglucosidase alfa plus miglustat with other enzyme replacement therapies for late-onset Pompe disease: a network meta-analysis utilizing patient-level and aggregate data.","authors":"Simon Shohet, Noemi Hummel, Shuai Fu, Ian Keyzor, Alasdair MacCulloch, Neil Johnson, Jeff Castelli, Ilona Czarny-Ozga, Tahseen Mozaffar, Howard Thom","doi":"10.57264/cer-2024-0045","DOIUrl":"10.57264/cer-2024-0045","url":null,"abstract":"<p><p><b>Aim:</b> Late-onset Pompe disease is characterized by progressive loss of muscular and respiratory function. Until recently, standard of care was enzyme replacement therapy (ERT) with alglucosidase alfa. Second-generation ERTs avalglucosidase alfa (aval) and cipaglucosidase alfa with miglustat (cipa+mig) are now available. Without head-to-head trials comparing aval with cipa+mig, an indirect treatment comparison is informative and timely for understanding potential clinical differentiation. <b>Materials & methods:</b> A systematic literature review was performed to identify relevant studies on cipa+mig and aval. Using patient-level and aggregate published data from randomized controlled trials (RCTs) and phase I/II and open-label extension (OLE) trials, a multi-level network meta-regression was conducted, adjusting for various baseline covariates, including previous ERT duration, to obtain relative effect estimates on 6-minute walk distance (6MWD, meters [m]) and forced vital capacity (FVC, % predicted [pp]). Analyses of two networks were conducted: Network A, including only RCTs, and network B, additionally including single-arm OLE and phase I/II studies. <b>Results:</b> Network B (full evidence analysis) showed that cipa+mig was associated with a relative increase in 6MWD (mean difference 28.93 m, 95% credible interval [8.26-50.11 m]; Bayesian probability 99.7%) and FVC (2.88 pp [1.07-4.71 pp]; >99.9%) compared with aval. The comparison between cipa+mig and aval became more favorable for cipa+mig with increasing previous ERT duration for both end points. Analysis of network A showed that cipa+mig was associated with a relative decrease in 6MWD (-10.02 m [-23.62 to 4.00 m]; 91.8%) and FVC (-1.45 pp [-3.01 to 0.07 pp]; 96.8%) compared with aval. <b>Conclusion:</b> Cipa+mig showed a favorable effect versus aval when all available evidence was used in the analysis.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240045"},"PeriodicalIF":1.9,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11426283/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142288757","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":"Comparative efficacy of diroximel fumarate, ozanimod and interferon beta-1a for relapsing multiple sclerosis using matching-adjusted indirect comparisons.","authors":"Tammy Jiang, Mathura Shanmugasundaram, Ivan Božin, Mark S Freedman, James B Lewin, Changyu Shen, Tjalf Ziemssen, Douglas L Arnold","doi":"10.57264/cer-2023-0161","DOIUrl":"10.57264/cer-2023-0161","url":null,"abstract":"<p><p><b>Aim:</b> Diroximel fumarate (DRF), ozanimod (OZA) and interferon beta-1a (IFN) are disease-modifying therapies approved for the treatment of relapsing multiple sclerosis. No randomized trials have compared DRF versus OZA and IFN. We compared DRF versus OZA and DRF versus IFN using matching-adjusted indirect comparisons for efficacy outcomes, including annualized relapse rate (ARR), 12- and 24-week confirmed disability progression (CDP) and absence of gadolinium-enhancing (Gd+) T1 lesions and new/newly enlarging T2 lesions. <b>Patients & methods:</b> We used individual patient data from EVOLVE-MS-1 (NCT02634307), a 2-year, open-label, single-arm, phase III study of DRF (n = 1057) and aggregate data from RADIANCE (NCT02047734), a 2-year, double-blind, phase III study that compared OZA 1 mg once daily (n = 433) and intramuscular IFN 30 μg once weekly (n = 441). To account for cross-trial differences, the EVOLVE-MS-1 population was restricted to those who met the inclusion/exclusion criteria for RADIANCE, then weighted to match the average baseline characteristics of RADIANCE. <b>Results:</b> After weighting, DRF and OZA had similar ARRs (0.18 and 0.17, respectively), with a rate difference (DRF vs OZA) of 0.01 (95% confidence interval [CI]: -0.04 to 0.06). DRF had a lower ARR than IFN (0.18 and 0.28, respectively), with a rate difference (DRF vs IFN) of -0.10 (95% CI: -0.16 to -0.04) after weighting. Outcomes for 12- and 24-week CDP favored DRF versus OZA; 12-week CDP favored DRF versus IFN, but there was not strong evidence favoring DRF over IFN for 24-week CDP. Compared with OZA and IFN, DRF had higher proportions of patients without Gd+ T1 lesions and patients without new/newly enlarging T2 lesions. <b>Conclusion:</b> Disability progression and radiological outcomes were favorable for DRF versus OZA, although no differences were observed in ARR. Clinical and radiological outcomes generally favored DRF versus IFN. These findings may be informative for patients and clinicians considering different treatment options for MS.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e230161"},"PeriodicalIF":1.9,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11428343/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142000068","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 systematic review of health-related quality of life outcomes in patients with advanced breast cancer treated with palbociclib.","authors":"Imtiaz A Samjoo, Alexandra Hall, Connie Chen, Bao-Ngoc Nguyen, Meaghan Bartlett, Mary Lou Smith, Nadia Harbeck, Joseph C Cappelleri, Meghan Karuturi, Doris Makari, Lillian Shahied Arruda, Rickard Sandin, Kent Hanson, Justin Doan","doi":"10.57264/cer-2024-0111","DOIUrl":"10.57264/cer-2024-0111","url":null,"abstract":"<p><p><b>Aim:</b> To evaluate the impact of palbociclib treatment on health-related quality of life (HRQoL) in patients with hormone receptor-positive, human epidermal growth factor 2-negative advanced breast cancer (HR+/HER2- aBC) or metastatic breast cancer (mBC) in both the clinical and real-world setting. <b>Materials & methods:</b> A systematic literature review was conducted to identify clinical trials and real-world evidence studies up to June 2023 that reported HRQoL outcomes in patients with HR+/HER2- aBC or mBC treated with Palbociclib. <b>Results:</b> 15 unique studies reported across 35 records were identified. Of these, seven were randomized controlled trials (RCTs), three were single-arm clinical trials and five were real-world evidence (RWE) studies. HRQoL was generally found to be maintained in patients with HR+/HER2- aBC or mBC across RCTs, single-arm clinical trials and RWE studies. HRQoL measures across instruments, study types and line of therapy, were largely reported to be at least maintained if not improved from baseline among patients treated with palbociclib and were observed to be comparable or better in the palbociclib group versus monotherapy control arm in RCTs. Similar results were seen for treatment-related outcomes (e.g., sexual functioning, upset by hair loss, systemic therapy side effects etc.), and important individual patient outcomes, including pain, fatigue and physical functioning. Findings were also consistent across key clinical characteristics (visceral metastases, neutropenia), as well as patient populations often underrepresented in clinical trials (Asian patients, older adults). <b>Conclusion:</b> Overall, current evidence suggests that HRQoL is largely preserved with the addition of palbociclib to endocrine therapy in patients with HR+/HER2- aBC or mBC across study types and populations.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240111"},"PeriodicalIF":1.9,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11426284/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142288746","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 pharma can amplify product value with implementation science.","authors":"Melvin Skip Olson, Leah L Zullig, Sabina De Geest","doi":"10.57264/cer-2024-0076","DOIUrl":"10.57264/cer-2024-0076","url":null,"abstract":"<p><p>Achieving blockbuster status requires more than clinical trial success. Crucial barriers often include real-world factors like patient acceptance, prescriber behavior and timely and full reimbursement. Implementation science can be used to identify such barriers, develop strategies to overcome them, as well as test their effect. Used correctly and at the right time, implementation science can amplify product value and lead to a triple win for patients, healthcare systems and pharma. Three easy steps that focus on context, strategies and outcomes, can be followed by pharma to bring implementation thinking and research into their processes. A 'what if' case study is shared to give an indication of how this might work and the impact it might have.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240076"},"PeriodicalIF":1.9,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11426286/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142140188","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-2024-0126","DOIUrl":"10.57264/cer-2024-0126","url":null,"abstract":"","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240126"},"PeriodicalIF":1.9,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11367562/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141874975","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}