Browsing by Subject "Real-world evidence"

Now showing 1 - 6 of 6
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    Correction to: Real-World Treatment Patterns in Patients with Vitiligo in the United States
    (Springer, 2023) Rosmarin, David; Soliman, Ahmed M.; Li, Chao; Dermatology, School of Medicine
    Correction to: Dermatol Ther (Heidelb) (2023) 13:2079–2091 10.1007/s13555-023-00983-3 Authors would like to update the middle name of co-author as Ahmed M. Soliman. The original article has been corrected.
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    Disease Course, Treatment Patterns and Goals Among Patients with Non-segmental Vitiligo Across Europe and the United States
    (Springer, 2024) Rosmarin, David; Soliman, Ahmed M.; Marwaha, Simran; Piercy, James; Camp, Heidi S.; Anderson, Peter; Ezzedine, Khaled; Dermatology, School of Medicine
    Introduction: There is currently a lack of research regarding disease course and burden as well as treatment patterns and goals in patients with non-segmental vitiligo (NSV). The aim of this analysis was to evaluate disease course, treatment patterns and goals in patients with NSV. Methods: This analysis used secondary data from the Adelphi Real World Vitiligo Disease Specific Programme™ 2021, specifically, a survey of physicians and their adult and adolescent patients with NSV. Physicians categorized patients by the extent of NSV at time of survey completion as mild, moderate or severe/very severe. Physician-reported patient information included demographics, current/previously prescribed NSV therapies, treatment satisfaction and the Vitiligo Noticeability Scale (VNS). Patients completed a survey on treatment satisfaction and the VNS. Treatment pattern data were stratified by disease extent and Fitzpatrick skin type. Results: At survey completion, physicians reported that 38, 50 and 12% of patients (N = 1865) had improving, stable and deteriorating/progressing disease, respectively. Most patients (96%) with mild disease at treatment initiation still had mild disease at the time of survey completion. More than half of patients with moderate disease (62%) or severe/very severe disease (57%) at treatment initiation still had moderate or severe/very severe disease at survey completion. Topical calcineurin inhibitors (TCIs) were the most common treatment in 40% of patients followed by phototherapy in 30%. Patients hoped for re-pigmentation (mild 56%, moderate 62%, severe/very severe 66%), reduction (mild 50%, moderate 56%, severe/very severe 49%) or cessation of affected areas with vitiligo (mild 48%, moderate 54%, severe/very severe 43%). Conclusion: The study findings indicate that a significant proportion of patients with NSV are not improving on current treatments, most commonly TCIs and phototherapy. The results highlight the unmet need for novel and effective therapies to substantially improve re-pigmentation, an important treatment goal for patients with NSV.
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    Real-World Treatment Patterns in Patients with Vitiligo in the United States
    (Springer, 2023) Rosmarin, David; Soliman, Ahmed M.; Li, Chao; Dermatology, School of Medicine
    Introduction: Vitiligo is an autoimmune disorder resulting in skin depigmentation, with limited approved treatment options. This study evaluated medication utilization and treatment patterns among patients in the first year following vitiligo diagnosis. Methods: This retrospective analysis of claims data from the Merative® MarketScan Research Databases included patients aged ≥ 12 years newly diagnosed with vitiligo. Patients were identified between October 1, 2016, and April 30, 2021, and had ≥ 12 months of continuous enrollment pre- and post-vitiligo diagnosis. Medication use, treatment line of therapy, time to and number of medication claims, and length of therapy were reported in the 12 months post-vitiligo diagnosis. Results are reported separately for treatment initiators post-vitiligo diagnosis, patients with moderate-to-severe vitiligo, and adolescents (aged 12-17 years). Results: A total of 19,335 patients were included in the analysis, with half (N = 9648, 49.9%) not receiving any treatment during the 12-month follow-up. Switching was minimal among treatment initiators (N = 5845) in the 12 months post-vitiligo diagnosis, with the most frequent first-line treatments being high-potency topical corticosteroids (25.4%), oral corticosteroids (23.1%), and topical calcineurin inhibitors (TCI, 14.7%). Adolescents initiating treatment (N = 486) most frequently received TCI (30.9%) as first-line therapy. Patients with moderate-to-severe vitiligo (N = 3462) were very likely to receive treatment during follow-up, with only 1.5% not receiving treatment. Among patients with no vitiligo treatment prior to diagnosis, time to first medication claim ranged from 51.9 days (standard deviation [SD], 84.0) for TCI to 178.6 days (SD 116.0) for systemic immunosuppressants; mean total days supplied ranged from 14.4 days (SD 27.1) for oral corticosteroids to 121.0 (SD 114.0) for immunosuppressants. Conclusion: In this real-world study, a high proportion of patients did not receive any treatment. Among those receiving treatment, most were unlikely to switch or use a combination of treatments within the first year of vitiligo diagnosis.
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    Regulatory Acceptable Real-World Effectiveness Endpoints: How Clarity Can Best be Achieved in the United States
    (2023-12) Beyrer, Julie; Hess, Lisa M.; Nan, Hongmei; Lederer, Nirosha M.
    Background: The therapies many patients need do not exist today. Real-world evidence (RWE) can accelerate patient access to treatment. However, lack of clarity on regulatory acceptable realworld effectiveness endpoints can result in delayed or lost new therapy opportunities to bring new treatments to patients. Research question: How can clarity on regulatory acceptable real-world effectiveness endpoints in the United States best be achieved? Methods: A qualitative research study was conducted by interviewing expert informants from diverse stakeholder types to explore their perceptions of gaps, solutions, action needed, and determinants for achieving clarity. Themes were derived using an inductive coding approach, followed by an appraisal of the potential macro-level solutions using Bardach's criteria for policy analysis. Results: Four macro-level solutions were identified: Food and Drug Administration (FDA) Advancing RWE Program, FDA report or dashboard for stakeholders to more easily locate FDA reviews and decisions about RWE, a tool to help stakeholders apply existing related FDA guidance, and FDA guidance on real-world effectiveness endpoints. Plan for Change: Recommended actions for implementing the four solutions and potential evaluation measures were derived from interview themes and Expert Recommendations for Implementing Change. Of the four solutions, an immediate opportunity that FDA and non-FDA stakeholders could initiate is a tool to help stakeholders apply existing FDA guidance, precedent, and endpoint validation principles from the scientific literature. Conclusion: Two solutions are already in progress to increase stakeholders experiences with RWE for regulatory decision making (e.g., FDA Advancing RWE Program and FDA report on RWE submitted for regulatory review). Parallel implementation of other solutions is likely needed (e.g., developing a tool to help stakeholders navigate the existing guidance and precedents on real-world effectiveness endpoints), with regulatory guidance on real-world effectiveness endpoints to follow. Good engagement and transparency across the stakeholder communities are essential to make the most meaningful impact.
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    The Society for Cardiovascular Magnetic Resonance Registry at 150,000
    (Elsevier, 2024-07-04) Tong, Matthew S.; Slivnick, Jeremy A.; Sharif, Behzad; Kim, Han W.; Young, Alistair A.; Sierra-Galan, Lilia M.; Mukai, Kanae; Farzaneh-Far, Afshin; Al-Kindi, Sadeer; Chan, Angel T.; Dibu, George; Elliott, Michael D.; Ferreira, Vanessa M.; Grizzard, John; Kelle, Sebastian; Lee, Simon; Malahfji, Maan; Petersen, Steffen E.; Polsani, Venkateshwar; Toro-Salazar, Olga H.; Shaikh, Kamran A.; Shenoy, Chetan; Srichai, Monvadi B.; Stojanovska, Jadranka; Tao, Qian; Wei, Janet; Weinsaft, Jonathan W.; Wince, W. Benjamin; Chudgar, Priya D.; Judd, Matthew; Judd, Robert M.; Shah, Dipan J.; Simonetti, Orlando P.; Medicine, School of Medicine
    Background: Cardiovascular magnetic resonance (CMR) is increasingly utilized to evaluate expanding cardiovascular conditions. The Society for Cardiovascular Magnetic Resonance (SCMR) Registry is a central repository for real-world clinical data to support cardiovascular research, including those relating to outcomes, quality improvement, and machine learning. The SCMR Registry is built on a regulatory-compliant, cloud-based infrastructure that houses searchable content and Digital Imaging and Communications in Medicine images. The goal of this study is to summarize the status of the SCMR Registry at 150,000 exams. Methods: The processes for data security, data submission, and research access are outlined. We interrogated the Registry and presented a summary of its contents. Results: Data were compiled from 154,458 CMR scans across 20 United States sites, containing 299,622,066 total images (∼100 terabytes of storage). Across reported values, the human subjects had an average age of 58 years (range 1 month to >90 years old), were 44% (63,070/145,275) female, 72% (69,766/98,008) Caucasian, and had a mortality rate of 8% (9,962/132,979). The most common indication was cardiomyopathy (35,369/131,581, 27%), and most frequently used current procedural terminology code was 75561 (57,195/162,901, 35%). Macrocyclic gadolinium-based contrast agents represented 89% (83,089/93,884) of contrast utilization after 2015. Short-axis cines were performed in 99% (76,859/77,871) of tagged scans, short-axis late gadolinium enhancement (LGE) in 66% (51,591/77,871), and stress perfusion sequences in 30% (23,241/77,871). Mortality data demonstrated increased mortality in patients with left ventricular ejection fraction <35%, the presence of wall motion abnormalities, stress perfusion defects, and infarct LGE, compared to those without these markers. There were 456,678 patient-years of all-cause mortality follow-up, with a median follow-up time of 3.6 years. Conclusion: The vision of the SCMR Registry is to promote evidence-based utilization of CMR through a collaborative effort by providing a web mechanism for centers to securely upload de-identified data and images for research, education, and quality control. The Registry quantifies changing practice over time and supports large-scale real-world multicenter observational studies of prognostic utility.
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    Using multiple imputation of real-world data to estimate clinical remission in pediatric inflammatory bowel disease
    (Becaris, 2023) Zhang, Nanhua; Liu, Chunyan; Steiner, Steven J.; Colletti, Richard B.; Baldassano, Robert; Chen, Shiran; Cohen, Stanley; Kappelman, Michael D.; Saeed, Shehzad; Conklin, Laurie S.; Strauss, Richard; Volger, Sheri; King, Eileen; Lo, Kim Hung; Pediatrics, School of Medicine
    Aims: To evaluate the performance of the multiple imputation (MI) method for estimating clinical effectiveness in pediatric Crohn's disease in the ImproveCareNow registry; to address the analytical challenge of missing data. Materials & methods: Simulation studies were performed by creating missing datasets based on fully observed data from patients with moderate-to-severe Crohn's disease treated with non-ustekinumab biologics. MI was used to impute sPCDAI remission statuses in each simulated dataset. Results: The true remission rate (75.1% [95% CI: 72.6%, 77.5%]) was underestimated without imputation (72.6% [71.8%, 73.3%]). With MI, the estimate was 74.8% (74.4%, 75.2%). Conclusion: MI reduced nonresponse bias and improved the validity, reliability, and efficiency of real-world registry data to estimate remission rate in pediatric patients with Crohn's disease.