The 2023 launch class exceeded Trinity Life Sciences’ expectations with only 50% underperforming their pre-launch first year forecasts while 39% overperformed, indicating an improvement from prior years (54% and 35%, respectively, for 2020–2023). The needle is moving.
This retrospective patient medical chart study aimed to assess the treatment patterns and real-world practice outcomes of NMIBC patients who were treated with BCG. Authors Sam Chang, Kyle Martin, Dan Shaffer, Amy Guo, Nandini Hadker Journal ISPOR Meeting; May 2020 View publication…
This analysis of real-world hospital data aimed to characterize the treatment regimens for inpatients experiencing rCDI, including both hospital- and community-acquired recurrence, and to compare these regimens to current treatment guidelines. Authors Gavin Miyasato, Hannah Li, Dushyant Katariya, Kevin Francis, Nandini Hadker Journal ISPOR Meeting; May 2020 View publication…
Network meta-analysis (NMA) compares multiple health technologies when there is a lack of direct evidence with randomized controlled trials (RCT) alone. While supplementing RCTs with robust real-world evidence (RWE) in NMAs has become standard practice, NMAs of RWE alone are now being used to gain insights into the patient experience in clinical practice. This SLR aimed to assess the quality of published RWE-only NMAs and evaluate the statistical methods used to account for clinical and statistical heterogeneity. Authors Nambiar S,…
This study analyzed direct medical costs of PWS patients and compared these to non-PWS patients; cost variation between those receiving growth hormone (GH) and those not was also evaluated. Authors Justin Li, David Yin, Qian Meng, Abigail Silber, Sina Aghsaei, Kevin Francis, Nandini Hadker, Theresa Strong, Jennifer Miller, Merlin Butler, Ann Manzardo, Michael Yeh, Shawn Czado Journal ISPOR Meeting; May 2020 View publication…
The purpose of this systematic literature review (SLR) was to assess current data on burden of survival for pediatric CXTpatients receiving platinum-based regimens in terms of hearing loss, specifically related to costs and quality of life (QoL)-based metrics. Authors Hennegan K, Silber A, Yuan A, Miyasato G, Li J, Soneji Y, Danker W Journal ISPOR Meeting; May 2020 View publication…
The purpose of this systematic literature review (SLR) was to assess current data on burden of survival for pediatric CXTpatients receiving platinum-based regimens in terms of hearing loss, specifically related to costs and quality of life (QoL)-based metrics. Authors Kalin Hennegan, Abigail Silber, Sumudu Dehipawala, Kevin Chithran, Donna Lockhart Journal ISPOR Meeting; May 2020 View publication…
Chronic Graft-Versus-Host Disease (cGVHD) is a complication of hematopoietic cell transplantation (HCT). While the clinical outcomes of cGVHD are well documented, few studies have assessed its treatment practices in the real-world. The objectives of this study are to quantify the prevalence of cGVHD, to examine provider prescribing patterns, and to evaluate the healthcare cost and resource utilization (HCRU) in a real-world US cGVHD population. Authors Carlos Bachier, Sanjay Aggarwal, Kalin Hennegan, Andrew Milgroom, Kevin Francis, Marcello Rotta Journal Blood; American…
Prader-Willi syndrome (PWS) is a complex orphan endocrine disease characterized by hyperphagia and abnormal food-related behaviors that contribute to severe morbidity and early mortality along with a significant burden on patients and caregivers. Life-long medical care is required but the consistency of services rendered to this population has not been evaluated. This study characterized use of US hospital care, specialty physician care, and growth hormone (GH) therapy for PWS patients at different life stages. Authors Merlin Butler, Ann Manzardo, Theresa…
Prader-Willi syndrome (PWS) is a complex developmental genetic disorder associated with hypotonia, poor feeding in neonates, onset of hyperphagia in early childhood, and shorter overall life expectancy. Prior epidemiology studies of PWS have examined smaller populations, with limited research in a US population. The aim of this study was to provide a contemporary estimate of PWS prevalence and annual all-cause mortality in the US using a large administrative medical claims dataset. Authors Shawn McCandless, Marissa Suh, David Yin, Michael Yeh,…
Prader-Willi syndrome (PWS) is a rare, complex multi-system genetic disorder characterized by hyperphagia and abnormal food-related behaviors that contribute to severe morbidity and early mortality and to a significant burden on patients and caregivers. The hyperphagia seen in people with PWS can result in significant obesity. This study assessed rates of comorbidities associated with obesity, including type II diabetes (T2D), cardiovascular disease (CVD), and sleep apnea (SA) in a large US PWS vs. a non-PWS cohort. Authors Diane Stafford, Justin…
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