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…
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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,…
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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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The purpose of this study was to systematically assess the prevalence, clinical consequences, and reporting of treatment-emergent hypophosphatemia within literature investigating IVI therapies marketed in the United States (US)
Authors
John Glaspy, Michelle Lim-Watson, Michael A Libre, Swagata Karkare, Nandini Hadker, Aleksandra Bajic-Lucas, William Strauss, Naomi Dahl
Journal
Therapeutics and Clinical Risk Management
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Recent developments in digitally assisted vitreo-retinal surgery (DAVS) and heads-up setup were introduced to the global ophthalmic surgical community to enhance visualization with digital signal amplification, higher resolution, and increased depth of field, while addressing some of the ergonomic challenges associated with conventional microscopes
Authors
Leighton Morris, William Wiley, Van Sandwick, Chiraag Lathia, Hang Cheng, Dina Abulon, Nandini Hadker, Kalin Hennegan
Journal
ISPOR EU 2020
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While technological or procedural advances have resulted in low complication and re-operation rates, there are limitations of existing technologies — including but not limited to: surgical endo-illumination challenges, need for in-procedure adjustments, and additional information/support provided by other surgical devices — all of which can create challenges for surgeons during procedures
Authors
Leighton Morris, Alan Franklin, Van Sandwick, Chiraag Lathia, Hang Cheng, Nandini Hadker, Kalin Hennegan
Journal
ISPOR EU 2020
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July 06, 2021
Executive Summary
The Chinese National Reimbursement Drug List (NRDL) is managed and negotiated by the National Healthcare Security Administration (NHSA) and drugs listed on the NRDL are reimbursed by the public insurance
Since 2017, on average, a significant price discount of 44-61% was required for products to achieve a positive listing on the NRDL
Based on average 2018 sales data, products included on the 2017 NRDL were able to achieve an increase in sales revenue (188%) despite…
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June 02, 2021
Executive Summary
The Medical Fund Bill would increase drug spending up to 4% of the total healthcare spending in PolandThe reimbursement applications can be reviewed as quickly as 180 days, reducing the typical time-to-market of two – three yearsThe criteria for “innovativeness” that need to be met for inclusion on the list are still not fully fleshed out to dateOnly four of the 12 candidates on the list have been approved by the Health Minister to secure reimbursement…
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May 25, 2021
Executive Summary
Branded novel combinations are increasingly entering the oncology market but bring a unique set of challenges to gain access and pricing successDynamics beyond demonstrating synergistic efficacy should be taken into consideration to optimize combination price / access outcomes
Trinity’s Take: In this article, Trinity uses select combination case studies to highlight the key elements to consider when thinking through price / access strategy for a novel combination
Evaluation of the Novel Combinations Landscape
The pursuit of…
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Communicating a compelling value story to clinicians and payers is key to launching a product successfully and optimizing value throughout the product lifecycle.
But how exactly does one generate evidence that will educate stakeholders on the disease state, shape the future market and support your value narrative?What are the ‘nuts and bolts’ of designing and conducting such studies?How do you distill down to the key value messages from a complex data set to be most impactful to clinical and payer…
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