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Rare Disease Policy in High-Income Countries: An Overview of Achievements, Challenges, and Solutions.
Value Health
January 2025
Canadian Organization for Rare Disorders.
Objectives: To provide an overview of policy initiatives in high-income countries aimed at supporting the development and accessibility of treatments for rare diseases.
Methods: We examine how legislative, research, and pricing policies in high-income countries address barriers that have historically hindered innovation and access to rare disease treatments. By analysing examples from the EU, UK, US, Canada, Japan, and Australia, the article identifies ongoing initiatives, outlines current challenges, and explores proposed solutions to foster a sustainable, innovative, and accessible rare disease treatment ecosystem.
2025 SWHR Women's Health Research Agenda: Prioritizing Uterine Fibroids, Lupus, and Metabolism.
J Womens Health (Larchmt)
January 2025
Society for Women's Health Research, Washington, DC, USA.
Women face unique and multifaceted challenges throughout their lifespans, shaped by biological, societal, and health care-related factors. These challenges have led to gender disparities in disease burden, access to care, and representation in medical research, underscoring the need to increase targeted investments in women's health. Historically, research on diseases that disproportionately affect women has been underfunded, hindering progress in closing gender health gaps.
View Article and Find Full Text PDFBayesian Borrowing With Multiple Heterogeneous Historical Studies Using Order Restricted Normalized Power Prior.
Stat Med
February 2025
U.S. Food and Drug Administration, Silver Spring, Maryland.
The recent U.S. Food and Drug Administration guidance on complex innovative trial designs acknowledges the use of Bayesian strategies to incorporate historical information based on clinical expertise and data similarity.
View Article and Find Full Text PDFTransgressive hybrids as hopeful holobionts.
Microbiome
January 2025
Department of Biological Sciences, Clemson University, Clemson, SC, 29631, USA.
Background: Hybridization between evolutionary lineages has profound impacts on the fitness and ecology of hybrid progeny. In extreme cases, the effects of hybridization can transcend ecological timescales by introducing trait novelty upon which evolution can act. Indeed, hybridization can even have macroevolutionary consequences, for example, as a driver of adaptive radiations and evolutionary innovations.
View Article and Find Full Text PDFInfant birth weight in Brazil: A cross-sectional historical approach.
Soc Sci Med
January 2025
University of California, Riverside, 900 University Ave, Riverside, CA, 92521, United States. Electronic address:
In 1888, Brazil became the last country in the Americas to abolish slavery. Historians have outlined the racialized health disparities of people of African descent in the post-abolition period. Epidemiologists have shown that twenty-first-century health disparities continue to mirror patterns from over a century ago.
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