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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.
The disparities and development trajectories of nations in achieving the sustainable development goals.
Nat Commun
January 2025
State Key Laboratory of Urban and Regional Ecology, Research Center for Eco-Environmental Sciences, Chinese Academy of Sciences, Beijing, China.
The Sustainable Development Goals (SDGs) provide a comprehensive framework for societal progress and planetary health. However, it remains unclear whether universal patterns exist in how nations pursue these goals and whether key development areas are being overlooked. Here, we apply the product space methodology, widely used in development economics, to construct an 'SDG space of nations'.
View Article and Find Full Text PDFEmerging trends in managed care pharmacy: A mixed-method study.
J Manag Care Spec Pharm
January 2025
Academy of Managed Care Pharmacy Foundation, Alexandria, VA.
Background: Over the past 5 years, managed care pharmacy has been shaped by a global pandemic, advancements in generative artificial intelligence (AI), Medicare drug price negotiation policies, and significant therapeutic developments. Collective intelligence methods can be used to anticipate future developments in practice to help organizations plan and develop new strategies around those changes.
Objective: To identify emerging trends in managed care pharmacy.
Interpretable adenylation domain specificity prediction using protein language models.
bioRxiv
January 2025
Computational Biology Department, School of Computer Science, Carnegie Mellon University, Pittsburgh, PA, USA.
Natural products have long been a rich source of diverse and clinically effective drug candidates. Non-ribosomal peptides (NRPs), polyketides (PKs), and NRP-PK hybrids are three classes of natural products that display a broad range of bioactivities, including antibiotic, antifungal, anticancer, and immunosuppressant activities. However, discovering these compounds through traditional bioactivity-guided techniques is costly and time-consuming, often resulting in the rediscovery of known molecules.
View Article and Find Full Text PDFGlobal insight into rare disease and orphan drug definitions: a systematic literature review.
BMJ Open
January 2025
Centre for Public Health, Institute of Clinical Sciences B, Royal Victoria Hospital, Queen's University Belfast School of Medicine, Dentistry and Biomedical Sciences, Belfast, UK.
Objectives: This study sheds light on the available global definitions, classifications, and criteria used for rare diseases (RDs), ultrarare diseases (URDs), orphan drugs (ODs) and ultraorphan drugs (UODs) and provides insights into the rationale behind these definitions.
Design: A systematic literature review was conducted to identify existing definitions and the criteria used to define RDs, ODs and their subtypes.
Data Sources: Searches were performed in the PubMed/Medline, Embase, Scopus and Web of Science (Science and Social Sciences Citation Index) databases covering articles published from 1985 to 2021.
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