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Orphan Lung Diseases A Clinical Guide to Rare Lung Disease
The second edition comprehensively reviews the wide field of rare pulmonary diseases. Issues such as less common diseases affecting the airways,...
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A comprehensive study of the rare diseases and conditions targeted by orphan drug designations and approvals over the forty years of the Orphan Drug Act
BackgroundRare diseases affect more than 30 million Americans. The passage of the Orphan Drug Act (ODA) in the United States in 1983 represented a...
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Issues, Challenges and Opportunities for Economic Evaluations of Orphan Drugs in Rare Diseases: An Umbrella Review
Background and ObjectivesThere are significant challenges when obtaining clinical and economic evidence for health technology assessments of rare...
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An ‘Adaptive Treatment Strategy’ for Oral Vancomycin in Patients with the Orphan Disease Primary Sclerosing Cholangitis
Decision-making in clinical medicine ideally is based upon evidence from randomized, placebo-controlled trials (RCTs) and subsequent systematic...
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Analysis of Incentive Policies and Initiatives on Orphan Drug Development in China: Challenges, Reforms and Implications
ObjectivesRare diseases are a global public health issue with a more pressing situation in China. Unfortunately, the relevant research and...
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Angeborene Stoffwechselerkrankungen – Orphan Diseases aus der Sicht der Pädiatrie
Orphan diseases are rare or ultra-rare diseases with a prevalence of less than 5 individuals in 10,000 inhabitants according to an EU-wide...
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The importance of the policy framework on orphan drug accessibility
PurposeAs orphan drugs are agents for treating rare diseases, the pharmaceutical industry usually has little interest in develo** them without...
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Implementation status of pharmacological studies in the development of orphan drugs
BackgroundThe nonclinical as well as clinical development of orphan drugs is difficult, owing to unknown pathophysiology and the absence of animal...
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The Challenge for Orphan Drugs Remains: Three Case Studies Demonstrating the Impact of Changes to NICE Methods and Processes and Alternative Mechanisms to Value Orphan Products
BackgroundThe National Institute for Health and Care Excellence (NICE) is responsible for ensuring that patients in England and Wales can access...
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Orphan medical devices have come a long way
BackgroundIn many countries worldwide orphan drug regulations are installed but only the United States of America and Japan have an orphan device...
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Current status and trend of clinical development of orphan drugs in China
BackgroundRare diseases have been increasingly recognized as unmet medical and health needs worldwide; a growing demand for the development of orphan...
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Special FDA designations for drug development: orphan, fast track, accelerated approval, priority review, and breakthrough therapy
BackgroundOver the past decades, US Congress enabled the US Food and Drug Administration (FDA) to facilitate and expedite drug development for...
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How to START? Four pillars to optimally begin your orphan drug development
Drug development is a complex, resource intensive and long process in any disease area, and develo** medicines to treat rare diseases presents even...
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Costs of orphan medicinal products: longitudinal analysis of expenditure in Wales
BackgroundThe Orphan Regulation ((EC) No 141/2000) has successfully redirected private and public investment towards previously neglected areas...
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The actual status of drug prices and adjustment factors for drug price calculation: an analysis of ultra-orphan drug development in Japan
BackgroundExtremely high prices facilitate drug development for ultra-rare diseases (ultra-orphan drugs). However, various problems arise in terms of...
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Trends in orphan medicinal products approvals in the European Union between 2010–2022
BackgroundOver the last twenty years of orphan drug regulation in Europe, the regulatory framework has increased its complexity, with different...
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How do patients and other members of the public engage with the orphan drug development? A narrative qualitative synthesis
BackgroundThe diversity of patient experiences of orphan drug development has until recently been overlooked, with the existing literature reporting...
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Exploring alternative financing models and early access schemes for orphan drugs: a Belgian case study
BackgroundAlthough some jurisdictions have implemented particular adjustments to accommodate often-expensive orphan drugs in their healthcare...
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Pharmacovigilance for rare diseases: a bibliometrics and knowledge-map analysis based on web of science
ObjectivesThe aims of this paper is to search and explore publications in the field of pharmacovigilance for rare diseases and to visualize general...
