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Orphan Medical Devices and Pediatric Cardiology – What Interventionists in Europe Need to Know, and What Needs to be Done
Medical devices include a great diversity of technologies, which are evaluated and approved in the European Union (EU) according to a revised law...
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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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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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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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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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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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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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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 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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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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Exploring the feasibility of using the ICER Evidence Rating Matrix for Comparative Clinical Effectiveness in assessing treatment benefit and certainty in the clinical evidence on orphan therapies for paediatric indications
BackgroundThe evaluation of clinical evidence takes account of health benefit (efficacy and safety) and the degree of certainty in the estimate of...
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Lifecycle management of orphan drugs approved in Japan
BackgroundThe development of orphan drugs (ODs) is challenging from both development and business perspectives because of their small patient...
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Is an Orphan Drug’s Cost-Effectiveness Associated with US Health Plan Coverage Restrictiveness?
Background and ObjectivesOrphan drugs’ high prices raise questions about whether their costs are worth their benefits. We examined the association...
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Patients, payers and developers of Orphan Medicinal Products: lessons learned from 10 years’ multi-stakeholder dialogue on improving access in Europe via MoCA
BackgroundThe Mechanism of Coordinated Access to Orphan Medicinal Products (MoCA) was established in 2013 with the intention of develo** a...
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Prices of Orphan Drugs in Four Western European Countries Before and After Market Exclusivity Expiry: A Cross-Country Comparison of List Prices and Purchase Prices
BackgroundIncreasing pharmaceutical expenditure challenges the sustainability and accessibility of healthcare systems across Europe. Confidentiality...
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Postmarketing safety of orphan drugs: a longitudinal analysis of the US Food and Drug Administration database between 1999 and 2018
BackgroundInformation about the specific regulatory environment of orphan drugs is scarce and inconsistent. Uncertainties surrounding the...
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Analysis of patient access to orphan drugs in Turkey
BackgroundRare diseases are life-threatening, serious, and chronic conditions that require complex care and have a low prevalence. An estimated one...
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FDA orphan products clinical trial grants: assessment of outcomes and impact on rare disease product development
BackgroundThe Office of Orphan Products Development (OOPD) of the United States (U.S.) Food and Drug Administration (FDA) has awarded over 700 grants...
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The impact of early phase price agreements on prices of orphan drugs
BackgroundInnovative orphan drugs often have an incremental cost-effectiveness ratio (ICER) which is higher than the maximum threshold for...
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