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O – Orphan Product Approach
When government takes an active role in meeting socio-economic needs for technological innovation that lack sufficient free market incentives, the...
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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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Advancing rare disease treatment: EMA’s decade-long insights into engineered adoptive cell therapy for rare cancers and orphan designation
Adoptive cell therapy (ACT), particularly chimeric antigen receptor (CAR)-T cell therapy, has emerged as a promising approach for targeting and...
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Orphan drug development: Challenges, regulation, and success stories
Rare diseases, also known as orphan diseases, are diseases with low occurrence in the population. Develo** orphan drugs is challenging because of...
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Regulatory Considerations Toward Orphan Drug Designation and Orphan Drug Exclusivity in the United States and European Union: Structural Similarity, Clinical Superiority/Significant Benefit, and Case Studies
The U.S. Food and Drug Administration and European Commission have developed successful orphan drug legislation to promote the research, development,...
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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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Analysis on the Marketing Trend and Approval Lag of Imported Orphan Drugs from 2010 to 2021 in China
BackgroundIn order to meet the unmet needs of rare disease patients in China, importing orphan drugs is an important way. The objectives of this...
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Orphan drug development in alpha-1 antitypsin deficiency
Alpha-1 antitrypsin deficiency (AATD, OMIM #613490) is a rare metabolic disorder affecting lungs and liver. The purpose of this study is to assess...
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Towards ethical drug pricing: the European Orphan Genomic Therapies Fund
An increasing number of novel genomic therapies are expected to become available for patients with rare or ultra-rare diseases. However, the primary...
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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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Pharmacological Profile of FDA-Approved Orphan Drugs in the Year 2022
Purpose of ReviewThis manuscript aimed to provide a scientific report of recently FDA-approved orphan drugs for hel** patients, researchers,...
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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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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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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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Orphan Drugs and Rare Diseases
There are approximately 8,000 recognized rare diseases affecting close to 30 million people in the United States. Sponsors choosing to engage in...
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Activation of orphan receptor GPR132 induces cell differentiation in acute myeloid leukemia
Blocked cellular differentiation is a critical pathologic hallmark of acute myeloid leukemia (AML). Here, we showed that genetic activation of the...
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The Impact of US Medical Product Regulatory Complexity on Innovation: Preliminary Evidence of Interdependence, Early Acceleration, and Subsequent Inversion
PurposeIs the complexity of medical product (medicines and medical devices) regulation impacting innovation in the US? If so, how?
Methods ...
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Expert System for the Diagnosis of Orphan Diseases
Diagnosis of orphan (rare), in particular hereditary diseases, is associated with difficulties due to the diversity of pathology and polymorphism...