Results 11 to 20 of about 3,868,024 (353)
The drug development pipeline for glioblastoma-A cross sectional assessment of the FDA Orphan Drug Product designation database. [PDF]
PLoS ONE, 2021 BackgroundGlioblastoma (GBM) is the most common malignant brain tumour among adult patients and represents an almost universally fatal disease. Novel therapies for GBM are being developed under the orphan drug legislation and the knowledge on the ...
Pascal Johann, Dominic Lenz, Markus Ries
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Establishing a reasonable price for an orphan drug [PDF]
Cost Effectiveness and Resource Allocation, 2020 Background This paper addresses the question of what a reasonable price for an orphan drug is. The research proposes a way to adjust an established payer/HTA body incremental cost-effectiveness threshold (CET) to take account of differences in patient ...
Mikel Berdud +2 more
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Precision Medicines’ Impact on Orphan Drug Designation [PDF]
Clinical and Translational Science, 2019 The incentives provided under the Orphan Drug Act (ODA) have been credited for catalyzing the marketing approval of drugs for the treatment of rare diseases by the US Food and Drug Administration.
Christine M. Mueller +2 more
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Orphanet Journal of Rare Diseases, 2021 Background Orphan drug designations are a useful proxy to investigate trends in rare disease drug development. Drug developers must receive a designation before they are eligible for the economic incentives of the Orphan Drug Act in the United States. We
Kathleen L. Miller +2 more
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Orphan drug clinical development
Therapies, 2020 Clinical development for orphan drugs is extremely demanding but fascinating. There is no single aspect that is really specific to it but instead it gathers most of the hurdles: design, outcomes, recruitment, ethics, cost, probability and predictability for success.
Joëlle Micallef +4 more
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Visual Research of Global Orphan Drug from a Bibliometric Perspective [PDF]
Drug Design, Development and TherapyXiaoya Wen,1,* Guangzhi Jin,2,* Chunxing Wu3 1Department of Pharmacy, The Fourth People’s Hospital of GuiYang, Guiyang, Guizhou Province, 550007, People’s Republic of China; 2Department of Hemodialysis Room, The Second People’s Hospital ...
Wen X, Jin G, Wu C
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Predictors of orphan drug approval [PDF]
Orphanet Journal of Rare Diseases, 2010 Results More orphan drugs were developed in the US during the first ten years of the US Orphan Drug Act (1983-1992, N=73) and during the first ten years of the EU Regulation on Orphan Medicinal products (2000-2009, N=112) than in the EU (2000-2009, N=59). Orphan drug approval was strongly associated with previous experiences of the sponsor in obtaining
Heemstra Harald
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PLoS ONE, 2016 BackgroundConcerns about the high cost of orphan drugs has led to questions being asked about the generosity of the incentives for development, and associated company profits.MethodsWe conducted a retrospective, propensity score matched study of publicly-
Dyfrig A Hughes, Jannine Poletti-Hughes
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Orphan drugs' clinical uncertainty and prices: Addressing allocative and technical inefficiencies in orphan drug reimbursement. [PDF]
Front Pharmacol, 2023 Legislations incentivising orphan drug development and scientific advances have made orphan drugs pharma’s high-end favourite for the past two decades. Currently, around 50% of new marketing authorizations are for orphan drugs. For third-party healthcare
Eichler HG +3 more
europepmc +2 more sources
BMJ Open, 2019 Objective To determine differences in the characteristics of cancer drugs designated as orphan drugs by the Food and Drug Administration (FDA) and European Medicines Agency (EMA).Design and setting Identification of all cancer drugs (initial or ...
Aaron S Kesselheim +1 more
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