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Expert Opinion on Orphan Drugs, 2021
The Orphan Drug Act was created to stimulate the development of drugs and biologics for rare diseases. Investigating products that have received orphan drug designation provide a greater understanding of rare disease drug development, as well as the repositioning business models of developers.We used a dataset containing all orphan drug designations ...
Kathleen L, Miller +3 more
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The Orphan Drug Act was created to stimulate the development of drugs and biologics for rare diseases. Investigating products that have received orphan drug designation provide a greater understanding of rare disease drug development, as well as the repositioning business models of developers.We used a dataset containing all orphan drug designations ...
Kathleen L, Miller +3 more
openaire +2 more sources
Evaluation of Orphan Products by the U.S. Food and Drug Administration
International Journal of Technology Assessment in Health Care, 1992AbstractOrphan drug products generally are used in treating or preventing rare diseases. The small number of patients available for study may create special problems in the evaluation of these products. This paper examines some of the special problems that are associated with the design and implementation of studies to evaluate the safety and efficacy ...
John V. Kelsey, Marlene E. Haffner
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Orphan Drugs: The Question of Products Liability
American Journal of Law & Medicine, 1985AbstractOrphan drugs, essential for die treatment of persons widi rare diseases, generally are unprofitable for manufacturers to develop and market. While congressional and administrative efforts to promote die development of orphan drugs have met widi modest success, application of products liability doctrine to orphan drug sponsors could subvert ...
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Orphan drugs and paediatric medicinal products
Pharmaceuticals, Policy and Law, 2013While the safety and availability of medicinal products for the majority of adult patients has steadily improved in recent decades, for children and people suffering from rare diseases (orphan diseases) there is a lack of approved medicinal products for these patient populations.
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Orphan drug product regulation – United States
Int. Journal of Clinical Pharmacology and Therapeutics, 2002The legislative history of the United States Orphan Drug Act began with rare, unanimous approval by the United States Congress. The Act, mid consequently the Regulations, have evolved since then. The two-stage process of Orphan Drug designation and approval is outlined, as well as the incentives that are offered to commercial companies for their ...
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International Journal of Technology Assessment in Health Care, 2022
IntroductionIn 2018 the Dutch Ministry of Health (MoH) introduced a new policy regarding the conditional reimbursement (CR) of drugs in the basic health insurance package. This new policy offers patients with a serious, often rare disease for which no effective treatment is yet available, the possibility of obtaining controlled access to new promising ...
Floor van Heesch, Marijke De Vries
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IntroductionIn 2018 the Dutch Ministry of Health (MoH) introduced a new policy regarding the conditional reimbursement (CR) of drugs in the basic health insurance package. This new policy offers patients with a serious, often rare disease for which no effective treatment is yet available, the possibility of obtaining controlled access to new promising ...
Floor van Heesch, Marijke De Vries
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Current status of orphan disease drug development.
Current opinion in pediatrics, 1994The Orphan Drug Act has successfully stimulated the production of many orphan products for a number of orphan diseases. The success of its exclusive marketing provision in bringing otherwise unprofitable products to market has attracted the attention of ...
J. Thoene
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Journal of Medicinal Chemistry, 2023
The master transcription factor receptor retinoic acid receptor-related orphan receptor γt (RORγt) regulates the differentiation of T-helper 17 (Th17) cells and the production of interleukin-17 (IL-17).
Lixue Lu +15 more
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The master transcription factor receptor retinoic acid receptor-related orphan receptor γt (RORγt) regulates the differentiation of T-helper 17 (Th17) cells and the production of interleukin-17 (IL-17).
Lixue Lu +15 more
semanticscholar +1 more source
Clinical Pharmacology & Therapeutics, 2010
Many neurological diseases or conditions are rare disorders. The Orphan Drug Act (ODA) of 1983 was promulgated to promote the development of products for such conditions. In this Opinion piece, we discuss how the ODA has affected neurological diseases, note how current and future sponsors (any person(s) or entity (i.e., academic, corporate body ...
K A Burke +3 more
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Many neurological diseases or conditions are rare disorders. The Orphan Drug Act (ODA) of 1983 was promulgated to promote the development of products for such conditions. In this Opinion piece, we discuss how the ODA has affected neurological diseases, note how current and future sponsors (any person(s) or entity (i.e., academic, corporate body ...
K A Burke +3 more
openaire +3 more sources
Dutch hospital’s manufacture of drug for rare condition was legal, says inspectorate
British medical journal, 2018A five month investigation by Dutch health inspectors into a leading university hospital’s cost price production of orphan drug chenodeoxycholic acid (CDCA) has concluded that such “small scale” preparation is legal.
T. Sheldon
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