Results 241 to 250 of about 68,610 (290)
Some of the next articles are maybe not open access.
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
openaire +1 more source
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
openaire +1 more source
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
openaire +2 more sources
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
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 ...
openaire +2 more sources
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 ...
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 ...
M E, Haffner, J V, Kelsey
openaire +2 more sources
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.
openaire +2 more sources
Mode 2 knowledge production: evidence from orphan drug networks
Science and Public Policy, 2007This paper seeks to contribute to the understanding of public participation in the process of science policy-making and the lay public's contribution to scientific knowledge production. Nowotny et al (2001) claim that the traditional boundaries between expert and lay knowledge are being transgressed and science and technology are thereby enriched.
openaire +3 more sources
Plant molecular farming: a promising frontier for orphan drug production
Biotechnology LettersOrphan diseases comprise a range of disorders that individually affect a small percentage of people, but collectively impact millions of people worldwide. Patients with this disorder often face significant challenges in diagnosis, treatment, and access to care due to their rare nature and limited understanding and treatment options.
Balamurugan Shanmugaraj +2 more
openaire +2 more sources
2017
Over 30 years ago, the United States (US) Congress passed the Orphan Drug Act (ODA) to encourage the development of products for rare diseases or conditions ("orphan products"). The Act provided incentives to sponsors for developing products with orphan designation and established a grant program to fund studies of orphan products.
openaire +2 more sources
Over 30 years ago, the United States (US) Congress passed the Orphan Drug Act (ODA) to encourage the development of products for rare diseases or conditions ("orphan products"). The Act provided incentives to sponsors for developing products with orphan designation and established a grant program to fund studies of orphan products.
openaire +2 more sources