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HemaSphere, Volume 10, Issue S1, June 2026.
wiley   +1 more source

Guiding the willingness to pay for repurposed tiratricol in the treatment of Allan-Herndon-Dudley syndrome using a cost-based-plus pricing framework

Manders EA, Keestra S, Bannenberg WJ, de Visser SJ, Hollak CE.   +4 more
europepmc   +1 more source

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Drugs and biologics receiving FDA orphan drug designation: an analysis of the most frequently designated products and their repositioning strategies

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
exaly   +3 more sources

Europe finally agrees to encourage orphan-drug production

Lancet, The, 2000
Arthur Rogers
exaly   +2 more sources

Evaluation of Orphan Products by the U.S. Food and Drug Administration

International Journal of Technology Assessment in Health Care, 1992
AbstractOrphan 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

PP147 Conditional Reimbursement Of Medicinal Products, A Procedure For Orphan Drugs, Conditionals and Exceptionals

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

Orphan Drugs: The Question of Products Liability

American Journal of Law & Medicine, 1985
AbstractOrphan 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

Orphan drug product regulation – United States

Int. Journal of Clinical Pharmacology and Therapeutics, 2002
The 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