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Orphans in the Market: The History of Orphan Drug Policy [PDF]
Social History of Medicine, 2017 Summary This paper examines the history of orphan drug policy, from the emergence of ‘orphans’ in the American pharmaceutical market in the 1960s, through the debates and agitations that resulted in the passage of the US Orphan Drug Act of 1983, to attempts in the 1990s to prevent abuse of that Act and restore its original intentions ...
Koichi Mikami
openaire +5 more sources
Do investors value the FDA orphan drug designation?
Orphanet Journal of Rare Diseases, 2017 Background The Orphan Drug Act is an important piece of legislation that uses financial incentives to encourage the development of drugs that treat rare diseases.
Kathleen L. Miller
doaj +2 more sources
Orphan Drug Prices and Epidemiology of Rare Diseases: A Cross-Sectional Study in Italy in the Years 2014-2019. [PDF]
Front Med (Lausanne), 2022 Introduction It is well acknowledged that the price of orphan drugs is normally higher than that resulting from the value-based pricing. A correlation between the cost of therapy for orphan drugs and the epidemiology (prevalence and incidence) of the ...
Villa F +9 more
europepmc +2 more sources
The impact of the rare disease and Orphan Drug Act in Taiwan. [PDF]
J Food Drug Anal, 2021 The Rare Disease and Orphan Drug Act (the Act) was enacted in 2000 in Taiwan for the facilitation of the research, development, and accessibility of orphan drugs and special nutritional foods; for the prevention and early diagnosis of rare diseases; and ...
Hsiang NC +4 more
europepmc +2 more sources
A systematic review of moral reasons on orphan drug reimbursement. [PDF]
Orphanet J Rare Dis, 2021 Background The number of market approvals of orphan medicinal products (OMPs) has been increasing steadily in the last 3 decades. While OMPs can offer a unique chance for patients suffering from rare diseases, they are usually very expensive. The growing
Zimmermann BM +2 more
europepmc +2 more sources
Drugs and biologics receiving FDA orphan drug designation: an analysis of the most frequently designated products and their repositioning strategies. [PDF]
Expert Opin Orphan Drugs, 2022 Background 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 ...
Miller KL, Kraft S, Ipe A, Fermaglich L.
europepmc +2 more sources
Racial and ethnic disparities in COVID-19 mortality in the United States
Journal of Ideas in Health, 2022 Background: Among COVID-19–associated deaths reported in the United States (U.S.), minority communities were disproportionately represented. The objective was to assess differences in mortality by race and ethnicity among patients with coronavirus ...
Francisco Javier Prado-Galbarro +4 more
doaj +1 more source
The European journal of health economics : HEPAC : health economics in prevention and care, 2023 Background Over the past decades, US Congress enabled the US Food and Drug Administration (FDA) to facilitate and expedite drug development for serious conditions filling unmet medical needs with five special designations and review pathways: orphan ...
D. Michaeli +4 more
semanticscholar +1 more source
British medical journal, 2023 Objective To analyze the US Food and Drug Administration (FDA) approval, trials, unmet needs, benefit, and pricing of ultra-rare (200 000 citizens) orphan cancer drug indications and non-orphan cancer drug indications.
T. Michaeli, H. Jürges, D. Michaeli
semanticscholar +1 more source