Results 211 to 220 of about 22,997 (249)

The Impact of the Orphan Drug Act on the Development and Advancement of Neurological Products for Rare Diseases: A Descriptive Review

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, S N, Freeman, M A, Imoisili, T R, Coté   +3 more
openaire   +2 more sources

Mode 2 knowledge production: evidence from orphan drug networks

Science and Public Policy, 2007
This 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

Incentivizing Orphan Product Development: United States Food and Drug Administration Orphan Incentive Programs

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

The Orphan Drug Act and the Federal Government's Orphan Products Development Program.

Public health reports (Washington, D.C. : 1974), 1984
Through the combined efforts of agencies and organizations in the public and private sector, drugs have been made available that would not have been at hand without a specific focus on the orphan drug issue. It is anticipated that these cooperative efforts will continue beyond the first enthusiastic burst engendered by the inception of new and ...
openaire   +1 more source

Orphan products: drugs and devices for rare diseases.

Nursing economic$, 1993
The issues of orphan products include an inadequate definition of orphan status that hampers implementation of the legislation, difficulties in evaluating the products themselves in order to ensure safety and effectiveness, neglect in developing orphan devices, and conflict over creating unnecessary monopolies and excessive profits.
openaire   +1 more source

Survey Indicates 133 Orphan Drug Products in Development for 96 Relatively Rare Disorders

JAMA: The Journal of the American Medical Association, 1989
ABSTRACT EIGHTY-FIVE companies have 133 drugs in various stages of development for 96 rare diseases.This is the first comprehensive listing of orphan drugs since the Orphan Drug Act became law in 1983. The list was compiled by the Pharmaceutical Manufacturers Association in response to a request by the National Commission on Orphan Diseases (JAMA. 1989;
openaire   +1 more source

The Orphan Drug Products—Another Solution?

Drug Intelligence & Clinical Pharmacy, 1983
openaire   +1 more source

Successfully Navigating Food and Drug Administration Orphan Drug and Rare Pediatric Disease Designations for AAV9-hPCCA Gene Therapy: The National Institutes of Health Platform Vector Gene Therapy Experience

Human Gene Therapy, 2023
Charles Venditti, Richa Lomash, Manoli Irini   +2 more
exaly  

Orphan Drug Development in Cardiovascular Medicine

Circulation: Cardiovascular Quality and Outcomes, 2020
Javed Butler, Muthiah Vaduganathan, Muhammad Khan   +2 more
exaly  

The Reform of the Orphan Drug Assessment System in China

Journal of Pharmaceutical Innovation, 2021
Xiaoying Zheng, Lin Wang, Qiang Zheng
exaly