Results 121 to 130 of about 110,217 (215)
Arbor: Ciencia, Pensamiento y Cultura, 2018 Orphan drugs are intended for the treatment of diseases of low prevalence, also known as rare diseases. Research and development into new therapies for this heterogeneous group of diseases presents a number of well-recognized difficulties. One of these is that the small number of patients affected jeopardizes the economic return on the investment made ...
Fontanet Sacristán, Juan Manuel +1 more
openaire +1 more source
Using Artificial Intelligence to Advance the Research and Development of Orphan Drugs
BusinessesWhile artificial intelligence has successful and innovative applications in common medicine, could its application facilitate research on rare diseases? This study explores the application of artificial intelligence (AI) in orphan drug research, focusing
Carla Irissarry, Thierry Burger-Helmchen
doaj +1 more source
Regulating Rare Disease: Safely Facilitating Access to Orphan Drugs [PDF]
, 2018 While approximately one in ten Americans suffers from a rare disease, only 5 percent of rare diseases have a U.S. Food and Drug Administration (FDA) approved treatment.
Bannister, Julien B.
core +1 more source
Two-stage public-private partnership proposal for R&D on neglected diseases [PDF]
We propose a 2-stage procurement model of public-private partnership to provide better incentives for R&D on neglected diseases. The model combines advance market commitment, subsidized clinical trials, and rewards based on therapeutical contributions of
Brigitte Granville, Eshref Trushin
core
Adenylosuccinic Acid: An Orphan Drug with Untapped Potential. [PDF]
Pharmaceuticals (Basel), 2023 Rybalka E +5 more
europepmc +1 more source
How to START? Four pillars to optimally begin your orphan drug development. [PDF]
Orphanet J Rare Dis, 2023 Jonker AH +5 more
europepmc +1 more source
A General-Equilibrium Analysis of Public Policy for Pharmaceutical Prices [PDF]
Retail sales of prescription drugs totaled $154.5 billion in 2001. The National Institute for Health Care Management estimates annual sales will exceed $400 billion by the year 2010.
Kelton, Christina M.L. +1 more
core
Orphan drugs' clinical uncertainty and prices: Addressing allocative and technical inefficiencies in orphan drug reimbursement. [PDF]
Front Pharmacol, 2023 Eichler HG +3 more
europepmc +1 more source
A qualitative study on the views of experts on the social impact of the high-priced orphan drug nusinersen. [PDF]
Explor Res Clin Soc Pharm, 2023 Rosenberg S +3 more
europepmc +1 more source