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
"Is the Copy Better than the Original? The Regulation of Orphan Drugs: a US-EU Comparative Perspective" [PDF]
, 2012 The US Orphan Drug Act of 1983 pioneered the regulation of this type of medicines, and its success encouraged other countries to enact similar legislation. Among these new orphan drug laws is the one that was drafted in the European Union in 2000.
Garcia, Anton Leis
core
Adenylosuccinic Acid: An Orphan Drug with Untapped Potential. [PDF]
Pharmaceuticals (Basel), 2023 Rybalka E +5 more
europepmc +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
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
Advancing hypoparathyroidism treatment: FDA approval of Palopegteriparatide as a promising orphan drug. [PDF]
Ann Med Surg (Lond)Shahid SR +4 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