Results 131 to 140 of about 110,217 (215)

Advances in orphan drug development for alpha-1 antitrypsin deficiency: a 2025 update from the FDA and EMA. [PDF]

Ther Adv Respir Dis
Höger P, Ries M, Olivares Rivera A, Ersöz H, Buschulte K, Fähndrich S, Kontogianni K, Herth F, Trudzinski FC.   +8 more
europepmc   +1 more source

Is It Possible to Have Cheaper Drugs and Preserve the Incentive to Innovate: Reforming the Drug Approval Process According to Market Principles [PDF]

This paper argues that drugs are expensive not because of a lack of competition among research-based pharmaceutical companies, but because of a lack of competition in the drug approval process.
Corinne Sauer, Robert Sauer
core  

Correction to: Analysis of the first ten years of FDA’s rare pediatric disease priority review voucher program: designations, diseases, and drug development

Orphanet Journal of Rare Diseases
Catherine Mease, Kathleen L. Miller, Lewis J. Fermaglich, Jeanine Best, Gumei Liu, Erika Torjusen   +5 more
doaj   +1 more source

Advances in orphan drug development: Time to change the status and stereotype. [PDF]

Perspect Clin Res, 2022
Thakur S.
europepmc   +1 more source

Determinants of orphan drug health technology assessment in South Korea: an empirical analysis. [PDF]

Front Pharmacol
Lee SH, Lee SK, Lee JH, Bang JS.
europepmc   +1 more source

How do patients and other members of the public engage with the orphan drug development? A narrative qualitative synthesis. [PDF]

Orphanet J Rare Dis, 2023
Frost J, Hall A, Taylor E, Lines S, Mandizha J, Pope C.   +5 more
europepmc   +1 more source

Concurrent nanotherapeutics and regulatory updates for the management of amyotrophic lateral sclerosis: a focused review for orphan drug (Tofersen). [PDF]

Orphanet J Rare Dis
Kumar A, Shukla S, Rai A, Pathak P, Narayan KP.   +4 more
europepmc   +1 more source

Trends in orphan drug use and spending among children and adolescents during 2010-2020 in Korea. [PDF]

Front Pharmacol, 2022
Oh JY, Lee JY, Kim DS.
europepmc   +1 more source

Rare disease patients in India are rarely involved in international orphan drug trials. [PDF]

PLOS Glob Public Health, 2022
Chakraborty M, Choudhury MC, Chakraborty I, Saberwal G.   +3 more
europepmc   +1 more source

Predictive Analysis for First Submission of Generic Drug Application for Orphan Drug Products Using Random Survival Forest. [PDF]

Clin Transl Sci
Hopefl R, Wang J, Mohan AR, Sun WJ, Kim MJ, Hu M, Fang L.   +6 more
europepmc   +1 more source