Results 111 to 120 of about 109,656 (184)

Labeling Pediatric Products: BPCA Efforts and Orphan Drug Opportunities. [PDF]

J Pediatr Pharmacol Ther
Wei H, Burckart GJ, Taylor-Zapata P, Wang Y.   +3 more
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

Orphan Drug Use in Patients With Rare Diseases: A Population-Based Cohort Study. [PDF]

Front Pharmacol, 2022
Gorini F, Santoro M, Pierini A, Mezzasalma L, Baldacci S, Bargagli E, Boncristiano A, Brunetto MR, Cameli P, Cappelli F, Castaman G, Coco B, Donati MA, Guerrini R, Linari S, Murro V, Olivotto I, Parronchi P, Pochiero F, Rossi O, Scappini B, Sodi A, Vannucchi AM, Coi A.   +23 more
europepmc   +1 more source

Debates over orphan drug pricing: a meta-narrative literature review. [PDF]

Orphanet J Rare Dis
Hanchard MS.
europepmc   +1 more source

Discrepancies between FDA documents and ClinicalTrials.gov for Orphan Drug-related clinical trial data. [PDF]

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

A systematic review of moral reasons on orphan drug reimbursement. [PDF]

Orphanet J Rare Dis, 2021
Zimmermann BM, Eichinger J, Baumgartner MR.   +2 more
europepmc   +1 more source

Orphan Drug Prices and Epidemiology of Rare Diseases: A Cross-Sectional Study in Italy in the Years 2014-2019. [PDF]

Front Med (Lausanne), 2022
Villa F, Di Filippo A, Pierantozzi A, Genazzani A, Addis A, Trifirò G, Cangini A, Tafuri G, Settesoldi D, Trotta F.   +9 more
europepmc   +1 more source

Global insight into rare disease and orphan drug definitions: a systematic literature review. [PDF]

BMJ Open
Abozaid GM, Kerr K, Alomary H, Al-Omar HA, McKnight A.   +4 more
europepmc   +1 more source

From care to cure: a patient engagement framework for rare disease and orphan drug research. [PDF]

Ther Adv Rare Dis
Awada N, Varughese A.
europepmc   +1 more source