Results 141 to 150 of about 110,217 (215)

The impact of the rare disease and Orphan Drug Act in Taiwan. [PDF]

J Food Drug Anal, 2021
Hsiang NC, Huang WF, Gau CS, Tsai TW, Chang LC.   +4 more
europepmc   +1 more source

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

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

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

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

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

Five-Year Sales for Newly Marketed Prescription Drugs With and Without Initial Orphan Drug Act Designation.

JAMA, 2023
Tu SS, Nagar S, Kesselheim AS, Lu Z, Rome BN.   +4 more
europepmc   +1 more source