Detalhe da pesquisa
1.
Advancing rare disease treatment: EMA's decade-long insights into engineered adoptive cell therapy for rare cancers and orphan designation.
Gene Ther
; 2024 Mar 14.
Artigo
em Inglês
| MEDLINE | ID: mdl-38480914
2.
The European landscape for gene therapies in orphan diseases: 6-year experience with the EMA Committee for Orphan Medicinal Products.
Mol Ther
; 31(12): 3414-3423, 2023 Dec 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-37794679
3.
[Evidence-based health care with pharmaceuticals for rare diseases: the role of digitalisation]. / Evidenzbasierte Arzneimittelversorgung bei Seltenen Erkrankungen: die Rolle der Digitalisierung.
Bundesgesundheitsblatt Gesundheitsforschung Gesundheitsschutz
; 65(11): 1170-1177, 2022 Nov.
Artigo
em Alemão
| MEDLINE | ID: mdl-36264322
4.
First-line tandem high-dose chemotherapy and autologous stem cell transplantation versus single high-dose chemotherapy and autologous stem cell transplantation in multiple myeloma, a systematic review of controlled studies.
Cochrane Database Syst Rev
; 10: CD004626, 2012 Oct 17.
Artigo
em Inglês
| MEDLINE | ID: mdl-23076906
5.
Licensing of Orphan Medicinal Products-Use of Real-World Data and Other External Data on Efficacy Aspects in Marketing Authorization Applications Concluded at the European Medicines Agency Between 2019 and 2021.
Front Pharmacol
; 13: 920336, 2022.
Artigo
em Inglês
| MEDLINE | ID: mdl-36034814
6.
Defining Satisfactory Methods of Treatment in Rare Diseases When Evaluating Significant Benefit-The EU Regulator's Perspective.
Front Med (Lausanne)
; 8: 744625, 2021.
Artigo
em Inglês
| MEDLINE | ID: mdl-34513895
7.
Assessment of significant benefit for orphan medicinal products by European regulators may support subsequent relative effectiveness assessments by health technology assessment organizations.
Drug Discov Today
; 25(7): 1223-1231, 2020 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-32344040
8.
Comparative analysis of the scope of European Union paediatric investigation plans with corresponding orphan designations.
Arch Dis Child
; 103(5): 427-430, 2018 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-29089318
9.
Establishing rarity in the context of orphan medicinal product designation in the European Union.
Drug Discov Today
; 23(3): 681-686, 2018 Mar.
Artigo
em Inglês
| MEDLINE | ID: mdl-28647377
10.
Demonstrating significant benefit of orphan medicines: analysis of 15 years of experience in Europe.
Drug Discov Today
; 23(1): 90-100, 2018 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-29024805
11.
Evolving prevalence of haematological malignancies in orphan designation procedures in the European Union.
Orphanet J Rare Dis
; 12(1): 17, 2017 01 21.
Artigo
em Inglês
| MEDLINE | ID: mdl-28109318
12.
Clinical pharmacology of tyrosine kinase inhibitors becoming generic drugs: the regulatory perspective.
J Exp Clin Cancer Res
; 33: 15, 2014 Feb 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-24502453
13.
Establishing medical plausibility in the context of orphan medicines designation in the European Union.
Orphanet J Rare Dis
; 9: 175, 2014 Dec 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-25475155
14.
Defining orphan conditions in the context of the European orphan regulation: challenges and evolution.
Nat Rev Drug Discov
; 18(7): 479-480, 2019 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-30940922
15.
Erratum to: Establishing medical plausibility in the context of orphan medicines designation in the European Union.
Orphanet J Rare Dis
; 10: 129, 2015 Oct 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-26444545