194 related articles for article (PubMed ID: 30486835)
1. Oncologic orphan drugs approved in the EU - do clinical trial data correspond with real-world effectiveness?
Schuller Y; Biegstraaten M; Hollak CEM; Klümpen HJ; Gispen-de Wied CC; Stoyanova-Beninska V
Orphanet J Rare Dis; 2018 Nov; 13(1):214. PubMed ID: 30486835
[TBL] [Abstract][Full Text] [Related]
2. Factors Contributing to the Efficacy-Effectiveness Gap in the Case of Orphan Drugs for Metabolic Diseases.
Schuller Y; Hollak CEM; Gispen-de Wied CC; Stoyanova-Beninska V; Biegstraaten M
Drugs; 2017 Sep; 77(13):1461-1472. PubMed ID: 28752290
[TBL] [Abstract][Full Text] [Related]
3. Orphan drugs, orphan diseases. The first decade of orphan drug legislation in the EU.
Joppi R; Bertele' V; Garattini S
Eur J Clin Pharmacol; 2013 Apr; 69(4):1009-24. PubMed ID: 23090701
[TBL] [Abstract][Full Text] [Related]
4. Trends in orphan medicinal products approvals in the European Union between 2010-2022.
Bouwman L; Sepodes B; Leufkens H; Torre C
Orphanet J Rare Dis; 2024 Feb; 19(1):91. PubMed ID: 38413985
[TBL] [Abstract][Full Text] [Related]
5. Clinical evidence for orphan medicinal products-a cause for concern?
Picavet E; Cassiman D; Hollak CE; Maertens JA; Simoens S
Orphanet J Rare Dis; 2013 Oct; 8():164. PubMed ID: 24131572
[TBL] [Abstract][Full Text] [Related]
6. 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.
Naumann-Winter F; Wolter F; Hermes U; Malikova E; Lilienthal N; Meier T; Kalland ME; Magrelli A
Front Pharmacol; 2022; 13():920336. PubMed ID: 36034814
[No Abstract] [Full Text] [Related]
7. Determinants for successful marketing authorisation of orphan medicinal products in the EU.
Putzeist M; Heemstra HE; Garcia JL; Mantel-Teeuwisse AK; Gispen-De Wied CC; Hoes AW; Leufkens HG
Drug Discov Today; 2012 Apr; 17(7-8):352-8. PubMed ID: 22094244
[TBL] [Abstract][Full Text] [Related]
8. Cancer drugs for solid tumors approved by the EMA since 2014: an overview of pivotal clinical trials.
Lasala R; Logreco A; Romagnoli A; Santoleri F; Musicco F; Costantini A
Eur J Clin Pharmacol; 2020 Jun; 76(6):843-850. PubMed ID: 32125472
[TBL] [Abstract][Full Text] [Related]
9. [Cancer: Is it really so different? Particularities of oncologic drugs from the perspective of the pharmaceutical regulatory agency].
Enzmann H; Broich K
Z Evid Fortbild Qual Gesundhwes; 2013; 107(2):120-8. PubMed ID: 23663906
[TBL] [Abstract][Full Text] [Related]
10. The European challenges of funding orphan medicinal products.
Szegedi M; Zelei T; Arickx F; Bucsics A; Cohn-Zanchetta E; Fürst J; Kamusheva M; Kawalec P; Petrova G; Slaby J; Stawowczyk E; Vocelka M; Zechmeister-Koss I; Kaló Z; Molnár MJ
Orphanet J Rare Dis; 2018 Nov; 13(1):184. PubMed ID: 30396361
[TBL] [Abstract][Full Text] [Related]
11. Single-arm trials supporting the approval of anticancer medicinal products in the European Union: contextualization of trial results and observed clinical benefit.
Mulder J; Teerenstra S; van Hennik PB; Pasmooij AMG; Stoyanova-Beninska V; Voest EE; de Boer A
ESMO Open; 2023 Apr; 8(2):101209. PubMed ID: 37054504
[TBL] [Abstract][Full Text] [Related]
12. Comparing access to orphan medicinal products in Europe.
Zamora B; Maignen F; O'Neill P; Mestre-Ferrandiz J; Garau M
Orphanet J Rare Dis; 2019 May; 14(1):95. PubMed ID: 31053154
[TBL] [Abstract][Full Text] [Related]
13. Assessing the benefit of cancer drugs approved by the European Medicines Agency using the European Society for Medical Oncology Magnitude of Clinical Benefit Scale over time.
Thomson S; Witzke N; Gyawali B; Delos Santos S; Udayakumar S; Cardone C; Cheung MC; Chan KKW
Eur J Cancer; 2021 Jun; 150():203-210. PubMed ID: 33932727
[TBL] [Abstract][Full Text] [Related]
14. Estimating the budget impact of orphan drugs in Sweden and France 2013-2020.
Hutchings A; Schey C; Dutton R; Achana F; Antonov K
Orphanet J Rare Dis; 2014 Feb; 9():22. PubMed ID: 24524281
[TBL] [Abstract][Full Text] [Related]
15. Observations on Three Endpoint Properties and Their Relationship to Regulatory Outcomes of European Oncology Marketing Applications.
Liberti L; Stolk P; McAuslane JN; Schellens J; Breckenridge AM; Leufkens H
Oncologist; 2015 Jun; 20(6):683-91. PubMed ID: 25948678
[TBL] [Abstract][Full Text] [Related]
16. [The role of real-world evidence for the study of rare diseases epidemiology and the post-marketing evaluation of orphan drugs.].
Crisafulli S; Trifirò G
Recenti Prog Med; 2022; 113(7):425-429. PubMed ID: 35852077
[TBL] [Abstract][Full Text] [Related]
17. Orphan Medicinal Products for the Treatment of Pancreatic Cancer: Lessons Learned From Two Decades of Orphan Designation.
Mulder J; van Rossum T; Mariz S; Magrelli A; de Boer A; Pasmooij AMG; Stoyanova-Beninska V
Front Oncol; 2021; 11():809035. PubMed ID: 34988030
[TBL] [Abstract][Full Text] [Related]
18. Costs of orphan medicinal products: longitudinal analysis of expenditure in Wales.
Pijeira Perez Y; Wood E; Hughes DA
Orphanet J Rare Dis; 2023 Nov; 18(1):342. PubMed ID: 37915031
[TBL] [Abstract][Full Text] [Related]
19. Folic acid supplementation and malaria susceptibility and severity among people taking antifolate antimalarial drugs in endemic areas.
Crider K; Williams J; Qi YP; Gutman J; Yeung L; Mai C; Finkelstain J; Mehta S; Pons-Duran C; Menéndez C; Moraleda C; Rogers L; Daniels K; Green P
Cochrane Database Syst Rev; 2022 Feb; 2(2022):. PubMed ID: 36321557
[TBL] [Abstract][Full Text] [Related]
20. Evidence supporting regulatory-decision making on orphan medicinal products authorisation in Europe: methodological uncertainties.
Pontes C; Fontanet JM; Vives R; Sancho A; Gómez-Valent M; Ríos J; Morros R; Martinalbo J; Posch M; Koch A; Roes K; Oude Rengerink K; Torrent-Farnell J; Torres F
Orphanet J Rare Dis; 2018 Nov; 13(1):206. PubMed ID: 30442155
[TBL] [Abstract][Full Text] [Related]
[Next] [New Search]
