169 related articles for article (PubMed ID: 36249759)
21. 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]
22. High-cost drugs for rare diseases: their expenditure and value based on a regional area-based study.
Manea S; Visonà Dalla Pozza L; Minichiello C; Altieri L; Mazzucato M; Bonin M; De Ambrosis P; Borgonovi E; Facchin P
Health Serv Manage Res; 2024 Feb; 37(1):52-60. PubMed ID: 36627202
[No Abstract] [Full Text] [Related]
23. Italian cancer figures--Report 2015: The burden of rare cancers in Italy.
; Busco S; Buzzoni C; Mallone S; Trama A; Castaing M; Bella F; Amodio R; Bizzoco S; Cassetti T; Cirilli C; Cusimano R; De Angelis R; Fusco M; Gatta G; Gennaro V; Giacomin A; Giorgi Rossi P; Mangone L; Mannino S; Rossi S; Pierannunzio D; Tavilla A; Tognazzo S; Tumino R; Vicentini M; Vitale MF; Crocetti E; Dal Maso L
Epidemiol Prev; 2016; 40(1 Suppl 2):1-120. PubMed ID: 26951748
[TBL] [Abstract][Full Text] [Related]
24. A population-based registry as a source of health indicators for rare diseases: the ten-year experience of the Veneto Region's rare diseases registry.
Mazzucato M; Visonà Dalla Pozza L; Manea S; Minichiello C; Facchin P
Orphanet J Rare Dis; 2014 Mar; 9():37. PubMed ID: 24646171
[TBL] [Abstract][Full Text] [Related]
25. Orphan medicinal products in Europe and United States to cover needs of patients with rare diseases: an increased common effort is to be foreseen.
Giannuzzi V; Conte R; Landi A; Ottomano SA; Bonifazi D; Baiardi P; Bonifazi F; Ceci A
Orphanet J Rare Dis; 2017 Apr; 12(1):64. PubMed ID: 28372595
[TBL] [Abstract][Full Text] [Related]
26. 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]
27. The Evolution of AIFA Registries to Support Managed Entry Agreements for Orphan Medicinal Products in Italy.
Xoxi E; Facey KM; Cicchetti A
Front Pharmacol; 2021; 12():699466. PubMed ID: 34456724
[TBL] [Abstract][Full Text] [Related]
28. Availability and Accessibility of Orphan Medicinal Products to Patients in Slovakia in the Years 2010-2019.
Foltanova T; Majernik A; Malikova E; Kosirova S
Front Pharmacol; 2022; 13():768325. PubMed ID: 35153774
[No Abstract] [Full Text] [Related]
29. A multi-stakeholder multicriteria decision analysis for the reimbursement of orphan drugs (FinMHU-MCDA study).
de Andrés-Nogales F; Cruz E; Calleja MÁ; Delgado O; Gorgas MQ; Espín J; Mestre-Ferrándiz J; Palau F; Ancochea A; Arce R; Domínguez-Hernández R; Casado MÁ;
Orphanet J Rare Dis; 2021 Apr; 16(1):186. PubMed ID: 33902672
[TBL] [Abstract][Full Text] [Related]
30. Access and pricing of medicines for patients with rare diseases in the European Union: an industry perspective.
Tafuri G; Bracco A; Grueger J
Expert Rev Pharmacoecon Outcomes Res; 2022 Apr; 22(3):381-389. PubMed ID: 34930086
[TBL] [Abstract][Full Text] [Related]
31. [The analysis of detection of rare diseases not included into listings of orphan diseases with preferential medication support].
Komarov IA; Krasilnikova EI; Zhulev IA; Zinchenko RF
Probl Sotsialnoi Gig Zdravookhranenniiai Istor Med; 2020 Sep; 28(5):893-902. PubMed ID: 33161658
[TBL] [Abstract][Full Text] [Related]
32. Off-label use of orphan medicinal products: a Belgian qualitative study.
Dooms M; Cassiman D; Simoens S
Orphanet J Rare Dis; 2016 Oct; 11(1):144. PubMed ID: 27793155
[TBL] [Abstract][Full Text] [Related]
33. Are rare diseases still orphans or happily adopted? The challenges of developing and using orphan medicinal products.
Dear JW; Lilitkarntakul P; Webb DJ
Br J Clin Pharmacol; 2006 Sep; 62(3):264-71. PubMed ID: 16934041
[TBL] [Abstract][Full Text] [Related]
34. 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]
35. 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]
36. Drug development for exceptionally rare metabolic diseases: challenging but not impossible.
Putzeist M; Mantel-Teeuwisse AK; Wied CC; Hoes AW; Leufkens HG; de Vrueh RL
Orphanet J Rare Dis; 2013 Nov; 8():179. PubMed ID: 24237580
[TBL] [Abstract][Full Text] [Related]
37. How Can We Optimize the Value Assessment and Appraisal of Orphan Drugs for Reimbursement Purposes? A Qualitative Interview Study Across European Countries.
Blonda A; Denier Y; Huys I; Kawalec P; Simoens S
Front Pharmacol; 2022; 13():902150. PubMed ID: 35928274
[No Abstract] [Full Text] [Related]
38. Development and validation of COMPASS: clinical evidence of orphan medicinal products - an assessment tool.
Picavet E; Cassiman D; Aertgeerts B; Simoens S
Orphanet J Rare Dis; 2013 Oct; 8():157. PubMed ID: 24107467
[TBL] [Abstract][Full Text] [Related]
39. Drug Repurposing for Rare Diseases: A Role for Academia.
van den Berg S; de Visser S; Leufkens HGM; Hollak CEM
Front Pharmacol; 2021; 12():746987. PubMed ID: 34744726
[No Abstract] [Full Text] [Related]
40. Independent Registries Are Cost-Effective Tools to Provide Mandatory Postauthorization Surveillance for Orphan Medicinal Products.
Sirrs SM; Arthus MF; Bichet DG; Rockman-Greenberg C; LeMoine K; Morel CF; Lachmann R; Lynd LD; Wasim S; West ML; Hollak C
Value Health; 2021 Feb; 24(2):268-273. PubMed ID: 33518033
[TBL] [Abstract][Full Text] [Related]
[Previous] [Next] [New Search]
