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内部出版物
CNKI学位论文(117)
知网期刊论文(69)
在“
SpringerLink电子期刊
”中,
命中:
33
条,耗时:0.0159646 秒
在所有数据库中总计命中:
186
条
1.
Are
product
s with an
orphan
designation for oncology indications different from
product
s for other rare indications? A retrospective analysis of European
orphan
designations granted between 2002-2012
作者:
Kim Pauwels
;
Isabelle Huys
;
Minne Casteels…
关键词:
Orphan
designation
;
Orphan
medicinal
product
;
Neoplastic disorders
;
Cancer
;
EMA
刊名:
Orphan
et Journal of Rare Diseases
出版年:2017
2.
Evolving prevalence of haematological malignancies in
orphan
designation procedures in the European Union
作者:
Benedetta Polsinelli
;
Stelios Tsigkos…
关键词:
European
orphan
regulation
;
Committee for
orphan
medicinal
product
s
;
Orphan
designation
;
Haematological malignancies
;
Prevalence
;
Market exclusivity
刊名:
Orphan
et Journal of Rare Diseases
出版年:2017
3.
Principles for consistent value assessment and sustainable funding of
orphan
drugs in Europe
作者:
Laura Gutierrez
;
Julien Patris
;
Adam Hutchings…
关键词:
Orphan
medicinal
product
s
;
Rare diseases
;
Value assessment
刊名:
Orphan
et Journal of Rare Diseases
出版年:2015
4.
Entwicklung von
Orphan
Drugs
作者:
Univ.-Prof. Dr. Thomas Stulnig
关键词:
Seltene Erkrankungen
;
Medikamente
;
Morbus Gaucher
;
Lysosomale Speicherkrankheit
;
Stoffwechselerkrankung
;
Rare diseases
;
Orphan
drug development
;
Gaucher disease
;
Lysosomal storage diseases
;
Metabolic diseases
刊名:Wiener klinisches Magazin
出版年:2015
5.
Use of biomarkers in the context of
orphan
medicines designation in the European Union
作者:
Stelios Tsigkos (1)
Jordi Llinares (1)
Segundo Mariz (1)
Stiina Aarum (1)
Laura Fregonese (1)
Bozenna Dembowska-Baginska (2)
Rembert Elbers (4)
Pauline Evers (2)
Tatiana Foltanova (3)
Andre Lhoir (2)
Ana Corrêa-Nunes (2)
Daniel O’Connor (2)
Albertha Voordouw (5)
Kerstin Westermark (2)
Bruno Sepodes (2) (6)
关键词:
Orphan
medicinal
product
designation
;
Distinct medical entity
;
Biomarker
刊名:
Orphan
et Journal of Rare Diseases
出版年:2014
6.
Reconciling uncertainty of costs and outcomes with the need for access to
orphan
medicinal
product
s: a comparative study of managed entry agreements across seven European countries
作者:
Thomas Morel (1)
Francis Arickx (2)
Gustaf Befrits (3)
Paolo Siviero (4)
Caroline van der Meijden (5)
Entela Xoxi (4)
Steven Simoens (1)
关键词:
Managed entry agreements
;
Orphan
drugs
;
Orphan
medicinal
product
s
;
Access
;
Reimbursement
;
Risk sharing
;
Uncertainty
;
Health technology assessment
;
Payers
;
Pricing
;
Performance
刊名:
Orphan
et Journal of Rare Diseases
出版年:2013
7.
Clinical evidence for
orphan
medicinal
product
s-a cause for concern?
作者:
Eline Picavet (11)
David Cassiman (12)
Carla E Hollak (13)
Johan A Maertens (14)
Steven Simoens (11)
关键词:
Quality assessment
;
Quality tool
;
Pivotal study
刊名:
Orphan
et Journal of Rare Diseases
出版年:2013
8.
Estimating the budget impact of
orphan
drugs in Sweden and France 2013-020
作者:
Adam Hutchings (1)
Carina Schey (2)
Richard Dutton (3)
Felix Achana (4)
Karolina Antonov (5)
关键词:
Orphan
medicines
;
Budget impact
;
Health economics
;
Europe
刊名:
Orphan
et Journal of Rare Diseases
出版年:2014
9.
Development and validation of COMPASS: clinical evidence of
orphan
medicinal
product
s -an assessment tool
作者:
Eline Picavet (1)
David Cassiman (2)
Bert Aertgeerts (3) (4)
Steven Simoens (1)
关键词:
Quality assessment
;
Quality assessment tool
;
Quality of reporting
刊名:
Orphan
et Journal of Rare Diseases
出版年:2013
10.
Drug development for exceptionally rare metabolic diseases: challenging but not impossible
作者:
Michelle Putzeist (15) (16)
Aukje K Mantel-Teeuwisse (15)
Christine C Gispen-de Wied (16)
Arno W Hoes (17)
Hubert GM Leufkens (15) (16)
Remco LA de Vrueh (18)
关键词:
Rare disease
;
Orphan
medicinal
product
;
Inherited metabolic disease
;
Prevalence
;
Preclinical proof of concept
刊名:
Orphan
et Journal of Rare Diseases
出版年:2013
1
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