Recent advances in methodology for clinical trials in small populations: the InSPiRe project-Reference-Cited by-同舟云学术

Recent advances in methodology for clinical trials in small populations: the InSPiRe project

Published:2018-10-25 Issue:1 Volume:13 Page:
ISSN:1750-1172
Container-title:Orphanet Journal of Rare Diseases
language:en
Short-container-title:Orphanet J Rare Dis

Author:

Friede Tim,Posch Martin,Zohar Sarah,Alberti Corinne,Benda Norbert,Comets Emmanuelle,Day Simon,Dmitrienko Alex,Graf Alexandra,Günhan Burak Kürsad,Hee Siew Wan,Lentz Frederike,Madan Jason,Miller Frank,Ondra Thomas,Pearce Michael,Röver Christian,Toumazi Artemis,Unkel Steffen,Ursino Moreno,Wassmer Gernot,Stallard Nigel^ORCID

Funder

Seventh Framework Programme

Publisher

Springer Science and Business Media LLC

Subject

Pharmacology (medical),Genetics(clinical),General Medicine

Link

http://link.springer.com/content/pdf/10.1186/s13023-018-0919-y.pdf

Reference52 articles.

1. European Union. Regulation (EC) No 141/2000 of the European Parliament and of the Council of 16 December 1999 on Orphan Medicinal Products. 2015. http://eur-lex.europa.eu/LexUriServ/LexUriServ.do?uri=OJ:L:2000:018:0001:0005:en:PDF . Accessed 1 Aug 2018.

2. Rare Diseases Act of 2002. https://history.nih.gov/research/downloads/PL107-280.pdf . Accessed 1 August 2018.

3. World Bank Population total. http://api.worldbank.org/v2/en/indicator/SP.POP.TOTL?downloadformat=excel . Accessed 1 Aug 2018.

4. Food and Drug Administration. Rare diseases: Common issues in drug development - Guidance for Industry. 2015. https://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM458485.pdf . Accessed 1 Aug 2018.

5. European Union. HEALTH-2013-INNOVATION-1 Call. 2015. https://ec.europa.eu/research/participants/portal/desktop/en/opportunities/fp7/calls/fp7-health-2013-innovation-1.html . Accessed 1 Aug 2018.

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