Applicability and added value of novel methods to improve drug development in rare diseases

Applicability and added value of novel methods to improve drug development in rare diseases

Abstract Background The ASTERIX project developed a number of novel methods suited to study small populations. The objective of this exercise was to evaluate the applicability and added value of novel methods to improve drug development in small populations, using real world drug development program...

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Bibliographic Details
Main Authors: Marian Mitroiu, Katrien Oude Rengerink, Caridad Pontes, Aranzazu Sancho, Roser Vives, Stella Pesiou, Juan Manuel Fontanet, Ferran Torres, Stavros Nikolakopoulos, Konstantinos Pateras, Gerd Rosenkranz, Martin Posch, Susanne Urach, Robin Ristl, Armin Koch, Spineli Loukia, Johanna H. van der Lee, Kit C. B. Roes
Format: Article
Language:English
Published: BMC 2018-11-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Orphan
Rare condition
Clinical trials
Small population
Statistical methods
Online Access:http://link.springer.com/article/10.1186/s13023-018-0925-0

Internet

http://link.springer.com/article/10.1186/s13023-018-0925-0

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