Articles citing this article

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Cited article:

Using a meta-narrative literature review and focus groups with key stakeholders to identify perceived challenges and solutions for generating robust evidence on the effectiveness of treatments for rare diseases

Kylie Tingley, Doug Coyle, Ian D. Graham, et al.
Orphanet Journal of Rare Diseases 13 (1) (2018)
https://doi.org/10.1186/s13023-018-0851-1

Passer des modèles in vitro et animaux à l’homme : les spécificités des maladies rares sont-elles un frein ? L’établissement de la preuve de concept dans le traitement des maladies rares est-il une difficulté ?

Joëlle Micallef
La Presse Médicale 39 (5) 24 (2010)
https://doi.org/10.1016/j.lpm.2010.02.025

Evaluation and Validation of Diagnostic Tests for Guiding Therapeutic Decisions

Paul Landais, Valérie Méresse, Jean-Claude Ghislain, et al.
Thérapie 64 (3) 195 (2009)
https://doi.org/10.2515/therapie/2009028

Safer, Better, More Appropriate: Clinical Trial Design for Pediatric Drug Labels

Susan K. McCune, Lisa L. Mathis, David M. Cocchetto, Karen Ball and W. Rodriguez
Drug Information Journal 40 (2) 185 (2006)
https://doi.org/10.1177/009286150604000207