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[Orphan diseases: evaluations of new treatments].

Authors
  • Bergmann, J F
  • Delcey, V
  • Champion, K
Type
Published Article
Journal
La Revue de Médecine Interne
Publisher
Elsevier
Publication Date
Oct 01, 2007
Volume
28 Suppl 2
Identifiers
PMID: 18228689
Source
Medline
License
Unknown

Abstract

The new treatments of orphan diseases must be assessed according to the methodologic rules of therapeutic trials, in order to ensure clinically and statistically significant evaluations: randomized controled trials, single primary end-point, statistically significant and clinically relevant efficience. Although these rules may be relaxed in orphan diseases, they have to maintain the standard of therapeutic efficiency.

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