title: [Orphan diseases: evaluations of new treatments].
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.
authors.affiliation: Service de Médecine Interne A, Hôpital Lariboisière, 2, rue Ambroise Paré, 75475 Paris 10, France. jf.bergmann@lrb.aphp.fr
authors.forename: J F
authors.initials: JF
authors.lastname: Bergmann
authors.forename: V
authors.initials: V
authors.lastname: Delcey
authors.forename: K
authors.initials: K
authors.lastname: Champion
mesh_terms: D003627:Data Interpretation, Statistical; D004311:Double-Blind Method; D005776:Gaucher Disease / Q000628:therapy*; D006801:Humans; D015337:Multicenter Studies as Topic; D010919:Placebos; D016032:Randomized Controlled Trials as Topic; D035583:Rare Diseases / Q000628:therapy*
chemical_list: D010919:Placebos