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Sous presse. Epreuves corrigées par l'auteur. Disponible en ligne depuis le mardi 10 mars 2020
Doi : 10.1016/j.therap.2020.02.013
Received : 25 September 2019 ;  accepted : 22 October 2019
The challenges of pharmacoepidemiology of orphan drugs in rare diseases

Maryse Lapeyre-Mestre
 Service de pharmacologie médicale et clinique, faculté de médecine, CIC 1436, CHU et université Paul Sabatier – Toulouse 3, 31000 Toulouse, France 


Large electronic health records database available at a national level offer great opportunity for research in rare diseases and orphan drugs. Methods and data used in pharmacoepidemiology present a great potential for epidemiology, drug utilization studies, drug safety, drug effectiveness and pharmacoeconomics. This review presents the different sources of data in Europe, with a special focus on the French situation, with the recent implementation of SNDS (système national des données de santé [French national health data wharehouse]). Some examples are given. Development of rigorous and innovative methods must be encouraged in the future.

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Keywords : Rare disease, Orphan drug, Pharmacoepidemiology, Pharmacovigilance, Electronic health records

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