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Sous presse. Epreuves corrigées par l'auteur. Disponible en ligne depuis le jeudi 27 février 2020
Doi : 10.1016/j.therap.2020.02.014
Received : 16 September 2019 ;  accepted : 15 November 2019
Patients’ organizations in rare diseases and involvement in drug information: Illustrations with LMC France, the French Association of Chronic Myeloid leukemia

Mina Daban a , Clémence Lacroix b, Joëlle Micallef b,
a LMC France, 13010 Marseille, France 
b Inserm, service de pharmacologie clinique et pharmacovigilance, Inst Neurosci Syst, Aix-Marseille université, AP–HM, 13005 Marseille, France 

Corresponding author. Service de pharmacologie clinique, 254, rue Saint-Pierre, 13005 Marseille, France.Service de pharmacologie clinique254, rue Saint-PierreMarseille13005France

There are few areas in medicine in which patient-parent advocacy groups play such a central and prominent role as in the field of rare diseases. As illustrated by the French Association of Chronic Myeloid Leukemia (LMC France), its contribution is not only focused on its role as participants or key informant related to clinical studies but also on drug information and drug safety. The discovery of tyrosine kinase inhibitors against the BCR-ABL1 oncogenic fusion protein has revolutionized the management of CML, becoming a chronic illness rather than a life-threatening disease. Because ensuring ongoing well-being requires some knowledge, LMC France has built, in synergy with healthcare professionals from its scientific council, a CML-specific, patient-friendly knowledge base including resources and knowledge related to drug information (drug development, generics, pharmacovigilance) and drug safety using several educational tools such as videos, CML support, and CML drug sheets. To disseminate more largely, an e-university learning for regional key informants from LMC France and also CML patients and their caregivers was launched, including a large resources related to drug information and drug safety in synergy with hematologists and clinical pharmacologists.

The full text of this article is available in PDF format.

Keywords : Patients’ advocacy, Rare disease, Chronic myeloid leukemia, Pharmacology, Drug information, Orphan drugs

 LMC France, 13010 Marseille, France.

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