Drug repurposing: From the discovery of a useful pharmacological effect to making the treatment available to the patient.

Autor:	Deplanque D; Université Lille, Inserm, CHU Lille, centre d'investigation clinique (CIC) 1403, 59000 Lille, France. Electronic address: dominique.deplanque@univ-lille.fr., Fetro C; Fondation Maladies Rares, 75014 Paris, France., Ferry A; Laboratoires CTRS, 92200 Neuilly-sur-Seine, France., Lechat P; Université Paris-cité, service de pharmacologie et toxicologie, hôpital européen Georges-Pompidou, 75015 Paris, France; Agence générale des équipements et des produits de santé (AGEPS), Assistance publique-Hôpitaux de Paris, 75005 Paris, France., Beghyn T; APTEEUS SAS, campus Institut Pasteur, 59000 Lille, France., Bernard C; Agence générale des équipements et des produits de santé (AGEPS), Assistance publique-Hôpitaux de Paris, 75005 Paris, France., Bernasconi A; Laboratoires SERB, 75008 Paris, France., Bienayme H; ORPHELIA Pharma, 75005 Paris, France., Cougoule C; Institut de pharmacologie et de biologie structurale (IPBS), université de Toulouse, CNRS, université Toulouse III - Paul-Sabatier (UPS), 31400 Toulouse, France., Del Bano J; Aix-Marseille université, AP-HM, Inserm, DHUNE, Inst Neurosci Syst, service de pharmacologie clinique et pharmacovigilance, Thelonius Mind, 13000 Marseille, France., Demiot C; UR 20218-NeurIT, faculties of medicine and pharmacy, university of Limoges, 87025 Limoges, France., Lebrun-Vignes B; Service de pharmacologie et centre régional de pharmacovigilance, hôpital Pitié-Salpêtrière, groupe hospitalier, AP-HP, Sorbonne université, 75013 Paris, France.
Jazyk:	angličtina
Zdroj:	Therapie [Therapie] 2023 Jan-Feb; Vol. 78 (1), pp. 10-18. Date of Electronic Publication: 2022 Dec 05.
DOI:	10.1016/j.therap.2022.11.009
Abstrakt:	The repurposing of a medicine already on the market to a new indication could be an opportunity to respond rapidly to a therapeutic need not yet covered, particularly in the context of rare and neglected diseases, or health emergencies. However, at each stage, difficulties may arise that will prevent the repurposed drug from being provided to patients. Beyond fortuity or a systematic strategy to detect a useful pharmacological effect, the implementation of the preclinical and clinical stages is sometimes complicated by the difficulty of accessing the molecule and its pharmaceutical data. Furthermore, relevant clinical results will not always be sufficient to ensure that a marketing authorisation is obtained or that patients receive satisfactory care. In addition to describing these various obstacles, the round table provided an opportunity to put forward recommendations for overcoming them, in particular the creation of a public-private partnership structure with sufficient funding to be able to offer individualised support for projects up to and including the marketing application. (Copyright © 2022 Société française de pharmacologie et de thérapeutique. Published by Elsevier Masson SAS. All rights reserved.)
Databáze:	MEDLINE
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