title: The "Temporary Recommendations for Use": A dual-purpose regulatory framework for off-label drug use in France.
abstract: In 2012, following the Mediator(®) (benfluorex) scandal, France displayed the ambitious goal to implement a regulatory framework for controlling off-label drug use: the "Temporary Recommendations for Use" (RTUs). It aims to regulate the use of pharmaceuticals outside the scope of a marketing authorization (MA) by establishing a framework for patient monitoring and data collection. This is intended to ensure that the benefit/risk ratio is favorable for the indication approved by the RTU. The granting of an RTU enables the reimbursement of off-label drug use and encourages pharmaceutical companies to expand their MA. Between 2012 and 2014, the regulator framework for RTUs was amended twice in order to allow the bypassing of an MA for economic reasons, when a licensed alternative drug exists (so far, this is only illustrated by the bevacizumab (Avastin(®))/ranibizumab (Lucentis(®)) case). The primary purpose of the RTU framework is interesting by implementing an original national control for off-label uses that respond to a public health need. The secondary purpose is more controversial as it promotes off-label use. This has raised legal issues and has created a ground for litigation between pharmaceutical firms and health authorities. RTUs provide an interesting example for other countries that are exploring the possibility of regulating off-label drug use. At the same time, the processes surrounding the implementation of RTUs illustrate the difficulties of public policies to balance public health needs, safety and economic goals.
authors.affiliation: Law and Health Economics Department, Faculty of Pharmacy, Paris Descartes University, Sorbonne Paris Cité, 4 avenue de l'Observatoire, 75006 Paris, France; Institut Droit et Santé (INSERM UMR-S 1145), Paris Descartes University, Sorbonne Paris Cité, 45 rue des Saints-Pères, 75006 Paris, France; Pharmacy Department, General Agency of Equipments and Health Products (AGEPS), 7 rue du Fer-à-Moulin, 75005 Paris, France. Electronic address: albane.degrassat-theas@parisdescartes.fr.
authors.forename: Albane
authors.initials: A
authors.lastname: Degrassat-Théas
authors.affiliation: Law and Health Economics Department, Faculty of Pharmacy, Paris Descartes University, Sorbonne Paris Cité, 4 avenue de l'Observatoire, 75006 Paris, France; Institut Droit et Santé (INSERM UMR-S 1145), Paris Descartes University, Sorbonne Paris Cité, 45 rue des Saints-Pères, 75006 Paris, France; Pharmacy Department, General Agency of Equipments and Health Products (AGEPS), 7 rue du Fer-à-Moulin, 75005 Paris, France.
authors.forename: François
authors.initials: F
authors.lastname: Bocquet
authors.affiliation: Pharmacy Department, General Agency of Equipments and Health Products (AGEPS), 7 rue du Fer-à-Moulin, 75005 Paris, France.
authors.forename: Martine
authors.initials: M
authors.lastname: Sinègre
authors.affiliation: Law and Health Economics Department, Faculty of Pharmacy, Paris Descartes University, Sorbonne Paris Cité, 4 avenue de l'Observatoire, 75006 Paris, France; Institut Droit et Santé (INSERM UMR-S 1145), Paris Descartes University, Sorbonne Paris Cité, 45 rue des Saints-Pères, 75006 Paris, France.
authors.forename: Jérôme
authors.initials: J
authors.lastname: Peigné
authors.affiliation: Institut Droit et Santé (INSERM UMR-S 1145), Paris Descartes University, Sorbonne Paris Cité, 45 rue des Saints-Pères, 75006 Paris, France; Pharmacy Department, General Agency of Equipments and Health Products (AGEPS), 7 rue du Fer-à-Moulin, 75005 Paris, France.
authors.forename: Pascal
authors.initials: P
authors.lastname: Paubel
mesh_terms: D004348:Drug Labeling / Q000331:legislation & jurisprudence*; D005602:France; D006801:Humans; D056687:Off-Label Use / Q000331:legislation & jurisprudence*; D011050:Policy Making; D011634:Public Health
keywords: Marketing authorization; Off-label drug use; Policy development; Temporary Recommendations for Use