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World J Clin Oncol. Apr 10, 2016; 7(2): 253-257
Published online Apr 10, 2016. doi: 10.5306/wjco.v7.i2.253
Off-label use of targeted therapies in oncology
Dominique Levêque
Dominique Levêque, Division of Pharmacy, hôpital Hautepierre, 67000 Strasbourg, France
Author contributions: Levêque D wrote the paper.
Conflict-of-interest statement: There is no conflict of interest related to this work
Open-Access: This article is an open-access article which was selected by an in-house editor and fully peer-reviewed by external reviewers. It is distributed in accordance with the Creative Commons Attribution Non Commercial (CC BY-NC 4.0) license, which permits others to distribute, remix, adapt, build upon this work non-commercially, and license their derivative works on different terms, provided the original work is properly cited and the use is non-commercial. See: http://creativecommons.org/licenses/by-nc/4.0/
Correspondence to: Dominique Levêque, PhD, Division of Pharmacy, hôpital Hautepierre, avenue Molière, 67000 Strasbourg, France. dominique.leveque@chru-strasbourg.fr
Telephone: +33-3-88128213 Fax: +33-3-88127804
Received: September 2, 2015
Peer-review started: September 8, 2015
First decision: October 16, 2015
Revised: October 23, 2015
Accepted: December 29, 2015
Article in press: January 4, 2016
Published online: April 10, 2016
Processing time: 217 Days and 23.1 Hours
Abstract

Off-label use is defined by the prescription of a marketed drug outside the conditions described in the summary of product characteristics. In oncology, off-label prescribing of targeted therapies may occur in patients with other tumor types expressing the same target. Agents associated to phenotypic approaches such as therapies against the tumoral vasculature (anti-angiogenic drugs) and new immunotherapies (checkpoint inhibitors) also carry the potential of alternative indications or combinations. Off-label use of targeted therapies is little documented and appears to be in the same range than that regarding older drugs with wide variations among agents. When compared with older agents, off-label use of targeted therapies is probably more rational through tumoral genotyping but is faced with a limited clinical support, reimbursement challenges related to the very high pricing and the cost of genotyping or molecular profiling, when applicable.

Keywords: Targeted therapy; Monoclonal antibody; Off-label anticancer drug use; Reimbursement; Enzyme inhibitor

Core tip: Off-label use is defined by the prescription of a marketed drug outside the conditions described in the summary of product characteristics. This review is the first one focussing on the off-label use of targeted therapies in oncology. When compared with older agents, off-label use of targeted therapies is probably more rational through tumoral genotyping but is faced with a limited clinical support, reimbursement challenges related to the very high pricing and the cost of genotyping or molecular profiling, when applicable.