Translational Research: Precision Medicine, Personalized Medicine, Targeted Therapies: Marketing or Science?

¹ UMR 850 INSERM, CHU Limoges, Université de Limoges, Limoges, France
² Laboratoire Merck Serono SAS, Lyon, France
³ Aix Marseille Université; Assistance Publique – Hôpitaux de Marseille, Service d’Oncologie Multidisciplinaire et Innovations Thérapeutiques, Marseille, France
⁴ Laboratore Novartis Pharma SAS, Rueil Malmaison, France
⁵ Inserm-Transfert, Paris, France
⁶ Laboratoire Lilly France, Neuilly-sur-Seine, France
⁷ Institut Universitaire de Cancérologie, Toulouse, France
⁸ LEEM, Paris, France
⁹ ARIIS, Paris, France
¹⁰ Laboratoire Amgen, Neuilly-sur-Seine, France
¹¹ Imperial College, London, Royaume-Uni
¹² Institut Pasteur, Lille, France
¹³ Laboratoire Lundbeck SAS Issy-les-Moulineaux France
¹⁴ Université Lyon 1, HCL, Lyon, France
¹⁵ Institut National du Cancer, Boulogne Billancourt, France
¹⁶ Assistance publique – Hôpitaux de Paris, Paris, France
¹⁷ Université Paris 7, Paris, France
¹⁸ Inserm, Paris, France
¹⁹ HAS, Saint Denis la Plaine, France
²⁰ Laboratoire Roche Diagnostics, Meylan, France
²¹ ANSM, Saint Denis, France
²² CNAMTS, Paris, France
²³ Faculté de Médecine Paris-Sud, Le Kremlin Bicêtre, France

Correspondence and offprints: Pierre Marquet, UMR-S850 INSERM, Université de Limoges, Service de Pharmacologie, Toxicologie et Pharmacovigilance, CHU de Limoges, 87042 Limoges, France. E-mail: pierre.marquet@unilim.fr

Received: 17 December 2014
Accepted: 18 December 2014

Abstract

Personalized medicine is based on: 1) improved clinical or non-clinical methods (including biomarkers) for a more discriminating and precise diagnosis of diseases; 2) targeted therapies of the choice or the best drug for each patient among those available; 3) dose adjustment methods to optimize the benefit-risk ratio of the drugs chosen; 4) biomarkers of efficacy, toxicity, treatment discontinuation, relapse, etc. Unfortunately, it is still too often a theoretical concept because of the lack of convenient diagnostic methods or treatments, particularly of drugs corresponding to each subtype of pathology, hence to each patient. Stratified medicine is a component of personalized medicine employing biomarkers and companion diagnostics to target the patients likely to present the best benefit-risk balance for a given active compound. The concept of targeted therapy, mostly used in cancer treatment, relies on the existence of a defined molecular target, involved or not in the pathological process, and/or on the existence of a biomarker able to identify the target population, which should logically be small as compared to the population presenting the disease considered. Targeted therapies and biomarkers represent important stakes for the pharmaceutical industry, in terms of market access, of return on investment and of image among the prescribers. At the same time, they probably represent only the first generation of products resulting from the combination of clinical, pathophysiological and molecular research, i.e. of translational research.