title: Equal access to innovative therapies and precision cancer care.
abstract: Patients with cancers of differing histologies that express certain biomarkers are likely to benefit from treatment with targeted therapies. However, targets can be present in malignancies other than those indicated by a drug's label, and as a result, affected patients will have no access to those potentially useful drugs. To tackle this issue, the French National Cancer Institute developed the AcSé Programme in 2013. This programme is designed to make treatment decisions or recommendations on the basis of the presence of relevant biomarkers for malignancies with no targeted therapies available and also aims to improve safety, and evaluate the efficacy of targeted drugs used outside of their approved indications. Patients across France have access to molecular testing in 28 molecular genetics centres and to targeted therapies within phase II trials provided no other trials exist in which they could reasonably be included. Trials include patients below the age of 18 if safe dosing data are available. As of January 2016, 183 French clinical sites and over 7,000 patients are participating in AcSé led trials. Proof of concept is being demonstrated through trials designed to investigate the effectiveness of crizotinib and vemurafenib in a wide variety of cancers.
authors.affiliation: Institut National du Cancer, 52 avenue André Morizet, F-92513 Boulogne-Billancourt, France.
authors.forename: Agnès
authors.initials: A
authors.lastname: Buzyn
authors.affiliation: Centre Léon Bérard, 28 Rue Laennec, F-69008, Lyon, France.|University of Lyon, 92 Rue Pasteur, F-69007 Lyon, France.
authors.forename: Jean-Yves
authors.initials: JY
authors.lastname: Blay
authors.affiliation: Institut National du Cancer, 52 avenue André Morizet, F-92513 Boulogne-Billancourt, France.
authors.forename: Natalie
authors.initials: N
authors.lastname: Hoog-Labouret
authors.affiliation: R&D Unicancer, 101 rue de Tolbiac, F-75654 Paris Cedex 13, France.
authors.forename: Marta
authors.initials: M
authors.lastname: Jimenez
authors.affiliation: Institut National du Cancer, 52 avenue André Morizet, F-92513 Boulogne-Billancourt, France.
authors.forename: Frédérique
authors.initials: F
authors.lastname: Nowak
authors.affiliation: Gustave Roussy, 114 rue Edouard Vaillant, F-94805, Villejuif, France and University Paris-SUD, Faculté de Médecine, 63 Rue Gabriel Péri, F-94270 Le Kremlin Bicêtre, France.|INSERM CSEP Team 2, 114 rue Edouard Vaillant, F-94805, Villejuif, France.
authors.forename: Marie-Cécile Le
authors.initials: MC
authors.lastname: Deley
authors.affiliation: Centre Léon Bérard, 28 Rue Laennec, F-69008, Lyon, France.
authors.forename: David
authors.initials: D
authors.lastname: Pérol
authors.affiliation: R&D Unicancer, 101 rue de Tolbiac, F-75654 Paris Cedex 13, France.
authors.forename: Christian
authors.initials: C
authors.lastname: Cailliot
authors.affiliation: ARC Foundation for Cancer Research, 9 Rue Guy Moquet, F-94805 Villejuif, France.
authors.forename: Jacques
authors.initials: J
authors.lastname: Raynaud
authors.affiliation: Gustave Roussy, 114 rue Edouard Vaillant, F-94805, Villejuif, France and University Paris-SUD, Faculté de Médecine, 63 Rue Gabriel Péri, F-94270 Le Kremlin Bicêtre, France.
authors.forename: Gilles
authors.initials: G
authors.lastname: Vassal
mesh_terms: D000970:Antineoplastic Agents / Q000627:therapeutic use; D014408:Biomarkers, Tumor / Q000378:metabolism; D002986:Clinical Trials as Topic; D000077547:Crizotinib; D006297:Health Services Accessibility; D006801:Humans; D007211:Indoles / Q000627:therapeutic use; D009369:Neoplasms / Q000235:genetics / Q000628:therapy*; D056687:Off-Label Use; D057285:Precision Medicine / Q000592:standards*; D047428:Protein Kinase Inhibitors / Q000627:therapeutic use; D011720:Pyrazoles / Q000627:therapeutic use; D011725:Pyridines / Q000627:therapeutic use; D013449:Sulfonamides / Q000627:therapeutic use; D000077484:Vemurafenib
chemical_list: D000970:Antineoplastic Agents; D014408:Biomarkers, Tumor; D007211:Indoles; D047428:Protein Kinase Inhibitors; D011720:Pyrazoles; D011725:Pyridines; D013449:Sulfonamides; D000077484:Vemurafenib; D000077547:Crizotinib