Equal access to innovative therapies and precision cancer care

Agnès Buzyn, Jean Yves Blay, Natalie Hoog-Labouret, Marta Jimenez, Frédérique Nowak, Marie Cécile Le Deley, David Pérol, Christian Cailliot, Jacques Raynaud, Gilles Vassal

    Research output: Contribution to journalReview articlepeer-review

    41 Citations (Scopus)

    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.

    Original languageEnglish
    Pages (from-to)385-393
    Number of pages9
    JournalNature Reviews Clinical Oncology
    Volume13
    Issue number6
    DOIs
    Publication statusPublished - 1 Jun 2016

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