Groupe hospitalier mutualiste de Grenoble, institut Daniel Hollard, 8, rue Docteur-Calmette, CS10043, 38028 Grenoble cedex 1, France; ANSM, 93285 Saint-Denis, France.
Roche France, 92650 Boulogne-Billancourt, France.
Therapie. 2019 Feb;74(1):103-117. doi: 10.1016/j.therap.2018.12.002. Epub 2018 Dec 13.
In a context of perpetual evolution of treatments, access to therapeutic innovation is a major challenge for patients and the various players involved in the procedures of access to medicines. The revolutions in genomic and personalized medicine, artificial intelligence and biotechnology will transform the medicine of tomorrow and the organization of our health system. It is therefore fundamental that France prepares for these changes and supports the development of its companies in these new areas. The recent "Conseil stratégique des industries de santé" launched by Matignon makes it possible to propose a regulatory arsenal conducive to the implementation and diffusion of therapeutic innovations. In this workshop, we present a number of proposals, our approach having remained pragmatic with a permanent concern to be effective in the short term for the patients and to simplify the procedures as much as possible. This was achieved thanks to the participation in this workshop of most of the players involved (industrial companies, "Agence nationale de sécurité du médicament et des produits de santé", "Haute Autorité de santé", "Institut national du cancer", "Les entreprises du médicament", hospitals, "Observatoire du médicament, des dispositifs médicaux et de l'innovation thérapeutique"…). The main proposals tend to favor the implementation of clinical trials on our territory, especially the early phases, a wider access to innovations by favoring early access programs and setting up a process called "autorisation temporaire d'utilisation d'extension" (ATUext) that make it possible to prescribe a medicinal product even if the latter has a marketing authorisation in another indication. In addition, we propose a conditional reimbursement that will be available based on preliminary data but will require re-evaluation based on consolidated data from clinical trials and/or real-life data. Finally, in order to better carry out these assessments, with a view to access or care, we propose the establishment of partnership agreements with health agencies/hospitals in order to encourage the emergence of field experts, in order to prioritize an ascending expertise closer to patients' needs and to real life.
在治疗方法不断发展的背景下，患者和参与药品准入程序的各方都面临着获得治疗创新的巨大挑战。基因组和个性化医学、人工智能和生物技术的革命将改变明天的医学和我们的医疗体系组织。因此，法国必须为这些变化做好准备，支持其公司在这些新领域的发展。最近由马提翁发起的“健康产业战略理事会”使得提出有利于实施和推广治疗创新的监管手段成为可能。在本次研讨会上，我们提出了一些建议，我们的方法仍然具有务实性，始终关注在短期内为患者提供有效性，并尽可能简化程序。这是通过邀请大多数相关方（工业公司、“国家药品和医疗产品安全局”、“高级卫生管理局”、“国家癌症研究所”、“药品企业”、医院、“药品、医疗器械和治疗创新观察站”……）参与本次研讨会而实现的。主要建议旨在促进在我国开展临床试验，特别是早期阶段的临床试验，通过扩大早期准入计划和建立所谓的“扩展临时使用授权”（ATUext）程序，为创新药物提供更广泛的准入途径，从而可以在药物具有另一种适应症的上市许可的情况下开出处方。此外，我们建议实行有条件报销，该报销将根据初步数据提供，但需要根据临床试验和/或实际数据的汇总数据进行重新评估。最后，为了更好地进行这些评估，以便获得准入或护理，我们建议与卫生机构/医院建立伙伴关系协议，以鼓励出现领域专家，从而优先考虑更接近患者需求和现实生活的上行专业知识。
