Early access to health products in France: Major advances of the French "Conseil stratégique des industries de santé" (CSIS) to be implemented (modalities, regulations, funding).

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.

Leveraging EUnetHTA's conceptual framework to compare HTA decision drivers in France, Italy, and Germany from a manufacturer's point of view.

BACKGROUND: Health Technology Assessments (HTA) procedures differ substantially across the various European countries. We reviewed recent appraisals of a pharmaceutical manufacturer in three major European markets (France; Italy; Germany) and identified and categorized related decision drivers. METHODS: New marketing authorisation between January 2011 and August 2017, and Roche being the Marketing Authorization Holder, were included. Outcome of HTA appraisals by the Haute Autorité de Santé (HAS), Agenzia Italiana del Farmaco (AIFA), and Federal Joint Committee (Gemeinsamer Bundesausschuss, G-BA) were reviewed. Respective decision drivers were identified and commonalities and differences across the three countries were determined leveraging the EUnetHTA conceptual taxonomy (i.e. the 9 domains of the EUnetHTA core model). RESULTS: Within that time period Roche received European marketing authorization for eight new molecular entities (10 indications, respectively). Outcome of HTA appraisals was heterogeneous across the three countries. However, the four clinical domains of the EUnetHTA core model were driving the national HTA appraisals, with the clinical effectiveness domain being of most importance. Important drivers related to the other three clinical domains included the target patient population (subgroups, Germany), the current management of the condition (unmet need, Italy), the regulatory status (Orphan Designation, Germany), as well as safety considerations (all three countries). Average time between EMA approval and full commercial availability of new medicines was 63 (Germany), 459 (Italy), and 557 days (France). CONCLUSIONS: The clinical domains of the EUnetHTA framework are mainly driven by national HTA appraisals, providing a suitable starting point for further developing a joint European view on value and evidence. Underlying topics and issues still reveal considerable differences.