Therapie最新文献

Precision medicine aims to integrate individual patient characteristics into therapeutic decisions. Metabolomics and pharmaco-metabolomics enable direct exploration of the biological phenotype through comprehensive analysis of the metabolites present in an organism. These approaches provide an integrative perspective that considers the interactions among the genome, environment, microbiota, and lifestyle, paving the way for better prediction of treatment efficacy and the prevention of adverse events. However, their implementation faces technical, analytical, organizational, and regulatory hurdles. The Giens 2025 Workshops brought together academic, institutional, and industrial experts to identify the ambitions of pharmaco-metabolomics and the obstacles to its development, ultimately proposing key recommendations. These recommendations focus on standardizing practices, enhancing professional training, improving data interoperability, and increasing the accessibility of analytical tools. Thus, pharmaco-metabolomics is positioned as a significant lever for integrating the biological phenotype into personalized medicine by 2030.

Recognising the pivotal role of health products in our society, the challenges around accessing reliable information in a fast-expanding digital environment, and the essential role played by health literacy in enabling citizens to make informed choices, this round table session was convened to identify strategies for improving health product education and understanding among the general public. Having discussed the concept of health literacy and those initiatives already in place, the participants put forward ten recommendations (R) for improving health product literacy: definition of a consensus-based common core of minimum knowledge about medicines to provide a benchmark level of public understanding (R1); development of a multimodal information campaign coordinated at national, local and individual levels (R2); implementation of long-term interventions with repeated, age-appropriate, and context-sensitive messaging (R3); early introduction of health product education early in schools via lessons and health services (R4); promotion of high-quality education by embracing psychosocial skills and co-constructing messages with public contributors (R5); proactive engagement in digital environments to ensure visibility of reliable content (R6); promotion and upscaling of existing initiatives and educational content (R7); introduction of a trusted structure that centralises validated content (R8); using prescriptions to deliver personalised information (R9); and contribution to national discussions around health-related misinformation (R10).

Level V clinical added value (CAV) refers to a lack of CAV improvement of a new medicine, as assessed by the French Transparency Committee, compared to a standard of care therapeutic strategy. In 2024, 56% of medicines evaluated in primary registration or extended indication (excluding simplified procedures) were assigned an CAV V, compared with 44% in 2023. Obtaining an CAV V sometimes leads to the product not being available to patients, especially in the event of non-inclusion on the costly drug list for hospital products or after failure of negotiation between the drug manufacturers and the Health Products Economic Committee. The Giens workshops wished to devote a round table to the study of these drugs and analyse the different situations leading to CAV V to make proposals allowing access for patients to these drugs that are the subject of very specific CAV V situations. Three situations could benefit from specific attention: (i) Medicinal products for which the submitted clinical data does not allow the French Health Authority (HAS), at the time of evaluation, to decide on the quantity of clinical effect; (ii) Drugs for which the clinical effect is low or for which clinical relevance is discussed; (iii) Drugs for which no added clinical benefit is demonstrated but for which non-clinical benefits (such as organisational impact on care, or long-term savings) are identifiable. Following the analysis of these different situations and using examples, the members of the round table made eight concrete proposals to allow patients to access these products, in certain blocking situations.

Health technology assessment (HTA) informs public decision-making regarding the use of medical innovations. In France, the national health authority (Haute Autorité de santé, HAS) is the principal body responsible for these assessments. In hospitals, a specific form of HTA-hospital-based HTA (HB-HTA)-has been developed to evaluate technologies in their local context, contributing to better allocation of resources and judicious management of innovation. However, HB-HTA remains underdeveloped in France. In this context, round table no. 4 of the "Ateliers de Giens" (Giens Workshops), a meeting of experts from the fields of academia, medicine, hospital management and industry, looked at the obstacles and solutions that could enable HB-HTA structures in France to do more and do better. The round table identified several major findings that are hampering the development of HB-HTA. First, HB-HTA remains limited regarding its scope of application and number of potential beneficiaries. As a result, the activity suffers from a lack of recognition at an institutional and strategic level. The situation is further hampered by a marked lack of human, organizational and financial resources, as well as extremely limited dissemination of the findings, both internally and externally. Finally, the absence of harmonized assessment methods harms the informative value and impact of these procedures. To address these challenges, the round table formulated five recommendations: (1) expanding the uses of HB-HTA in the health system; (2) increasing recognition for HB-HTA by demonstrating its usefulness; (3) funding of HB-HTA pilot centers and creating the conditions for sustainable funding; (4) structuring the sharing of HB-HTA assessments; and (5) promoting a unified HB-HTA methodology and supporting the training of those involved.

Clinical research provides an important gateway to therapeutic innovation and, in many cases, national participation in trials can facilitate the future availability of new treatments in the country concerned. Clinical research activity in general is declining in France, especially in terms of industry-led trials; the only exception being early-phase cancer trials. There is an urgent need to boost the level of French participation in major international trials, although this will require a marked improvement in recruitment performance. Although the many factors contributing to the mediocre performance of France merit discussion, the overriding necessity is to bring forward solutions for increasing the level of participation in clinical trials going forward. It is against this background that we propose these recommendations for facilitating and increasing overall enrolment. There are a number of potential ways in which improvements could be achieved, beginning with identifying eligible patients and offering them the opportunity to take part in clinical trials, either through greater engagement by healthcare professionals or the development of decentralised trials. Other opportunities include optimising territorial coverage through inter-hospital networks or clinical trials centre networks, strengthening and improving clinical trials centre organisational structures by introducing AI-driven patient pre-screening tools, and lastly reprofiling some of the roles involved in clinical research and the provision of associated training. While some of these recommendations require regulatory changes, the majority could be implemented immediately, and would provide a rapid response to this major strategic challenge.