TY - JOUR T1 - Rapid access to innovative medicinal products while ensuring relevant health technology assessment. Position of the French National Authority for Health JF - BMJ Evidence-Based Medicine JO - BMJ EBM DO - 10.1136/bmjebm-2022-112091 SP - bmjebm-2022-112091 AU - Antoine Vanier AU - Judith Fernandez AU - Sophie Kelley AU - Lise Alter AU - Patrick Semenzato AU - Corinne Alberti AU - Sylvie Chevret AU - Dominique Costagliola AU - Michel Cucherat AU - Bruno Falissard AU - François Gueyffier AU - Jérôme Lambert AU - Etienne Lengliné AU - Clara Locher AU - Florian Naudet AU - Raphael Porcher AU - Rodolphe Thiébaut AU - Muriel Vray AU - Sarah Zohar AU - Pierre Cochat AU - Dominique Le Guludec Y1 - 2023/02/07 UR - http://ebm.bmj.com/content/early/2023/02/07/bmjebm-2022-112091.abstract N2 - In France, decisions for reimbursement taken by the Ministry of Health are based on appraisal by an independent Health Technology Assessment body (HTAb): the ‘Haute Autorité de santé’ (HAS). HAS grades the clinical added value of any medicinal product for which a manufacturer seeks reimbursement. This appraisal considers different types of clinical and patient-centred outcomes, including patient-reported ones. Under certain conditions, a concomitant economic assessment which accounts for patients’ preferences in the form of utility values is also performed.As providing fast access to breakthrough therapies is a critical expectation from patients, clinicians and health policy makers, the European Medicines Agency and the Food and Drug Administration have established various accelerated approval pathways. These procedures lead to conditional approvals frequently based on uncontrolled (ie, single arm) pivotal trials.1–4 Approvals based on uncontrolled trials are also motivated by randomisation sometimes being considered unfeasible or unethical, or because the pathophysiological rationale is assumed to be important when proving effectiveness (eg, a treatment with molecular target in oncology).5 6However, expected benefits based on uncontrolled trials as evidence are frequently not confirmed. The results of meta-epidemiological studies illustrate that appraisals based on such evidence entail a high level of uncertainty leading to ethical concerns.7 8 For patients, this may have deleterious consequences such as the use of products for which the benefits remain unknown,9 10 the overestimation of benefits with no further confirmation11 12 or the increased risks of adverse events.12 13 This high level of uncertainty may also impact health system sustainability.While ensuring rapid access to valuable treatments is of utmost importance to patients, maintaining an adequate balance with the performance of relevant HTA in this context is highly challenging. Thus, the French Minister of Health requested HAS to provide recommendations. A consultation of patient … ER -