Rapid access to innovative medicinal products while ensuring relevant health technology assessment. Position of the French National Authority for Health

The challenge of accelerated clinical developments 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...

Source: Evidence-Based Medicine - January 19, 2024 Category: Internal Medicine Authors: Vanier, A., Fernandez, J., Kelley, S., Alter, L., Semenzato, P., Alberti, C., Chevret, S., Costagliola, D., Cucherat, M., Falissard, B., Gueyffier, F., Lambert, J., Lengline, E., Locher, C., Naudet, F., Porcher, R., Thiebaut, R., Vray, M., Zohar, S., Coch Tags: Open access EBM analysis Source Type: research