title: [Assessment of actual benefits of new drugs by the Transparency Committee].
abstract: When a drug has been granted a marketing authorization, if the pharmaceutical company wants it to be covered by the National Health Insurance, the company has to submit a file with all the studies concerning the drug, especially drug-drug comparative studies, to be assessed by the Transparency Committee. Drugs are assessed on two criteria: actual or expected benefit (AB) and improvement in actual benefit (IAB). Actual benefit mainly takes into account the severity of the disease concerned, the level of efficacy relative to known side effects (risk-benefit ratio), and the place the drug is intended to take in the therapeutic strategy. At the end of the assessment, AB is considered as important, moderate, poor or insufficient (to justify inclusion of the drug on the list of products to be reimbursed). After actual benefit is determined, improvement of actual benefit is assessed, comparing the estimated benefit of this drug with one of drugs with the same indication that is already reimbursed, to assess whether this drug will improve the patient's disease. This can be assessed by direct comparison (two drugs compared in the same clinical trial) or by indirect comparison (separate studies with the same design). There are four levels of added value, from I (major improvement) to IV (minor improvement). Level V represents no improvement. This second assessment is always relative to another drug. It never provides an absolute score. However, IAB is very important for pharmaceutical companies, because it is a fundamental criterion to determine the price of the drug, which is discussed with the Economic Committee of Health Products in a final phase. Actual benefit and improvement in actual benefit are allocated for each indication of a drug.
authors.affiliation: Service de Médecine Interne, Centre Hospitalier Universitaire de l'Hôtel-Dieu, Assistance Publique-Hôpitaux de Paris, Paris, France. claire.le-jeunne@htd.aphp.fr
authors.forename: C
authors.initials: C
authors.lastname: Le Jeunne
mesh_terms: D002986:Clinical Trials as Topic; D004345:Drug Industry / Q000592:standards; D004358:Drug Therapy / Q000592:standards* / Q000639:trends*; D006801:Humans; D018570:Risk Assessment; D016896:Treatment Outcome