Rare malignant haemopathies: How to benefit from new therapeutic management [Hémopathies rares: Accès aux innovations mé dicamenteuses]

摘要

Most malignant haemopathies are considered rare cancers. Consequently, haematologists have organized their practice through clinical and biological networks and collaborative working groups dedicated to clinical research and organization of the therapeutic management of affected patients. These groups that function at a national scale are recognized as preferential partners by the French Society of Haematology, by the National Cancer Institute and by pharmaceutical laboratories. Therapeutic research protocols carried out with these groups constitute a first level of access to the most recently developed therapeutic procedures and medications. The second level is very common in haematology and consists in a temporary authorization of use (TAU), an intermediate step between the filing for marketing authorization and the final availability of a new medication. Though this procedure remains to be improved, it constitutes obviously an important advance for health authorities, doctors, patients and industrialists. The third level of access is based on the possibility of use independently from marketing authorization, another mainspring of innovation. The rational and coherent use of these procedures using classifications and guidelines is being studied currently.