Advanced therapy medicinal products—Perspectives, opportunities and challenges: Statement of the German Society of Pediatrics and Adolescent Medicine (DGKJ)

Advanced therapy medicinal products (ATMP), such as somatic gene therapy and cell therapy, have a high therapeutic potential for diseases that begin very early in life and that were previously untreatable. They are often licensed at a very early stage of development when the efficacy has been shown in a few affected patients and a previously unprecedented therapeutic success is revealed, especially if the therapy is effective before organ damage occurs. This gives rise to new pharmaceutical legal issues and ethical questions for pediatricians. In order to adequately use the new treatment options the diagnosis must be made earlier than before or new screening methods must be available. It is conceivable that the newborn screening will be divided into time-critical diseases in the first 72 h after birth and genetic screening, e.g. in the 4th–5th weeks of life. ATMP were not used in sufficient numbers when they were licensed, so that the necessary knowledge on efficacy and safety is not yet available (benefit-risk ratio). That is why they are used under strict conditions in specialized treatment centers according to quality criteria that the Federal Joint Committee (G-BA) determines after consulting the specialist competent societies. The expenditure of therapy and documentation of the clinical course in registers is considerable and must be reimbursed accordingly. The value of an ATMP becomes clear when it is used more widely after approval, just like the safety of a drug is not fully known with the approval. New challenges and opportunities arise for pediatrics.