[Clinical trials for children]

The 12th AMG amendment and the EU Directive 2001/20/EC set down clear legal guidelines on clinical studies of drug products in children. As a consequence there are new prospects for an increasing involvement of the pharmaceutical industry in developing drugs for use in children. BfArM has acted on this development by setting up a committee of experts on drug approval for use in children-similar to committees at the EMEA and FDA working on the same issue. Another useful step consists in the strengthening of pharmaco-vigilance measures. Treatment standards have been developed on the basis of the research instrument "clinical therapy optimization studies", frequently conducted as co-operative national or international studies joining many trial sites, and may definitely benefit from the new requirements. Clearly defined terms and regulations, such as "sponsor responsibility", "trial approval", "investigational medical product", "insurance", and "monitoring" describe problems that can or at times cannot be solved by such research groups. The sponsor's responsibilities in an international trial e. g. cannot usually be shouldered by a single scientist or treatment center. When in the future practically every single research question on drug treatment is governed by those regulations, costs will soar and fewer questions will be asked in clinical studies. So far, the AMG amendment has not given an assessment of benefit and risk regarding treatment quality and drug safety in children. Such an assessment depends on the willingness of all concerned to explore the scope of the legal regulations in order to let children participate in scientific progress where it is needed and where it makes sense.