Novel Clinical Trial Designs for Innovative Therapies

There is a paradigm shift in oncology from the development of classical intravenously applied cytotoxic anticancer drugs to so-called targeted therapies with molecules that are often administered orally on a chronic daily basis. The dose-limiting toxicities of targeted therapies are frequently markedly different from those of cytotoxic anticancer drugs. In addition, the activity levels of therapies targeted against defined cellular markers may significantly improve if patient pool enrichment is carried out on the basis of the presence of that specific marker in the tumor tissue. Alternative strategies for clinical development are needed for the swift and safe testing of these novel therapies.