The European Medicines Agency (EMEA) guideline on oncology drug development

6636 Background: The scientific assessment and positive opinion of the EMEA is mandatory for the approval of new oncology drugs in the European Union. One of the tasks of the EMEA is to provide guidance on the conduct of the various tests and trials necessary for approval. The EMEA has recently revised its guideline on the clinical development of new anticancer drugs. The revised version includes specific guidance on the development of non-cytotoxic (i.e., cytostatic) agents (http://www.emea.europa.eu/pdfs/human/ewp/020595en.pdf). Specific guidance is given about methodological issues using progression-free survival (PFS) as primary endpoint in confirmatory trials for registration (http://www.emea.europa.eu/pdfs/human/ewp/26757506en.pdf). Methods: The key elements of the revised guideline are described with particular reference to requirements for approval. Results: The early stages of clinical drug development have to be tailored according to the assumed pharmacology of the individual compound as defined...