Discovery and development of molecularly targeted drugs: regulatory perspectives

Abstract

Registration aims at ensuring that the requirements of quality, safety and efficacy for new medicines are met but how much evidence should be generated before considering that a favourable benefit/risk has been demonstrated may be controversial. Regulatory agencies tend to allow some uncertainty (to be resolved after registration) for potentially needed (as opposed to 'me too') substances for serious diseases and, in order to clarify the requirements, they issue 'guidance' documents. The 'Note for Guidance on Evaluation of Anticancer Medicinal Products in Man' of the European Medicines Agency (EMEA) came into operation in July 2003 but an update is announced by means of a Concept Paper both to address methodological aspects (including in relation to the new mechanisms of anticancer agents) and to integrate the new regulatory framework resulting from the recent Review of the European Legislation on Medicinal Products.