Chapter 6 - Clinical trials and off-label use of drugs

The contribution aims to analyze a topic closely related to the subject of experimentation, in terms of protection of patients undergoing drug therapy: the use of off-label drugs, that is, in summary, the possibility to use a medicine for therapeutic indications not covered within the marketing authorisation (M.A.) of the drug. Moving from European discipline on medicines for human use, the analysis focuses on the peculiarities of the Italian legislation, representing in Europe an emblematic case of off-label rules. A particular focus is dedicated to the case of drugs Avastin^® and Lucentis^®, which in recent years has highlighted the limits of that national legislation, including the possible contrast with European regulations. In particular, the choice of legislator, recently upheld by the Italian Constitutional Court, to submit the possibility, as well as the definition of what are the minimum requirements of safety and efficacy for a drug “off-label” may be prescribed to patients, are the main issues on which we intend to stimulate reflection on the reader.