Off-Label Use of Medicines: Legal Aspects

In Europe, subject to certain exemptions explained later, no medicine can be marketed for human use without a Marketing Authorisation granted either by a Member State competent authority or by the European Commission. The regulatory system exists to protect patients by ensuring that marketed medicines meet acceptable standards of safety, quality and efficacy in their indications. Nonetheless, for a range of reasons use of medicines outside their authorised indications, commonly known as offlabel use, and use of unlicensed medicines (i.e. medicines without a marketing authorisation) are common. This chapter outlines the definition of a medicine and the current regulatory framework; reviews the legal position of prescribers of off-label use and the use of unlicensed medicines; considers special populations and therapeutic areas where off-label use or the use of unlicensed medicines is common; and provides some general guidance for prescribers considering off-label use or the use of unlicensed medicines.