Old drug, new clinical use, no man’s land for the indication: an awareness call from European experts

Abstract

Licence holders of a new medical agent who apply for marketing authorisation in Europe have two choices: They may either apply to a national health authority (which limits authorisation to the authority’s country) or choose the centralised procedure with the European Medicines Agency (EMA) to apply for authorisation for all countries belonging to the European Union (EU) as well as Liechtenstein, Iceland and Norway. For anticancer drugs, as well as several other categories, the central authorisation approach through EMA is compulsory since 2005.1 On application, EMA will launch a content-defined and timeline-defined verifying procedure (figure 1), which will lead to a recommendation. The agency has no legislative or other decisive power. EMA’s recommendation will then be submitted to the European Commission, which will take the legal binding decision for marketing authorisation, based on EMA’s recommendation. This is not synonymous but a precondition for pricing and reimbursement, as the latter remains within the competence of member states and their national and/or regional health authorities (with requirements, procedures and decisions varying according to each country). Having obtained marketing authorisation by the European Commission following EMA’s recommendation, the drug’s marketing authorisation holder (MAH) must thus proceed applying individually for pricing and reimbursement in each country he wishes to commercialise the drug. Figure 1 Procedure and timelines for first drug marketing approval by EMA. CHMP, Committee for Medicinal Products for Human Use. Source: ema.europa.eu The EMA marketing authorisation recommendation will precisely define the indication(s), the exact composition in active substance and excipients, patient and healthcare professional information and even packaging. It has to be renewed in regular intervals (usually every 5 years). There are costs for a company related to the submission of an application for marketing authorisation and for any other changes to it after approval, including for regular renewal. Cancer (as well as …