Are we ready? What is missing and what is needed? A regulator’s perspective

Background The lack of clinical data for orphan drugs and the development of “precision medicines” or advanced therapies have required a new regulatory approach to guarantee a marketing authorization for new and innovative medicines. What is actually changing in the scenario is the way Agencies are managing the regulatory procedures to ensure a rapid access to treatment for patients with unmet medical needs. In the last few years EMA and National Agencies have provided a lot of scientific advices (SA) to companies and applicants about orphan designation, CTs design, marketing authorisation procedures and HTA evaluations. Currently the global scenario is already changing as we are issuing licenses for marketing authorization under exceptional circumstances and conditional marketing authorizations. The legislative framework is also changing with the new Pharmacovigilance Directive and the new Regulation on Clinical Trials entering into force progressively by 2015.