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NEW APPROACHES TO EVIDENCE BUILDING IN MEDICINE: THE REGULATORY PERSPECTIVE

Modern medicine is moving towards more specific individualised therapies that can target diseases in individuals with specific characteristics.  For each disease it is now possible to genotype and divide patients into different groups that will respond differently to the same treatment aimed at treating the overall disease.  This will lead at a more effective therapeutic approach that will maximise the benefit of a given treatment and will limit the potential occurrence of serious adverse events.

The need for tailored therapies will necessarily transform the knowledge cycle of therapeutic innovation that goes from cutting-edge discovery through comprehensive development and regulation to clinical application and utilisation. The Regulatory bodies have adapted their regulations to the changing scenario issuing different regulations for granting marketing authorisation to drugs aimed at specific patient populations or conditions. The European Medicine Agency has been the most innovative Agency in the field issuing regulations such as the Conditional Marketing Authorisation, the Marketing Authorisation Under Exceptional Circumstances (Directive 2001/83EC) and Post-authorisation Efficacy Studies (PAES).  These regulations together with the new pharmacovigilance legislation and the implementation of the risk management plans, periodic safety update reports (PSURS) and the legislation on the five year renewal of marketing authorisation have all been the precursors of the newly launched program of adaptive licensing.  This new program will rely significantly to a process of evidence building through the acquisition of real world data.