CHAPTER 2: CLINICAL TRIALS OF ADVANCED THERAPY MEDICINALPRODUCTS: WHICH FUTURE FOR THE EUROPEAN REGULATION?

Abstract

In relation to the “hot” topic of regenerative medicine, European Union law establisheda specific legal framework for advanced therapy medicinal products, manufactured fromhuman or animal genes, cells or tissues. However, these medicinal products do not havealways to be submitted to clinical trials as legal requirements vary according to theintended use: market of the whole of Member States, compassionate use, and hospitalexemption. Moreover, specificities of these medicinal products make clinical trials diffi-cult to be conducted. This paper highlights the consecutive competition between regula-tions and products that takes roots in the tension between the challenges of patients safetyand quick access to innovative treatments. It also shows the regulatory flexibility currently favored to ensure the necessary balance between safety and availability, is not withoutlimits,