ACADEMIC GMP · The impact of Regulation (EC) No 1394/2007 on the development of Advanced Therapy Medicinal Products (ATMPs): an academic perspective

Advanced Therapy Medicinal Products (ATMPs) are medicinal products for human use, based on gene therapy, somatic cell therapy or tissue engineering. A rapidly growing area in translational research, they represent the ‘next generation’ of complex medicines for complex diseases and pose particular challenges to medicines regulation.Regulation (EC) No 1394/2007 has been designed to ensure the free movement of ATMPs within the European Union (EU), to facilitate their access to the EU market and to foster the competitiveness of European pharmaceutical companies while guaranteeing the highest level of health protection for patients. The regulation extends standards of current Good Manufacturing Practice (cGMP) to ATMPs to ensure their quality, safety and efficacy.Academic cGMP facilities are major contributors to the development of ATMPs. They respond to clinical needs and provide medicinal products in an environment which, albeit compliant with industrial standards, is by definition not industrial. They find themselves in a challenging position between various, sometimes conflicting interests in the transition of ATMPs from bench to bedside. European investigator-initiated multicenter trials on ATMPs critically depend on academic cGMP facilities.We will assess the impact of Regulation (EC) No 1394/2007 on academic cGMP facilities by: a) conducting a European survey among non-industry facilities in this sector; b) organising workshops and a major conference for targeted, collaborative discourse; c) establishing a web-based platform for information exchange; d) analysing publications and guidance from the perspective of better regulation principles; e) analysing innovation statistics in relation to ATMPs.These activities will yield comprehensive evidence and concrete suggestions to policy makers. The project will in itself foster a dialogue that will strengthen networks of translational research and thus contribute to research excellence in Europe.