The Need for Increased Clarity and Transparency in the Regulatory Pathway for Gene Medicines in the European Union

Abstract

At the October 2011 meeting of the European Society of Gene and Cell Therapy (ESGCT) in Brighton, UK, the society hosted an excellent symposium in collaboration with the Committee on Advanced Therapies (CAT) of the European Medicines Agency (EMA), with a view to discussing problems and road blocks that biotech companies face when trying to bring Advanced Therapy Medicinal Products (ATMP) through the regulatory process. Overshadowing the symposium was the recent decision by the EMA to reject approval of Amsterdam Molecular Therapeutic’s Glybera (alipogene tiparvovec) despite an approval recommendation by the CAT. Thus, despite the considerable professional expertise of the CAT and their attempts to guide sponsors through the regulatory pathway in the EU, the process remains fraught with uncertainty and it was the clear consensus of the symposium that additional clarification of the authority of the CAT in the decision-making process was urgently needed.