Toward a European harmonisation of the requirements for the first clinical use of Advanced Therapy Medicinal Products (ATMPs): Preclinical requirements for cell based medicinal products

摘要

The necessity of a consistent approach and a regulatory "memory" was addressed by the EU with the promulgation of the Regulation 1394/2007/EC and the creation of a new committee for the evaluation of the Marketing Authorisation Application (MAA) of ATMPs, the Committee for Advanced Therapies (CAT). However, the scientific background of the ATMPs is rapidly changing due to a high rate of innovation. In fact, often the time frame required to perform the clinical development, usually of at least 5 years, is such that the product or some of its assumption might be obsolete at the moment of the Marketing Application. To favour the Small and Medium Enterprises (SMEs) and academic sponsors, the Regulation contains provisions which are intended to help this transition and to smooth the regulatory hurdles.