The puzzling situation of hospital exemption for advanced therapy medicinal products in Europe and stakeholders' concerns

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In Europe, advanced therapy medicinal products (ATMPs), including cell and gene medicinal products , tissue-engineered products and combined ATMPs, are governed by Directive 2001/ 83/EC and Regulation 726/2004, amended by Regulation 1394/2007, which sets specific rules concerning their centralized marketing authorization (MA), supervision and pharmacovigilance. Nevertheless, ATMPs not intended to be marketed and not industrially prepared are beyond the scope of Directive 2001/83/EC, according to article 28 of Regulation 1394/2007. This is commonly called “hospital exemption” (HE) and is restricted to any ATMP “which is prepared on a non-routine basis according to specific quality standards, and used within the same Member State in a hospital under the exclusive professional responsibility of amedical practitioner, in order to comply with an individual medical prescription for a custom-made product for an individual patient.” Member States must ensure that the manufacture of ATMPs under HE is authorized by the competent national authority and that traceability, pharmacovigilance and specific quality standards are equivalent to those applying to ATMPs granted centralized MA.