Regulating advanced therapy medicinal products through the Hospital Exemption: an analysis of regulatory approaches in nine EU countries

Abstract

Aim: To study regulatory approaches for the implementation and utilization of the Hospital Exemption (HE) in nine EU countries. Materials & methods: Using public regulatory documentation and interviews with authorities we characterized the national implementation process of the HE, including national implementation characteristics and two outcomes: national licensing provisions and the amount of license holders. Results: National licensing provisions vary substantially among selected countries as a result of different regulatory considerations that relate to unmet medical needs, benefit/risk balance, and innovation. The amount of license holders per country is moderate (0–11). Conclusion: The HE facilitates HE utilization in clinical practice in some countries, yet safeguarding of public health and incentivizing commercial development is challenging.