Hospital Exemption for ATMPs: Our Recommendations

Advanced therapy medicinal products (ATMPs) are medicines for human use that are based on genes, tissues or cells. They offer groundbreaking new opportunities for the treatment of disease and injury, bringing with them the promise of treatment of severe, disabling or life-limiting conditions but also the promise of one-time disease-modifying and potentially curative treatments that can transform and save lives.

The principle of hospital exemption (HE) allows for the use of an ATMP without a marketing authorization under certain circumstances. This only applies in a hospital setting on a non-routine basis for an individual patient and when no centrally authorized treatment or clinical trial is available.

EUCOPE and its member companies developing ATMPs are pleased to propose the following recommendations for the good practice of hospital exemption, for the benefit of science, research, and ultimately, patients in Europe. The recommendations are part of a position paper elaborated by the EUCOPE Working Group on Gene and Cell Therapies. For more information on our work on ATMPs, please see here.

Appropriate use

• Limit the use of HE to situations when there are no centrally authorized ATMPs available in the Member State for the indication and there are no clinical trials available for the same indication with an ATMP in the Member State.

Long term follow-up

• The principles of long-term follow-up should apply to commercial and non-commercial manufacturers, including HE producers.

Transparency

• Need for publicly available information about HE product at EU and/or national levels (use and safety/efficacy profile).

Harmonisation

• Need for further harmonization of HE requirements/licenses and eligibility criteria across all Member States