On Monday, 19 February 2024, at the EUCROF24 conference in Prague our Head of Development Area, Arianna Bertolani, takes the stage to present an abstract authored by three experts from CVBF: Giovanni Migliaccio, Irisi Sukaj and Arianna Bertolani. This abstract provides a concise yet comprehensive view of the challenges and opportunities in the submission process for Advanced Therapy Medicinal Products (ATMPs) to the Clinical Trial Information System (CTIS) in the European Union and European Economic Area (EU/EEA).

In a world of constant medical innovation, ATMPs, based on genes, tissues, or cells, offer unique possibilities for treating long-term diseases and injuries. With a surge in ATMP development, it has become crucial to understand the intricacies of submitting clinical trials involving these innovative products.

CVBF, as a Clinical Research Organization (CRO), has extensive experience in managing complex clinical projects, from the start-up phase to the submission on CTIS, ATMP projects included.

The challenges of submitting ATMPs to CTIS span various stages of the process. From the scarcity of suitable animal models during the preclinical phase to limitations in manufacturing capacity and the stringent requirements of Good Manufacturing Practice (GMP), the road to CTIS submission is full of challenges. Furthermore, the relative inexperience of various stakeholders can directly affect the preparation of the essential documents for CTIS submission.

In the European landscape, regulatory requirements and feedback from member state competent authorities exhibit a significant level of heterogeneity. However, the European Medicines Agency (EMA) has come forward to support non-profit developers. EMA’s provision of scientific advice and the reduction or waiving of fees for ATMPs underscore their commitment to facilitating the submission process.

Since its launch on 31 January 2022, the use of CTIS to submit applications for clinical trials is increasing, yet the unique features, functional demands, and challenges of submitting ATMPs may not be fully understood by all stakeholders. Experience with this system will be paramount to evaluate its future development and refinement.

To follow this presentation and much more, we invite you to come and join us at the EUCROF Conference, scheduled on 19-20 February 2024, in the beautiful city of Prague.