It argues that this causes a translational gap between development of these medicines and reaching later regulatory milestones, and that regulators need to continue working towards closing this gap in a proactive manner.
The paper emphasises the numerous ways in which organisations developing advanced-therapy medicines can engage in dialogue with the Agency and receive guidance on regulatory procedures. These include scientific advice, meetings with the Innovation Task Force, and classification or certification of medicines as advanced-therapy medicinal products by the CAT. The CAT has also run focus groups to discuss aspects of the development of advanced-therapy medicines with its stakeholders, and organised a scientific workshop with learned societies.
Advanced-therapy medicines are medicines that are made from genes and cells. They may offer groundbreaking new treatment opportunities for many diseases and injuries. All advanced-therapy medicines intended for marketing in more than one EU Member State are authorised centrally via the European Medicines Agency, following scientific evaluation by the CAT.
The analysis presented in the paper, which looked at clinical studies in the EU Drug Regulating Authorities Clinical Trials (EudraCT) database, found that over three-quarters of the advanced-therapy medicines under development were cell-based medicines. Most medicines were being studied in cancer, followed by conditions affecting the heart, blood vessels and blood.