Today, a Call to Action was announced urging all those involved in the new EU Joint Clinical Assessment (JCA) to recognize and use all types of available clinical evidence, including single-arm trials and real-world evidence, in the assessment of Advanced Therapy Medicinal Products (ATMPs), which include cell and gene therapies.

This Call to Action is an initiative of the Alliance for Regenerative Medicine, Cancer Patients Europe and Fondazione Telethon, and co-signed by 33 not-for-profit organisations, including patient groups, scientific societies, research foundations and medical institutes. Currently, there are several clinical trials underway examining the safety and efficacy of genetic therapies, which are examples of ATMPs, in people with cystic fibrosis. It is therefore very likely that genetic therapies will be used in the future for the treatment of CF. For this reason, CF Europe finds it very important to endorse this Call to Action, so that all types of available clinical evidence on ATMPs can be utilized appropriately to provide people with timely access to these innovative and life-saving medications. If not, the new EU Joint Clinical Assessment will become an additional barrier for patient access to transformative advanced therapies if the Health Technology Assessment (HTA) Coordination Group does not modernize its approach to evaluating these medicines.

Read the full Call to Action here, and the press release here.