We sign the all to action on the imlementation of the EU Joint Clinical Assessment for ATMPS!
SMA Europe co-signs the Call to action on the implementation of the EU Joint Clinical Assessment for ATMPS.
The new EU Joint Clinical Assessment (JCA) will become an additional barrier to patient access to transformative advanced therapies if the Health Technology Assessment (HTA) Coordination Group does not modernize its approach to evaluating these medicines, warned 33 not-for-profit organisations, including patient groups, scientific societies, research foundations and medical institutes, in a Call to Action announced on 9 July 2024.
The organisations urged all those involved in the 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.
Recognising that randomised control trials are often unfeasible and unethical for ATMPs, which often target rare or ultra-rare diseases, they called on the EU HTA Coordination Group to:
· Revise the JCA methodological guidance to avoid discrediting evidence from single-arm trials
· Take a more pragmatic approach to running JCAs of ATMPs by using real-world data to fill evidence gaps
The signatories of the Call to Action are working together to bring these issues to the attention of the European Commission, the Coordination Group and members at the national level, to ensure that the voices of patients and all stakeholders in the ATMP ecosystem are heard, and, more importantly, acted upon.