JCA should not hamper access to transformative ATMPs
The EHC joins a group of patients, clinicians, academic medical centres, and therapeutic developers and manufacturers in signing a call to action for the successful roll-out of the Joint Clinical Assessment (JCA).
With the adoption of the Implementing Act on the JCA for medicinal products and the HTA Coordination Group’s Methodological and Practical Guidelines on direct and indirect comparison, the implementation of the JCA under the EU HTA Regulation is entering a critical phase. The EHC and other signatories are concerned that European patients with few or no treatment alternatives will experience further delays and limitations in access to potentially life-changing medicines. This would defeat the purpose of the EU HTA and JCA which aspire to increase and accelerate patient access.
Therefore, the joint statement urges the members of the HTA Coordination Group and its relevant subgroups, and JCA assessors to recognise all types of available evidence including single-arm trials and RWE, and to use the JCA report to describe, rather than judge, any resulting uncertainty as to the treatments’ benefits, as called for by the HTA Regulation. A significant portion of outstanding uncertainty can be addressed at the national level during the appraisal phase and through the collection of RWE.
In so doing, the Coordination Group can lead the development of a fit-for-purpose JCA system that efficiently addresses the needs of healthcare systems, without obstructing patient access to transformative therapies.
Jim is aged 66 and has Haemophilia A with a high titer inhibitor which he developed at age 14.
Maria Elisa Mancuso (MD, PhD) is a Haematologist and works as a Senior Haematology Consultant at the Center for Thrombosis and Haemorrhagic Diseases of IRCCS Humanitas Research Hospital in Rozzano, Milan, Italy. She is Adjunct Clinical Professor at Humanitas University. She obtained a post-degree in Clinical and Experimental Haematology and a PhD in Clinical Methodology. She is involved in clinical research and has published several original articles in peer-reviewed journals a The Lancet, Blood, Journal of Thrombosis and Haemostasis, Haematologica, Thrombosis and Haemostasis, British Journal of Haematology and Haemophilia. She is reviewer for several peer-reviewed journals and member of the Editorial Board of JTH. She is a member of several scientific societies (ISTH, WFH, ASH, EAHAD, SISET, AICE) and was a medical member of the Inhibitor Working Group of the European Hemophilia Consortium. She is co-chair of the ADVANCE Study Group. She has acted also as co-chair of the Scientific and Standardization Subcommittee of ISTH on FVIII, FIX and rare bleeding disorders. She has been involved as principal and co-investigator in several clinical trials, and she takes care of both children and adults with hemophilia and other congenital bleeding disorders with a specific scientific interest in novel therapies, prophylaxis, inhibitors, and chronic hepatitis C.
As a patient with factor II deficiency, the diagnostic and treatment of rare bleeding disorders is a matter dear to my heart. My motivation to participate in the work of the ERIN committee is to improve both diagnostic and treatment for patients with rare bleeding disorders across Europe.
Economist and financial expert by profession, executive coach and trainer by passion and haemophilia advocate by every drop of my blood through my son (who has severe haemophilia A with inhibitors). Bringing a good decade of practical experience from the corporate insurance world, laser focus, growth mindset and resilience from my own experience, offering you anything I can just do, in hope that together we can make life more fulfilled for those impacted by bleeding disorders.
Amy Owen-Wyard is a Registered Mental Health Nurse. With experience working with children, young people, their families and adults with severe and enduring mental health conditions. Amy was also involved in a service improvement to provide a holistic care approach for those in general hospitals to support both their mental and physical health, whilst sharing her expertise and knowledge in mental health with the wider multidisciplinary team.
