On 19th February 2020 EHC took part in “Advanced medicinal therapies : Step up the healthcare revolution now” event at the European Parliament and here is what we took back home from this meeting.
The event dedicated to Advanced Medicinal Therapies (ATMPs) was organized by EuropaBio and took place at the European Parliament. ATMPs are treatment options using human genes, cells and tissues and offering unprecedented results in the treatment of rare diseases and genetic conditions. These treatment options are unique in addressing the disease at its very root and offering the patients the prospect of definite curation. However, many questions and challenges are uprising due to the very fast development of ATMPs and yet unknown consequences on the long term for certain types of these therapies. The event was hosted by MEPs Claudia Gamon (RE) and Tomislav Sokol (EPP), who by the way, had attended EHC Round Table on Markets and Access in December 2019.
The discussions brought together European Medicines Agency (EMA) representatives, healthcare providers, pharma industry and patient representatives. We mostly appreciated the presentations covering concrete applications of ATMPs for patients and the discussions on the need of a tailored and supportive approach for the development of ATMPs. To illustrate the latter, 2019 has been a significant year for advanced therapies in Europe with 260 ongoing clinical trials for ATMPs and the European Medicines Agency is expecting up to 20 new applications for the coming 2 years. Products approvals for gene therapy specifically are to double within the next 1-2 year(s), with foreseen continued improvements in the manufacturing and delivery of this treatment. Europe has been an active player in ATMPs development compared to the rest of the world but one should not forget that new treatment options go hand in hand with tailored approaches and functioning schemes for payment models and care delivery, therefore regulatory bodies have to adapt to the changing landscape and enhance innovation. From the regulator’s perspective, the main highlight of the discussions was the growing need for harmonization of rules at the EU level in manufacturing and clinical trials for ATMPs.
Haemophilia was on the programme of the event and served as a case study to illustrate gene therapy application for monogenic diseases. Mariette Driessens, The Netherlands, gave a captivating and realistic presentation on haemophilia and the implications of gene therapy in the treatment of this condition. The numerous advantages and promising perspectives of gene therapy were balanced by some ethical questions and skepticism from the patient perspective. For example, what about equality in access to advanced therapies for all patients, knowing that the largest part of worldwide haemophilia population lacks even basic care? or what about the risk of limiting access to factor concentrates for patients with already limited access to treatment, if gene therapy becomes the main focus of pharmaceutical industry? The high cost of treatment for the society was also mentioned and finally, the psychological impact of gene therapy on patients and their environment is also to be taken into consideration. Indeed, some patients are even questioning their identity shift if undergoing gene therapy and expressed concerns about being suddenly excluded from the heamophilia community if they were to be cured after living their whole life with the condition.
Speaking of gene therapy, EHC is organising a Round Table of Stakeholders on gene therapy in October 2020; follow our posts on social media and website for additional info.