ATMP-Workshop: CAR T cell therapies in Europe
Despite the potential of CAR T cell therapies, there are still significant hurdles in Europe to be overcome during their development, approval process, and reimbursement. On December 3 over hundred experts met at the invitation of BIO.NRW and Miltenyi Biotec at ATMP Workshop in Brussels to come up with solutions and make these novel therapies available to as many patients as possible.
"It is time to pool expertise from different disciplines, collaborate and develop strategies to overcome challenges of CAR T cell therapies”, requires Dr. Andrew Kaiser of Miltenyi Biotec. Under the patronage of the Ministry of Economic Affairs, Innovation, Digitalization, and Energy of the German Federal State of North Rhine Westphalia (NRW) scientists, physicians, healthcare economics experts, and members of European regulatory bodies discussed in Brussels the latest research results and strategies to promote the advancement of CAR T cell therapies.
Making personalized therapies available to patients not only imposes high requirements on their development it also depends on complex manufacturing and logistics. “Right now we see that capacities remain very limited and costs high. Hence, the number of patients that can be treated is limited”, explains Dr. Andrew Kaiser who is the coordinator of the CARAT consortium. “To address that we need automated, safe and effective solutions, that can be applied also at the point of care.” This is also one of the objectives of the CARAT initiative.
Not only the manufacture of cellular products requires new concepts, established approval procedures for common drug products need also to be adapted to meet the needs of personalized cell and gene therapies. “The dialogue between pharma companies, academia and regulators as well as the government is important in multiple aspects including early basic and clinical research, also including reimbursement issues to facilitate rapid development and access of patients to high potential cell and gene therapies,” states Professor Klaus Cichutek, President of the Paul-Ehrlich-Institut (PEI), Federal Institute for Vaccines and Biomedicines in Germany. In addition, the issue of reimbursement by health insurance providers is far from being solved. “Development of novel cellular therapies is a long and expensive process”, according to Prof. Dr. Christian Dierks, Dierks+Company. "To be available to many patient prices have to go down significantly. However, if these treatments will not be adequately reimbursed, their advancement will be slowed down or even cease." To fuel the process of their development, novel EU funding programs will be substantial to enhance the competitiveness of Europe in the development of advanced cell and gene therapies.
To regain scientific and economic competitiveness of Europe in the in the field of ATMP development the following action points were defined as outcome of the work shop:
- Novel funding programs of the EC are needed to enhance research in Europe and to support scientists and companies in the fight against cancer. New funding models are needed to support pre-clinical and clinical developments of ATMP up to the support of commercial translation and of Phase III trials (In alignment with funding schemes e.g. in the US, “flagship” funding initiatives, calls for multidisciplinary teams to tackle development of cancer treatments)
- More funding for research clusters of excellence (NIH-like structures)
- Dynamic regulatory environment is essential for boosting ATMP development: close exchange between researchers, pharma and biotech companies and the regulatory agencies, dynamic adaption to the unconventional nature of ATMP-therapies, more funding for regulatory agencies to accelerate the approval process of ATMPs in Europe
- Harmonization of rules related to ATMP manufacturing and trial applications between the European countries, in order to accelerate multi-centric trial approval overall Europe.