Masterarbeit

Life Cycle of CAR -T cell products: Legal framework and points to consider during the life of a CAR-T cell product

Dr. Helen Springer-Frauenhoff (2018)

Summary
Language: English
CAR-T is an extraordinary immunotherapeutic technology using T cells with chimeric antigen receptors that offers new treatment options to cancer patients. Promising pre-clinical and clinical trial data such as durable complete remission in otherwise treatment-refractory B cell malignancies suggest that the high medical need in treating leukaemia and lymphoma can finally be addressed. Two recent marketing approvals by FDA and EMA for CAR-T cell products accelerate and drive this field to the developmental highway, thereby attracting experts and public attention as well as taking advantage of large industrial investments and governmental funding.
However, this technology comes with the challenges of personalised medicine, sophisticated manufacturing, severe side effects and very high costs. These advanced therapeutic medicinal products (ATMP) greatly differ from conventional small molecule drugs and also to biologics.
This thesis collects the current legal European requirements that need to be followed during the entire life cycle of CAR-T products. Dissected into 15 phases, this thesis analyses how the specific peculiarities challenge the CAR-T products throughout their life cycle and regulatory assessments. Discussing major risks some of which could already be experienced with previous ATMPs intends to understand how CAR-T cell technology will perform in the future. Central questions are: Will CAR-T technology will be able to broaden its scope to other indications such as solid tumours as well? Will it bring return of investment to pharmaceutical companies and at the same time be affordable for the health communities and reimbursed?  These factors will be pre-requisites for CAR-T products to be established as standard therapy, and thus become a role model for the future generations of medicine. CAR T technology represents an innovative example that triggers reassessment of existing dogmas in drug development that even affect the regulatory field as well.
Pages: 56, Annexes: pages : -