Masterstudiengang "Drug Regulatory Affairs"

Master-Thesis

Regulatory requirements for CAR-T Cell Therapy against cancer - a comparison between the EU and the US ***

Dr. Ornella Fumagalli (Abschlußjahr: 2019)

Summary
Language: English
CAR-T cell therapy represents a significant turning point in the field of cancer treatment and has given a lot of hope to cancer patients. The therapy is based on stimulating the patient’s immune system and is designed to cure the disease with a single treatment.
While their complexity challenges our perception of what a drug is and their production can be challenging, the success of these novel therapies inspires the continued expansion of drug development boundaries.
In 2018, the first two products, Kymriah® (Novartis) and Yescarta® (Kite Pharma/Gilead), obtained regulatory approval in the EU for treatment of acute lymphoblastic leukaemia and diffuse large B-cell lymphoma, refractory to a standard chemotherapy regimen or relapsed after stem cell therapy.
In this master thesis the European and US regulatory environment and requirements for CAR-T cell therapy against cancer are summarised and the path to licensure of the two approved CAR-T cell therapies Kymriah and Yescarta analysed. Based on this, requirements and challenges for CAR-T therapy development are evaluated and a potential regulatory strategy for the EU and the US proposed. A final assessment on which ICH region to choose for CAR-T therapy development - the EU or the US - concludes the thesis.