Clinical Research: Phase 1 - Phase 4

Regulatory Challenges in Global CAR-T Cell Therapy Development

When exploring the complex and rigorous regulatory landscape for CAR-T cell therapies, sponsors may encounter challenges in their efforts to bring products to market

Since the historic approval of Kymriah® (tisagenlecleucel) in 2017, research on chimeric antigen receptor (CAR) T cell therapy has accelerated. There are more than 230 regenerative medicine companies in Europe and Israel, of which more than 140 are focused on cell therapies. Around the world, there were 452 clinical trials of genemodified and cell-based immunooncology therapies under way at the end of 2019 (1). Of these, 215 are in Phase II trials and 15 have progressed to Phase III trials.

With an etiology that involves an array of genetic interactions and dysfunction across multiple systems, cancer is one of the most scientifically complex and dynamic diseases. This complexity makes the design of oncology clinical trials, especially early-stage studies, challenging.

Regulations for Cell Therapy Products

In the EU, cell therapy products are subject to the EMA’s guideline titled Quality, non-clinical and clinical aspects of medicinal products containing genetically modified cells, which went into effect in November 2012 (3). A draft revision of this guideline was released in July 2018 to reflect the evolution of scientific and regulatory experience as well as current thinking on the requirements for non-clinical and clinical studies, including a focus on CAR T products. This draft was in public consultation until 31 July 2019, and the revision is still in progress.

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