Pre-approval and Post-approval Challenges in the Clinical Development and Reimbursement of CAR-T Cell Therapies

Developing CAR-T cell therapies, and cell and gene therapies in general, have unique challenges not observed with traditional drugs.

In light of the progress in cell therapy development, innovative development strategies need to be explored to accelerate development of the next generation of cell therapy products. On top of the crucial manufactory and logistical challenges, clinical development of CAR-Ts is facing unique statistical questions leading to authorization which need to be resolved for delivering the full treatment effect. Additionally, health care systems and payers face challenges associated with pricing and reimbursement that has resulted in the need for long-term registry studies designed to optimize treatment as well as provide a basis for outcomes-based agreements.

The Basel Biometric Section (BBS) is pleased to host a 2-day scientific meeting on this relevant and far-reaching topic by bringing together experts from the pharmaceutical industry, academia and the European/US regulatory bodies and health technology assessment (HTA) agencies and payers to present the current state of the art and discuss the statistical and other challenges and opportunities of CAR-T cell therapies.