November 2018. Kew K, Knight K, Karner C and Edwards S J.

Chimeric antigen receptor (CAR) T-cell therapy is a promising next generation immunotherapy for treatment of cancer. This review identified factors to be considered in NSW and nationally for assessment and implementation of this potentially lifesaving treatment. Accelerated regulatory approvals and clinical trials for its use in the treatment of various cancers are underway in several countries. Although positive response rates are high, long-term efficacy is unknown and serious safety issues have been identified. Manufacturers, regulators and research bodies are considering how best to assess the long-term efficacy, safety and cost of CAR T-cell therapy. There are lessons to be learned from other countries where processes have been adapted and initiatives undertaken to cope with the challenges posed by the wide range of CAR T-cell therapies coming through the pipeline.