Chimeric antigen receptor (CAR) T-cell therapy is offered as a once-only treatment for patients with certain cancers that are not responsive to standard treatment. While clinicians, patients and their families increasingly seek access to CAR T-cell therapy, there is no revenue stream to support access through public or private health systems.

The New South Wales (NSW) Ministry of Health and Victorian Department of Health and Human Services oversighted a health technology assessment (HTA) to explore the status and geography of regulatory frameworks supporting delivery of CAR T-cell therapy, evidence for the safety, efficacy and cost, clinical trials conducted or underway and manufacturing aspects.

CAR T-cell therapies are approved in the European Union and United States of America, and being considered in Australia, Canada, China and Japan. Efficacy, safety and cost-effectiveness is limited by the size and single-arm design of early stage trials and variation between them. While overall response ranges from 36–93 percent, early results for some cancers are less favorable. Durability of treatment effect is unknown, adverse events are common and can be life-threatening and risk of delayed onset toxicity remains unknown. Treatment requires access to approved manufacturing facilities (none in Australia) and specialist clinical staff.

CAR T-cell therapy is promising and demand is increasing, but the limited safety profile and evidence base should mitigate policy and investment decisions. Broader consideration should be given to developing, or identifying access to, manufacturing and clinical workforce capability and capacity to meet national demand. Australia is likely to encounter similar issues in other jurisdictions, such as limited evidence base and complex safety issues. Factors to be considered on a local and national basis for assessment and implementation include: (i) Regulatory support for industry; (ii) Strategies to manage uncertainties in long-term risks, benefits and costs; (iii) Access to accredited manufacturing facilities; (iv) Developing clinical and manufacturing workforce capability and capacity.