OBJECTIVES: Chimeric antigen receptor T-cell (CAR-T) therapies are innovative treatments that have shown remarkable efficacy in rare or hard-to-treat cancers. Studies involving CAR-T treatments often involve limited clinical evidence availability and small sample sizes that pose challenges in assessing the equity implications under current health technology assessment (HTA) processes. This review aimed to evaluate limitations within the clinical evidence base submitted to HTA agencies and document equity issues observed in CAR-T submissions.

METHODS: Published appraisals from NICE (UK), ZiN (Netherlands), SMC (Scotland), CADTH (Canada), HAS (France), MSAC (Australia), TLV (Sweden), AIFA (Italy), IQWiG/GBA (Germany) and NIPH (Japan) available on agency websites were screened (06/2023) to identify CAR-T submissions. Appraisals were reviewed and extracted in a standardized manner to analyze patient characteristics from clinical studies against critique documented by HTA agencies regarding equity concerns. Equity considerations for the same CAR-T therapy and indication were compared across agencies.

RESULTS: Fifty-one HTA appraisals were identified, comprising six CAR-T therapies over ten indications. Two of the ten agencies (NICE, CADTH) had a section within appraisal documents outlining equity issues. Twenty appraisals (39%) across four agencies (NICE, HAS, CADTH, MSAC) referenced equity considerations within the appraisal. Fourteen appraisals (27%) critiqued the underrepresentation in clinical evidence across demographics (e.g., age, ethnicity, socioeconomic status, geography). Common issues raised across appraisals involved: geography and access to treatment centers (39%), age restrictions (31%), ethnic minority underrepresentation (14%), and out-of-pocket costs (6%).

CONCLUSIONS: Equity considerations were documented in a substantial proportion of HTA appraisals involving CAR-T treatments, especially regarding geography and age restrictions. These findings emphasize the need for improved representation of diverse patient populations in CAR-T clinical studies and the importance of addressing equity concerns within HTA processes to expand access to these transformative therapies. Real-world evidence transportability can play a crucial role in informing decision-makers about patients traditionally underrepresented in clinical trials.