OBJECTIVES: The first two CAR-T cell therapies, Kymriah® (tisagenlecleucel) and Yescarta® (axicabtagene ciloleucel) received European marketing authorizations in August 2018 (Kymriah® for ALL and DLBCL and Yescarta® for DLBCL/PMBCL). These therapies offer potentially transformational benefits for patients with life‑threatening cancers from a single treatment administration. However, significant payer concern about their affordability exists and this research aims to evaluate how payer bodies have assessed these therapies to date.

METHODS: Publicly-available HTA evaluations of Kymriah® and Yescarta® by NICE, SMC, G‑BA, CIPM, AIFA, NLT, ZIN and HAS were screened and key information extracted (to 08/01/2020).

RESULTS: Twenty five HTA evaluations of CAR-T cell therapies in scope countries were identified (8 Kymriah® ALL, 8 Kymriah® DLBCL, 7 Yescarta® DLBCL/PMBCL, 1 Yescarta® DLBCL, and 1 Yescarta® PMBCL) completed at an average of 7.5 months after European marketing authorization. NICE recommended both Kymriah® (ALL and DLBCL) and Yescarta® (DLBCL/PMBCL) via the CDF. The SMC accepted Kymriah® (ALL and DLBCL) and Yescarta® in DLBCL/PMBCL based on resubmissions. Under HAS, Kymriah® attained ASMR III (ALL) and ASMR IV (DLBCL), and Yescarta® an ASMR III (DLBCL/PMBCL). The G-BA deemed that Kymriah® (ALL and DLBCL) and Yescarta® (DLBCL/PMBCL) offered an unquantifiable additional benefit, but these resolutions were time-limited for 1 year. The CIPM accepted Kymriah® in ALL and DLBCL, and Yescarta® in DLBCL/PMBCL. The NLT accepted Kymriah® (ALL) and Yescarta® (DLBCL/PMBCL) following a delay, but rejected Kymriah (DLBCL). The AIFA granted Kymriah® (ALL) and Yescarta® (DLBCL/PMBCL) innovative status. ZIN have placed Kymriah® (ALL and DLBCL) and Yescarta® (DLBCL/PMBCL) under a reimbursement lock mechanism.

CONCLUSIONS: CAR-T cell therapies have generally received rapid and positive assessments by European HTA bodies to date. Nevertheless, as their number and indications increase, payer reforms may be forthcoming (e.g. NICE budget impact threshold) to better contain costs.