OBJECTIVES : The first two CAR-T cell therapies, Kymriah^® (tisagenlecleucel) and Yescarta^® (axicabtagene ciloleucel) received European marketing authorization in August 2018 (Kymriah^® for ALL and DLBCL and Yescarta^® for DLBCL/PMBCL). These therapies offer transformational benefits for patients with life‑threatening cancers with few treatment alternatives from a single treatment administration. However, there has been significant payer concern about the affordability of these therapies, which can be cost‑effective at very high patient prices. This research aims to evaluate how payer bodies have assessed these therapies to date.

METHODS : All publicly-available published HTA evaluations of Kymriah^® and Yescarta^® by NICE, SMC, G‑BA, NLT, ZIN and HAS were screened and key information extracted (to 08/06/2019).

RESULTS : Seventeen completed HTA evaluations of CAR-T cell therapies were identified (6 Kymriah^® ALL, 5 Kymriah^® DLBCL, 6 Yescarta^® DLBCL/PMBCL) at an average of 5.4 months after European marketing authorization. Two negative appraisals were identified (SMC: Kymriah^® [DLBCL] and Yescarta^® [DLBCL/PMBCL]). NICE recommended Kymriah^® (ALL and DLBCL) and Yescarta^® (DLBCL/PMBCL) for inclusion in the CDF. Under HAS, Kymriah^® attained ASMR III (ALL) and ASMR IV (DLBCL), and Yescarta^® an ASMR III (DLBCL/PMBCL). The G-BA reported that Kymriah^® and Yescarta^® offered a unquantifiable added benefit in their relevant indications as orphan drugs in time-limited resolutions. The SMC recommended Kymriah^® (ALL). The NLT accepted Kymriah^® (ALL), while the decisions for Kymriah^® (DLBCL) and Yescarta^® (DLBCL/PMBCL) have been delayed. ZIN have placed Kymriah^® and Yescarta^® in a reimbursement lock while a price negotiations are ongoing.

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