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. There has been significant payer concern about the affordability of these therapies, which can be cost‑effective at very high patient prices (Kymriah’s^® UK list price: £282,000 per patient). 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, TLV, and HAS were screened and key information extracted (to 10/01/2019).

RESULTS : Eight HTA evaluations of CAR-T cell therapies in scope countries were identified (3 Kymriah^® ALL, 3 Kymriah^® DLBCL, 2 Yescarta^® DLBCL/PMBCL) at an average of 3.5 months after European marketing authorization. Only one negative appraisal was identified (NICE: Kymriah^® in DLBCL, draft guidance) but NICE also recommended Kymriah^® (in ALL) and Yescarta^® (DLBCL/PMBCL) for inclusion in the CDF.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 deemed Kymriah^® offered a considerable added benefit in ALL and DLBCL while Yescarta^® has not had a final resolution. The SMC and TLV have not yet completed evaluation of any CAR-T cell therapies.

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 may change their assessment procedures to better contain costs. The NICE budget impact threshold (introduced in April 2017) is a good example of this.