Evidence base for Yescarta and Kymriah

Was the evidence base for Yescarta and Kymriah sufficient to justify their cost and secure patient access across 6 markets



To determine whether the evidence base for high-cost CAR T-cell therapies tisagenlecleucel (Kymriah) and axicabtagene ciloleucel (Yescarta) was sufficient to be granted reimbursement by European HTA bodies (Transparency Committee (TC), France; G-BA, Germany; TLV, Sweden; NICE, UK) and ICER (US), and specifically how gaps in the current evidence are linked to access restrictions.


NICE, G-BA, TLV, ICER and TC HTA reports were identified for Yescarta in the diffuse large B-cell lymphoma (DLBCL) indication and Kymriah in the acute lymphoblastic leukaemia (ALL) and DLBCL indications. These reports were assessed against a pre-defined criterion to understand whether the clinical and economic evidence was sufficient at launch to secure reimbursement. Evidence gaps were identified, and recommendations to address these gaps were compared across the five countries.


Despite the high treatment costs, patients can access these treatments in all countries. Of the HTA bodies that consider cost-effectiveness, only ICER were certain of the cost-effectiveness of both drugs, whereas NICE and the TLV allowed access despite a high level of uncertainty. Prominent evidence gaps were identified for both Yescarta and Kymriah, with the primary concern being the lack of long-term overall and progression-free survival data, as well as the lack of comparative data generated by single-arm studies.  NICE, G-BA and the TC require additional evidence to be gathered to demonstrate both drugs long-term clinical value and cost-effectiveness.


Whilst these CAR-T treatments are perceived to be innovative, the UK, France and Germany granted restricted access due to the level of uncertainty surrounding the submitted clinical and economic evidence. European HTA bodies have implemented strategies to mitigate the risk of introducing these expensive drugs, including entry into the Cancer Drug Fund (UK), an innovative outcomes agreement in Germany or annual re-assessment by HAS (France).

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