PBI66 ARE HTA AGENCIES BUYING INTO THE CAR-T HYPE?

Abstract

The first chimeric antigen receptor T (CAR-T) cell therapies tisagenlecleucel-T and axicabtagene ciloleucel received marketing authorization in Europe in August 2018 for treatment of adult patients with severe hematologic malignancies. Both products received regulatory support under EMA’s PRIME scheme resulting in optimized trials from the regulatory perspective. The objective of this analysis was to evaluate the HTA agency view of these products by analysing health technology assessment (HTA) decisions on CAR-Ts and their rationale. All published HTAs for tisagenlecleucel-T and axicabtagene ciloleucel until May 2019 were identified, abbreviated reviews (IQWiG orphan) were excluded. HTA reports were analysed qualitatively to determine final recommendation and rationale, specifically looking at clinical and economic critiques. 30 HTAs from 13 HTA agencies on CAR-Ts were identified. Seven HTAs did not provide a recommendation (3 FIMEA, 1 NoMA, 1 AEMPS, 2 TLV). Of the assessments with a recommendation, the majority (78% [18/23]) recommended the use of CAR-T although five (22% [5/23]) of these restricted the recommended population. Five HTAs (22% [5/23]) did not recommend the use of CAR-Ts. Main positive comments were the innovative nature of the products and good clinical efficacy demonstrated in clinical studies. Like the EMA, HTA agencies also recognized the value of RWE that was used to provide context to the single-arm trials. Negative critiques were the lack of long-term data, inappropriate trial comparator and not being cost-effective. CAR-T products were positively received by the majority of HTA agencies due to innovative nature and good efficacy. Only in five cases a negative recommendation was given, mainly due to uncertain or lack of cost-effectiveness and one assessment due to lack of clinical benefit. These results highlight the importance of new innovative products in this disease area.