Because of considerable upfront costs, CAR-T(°) treatments are in the process of being reviewed by a number of countries to determine whether they represent value for money. Many countries have determined that the early evidence indicates a benefit, but there is uncertainty due to the single-arm nature of the trials and the lack of data in the longer term. The impact of hematopoietic stem cell transplantation in patients treated with CAR-T further adds to the uncertainty as to whether the CAR-T is responsible for the possible prolongation of survival or whether the stem cell transplant primarily contributes to this.
Clinicians have indicated that a proportion of patients in paediatric acute lymphoblastic leukaemia who receive CAR-T are likely to continue to receive stem cell transplants. In determining comparative and cost effectiveness, the influence of this choice of treatment pathway should be carefully considered.
(°) Chimeric antigen receptor-engineered T-lymphocytes
Last update: 25 February 2020