OBJECTIVES: Tisagenlecleucel, a CD19-directed autologous T-cell immunotherapy, produces high durable response rates with manageable safety in paediatric and young adult B-cell acute lymphoblastic leukaemia (ALL) patients refractory or in second or later relapse (r/r). The objective of this study is to estimate the budget impact of adopting tisagenlecleucel from the perspective of the National Health Service (NHS) in England.

METHODS: A budget impact model was developed in which a current treatment pathway (without tisagenlecleucel) was compared to a future scenario (including tisagenlecleucel). The number of tisagenlecleucel eligible patients was estimated using the population for England from the Office for National Statistics, data from Cancer Research UK, and published literature. Current treatment options included salvage chemotherapy (fludarabine-cytarabine-idarubicin) and blinatumomab. Tisagenlecleucel market share was assumed to increase over time. Treatment administration and dosing, subsequent stem cell transplantation, medical resources, and adverse event rates (grade ≥3, occurring in ≥5% of patients) were based on pivotal clinical trials. Associated costs were based on appropriate UK sources, such as NHS reference cost schedules, and leukapheresis, lymphodepletion, and bridging chemotherapy costs were also considered for tisagenlecleucel patients. The price of tisagenlecleucel was based on the U.S. wholesale acquisition cost converted to GBP.

RESULTS: Over a 3-year time horizon, 40 patients were estimated to meet the eligibility criteria for tisagenlecleucel each year. Based on predicted market share, 1, 16, and 20 patients were estimated to receive tisagenlecleucel in years 1-3, respectively. The cumulative 3-year budget impact was estimated to be £10,311,346 (year 1: £221,456, year 2: £4,464,553, year 3: £5,625,337).

CONCLUSIONS: Results of the budget impact analysis suggest that the introduction of tisagenlecleucel in England for the treatment of paediatric and young adult patients with r/r ALL is less than £6 million annually and well below the £20 million NHS budget impact threshold.