OBJECTIVES: Minimal Residual Disease (MRD) is a strong prognostic factor for patients with Philadelphia-chromosome-negative (Ph-) B-cell precursor acute lymphoblastic leukemia (BCP-ALL). The objective of this study is to estimate the cost-effectiveness of blinatumomab vs. chemotherapy in patients with MRD from a US payer perspective, using a Markov cohort modeling approach.

METHODS: A combined decision-tree and Markov cohort model was used to estimate the cost-effectiveness of blinatumomab in the BLAST trial vs. chemotherapy from a historical cohort (HC). MRD status was used to allocate transition probabilities for receiving hematopoietic stem cell transplant (HSCT), relapse, and death. Probability calculations were based on a competing risk framework. Patients from the HC study were matched to patients from BLAST using propensity score weighting. Utility values were based on EQ-5D data from BLAST applying US tariffs. Cost estimates were from published sources. One-way sensitivity analyses, scenario analyses, and probabilistic sensitivity analyses (PSAs) were conducted to test model robustness.

RESULTS: Blinatumomab yields an additional 2.47 life years and 2.05 quality-adjusted life years (QALYs) vs. chemotherapy. Blinatumomab has higher incremental costs vs. chemotherapy, $242,940; higher medication costs in the blinatumomab arm are partially offset by reduced post-relapse costs of $58,499. The ICER for blinatumomab vs. chemotherapy is $118,659/QALY gained. The costs of HSCT, MRD response rate for blinatumomab, and inpatient costs related to MRD status have the largest impact on the ICER. In PSAs, blinatumomab is cost-effective in 69% of simulations at an ICER threshold of $150,000/QALY.

CONCLUSIONS: Blinatumomab is cost effective vs. chemotherapy in ALL patients with MRD from a US healthcare perspective. Achieving MRD negativity with blinatumomab therapy is associated with better survival and improved QALYs. The results of these analyses may be useful for US healthcare payers in their deliberations regarding reimbursement decisions for this vulnerable population with limited treatment options.