Number needed to treat to avoid progression and death and cost analysis: zanubrutinib versus acalabrutinib in relapsed/refractory chronic lymphocytic leukemia.

[AIMS] In the absence of head-to-head comparative trials, this study aimed to compare zanubrutinib versus acalabrutinib in relapsed/refractory (R/R) chronic lymphocytic leukemia (CLL) by calculating number needed to treat (NNT) to avoid one disease progression or death and associated economic impact.

[METHODS] A health-economic model was developed from US payer perspective using efficacy data from matching-adjusted indirect comparison for overall R/R CLL in base-case analysis, and network meta-analysis for high-risk R/R CLL in the subgroup analysis. The NNT analysis included costs of drug acquisition, adverse event management, medical resource utilization, and subsequent treatment over 24 months. Deterministic sensitivity analyses assessed model uncertainty.

[RESULTS] In the base case, zanubrutinib versus acalabrutinib avoided one progression for every 10 patients treated (NNT = 10) and one death for every 15 patients treated (NNT = 15), yielding per-patient cost savings of $7,335 over 24 months. In high-risk R/R CLL subgroup, one progression was avoided per six patients treated (NNT = 6) and one death per 18 patients treated (NNT = 18), with cost savings of $11,533 per patient. Results were robust across sensitivity analyses.

[CONCLUSIONS] The NNT analysis demonstrates that treatment with zanubrutinib versus acalabrutinib is associated with more favorable clinical and economic outcomes in R/R CLL, especially in high-risk CLL patients.