OBJECTIVES: Hematopoietic stem cell transplantation (HSCT) plays a critical role in the management of acute lymphoblastic leukemia (ALL). In the era of tyrosine kinase inhibitors (TKIs) and their evolution from first to third generation, patients with ALL may attain complete responses and delay or avoid HSCT. The objective of this study is to assess the clinic-economic impacts of delayed or avoided HSCT in the treatment of patients with newly diagnosed (ND) Philadelphia chromosome-positive (Ph+) ALL receiving first-line ponatinib versus generic imatinib over a three-year time horizon from a US Commercial health plan perspective.

METHODS: A cost-consequence model (CCM) was developed using individual patient data on time-to-HSCT from the PhALLCON trial (NCT03589326). The number needed to treat (NNT) to avert one HSCT was calculated using restricted mean survival time at year three. The CCM considers epidemiology and costs associated with drug acquisition (assumed continuously accrued until HSCT; generic price for imatinib), HSCT, and graft-versus-host disease, informed by clinical trial results, published literature, and public databases.

RESULTS: In a one-million-member Commercial health plan, an estimated 4.6 patients will develop Ph+ ALL every year. Simulating these 4.6 patients over three years, treatment with ponatinib results in 1.2 averted HSCTs on average versus imatinib. By year three, the NNT to avert one HSCT with ponatinib is estimated to be 3.5 and patients treated with ponatinib experience on average up to 5.3 additional HSCT-free months versus imatinib. The incremental cost per month of delayed HSCT was $22,802 in year one and declined to $1,712 by year three with ponatinib. Total incremental per-member-per-month costs ranged from $0.05 to $0.04 from year one to three, respectively.

CONCLUSIONS: This analysis demonstrates that treatment of ND Ph+ ALL patients with ponatinib may offer more efficient care by delaying or avoiding HSCT and complications thereof at a modest cost versus treatment with generic imatinib.