Despite PFS Boost, missing data on 2nd Gen BTKi for R/R MCL may incur opportunity cost


The researchers highlight key findings from an early cost analysis of Phase 1/2 trials examining Burton’s tyrosine kinase inhibitors in patients with relapsed/refractory mantle cell lymphoma.

An early cost analysis based on Phase 1/2 clinical trials in patients with relapsed/refractory mantle cell lymphoma who received Burton’s tyrosine kinase inhibitors demonstrated that despite a benefit in terms of progression-free survival (PFS) with second-generation agents, a lack of according to results published in the Journal of Managed Care Specialty Pharmacy.

Cost-effectiveness analyzes aimed to compare ibrutinib (Imbruvica), acalabrutinib (Calquence) and zanubrutinib (Brukinsa) by estimating the incremental cost-effectiveness ratio (ICER) of the incremental cost to gain one year of life of PFS (PFLYg). In addition, the cost-utility ratio (ICUR) was assessed by the incremental cost to gain one quality-adjusted life year by PFS (PFQALYg).

Comparing acalabrutinib to ibrutinib, the 3-year ICER was $90,571 (probabilistic sensitivity analysis [PSA]), $88,588)/PFLYg with an ICUR of $117,098 (PSA, $110,063)/PFQALYg. Additionally, the comparison with zanubrutinib yielded a 3-year ICER of $58,422 (PSA, $58,907)/PFLYg and an ICUR of $73,027 (PSA, $73,634)/PFQALYg. The 5-year ICER estimate for acalabrutinib was $73,918 (PSA, $74,189)/PFLYg and the ICUR was $90,512 (PSA, $90,844)/PFQALYg. For zanubrutinib, the corresponding ICER and ICUR were $48,641 (PSA, $48,732)/PFLYg and $61,612 (PSA, $63,727)/PFQALYg.

“This ACE based on early phase trials of BTKIs in MCL R/R suggests an additional PFS advantage for second-generation BTKIs over ibrutinib, with acalabrutinib producing better additional PFLY and PFQALY results than zanubrutinib compared to ibrutinib, but at a higher incremental cost Both acalabrutinib and zanubrutinib were cost-effective compared to a WTP threshold of $150,000 Relative uncertainty due to lack of direct evidence from of trials may result in an opportunity cost or loss of health benefits. The economic gains associated with greater and more precise empirical certainty are likely to exceed the costs of a clinical trial,” the researchers wrote. .

The investigators estimated the effectiveness and costs of the treatment over a period of 3 to 5 years. Several comparators were included in the search, including ibrutinib versus acalabrutinib, ibrutinib versus zanubrutinib, and acalabrutinib versus zanubrutinib.

Further results of the analysis indicated that with zanubrutinib as comparator, the 3-year ICER for acalabrutinib was $144,633 (PSA, $134,964)/PFLYg and the ICUR was 197,227 $ (PSA, $166,109)/PFQALYg. Additionally, the 5-year estimates were $117,579 (PSA, $118,161)/PFQALYg and $136,144 (PSA, $136,818)/PFQALYg, respectively.

The researchers concluded that more data is needed to assess the relative cost effectiveness of BTK inhibitors.


Alrawashdh N, McBride A, Slack M, et al. Cost-effectiveness and value of information analyzes of Bruton’s tyrosine kinase inhibitors for the treatment of relapsed or refractory mantle cell lymphoma in the United States. J Manage Care Spec Pharm. 2022;8(4):390-400. doi:10.18553/jmcp.2022.28.4.390


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