Revumenib for Rare Acute Leukemias Is Cost-Neutral for Health Plans: Ivo Abraham, PhD, RN

Including revumenib (Revuforj; Syndax Pharmaceuticals) for relapsed/refractory acute leukemias with KMT2A translocation would be cost-neutral for a health plan with slight savings mainly due to lower administration costs, according to a recent budget impact analysis.

First-in-class targeted therapies, especially those addressing rare diseases such as revumenib, present a unique set of challenges to navigate, explained Ivo Abraham, PhD, RN, professor, Department of Pharmacy Practice and Science, R. Ken Coit College of Pharmacy, The University of Arizona, the lead author on the analysis.

As the landscapes for oncology, hematology, and immunology evolve, they are being characterized by a growing number of treatments for increasingly smaller, more specific patient populations, which strains traditional health care economic evaluation methods. In this instance, the KMT2A translocation might affect only 1.7 individuals per million patients. This small patient pool makes it difficult to demonstrate broad economic value through conventional cost-effectiveness analyses, which often rely on metrics like cost per quality-adjusted life year (QALY), Abraham noted.

“These are very difficult drugs to investigate,” he said. “They're very expensive to make, so our old methods—old in quotes—don't help us anymore…. Most treatments that come out would not be [considered] cost-effective” based on QALYs.

In this analysis, Abraham and his colleagues had to compare revumenib against all other treatments in the National Comprehensive Cancer Network guidelines for this patient population because there are no other specific treatments for this mutation. A total of 11 additional pharmacotherapies for relapsed/refractory acute leukemias were included as options. The absence of specific comparative treatments for many of these highly targeted therapies can complicate evaluations, because the analyses require conservative assumptions that can understate the true value proposition of the novel therapy, Abraham said.

The budget impact analysis can be a useful, albeit often overlooked, tool. Unlike cost-effectiveness analyses, the budget impact analysis examines the financial implications of introducing a new therapy onto a health plan's formulary. It shifts the conversation from whether a drug is "cost-effective" by traditional metrics to how it can be made "affordable" within a given budget.

“That’s a key point to make, because with our health care financing, with our thinking about how to do economic evaluations, we haven’t kept up with the phenomenal advances in science and clinical science, which we as a society want and value,” Abraham said.