Presentation (CTI)

OBJECTIVES: Under the IRA, CMS should consider therapeutic alternatives when negotiating drug prices, incorporating alternatives’ prices and comparative clinical effectiveness. CEA has been proposed to inform IRA price negotiations. However, a challenge remains: how to define therapeutic alternatives for value assessment. Existing approaches rely on guidelines, standards of care, trial comparators, and expert judgment to select one or more comparators, followed by pairwise CEA with the target drug. While pragmatic, it may not reflect real-world treatment choices. A patient-centered perspective instead asks: what options would patients receive in the absence of the target drug? These alternatives may include multiple treatments—or none—weighted by real-world practice patterns and patient preferences. We propose a comparator choice set framework to address this gap.
METHODS: We extend prior work on choice set-based value assessment by comparing two clinically relevant treatment choice sets: an expanded choice set that includes the target drug alongside all therapeutic alternatives, and a restricted choice set that excludes the target drug. Rather than pairwise comparisons, this framework evaluates the incremental value of adding the target drug to the choice set, capturing substitution across alternatives. We illustrate this by evaluating inclusion versus exclusion of ADT plus Xtandi® as first-line therapy for nmCRPC.
RESULTS: Traditional pairwise CEA yielded a wide range of value-based monthly prices for Xtandi® ($9390-$9853), depending on the selected comparator. Conversely, this produced a policy-relevant maximum value-based price of $9460 per month by accounting for treatment choice distributions; the MFP is $7004 per month.
CONCLUSIONS: We introduce a comparator choice set framework that evaluates the marginal value of adding a drug to the set of clinically appropriate alternatives. By moving beyond pairwise comparisons, the approach yields value estimates that are not driven by arbitrary comparator selection and provides a transparent basis for CMS drug price negotiations under the IRA.