Presentation (CTI)

OBJECTIVES: To compare the clinical and economic evidence, with a focus on real-world evidence (RWE), considered by the Centers for Medicare and Medicaid Services (CMS) in determining the Maximum Fair Prices (MFPs) for sacubitril/valsartan, rivaroxaban, and apixaban under the Inflation Reduction Act’s Medicare Drug Price Negotiation Program.
METHODS: A qualitative comparative analysis was conducted using CMS’s published MFP explanation documents. Clinical evidence was assessed based on therapeutic indications, comparative effectiveness, safety outcomes, and patient-centered data. Economic evidence included manufacturer-submitted data on research and development and costs, production and distribution costs, market pricing, and cost-effectiveness studies. The number of RWE studies cited was quantified relative to total evidence sources.
RESULTS: Sacubitril/valsartan’s MFP ($295) was informed by its clinical benefit in heart failure, supported by RWE demonstrating reduced hospitalizations and improved patient-reported outcomes. Rivaroxaban ($197) and apixaban ($231) were evaluated for stroke prevention and venous thromboembolism (VTE) treatment. Apixaban’s higher MFP was supported by a broader body of RWE, including over 10 studies out of 239 total references (~4.2%), compared to 6/227 (~2.6%) for rivaroxaban and 3/94 (~3.2%) for sacubitril/valsartan. CMS consistently prioritized RWE alongside randomized controlled trials to assess comparative effectiveness, safety in diverse populations, and treatment adherence in real-world settings.
CONCLUSIONS: RWE played a pivotal role in CMS’s holistic evaluation of clinical and economic value, particularly in differentiating between therapeutically similar agents. The integration of RWE into the negotiation framework underscores its growing importance in value-based pricing and policy decisions. This analysis highlights the need for robust real-world data to support future drug pricing negotiations and formulary decisions.