A Framework for Re-Thinking Evidence Generation Requirements in the United States: It’ Time to Meet Expectations of the Inflation Reduction Act

OBJECTIVES: Implications of the Inflation Reduction Act (IRA) are far-ranging across the US healthcare system, including how the Centers for Medicare & Medicaid (CMS) conduct pricing negotiations. While real-world evidence (RWE) is well-accepted by other US government agencies, its ability to influence CMS negotiations is unknown. We sought to explore the role of RWE in these negotiations, and determine if a framework could help inform generation of credible and meaningful RWE to inform pricing processes.

METHODS: We conducted a systematic literature review within Embase, PubMed, and relevant grey literature, limited to those published in English from September 2022 up to May 2024 that included the terms “IRA,” “RWE”, “health technology assessment”, “price negotiation”, and/or “innovation”. Relevant information was synthesized from identified sources

RESULTS: Our review identified 65 relevant articles, of which 20 mandated full review. Analyses indicated that: (1) CMS may have adopted a value framework for pricing negotiations; and (2) RWE has substantial value to support argumentation around drug effectiveness and benefits to support higher maximum fair price (MFP). This is because RWE more closely reflects actual product usage in clinical practice, therefore more closely reflecting expectations around effectiveness and other benefits. Results also informed development of a framework that includes mandating assessments of real-world data sources for their ability to inform negotiations at sentinel moments in product history (e.g., launch, key timepoints thereafter); our framework therefore allows for use of different sources to address different (but equally relevant) questions around magnitude and impact of product usage.

CONCLUSIONS: RWE represents a comprehensive and valuable means to assess therapy impact across broad patient populations in clinical practice. However, it is important to develop a rigorous and robust framework to encourage use of appropriate sources to meet required evidence needs (initial and subsequent), with resulting output used to inform MFP negotiations.