Presentation (CTI)

OBJECTIVES: Justifications for prices of the first 10 drugs selected in Inflation Reduction Act (IRA) Drug Price Negotiation Program have been released by Centers for Medicare & Medicaid Services (CMS). The objective of this study was to analyze references utilized in the initial CMS reports to provide insights into how future drug negotiations may be conducted.
METHODS: References from CMS' maximum fair price (MFP) explanations were catalogued to identify real-world evidence (RWE), cost-effectiveness analyses (CEAs), network meta-analyses (NMAs), and indirect treatment comparisons (ITCs). Titles and abstracts were screened to further categorize studies by country of origin, study sponsor, and inclusion of Medicare-specific data sources.
RESULTS: A total of 1,824 select references were submitted by CMS across 10 MFP justification reports. As an illustrative example, CMS cited the following for its MFP explanation for apixaban: 14 ITCs/NMAs, 65 CEAs, and 90 RWE studies; 60% of these were conference abstracts. The majority (62.5%; 105/168) were pharmaceutical industry-sponsored studies. Of the CEAs cited by CMS for apixaban, 91.0% (59/65) came from countries outside the United States (US) and the majority (75%) utilized quality-adjusted life-years (QALYs) in their results. Nearly a third (28.0%, 25/90) of the RWE studies originated from ex-US countries. Among the US-based RWE studies, 76.4% (55/72) utilized Medicare data.
CONCLUSIONS: This analysis shows that CMS seems willing to leverage economic models, and specifically studies reporting QALYs despite challenges in the US, as well as ex-US RWE, as part of their MFP justification. CMS’ openness to QALY-based economic evaluations may prompt manufacturers to publish CEAs from multiple countries to establish a consistent evidence base of value for money. Additionally, conducting RWE studies ex-US may help proactively build evidentiary base that may be leveraged in future CMS negotiations.