Impact of the use of cost-effectiveness analysis (CEA) on patient access to innovative medicines for cancer and rare diseases: A comparison of CEA versus non-CEA markets

Background Although previous studies have examined approaches to health technology assessment (HTA) and different resulting levels of patient access across individual markets, data focusing on the use of cost-effectiveness analysis (CEA) versus other primary approaches to value assessment (e.g., based on relative clinical effectiveness) were previously lacking in the published literature. This study aimed to identify how the use of CEA impacted national-level patient access decisions for innovative medicines. Methods National-level patient access decisions in 10 markets with established systems (CEA: Australia, Canada, England, Scotland, Sweden; non-CEA: France, Germany, Italy, Spain, United States [US]) were assessed for all first indications of oncology and orphan drugs with initial European Medicines Agency regulatory approval between 2016 and 2019, and all subsequent indications approved to December 2021. Corresponding US, Australian, and Canadian approvals for the respective drugs were also included. To ensure consistency across markets, a patient access decision was defined as the first applicable national-level access outcome decision recommending some positive level of patient access (either full access or restricted access) or the final negative recommendation (no access). Results The sample included 129 unique drug/indication pairs (70 individual drugs). Overall, 862 patient access decisions were identified. The number of patient access decisions was markedly higher in non-CEA (n = 510) than CEA markets (n = 352). The total number and proportion of full patient access decisions was also higher in non-CEA (n = 335 [65.7%]) than CEA (n = 124, [35.2%]) markets, with restricted patient access decisions more common in CEA (n = 150 [42.6%]) than non-CEA (n = 113 [22.2%]) markets. The types of patient access decisions (i.e., no vs. full vs. restricted vs. no access) also varied across agencies, even within CEA and non-CEA markets. Conclusions National-level positive patient access decisions for innovative oncology and orphan medicines were more limited in markets that primarily use CEA in value assessments than in those that do not. Patient access restrictions beyond the regulatory label were also applied more often in CEA than non-CEA markets at the national level, recognizing that additional (and heterogeneous) barriers that impact ultimate patient access may occur downstream of the national decision in some markets.