Does Accelerated Approval Result in Favorable Access: Examining the Burden of Evidence Necessary for Appropriate Payer Coverage of Accelerated Approval Products

OBJECTIVES: The FDA accelerated approval pathway allows patients early access to drugs treating serious conditions with high unmet need. Accelerated approval drugs have historically received varying payer coverage, ranging from not covered to coverage in line with the labeled indication. This analysis aims to understand the key factors influencing payer perceptions of the clinical evidence supporting use of accelerated approval products.

METHODS: Non-oncology, non-infectious disease accelerated approval products launched from 2014-2023 with ongoing confirmatory trials were isolated for analysis. Isolated products were then analyzed to determine the influence of supporting clinical evidence on the commercial payer coverage landscape. Key factors analyzed included trial design (e.g., population size, comparator, duration, endpoint selection), magnitude of effect, and safety & tolerability. Coverage was assessed across a representative sample of 10 payers for all therapies meeting the research criteria.

RESULTS: In total, 17 products met the selection criteria outlined. Endpoint selection was determined to be a lead indicator of coverage decisions. Specifically, of these 17, 7 products used objective endpoints as primary endpoint(s) in their pivotal trial(s), while 10 used subjective endpoints. The 7 products with objective endpoints consistently achieved more favorable payer coverage across the analyzed plans, although payers consider the totality of evidence when evaluating accelerated approval products.

CONCLUSIONS: Although the accelerated approval pathway was designed to allow patients access to therapies without robust supporting evidence at time of approval, payer coverage does not necessarily mirror this intent. The primary endpoint objectivity represents a leading factor contributing to the burden of evidence required to achieve favorable coverage. However, many other criteria are considered, creating a case-by-case review basis for coverage determination. Manufacturers seeking favorable coverage of accelerated approval products may increase likelihood of success by incorporating objective endpoints in their pivotal trial design.