Assessment of Coverage in England of Cancer Drugs Qualifying for US Food and Drug Administration Accelerated Approval

Abstract

The US Food and Drug Administration’s (FDA) accelerated approval process allows drugs to be approved based on clinical trial findings that would otherwise not be acceptable for use in the traditional FDA approval process (ie, indicating changes based on surrogate measures that are only reasonably likely to estimate actual clinical benefit).¹ Surrogate measures include biomarkers and other measurable physical properties that may be able to estimate the way a patient feels, functions, or survives. When surrogate measures are validated as being clinically meaningful, they can be substituted for traditional clinical outcomes as end points for clinical trials used for FDA approval, allowing those clinical trials to be conducted more quickly or among fewer patients. The accelerated approval process was developed to facilitate the approval of drugs that address unmet medical needs by allowing regulatory approval based on unvalidated surrogate measures; this process requires that the manufacturer commit to conducting confirmatory clinical trials after approval is granted. Accelerated approval is thus comparable to conditional approval; however, the drugs are formally designated as fully approved from the time of their first approval.