Abstract The accelerated approval (AA) pathway expedites treatment access for patients with unmet medical needs. However, evidence on the program’s impact are lacking. This study assessed real-world clinical outcomes for AA drugs in solid tumor cancers. A nationwide electronic health record (EHR)-derived de-identified database was utilized to explore the AA pathway’s impact in patients with selected solid tumors in 2011–2023. Included patients received AA or non-AA products with the same indication. Outcomes were real-world progression-free survival (rwPFS) and overall survival (OS). Population-level health impacts were estimated via market share and real-world progression-free life years (rwPFLYs) and life years (LYs). Twenty-three AA indications were studied. At study completion, 83% of indications had their clinical benefit verified while 17% had been withdrawn from the market. Overall, 78% of indications demonstrated significant improvements in rwPFS or OS, while none reported significantly worse rwPFS versus controls. In total, 44% and 48% of indications had 30% rwPFS and OS improvement, respectively. More indications with sample sizes 100 in the AA cohort demonstrated significant rwPFS gains (80% 12/15) versus cohorts 100 (38% 3/8). An estimated 118,107 patients received AA drugs during their AA window, resulting in 56,399 rwPFLYs and 50,848 LYs gained. Most solid tumor AA indications were associated with improved clinical outcomes, providing substantial US population-level health gains. This EHR analysis provides insight into whether the AA pathway appropriately targets promising beneficial therapies. Expanding beyond randomized control trial evidence and conventional primary endpoints may be necessary to verify clinical benefit, particularly in rare tumors.
William B. Wong (Mon,) studied this question.