Expedited pathway portfolios, trial complexity, and FDA approval timelines in oncology and neurology: A comparative analysis

Ahmed M Sayed, Nada M Ezzelarab and Nguyen Tien Huy

PLOS ONE, 2026, vol. 21, issue 8, 1-15

Abstract: FDA expedited programs are intended to facilitate development and review for therapies addressing serious conditions with unmet medical need, but their use may depend on whether the evidentiary package is compatible with regulatory acceleration. We compared FDA original approvals in oncology and neurology from 2015 to 2024 to evaluate whether differences in approval timing reflected expedited pathway portfolio intensity, pivotal evidence architecture, or alignment between the two. We conducted a retrospective comparative cohort study of 195 original FDA approvals, including 136 oncology products and 59 neurology products. Product-level regulatory data were curated from FDA approval materials. Pivotal or registrational trials supporting first approval were identified from FDA labels and review documents and enriched through exact National Clinical Trial identifier matching to AACT. The final evidence dataset included 221 curated product–trial links. Regulatory pathway-portfolio intensity was summarized using a Regulatory Bundling Index incorporating Fast Track, Breakthrough Therapy designation, Priority Review, Accelerated Approval, and Orphan designation. Outcomes included regulatory approval time, clinical development time, total time to approval, and pivotal evidence architecture. Oncology products had higher pathway intensity than neurology products, with median Regulatory Bundling Index 3 [IQR, 2–4] versus 2 [IQR, 0–3] (p

Date: 2026
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DOI: 10.1371/journal.pone.0355495

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