Use of FDA Expedited Programs and Development Timelines for New Molecular Entity Drug and New Biologic Approvals by Sponsor Experience.

Industry reports indicate that small biopharmaceutical companies are increasingly launching their own new molecular entities, with first-time companies expected to represent the primary share of blockbuster product launches within the decade. However, company experience may affect the use of Food and Drug Administration (FDA) expedited programs, clinical development times, and FDA review times for new molecular entities and new biologics. Hence, we conducted a cross-sectional study examining all new molecular entity (NME) drug and new biologic approvals by the FDA from 2015 through 2022. Sponsors were categorized as first-time when the product represented their inaugural approval or followed their first approval by no more than 12 months. We extracted data on expedited program use (accelerated approval, breakthrough therapy, fast track, and priority review), first-cycle approval status, Prescription Drug User Fee Act (PDUFA) deadline achievement status, development and regulatory review timelines, and patent counts from public FDA data. From 2015 through 2022, 355 NMEs and new biologics were approved: 131 (36.9%) by first-time companies and 224 (63.1%) by experienced companies. The proportion of approvals attributed to first-time companies increased significantly over time (27.3% in 2015-2016 vs. 43.7% in 2021-2022; P = 0.04). Despite their lack of prior regulatory experience, there were no statistically significant differences in expedited program use, first-cycle approval rates, PDUFA deadline achievement rates, clinical development times, or patent counts between first-time and experienced companies. These findings suggest that first-time pharmaceutical companies appear to successfully navigate FDA regulatory processes as effectively as experienced firms.