Industry comments also seek more specificity on the types of real-world data that could supplement a single adequate and well-controlled trial to demonstrate substantial evidence of efficacy, and clarification as to whether confirmatory evidence can come from the same single clinical study.

US FDA’s 72 approvals under streamlined submission program consistently come before the PDUFA goal date, Pink Sheet analysis shows, but benefit ranges from 2 weeks for priority review novel agents to 1.8 months for other priority applications.

Only two breakthrough-designated novel agents remain on the US FDA drugs center’s 2023 user fee calendar, while CBER stacks up on vaccines and gene therapies under both BTD and RMAT programs.

The advisory committee review of US WorldMeds’ eflornithine for neuroblastoma was remarkable for the unusual nature of the efficacy data to support the NDA – and also for who spoke on behalf of the agency and how effectively they walked the panel through a complex set of issues.