Analysis of the Real-Time Oncology Review (RTOR) Pilot Program for Approvals of New Molecular Entities

Abstract

The United States Food and Drug Administration (FDA) Oncology Center of Excellence (OCE) established the Real-Time Oncology Review (RTOR) pilot program in 2017 to streamline the review process for oncology drug applications with the applicant and the Agency agreeing upon a piecemeal strategy and timeline for module components. The Prescription Drug User Fee Act (PDUFA) review clock does not officially start until the final component is submitted. Participation requires careful planning of time and resources due to the multiple submissions and interactions with the FDA. Applicants must also meet certain criteria regarding the clinical trial design and development program to be eligible for RTOR. Publicly available databases (Drugs@FDA) and documents were searched for all RTOR applications, which revealed a total of 28 approved applications that participated from February 2018 to August 2020. Initial marketing applications were further reviewed to identify any potential advantages or limitations from participation in the pilot program. These four case studies demonstrated an individualized RTOR process reflecting the program’s pilot status. The FDA approved 3 out of the 4 applications approximately three to four months before the PDUFA goal date. The time savings is not guaranteed as other parts of the review may influence the overall timeline. However, the optional biweekly teleconferences increased communication and collaboration between the applicant and the FDA. The full impact of RTOR on applications remains undetermined as the number of approved applications that have participated in the pilot program is still relatively small. Supplementary Information The online version contains supplementary material available at 10.1007/s43441-021-00296-7.

Premarket Evidence and Postmarketing Requirements for Real-Time Oncology Review Indication Approvals

“…The US Food and Drug Administration (FDA) launched the Real-Time Oncology Review (RTOR) program in 2018 to support earlier review of oncology therapeutics expected to be a substantial improvement over available therapy based on straightforward study designs and easily interpreted end points. 1 Although RTOR applications have been associated with shorter median approval times, 2 , 3 little is known about the evidence supporting RTOR indication approvals. Accordingly, we examined FDA indication approvals that underwent RTOR; characterized their supporting evidence; and established, when applicable, whether postmarketing studies were required to confirm clinical effectiveness.…”