Feng Catherine, Virparia Riddhi, Mui Eric T-K
Pharmaceutical Industry Fellowship Program, Northeastern University, 360 Huntington Ave, R218 140 The Fenway, Boston, MA, 02115, USA.
Division of Pharmacotherapy & Experimental Therapeutics, UNC Eshelman School of Pharmacy, Chapel Hill, NC, USA.
Ther Innov Regul Sci. 2021 Jul;55(4):881-888. doi: 10.1007/s43441-021-00296-7. Epub 2021 Apr 28.
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.
美国食品药品监督管理局（FDA）卓越肿瘤中心（OCE）于2017年设立了实时肿瘤学审评（RTOR）试点项目，旨在简化肿瘤药物申请的审评流程，由申请人与该机构就模块组件的分步策略和时间表达成一致。处方药使用者付费法案（PDUFA）的审评时钟直到最后一个组件提交才正式开始。由于需要多次提交申请并与FDA进行互动，参与该项目需要仔细规划时间和资源。申请人还必须满足关于临床试验设计和开发计划的某些标准才有资格参与RTOR。我们检索了公开可用的数据库（Drugs@FDA）和文件，以查找所有RTOR申请，结果显示共有28份获批申请在2018年2月至2020年8月期间参与了该项目。我们对初始上市申请进行了进一步审查，以确定参与试点项目可能存在的任何潜在优势或局限性。这四个案例研究展示了一个个性化的RTOR流程，反映了该项目的试点性质。FDA在PDUFA目标日期前大约三到四个月批准了4份申请中的3份。节省的时间并不能得到保证，因为审评的其他部分可能会影响整体时间表。然而，可选的双周电话会议增加了申请人与FDA之间的沟通与合作。由于参与试点项目的获批申请数量仍然相对较少，RTOR对申请的全面影响仍未确定。
