Abstract 2623: Investigation into factors influencing probability of success in oncology clinical trials and trends over time

Abstract

Abstract The regulatory approval process for new therapies in oncology involves costly clinical trials that can span multiple years and historically have had a low probability of success. To derive estimates of the probability of success and other related risk characteristics in oncology clinical trials, we searched Informa Pharma Intelligence's Trialtrove and Pharmaprojects databases for oncology-specific drug-indication programs between 2001 and 2020. In total, we identified 10,083 oncology-specific drug-indication programs, which included 2,800 therapeutic agents (excluding biosimilars) tested across 98 indications. The features we considered included indication and indication group, clinical phase, follow-on indication status, presence and type of patient selection biomarker, drug modality, and accelerated approval status. In several cases, clinical success rates were higher than the benchmark oncology historical average of the phase 1 to approval success rate of 4.1% found in our sample set. For example, the use of pre-selection biomarkers in early clinical development was associated with a greater than two-fold increase in probability of approval from phase 1. Moreover, drugs with prior approvals in another indication were more than three times more likely to be successful from phase 1 than a candidate targeting a novel mechanism. In addition, we found that monoclonal antibodies were numerically more successful than other modalities in the historical data, while oncolytic viruses, vaccines, and bispecific antibodies have been less successful. In all cases, concomitant with the emergence of genomic technologies, an improved scientific understanding of cancer biology, and an evolving regulatory environment that includes a higher number of accelerated approvals, success rates in oncology have improved during the time period of this analysis and particularly in the past 5 years. Citation Format: Mary Jane Hinrichs, Shomesh E. Chaudhuri, Robert Bowden, Andrew W. Lo, David W. Jenkins. Investigation into factors influencing probability of success in oncology clinical trials and trends over time [abstract]. In: Proceedings of the American Association for Cancer Research Annual Meeting 2021; 2021 Apr 10-15 and May 17-21. Philadelphia (PA): AACR; Cancer Res 2021;81(13_Suppl):Abstract nr 2623.