• Laeeq Malik, Alex Mejia, Helen Parsons, Benjamin Ehler, Devalingam Mahalingam, Andrew Brenner, John Sarantopoulos, and Steven Weitman.
• Institute for Drug Development, Cancer Therapy and Research Center, University of Texas Health Science Center at San Antonio, San Antonio, TX, USA, Malikl@uthscsa.edu.
• Cancer Chemother. Pharmacol. 2014 Nov 1;74(5):1099-103.

PurposeDrug development in oncology is resource intensive and has a high failure rate. In this exploratory analysis, we aimed to identify the characteristics and outcomes of published Phase I studies associated with future Food and Drug Administration (FDA) approval.MethodsPhase I studies of approved and non-approved anticancer agents between 2000 and 2013 were retrospectively examined. Fisher's exact and chi-squared tests were used to compare the potential predictive measures.ResultsPhase I studies of 88 anticancer agents (54 approved and 34 non-approved by the FDA), treating a total of 4,423 subjects, were examined. The median number of patients in Phase I trials of approved and non-approved agents was 44.5 and 32, respectively. A total of 423 subjects (86 reporting studies) had a complete responses, and 342 subjects (80 reporting studies) had a partial responses (PR). A higher number of PR (P < 0.001), PR rate (P = 0.003) and longer PR duration (P = 0.001) were predictive of regulatory success.ConclusionsThese preliminary findings indicate that objective responses in Phase I trials may have predictive value for later regulatory approval.

17 keywords...

hide…