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01-11-2014 | Short Communication

Predicting success in regulatory approval from Phase I results

Authors: Laeeq Malik, Alex Mejia, Helen Parsons, Benjamin Ehler, Devalingam Mahalingam, Andrew Brenner, John Sarantopoulos, Steven Weitman

Published in: Cancer Chemotherapy and Pharmacology | Issue 5/2014

Abstract

Purpose

Drug 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.

Methods

Phase 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.

Results

Phase 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.

Conclusions

These preliminary findings indicate that objective responses in Phase I trials may have predictive value for later regulatory approval.

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Title: Predicting success in regulatory approval from Phase I results
Authors: Laeeq Malik
Alex Mejia
Helen Parsons
Benjamin Ehler
Devalingam Mahalingam
Andrew Brenner
John Sarantopoulos
Steven Weitman
Publication date: 01-11-2014
Publisher: Springer Berlin Heidelberg
Published in: Cancer Chemotherapy and Pharmacology / Issue 5/2014
Print ISSN: 0344-5704
Electronic ISSN: 1432-0843
DOI: https://doi.org/10.1007/s00280-014-2596-4

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