Predicting success in regulatory approval from Phase i results

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

Research output: Contribution to journalArticlepeer-review

7 Scopus citations


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.

Original languageEnglish (US)
Pages (from-to)1099-1103
Number of pages5
JournalCancer chemotherapy and pharmacology
Issue number5
StatePublished - Nov 2014


  • Phase I
  • Predictors
  • Regulatory
  • Response rates

ASJC Scopus subject areas

  • Pharmacology (medical)
  • Oncology
  • Cancer Research
  • Toxicology
  • Pharmacology


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