Assessing the predictive value of the rodent neurofunctional assessment for commonly reported adverse events in phase I clinical trials

Regul Toxicol Pharmacol. 2016 Oct:80:348-57. doi: 10.1016/j.yrtph.2016.05.002. Epub 2016 May 4.

Abstract

Central Nervous System (CNS)-related safety concerns are major contributors to delays and failure during the development of new candidate drugs (CDs). CNS-related safety data on 141 small molecule CDs from five pharmaceutical companies were analyzed to identify the concordance between rodent multi-parameter neurofunctional assessments (Functional Observational Battery: FOB, or Irwin test: IT) and the five most common adverse events (AEs) in Phase I clinical trials, namely headache, nausea, dizziness, fatigue/somnolence and pain. In the context of this analysis, the FOB/IT did not predict the occurrence of these particular AEs in man. For AEs such as headache, nausea, dizziness and pain the results are perhaps unsurprising, as the FOB/IT were not originally designed to predict these AEs. More unexpected was that the FOB/IT are not adequate for predicting 'somnolence/fatigue' nonclinically. In drug development, these five most prevalent AEs are rarely responsible for delaying or stopping further progression of CDs. More serious AEs that might stop CD development occurred at too low an incidence rate in our clinical dataset to enable translational analysis.

Keywords: Adverse events; Central nervous system; First-in-human; Functional observational battery; Methods; Neurobehavioural assessment; Predictive value; Translation.

MeSH terms

  • Animals
  • Behavior, Animal / drug effects*
  • Central Nervous System / drug effects*
  • Central Nervous System / physiopathology
  • Central Nervous System Diseases / chemically induced*
  • Central Nervous System Diseases / physiopathology
  • Clinical Trials, Phase I as Topic*
  • Dose-Response Relationship, Drug
  • Drug-Related Side Effects and Adverse Reactions / etiology*
  • Drug-Related Side Effects and Adverse Reactions / physiopathology
  • Humans
  • Mice
  • Rats
  • Reproducibility of Results
  • Risk Assessment
  • Species Specificity
  • Toxicity Tests / methods*