Analyzing Biomarkers in Drug Development
Biomarkers as indicators of biological and pathogenic processes have been used for decades to diagnose patients and prescribe treatment. In the past 10 years, however, they’ve substantially changed drug development practices. The demand for analyzing biomarkers in drug development has increased dramatically with the advancement of precision medicine. In the drug development context, biomarkers that are indicative of mechanism of action, target engagement, and clinical outcomes have been employed to assess both the safety and efficacy of an intervention. More specifically, biomarkers have been used either to select patients for clinical trials, to make go/no-go decisions, to support registration and label claims, or to treat patients.
NOAEL in IND Programs
Throughout my career as a toxicologist, I have observed that toxicology studies in drug development, are sometimes considered as being only a box-checking exercise on the path to the clinical trial. While this can be so in a limited respect, I think it is underappreciated that definitive toxicology studies, and related studies, serve a highly critical purpose. That is, they help identify potential safety issues and target organs in humans, to identify translational markers of potential toxicity in later human trials and most immediately and importantly they can provide high quality and reliable data on which to base the safe starting dose in humans. Without these definitive studies, or with poorly conducted or interpreted studies, the risk to trial participants and to eventual patients, is simply too high. So, the data from these studies, and particularly the NOAEL information is essential for the safe and effective development of new drugs.