Integration and Use of Biomarkers in Drug Development, Regulation and Clinical Practice: a US Regulatory Perspective

The US FDA encourages the integration of biomarkers in drug development and their appropriate use in clinical practice. It is believed that this approach will help alleviate stagnation and foster innovation in the development of new medical products, and, ultimately, lead to more personalized medicine. To facilitate the use of biomarkers in drug development and clinical practice, the FDA organized workshops, issued guidances, established a voluntary submission process, developed online educational tools and, most importantly, strives to ensure the integration of this information into drug labels, for example, via the update of existing labels, or the inclusion of appropriate language in new drug labels. A pilot process has been set up to qualify novel biomarkers that are not associated with specific drug products, but are of more common use (e.g., biomarkers for drug safety). In addition, the FDA has initiated the creation of various consortia that are working towards the identification and characterization of exploratory biomarkers in order to qualify them for a specific use.