Personalized medicine: challenges in biomarker-related clinical trial design

Author(s): Pei He

Personalized medicine becomes an area of great interest following the recent development in human genetics, proteomics and metabolomics. There is an increasing need to embed the scientific discoveries from basic medical research into real life clinical practices, such that a patient’s biological traits can be used to facilitate treatment. Those individualized biological traits, termed as biomarkers, are actively involved in developing personalized medicine and therefore bring out challenges to clinical trial designs. The necessity of biomarker validation and patient subgroup selection make the trial design more complex. This paper will first introduce different types of biomarkers and then review the challenges of clinical trial design with biomarkers from the clinical, statistical and regulatory perspectives.