[Current PMDA Activities for Use of Biomarkers in Drug Evaluation]

Biomarkers (BM) are gradually being recognized as useful tools to evaluate drugs from development through post-approval periods. In the past decade, practical use of BM has advanced particularly in the field of anti-cancer drug development. Regardless of the use of BM, approximately 10% of key clinical trials for new drug applications of anti-cancer drugs were conducted as multiregional clinical trials. In the era of globalization of drug development, common understanding regarding the usefulness and limitations of BM availabilities in drug evaluation will contribute to provide better evidence in multiple clinical trials. However, only two guidelines regarding BM, i.e., terminologies of pharmacogenomics (E15 guideline) and document format in BM qualification submission to regulatory agencies (E16 guideline), have been harmonized in the International Conference on Harmonization of Technical Requirements for Registration of Pharmaceuticals for Human Use (ICH) so far. It is important to strengthen international harmonization and collaboration among academia, industry, and regulatory agencies, followed by the establishment of an international guideline on the application of BM in drug evaluation. This article outlines the regulatory perspective on remaining challenges and current Pharmaceuticals and Medical Devices Agency (PMDA) activities for use of BM in drug evaluation.