A State-of-the-Art Roadmap for Biomarker-Driven Drug Development in the Era of Personalized Therapies

• Published in: Journal of personalized medicine Vol. 12; no. 5; p. 669
• Main Authors: Serelli-Lee, Victoria, Ito, Kazumi, Koibuchi, Akira, Tanigawa, Takahiko, Ueno, Takayo, Matsushima, Nobuko, Imai, Yasuhiko
• Format: Journal Article
• Language: English
• Published: Switzerland MDPI AG 21.04.2022; MDPI
• Subjects: biomarker-driven drug development; Biomarkers; Biotechnology; Biotechnology industry; data ecosystem; Disease; Drug development; Drug dosages; ecosystem for personalized therapies; genome medicine; Genomes; Next-generation sequencing; Oncology; Pathophysiology; Patients; personalized medicine; Precision medicine; real-world data/evidence; Regulatory approval; Review; Working groups; Japan; disease blueprint; genome medicine; Japan; ecosystem for personalized therapies; clinical trial renovation; data ecosystem; biomarker-driven drug development; real-world data/evidence; personalized medicine
• ISSN: 2075-4426; 2075-4426
• DOI: 10.3390/jpm12050669

Advances in biotechnology have enabled us to assay human tissue and cells to a depth and resolution that was never possible before, redefining what we know as the "biomarker", and how we define a "disease". This comes along with the shift of focus from a "one-drug-fits-all" to a "personalized approach", placing the drug development industry in a highly dynamic landscape, having to navigate such disruptive trends. In response to this, innovative clinical trial designs have been key in realizing biomarker-driven drug development. Regulatory approvals of cancer genome sequencing panels and associated targeted therapies has brought personalized medicines to the clinic. Increasing availability of sophisticated biotechnologies such as next-generation sequencing (NGS) has also led to a massive outflux of real-world genomic data. This review summarizes the current state of biomarker-driven drug development and highlights examples showing the utility and importance of the application of real-world data in the process. We also propose that all stakeholders in drug development should (1) be conscious of and efficiently utilize real-world evidence and (2) re-vamp the way the industry approaches drug development in this era of personalized medicines.