Serelli-Lee Victoria, Ito Kazumi, Koibuchi Akira, Tanigawa Takahiko, Ueno Takayo, Matsushima Nobuko, Imai Yasuhiko
Clinical Evaluation Sub-Committee, Medicinal Evaluation Committee, Japan Pharmaceuticals Manufacturers Association, 2-3-11, Nihonbashi Honcho, Chuo-ku, Tokyo 103-0023, Japan.
Eli Lilly Japan K.K., 5-1-28 Isogamidori, Chuo-ku, Kobe 651-0086, Japan.
J Pers Med. 2022 Apr 21;12(5):669. 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.
生物技术的进步使我们能够以前所未有的深度和分辨率检测人体组织和细胞，重新定义了我们所熟知的“生物标志物”以及我们对“疾病”的定义。这伴随着从“一刀切”方法向“个性化方法”的重点转移，使药物开发行业处于高度动态的环境中，必须应对这些颠覆性趋势。对此，创新的临床试验设计一直是实现生物标志物驱动的药物开发的关键。癌症基因组测序面板及相关靶向疗法的监管批准已将个性化药物带入临床。诸如下一代测序（NGS）等先进生物技术的可用性不断提高，也导致了大量真实世界基因组数据的外流。本综述总结了生物标志物驱动的药物开发的现状，并突出了显示真实世界数据在该过程中的实用性和重要性的实例。我们还建议药物开发中的所有利益相关者应（1）意识到并有效利用真实世界证据，以及（2）在这个个性化药物时代重塑该行业进行药物开发的方式。
