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[摘要]
自1960年以来,美国FDA推出的基础研究-发现-设计-临床前开发-临床研究等过程的新药研发的转化研究,这种"转化研究"的"万里挑一"的模式,可以说是最早的"转化研究",对近50年的新药发展起了积极作用。随着生命科学研究的发展及成果的应用,转化研究得到快速发展,转换医学模式成为国际医学健康领域的新概念和研究模式。在美国2010年提出"精准医学"概念之后,奥巴马在他的国情咨文中提出了"精确医学"计划,希望更接近治愈癌症和糖尿病等疾病,希望将以基因为特点的大数据信息用于精准个体化药物治疗。"精准医学"作为医学的未来是人类医学的变革,长期目标是为实现多种疾病的治愈提供有价值的信息。基于精准医疗四要素中"精确、准时、共享、个体化",笔者提出"精准药学"的概念,希望它在实现"精准医疗"中发挥重要的作用,而且具有不同于"精准医学"的研究目标和研究特征。"精准药物"治疗只有实现"精准诊断"的基础上,医疗应用相关的"精准药物"才能提出"精准治疗方案",才能实现精准的个体化治疗。在"大数据"时代,基因组学是精准医学和精准药学的共同基础。但药物研发中可以认为与健康人和病人的基因有关,更与疾病的病因有关,但有些功能性疾病和病毒、细菌、寄生虫等感染性疾病的治疗还与它们的感染、复制及其酶和蛋白等生化过程有关,也与药物的制剂技术和组合有关。因此需要更广阔的知识和视野去认识研发的难度和治疗的精准性,才能开发出疗效更好、更安全、更便利、更经济的新药。
[Key word]
[Abstract]
Since the 1960s, the USA-FDA launched a mode on new drug research and development (R&D) by evaluation process based on discovery, design, preclinical research, and clinical development. This "one in a million" is a "translational research" and used for nearly 50 years. It played an active role in the R&D of new drugs. With the development and application of life science in medical research, translational research developed rapidly, thereby the medical model became a new concept and research model in international medical and health field. After the United States put forward the "precision medicine" concept, US President Barack Obama proposed a "precision medicine" plan in the State Union, and used this plan in the treatment of cancer and diabetes and other diseases, and hoped the big data information would be applied for precision drug individualized therapy. As the future development of medicine, "precision medicine" will change human medicine, and long-term goal is to achieve a variety of diseases of valuable information. Based on the four elements in precision medicine such as "accurate, timely, shared, and individual", author proposes the concept of "precision pharmacy" in this paper, and wants it to achieve "precision medicine" playing an important role, but having difference with "precision medicine" in research objectives and research characteristics. "precision diagnosis" is base to in individualized treatment, the application of "precision diagnosis" and "precise drugs" in order to achieve accurate medical therapy. In the "big data" era, genomics is common ground precision medicine and precision pharmacy. However, drug development and patient can be considered healthy genes, but also with the etiology of the disease related, but treating some functional diseases and infection diseases (such as viruses, bacteria, parasites, and other infectious diseases, but also with their infection), the replication and enzyme and protein and other biochemical processes. Additionally, the treatment is yet related with preparation techniques and combinations of drugs. So just we need a broader understanding the difficulty and having a vision in order to developsafer, more convenient, and more economical drugs with better efficacy for precision treatment.
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