孤儿药研发相关的信息学研究进展
发布时间：2019-03-25 13:19
【摘要】：药物研发包括临床前研究和临床研究,在推动医药产业发展中发挥着至关重要的作用。由于研发各阶段投入成本高、周期长,药物毒性作用难预知等原因,新药开发的耗费巨大,成功率平均只有万分之一,周期可长达几年甚至几十年。而罕见病药物研发由于基因异质性高、病理机制未明、可参与临床试验的患者数量少等问题在药物研发上更是具有较大局限性。近年来信息学领域的飞速发展大大推动了药物研发的进程,基于计算机的理性药物设计、海量虚拟筛选、组合化学、高通量反应筛选系统等技术不断发展成熟,在新药研发的各个阶段均起到积极的促进作用;信息技术还有助于药物重定位这一新型药物开发模式的发展,通过药物警戒作用可对药物研发进行有效的反馈、补充。信息技术的发展还促进了药物研发模式向集成式研发转变。这对孤儿药研发起到了积极的促进作用。本文主要对孤儿药研发相关的信息学研究进展进行综述。
[Abstract]:Drug research and development, including pre-clinical research and clinical research, plays an important role in promoting the development of pharmaceutical industry. Due to the high cost of R & D, long cycle and difficult to predict the toxicity of drugs, the cost of new drug development is huge, the average success rate is only 1/10000, and the cycle can be as long as several years or even decades. However, drug development in rare diseases has great limitations due to its high gene heterogeneity, unclear pathological mechanism and the small number of patients who can participate in clinical trials. In recent years, the rapid development in the field of informatics has greatly promoted the process of drug research and development. Computer-based rational drug design, mass virtual screening, combinatorial chemistry, high-throughput reaction screening system and other technologies have been developed and matured. It has played a positive role in promoting the development of new drugs at every stage of the research and development of new drugs; Information technology also contributes to the development of a new drug development model, drug repositioning, which can provide effective feedback and supplement to drug development through pharmacovigilance. The development of information technology has also promoted the transformation of drug R & D model to integrated R & D. This has played a positive role in the development of orphan drugs. In this paper, the advances in informatics related to orphan drug development are reviewed.
【作者单位】： 中国医学科学院北京协和医学院 北京协和医院中心实验室;中国医学科学院北京协和医学院 北京协和医院信息管理处;中国医学科学院北京协和医学院 北京协和医院普通内科;
【基金】：国家重点研发计划精准医学研究重点专项“罕见病临床队列研究”资助项目(2016YFC0901500) 中国医学科学院医学与健康科技创新工程经费资助项目(2016-12M-2-004) 上海市出生缺陷防治重点实验室开放课题(16DZKF1007)
【分类号】：R95
本文编号：2447011
[Abstract]:Drug research and development, including pre-clinical research and clinical research, plays an important role in promoting the development of pharmaceutical industry. Due to the high cost of R & D, long cycle and difficult to predict the toxicity of drugs, the cost of new drug development is huge, the average success rate is only 1/10000, and the cycle can be as long as several years or even decades. However, drug development in rare diseases has great limitations due to its high gene heterogeneity, unclear pathological mechanism and the small number of patients who can participate in clinical trials. In recent years, the rapid development in the field of informatics has greatly promoted the process of drug research and development. Computer-based rational drug design, mass virtual screening, combinatorial chemistry, high-throughput reaction screening system and other technologies have been developed and matured. It has played a positive role in promoting the development of new drugs at every stage of the research and development of new drugs; Information technology also contributes to the development of a new drug development model, drug repositioning, which can provide effective feedback and supplement to drug development through pharmacovigilance. The development of information technology has also promoted the transformation of drug R & D model to integrated R & D. This has played a positive role in the development of orphan drugs. In this paper, the advances in informatics related to orphan drug development are reviewed.
【作者单位】： 中国医学科学院北京协和医学院 北京协和医院中心实验室;中国医学科学院北京协和医学院 北京协和医院信息管理处;中国医学科学院北京协和医学院 北京协和医院普通内科;
【基金】：国家重点研发计划精准医学研究重点专项“罕见病临床队列研究”资助项目(2016YFC0901500) 中国医学科学院医学与健康科技创新工程经费资助项目(2016-12M-2-004) 上海市出生缺陷防治重点实验室开放课题(16DZKF1007)
【分类号】：R95
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