罕见病药物临床研发的审评考虑

Appraisal Concerns for the Clinical Research and Development of Drugs for Rare Diseases

  • 摘要: 罕见病单病种发病率极低,病情复杂,诊断难度大,这些因素导致罕见病药物临床研发面临诸多困难。中国在罕见病用药上存在迫切的临床需求,只有鼓励新药研发,特别是中国拥有自主知识产权的新药创新,才是解决中国罕见病患者的用药困境的根本举措。为进一步提高罕见病药物临床研发效率,国家药品监督管理局药品审评中心组织撰写了《罕见疾病药物临床研发技术指导原则》。这是中国首个从临床研发技术层面,针对罕见病起草的指导原则。该指导原则为药物研发单位提供了罕见病药物研发的科学思路与框架,旨在指导药物研发单位,在遵循一般药物研发规律的前提下,以科学为基础,结合罕见病自身特点,实现罕见病药物的合理、高效研发。本文将结合该指导原则中的研发理念,阐述当前对罕见病药物临床研发的审评考虑。

     

    Abstract: The incidence of each of the rare disease is very low. The complexity and diagnosis difficulty of the rare disease lead to the difficulties in the clinical research and development (R&D) of drugs for rare diseases. There is an urgent clinical need for the drug development of rare diseases in China. Encouraging R&D of new drugs, particularly the innovative drugs with China's own independent intellectural property is the basis for solving the predicament in drug shortage in China.. In order to further improve the efficiency of clinical R&D of drugs for rare diseases, the National Medical Products Administration (NMPA), Center for Drug Evaluation (CDE) issued Technical Guidance for Clinical Research and Development of Drugs for Rare Diseases. This is the first guidance for rare diseases in China that is drafted from the standpoint of the clinical technology research and development.The guidance is the scientifitc thinking and framework for the drug developing enterprises to research and develop drugs for rare disease efficiently and appropriately by following drug developing protocols and relating to the special features of rare disease.This paper presents the concepts and rationale in the guidance for the appraisal of rare disease drug research and development.

     

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