对“以患者为中心”的罕见疾病药物研发的审评考虑

Consideration on " Patient-Centered" Drug Development for Rare Diseases

  • 摘要: 罕见病患者的临床需求远未被满足,目前仍有许多罕见病缺乏有效的治疗药物,同时罕见病的药物研发又面临着比常见多发疾病更大的挑战。近年来,“以患者为中心”的药物研发理念已被广泛采纳。国家药品监督管理局药品审评中心陆续发布了《以患者为中心的临床试验实施技术指导原则》等多项相关指导原则,以推进“以患者为中心”的药物研发模式。在罕见病药物研发领域,落实“以患者为中心”的药物研发理念,关注患者视角,倾听患者声音,将是帮助药物研发企业、研究者、监管机构深入了解罕见病和患者需求,提高罕见病药物研发精准度与研发效率的有效途径。未来,国家药品监督管理局药品审评中心将持续关注“以患者为中心”的理念融入罕见病药物研发领域,切实提高罕见病患者在药物研发过程中的参与度,发挥患者观点对药物研发的指导作用。

     

    Abstract: The unmet clinical needs of patients with rare diseases persist. Many rare diseases lack effective treatments, and drug development for rare diseases faces greater challenges than that for common multiple diseases. In recent years, the concept of " patient-centered" drug development has been widely adopted. The Center for Drug Evaluation(CDE) of the National Medical Products Administration has successively issued a series of relevant guiding principles, such as the Technical Guidelines for the Implementation of Patient-Centered Clinical Trials, to promote a " patient-centered" drug development model. The implementation of the " patient-centered" approach in rare disease drug research and development, with a focus on patient perspectives and active engagement, can effectively facilitate a comprehensive understanding of rare diseases and patient needs among drug research and development enterprises, researchers, and regulatory agencies. This approach also enhances the accuracy and efficiency of rare disease drug research and development. The CDE will continue to prioritize the integration of the " patient-centered" concept into rare disease drug research and development, effectively enhance the involvement of rare patients in the drug research and development process, and leverage the guiding role of patients′ perspectives on drug research and development.

     

/

返回文章
返回