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Journal of International Pharmaceutical Research. 2019, (09) Core

Challenges and solution to health technology assessment of rare diseasesChinese Full TextEnglish Full Text (MT)

XU Hao-peng;SHEN Yang;ZHU Chong;GONG Meng-chun;CHEN Li;ZHAO Kun;ZHANG Shu-yang;Chinese Academy of Medical Sciences ＆ Peking Union Medical College;Digital China Health Technologies Co.,Ltd.;Rare Disease Research Center,Chinese Academy of Medical Sciences;Takeda China;National Health Development and Research Center,National Health Commission of the People′s Republic of China;Peking Union Medical College Hospital,Chinese Academy of Medical Sciences;

Abstract: Rare diseases are diseases with a low incidence and prevalence. In 2018,China published its first list of 121 rare diseases,which set up the foundation for future rare diseases policy formulation. Health technology assessment（HTA）is a policy analysis to evaluate the cost-effectiveness,patient reported outcomes,and societal benefits of innovative technology from multiple dimensions. Due to the nature of rare diseases,there are a number of challenges to apply HTA in rare disease area,including the very low disease prevalence,high heterogenicity of the diseases and a relatively higher cost of innovative interventions. Hence,many of the developed countries are applying special methods and procedures for rare diseases HTA,such as the acceptance of lower-level evidence,a higher threshold of cost-effectiveness ratio,and complete exemption of HTA for orphan drugs. In China,the HTA system has not been formally established yet. Rare disease-related HTA is facing even more challenges. However,the publication of the first list of rare diseases is an important milestone to facilitate the establishment of an HTA process of rare disease interventions in China.
Keywords：

health technology assessment; rare disease; cost; effectiveness;

  • DOI：

    10.13220/j.cnki.jipr.2019.09.001

  • Series：

    (E) Medicine ＆ Public Health

  • Subject：

    Pharmaceutics

  • Classification Code：

    R95

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