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

Considerations on the health techology assessment for rare diseases and orphan drugs in ChinaChinese Full TextEnglish Full Text (MT)

LI Hong;WANG Xiao-yan;YUE Xiao-meng;WU Jiu-hong;College of Pharmacy,University of Cincinnati,Digital China Health Co.,Ltd;Digital China Health Co.,Ltd;Department of Pharmacy,Special Medical Center,Strategic Support Force of PLA;

Abstract: There are about 7000 kinds of rare diseases in the world;the estimated number of patients with rare diseases is 4.3% of the world population and it is therefore a serious public health issue. For rare diseases,two questions are critical:（1）research and development for innovative treatments/medicines（orphan drugs）;（2）access to launched orphan drugs in terms of availability and affordability. Many countries,including China,have paid significant attentions to the first question in terms of the regulatory review policy and financial incentive. As to the second question,there is a great difference in access to orphan drugs in different countries and regions due to many factors,including the different medical care systems and high drug expenses. Recently,health technology assessment（HTA）has played an important role in evaluating clinical and economic value of innovative medicines,including orphan drugs. This paper discusses how to do HTA evaluation on orphan drugs in China with several policy suggestions.
Keywords：

rare disease; orphan drug; health technology assessment（HTA）; medicine access strategies;

  • DOI：

    10.13220/j.cnki.jipr.2019.09.004

  • Series：

    (E) Medicine ＆ Public Health

  • Subject：

    Pharmaceutics

  • Classification Code：

    R95

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