Characteristics of drug policy and pharmacoeconomics study on rare diseases / 国际药学研究杂志

ABSTRACT

At present, we are facing challenges in the development of rare disease drugs. The paper introduces the ethical theories of rare disease treatment, the characteristics of pharmacoeconomic evaluation on orphan drugs, the risk of reimbursement in medical insurance funds, the international experiences on the balance between the accessibility and cost containment of high-cost orphan drugs, the orphan drug list promulgated by Hong Kong SAR of China in 2019 and the status quo of orphan drugs in China. The author also analyzes the international practice from different perspec-tives, such as, the Orphan Drug Act(ODA)in the United States, the new concept of high specific technology introduced by National Institute Health and Clinical Excellence(NICE)in the UK, the retrospective review of pharmacoeconomics of orphan drugs by National Centre for Pharmacoeconomics(NCPE)in Ireland, the principles of orphan drug evaluation by TLV in Sweden, a special research program on the cost of orphan drugs in Turkey, and the four measures to control the cost of high-priced drugs in the Republic of Korea. The aim of this paper is to introduce international experiences for reference on the formulation of drug policies in rare diseases and development of related pharmacoeconomics in China.