Plasma protein products for the treatment of rare diseases / 国际药学研究杂志

ABSTRACT

Plasma protein products are also called blood products. At present, more than 30 kinds of blood prod-ucts have been issued worldwide. Because of their particular source of raw materials as well as the safety, reliability and irreplaceability in the treatment of some diseases, many blood products are in short supply in the market. Rare diseases are usually caused by genetic deficiency, autoimmunity, allergy and so on, which imposes a huge burden on patients and their families. Orphan drugs generally include the products of diagnostic reagents, vaccines, medicines and medical devices used for the prevention, diagnosis and treatment of rare diseases and rare conditions. Since the adoption of the Orphan Drug Act(ODA)in the United States in 1983, many countries or regions have passed the relevant laws aiming to encourage enterprises to develop orphan drugs by means of the research funding, tax relief, priority approval and market monopoly. The pass of ODA has greatly promoted the development of related blood product enterprises. This paper sum-marizes the plasma protein products approved by the pharmaceutical regulatory departments of the United States, the Eu-ropean Union, Japan and Australia as orphans, including four categories and 18 varieties, accounting for 56% of all plas-ma protein products in the world. At present, China has not yet enacted legislation to define rare diseases and orphan drugs, which has restricted to a certain extent the development of domestic blood product enterprises. The policy divi-dends from ODA will help the development of domestic blood product enterprises and narrow the gap between the domes-tic enterprises and the international blood product giants.