1. World Health Organization (2006) The Selection and Use of Essential Medicines: Report of the WHO Expert Committee, 2005; (Including the 14th Model List of Essential Medicines); [WHO Expert Committee on the Selection and Use of Essential Medicines, Geneva, 7-11 March 2005]. World Health Organization, Geneva.
2. Orphan drugs: the regulatory environment
3. Rare Diseases, Drug Development, and AIDS: The Impact of the Orphan Drug Act
4. 马端, 李定国, 张学. 中国罕见病防治的机遇与挑战[J]. 中国循证儿科杂志, 2011, 6(2): 81-82.
5. Rare Disease Terminology and Definitions—A Systematic Global Review: Report of the ISPOR Rare Disease Special Interest Group