Date of Award

January 2021

Document Type

Open Access Thesis

Degree Name

Master of Public Health (MPH)


School of Public Health

First Advisor

Robert Makuch

Second Advisor

Joshua Wallach


Rare disease definition varies among countries/regions. China has published The First National List of Rare Diseases, including 121 kinds of rare diseases in 2018. It was the first time that China has defined rare diseases officially. China has also started releasing a range of encouraging regulations regarding orphan drug development and approval in recent years. Nevertheless, the FDA has introduced Orphan Drug Act in the 1980s, Japan has launched a government-led support initiative in the 1990s, and the European Parliament has adopted the Orphan Regulation in 2000. Among the 121 rare diseases, 58 of them have approved treatments in the US, the EU, or Japan, with 124 drugs in total. Until 2020, only 68 drugs have been approved in China, meaning many remaining rare diseases have no treatment yet in China, and there is still a long way to go for orphan drug approval. Also, as orphan drugs usually have dramatically high prices, being listed in the National Reimbursement Drug List (NRDL) would help decrease the economic burden for patients significantly. However, among the 68 approved drugs, only 38 have been included in the NRDL. This study showed that successful orphan drug approval in China is associated with two significant characteristics, registration status in the National Rare Diseases Registry System, and the sponsor size. Orphan drug reimbursement is associated with orphan drug substances. The information may help drug developers and regulators in future orphan drug market access programs in China.


This is an Open Access Thesis.

Open Access

This Article is Open Access