Medical experts estimate that about 10 million Chinese patients have some type of rare disease. Specific legislation on orphan drugs is still lacking in China. However, since 2009, a fast approval process for drugs related to some rare diseases has been in effect. A separate regulation specifies that drugs for rare diseases are eligible for reduced clinical trial requirements.
For the past several years, Shanghai has covered the treatment of 12 identified rare diseases. Coverage is approximately $32,200 per year and per patient. The Shanghai Rare Disease Society, founded in early 2011, also works to promote legislation, research and insurance coverage for rare diseases.
Other local and provincial governments are showing more interest in rare diseases, too. The Peking Union Medical College Hospital set up a foundation in 2010 to support lymphangioleiomyomatosis (LAM) and tuberous sclerosis complex (TSC) patients. In October 2011, Shandong Province founded a Rare Disease Association. Qingdao, in Shandong, approved a 2012 proposal to cover a capped amount of the treatment fees for all diseases, including rare diseases. At least a portion of hemophilia treatment is covered in many provinces as well.
There is also an increasing number of patient advocacy groups, such the China Dolls Association, Hemophilia Association of China, LAM China and the phenylketonuria (PKU) Union. The Rare Disease Office of China Charity Foundation was established in 2008. The Chinese Organization for Rare Disorders has brought 20,000 people with 33 different rare diseases together on the internet. In 2013, 17 medical institutions from 13 different provinces established the China Rare Diseases Prevention and Treatment Alliance. The organization collects data on rare diseases in China, runs epidemiological studies and works towards better treatments.
China lacks doctors experienced in identifying rare diseases. Approximately one-third of those with rare diseases need to see 4 or more doctors before receiving a correct diagnosis, while almost half remain diagnosed incorrectly. Three-fourths of rare disease patients are unable to receive regular, modern treatment.