China Rare Disease List Likely to Spur Development of Orphan Drugs

In May, the Chinese government published its first national list of rare diseases, a welcome development for foreign pharmaceutical companies eager to market treatments for their products there.

The list of 121 diseases identified as rare was published by five Chinese government agencies, the State Drug Administration and the National Health Commission among them. Of the diseases on the list, 110 can be treated with rare disease drugs already approved in the United States and the other Western countries.

The definition of rare diseases varies from country to country, though they are often genetic in nature and costly to treat or control. The World Health Organization defines diseases found in between 0.65% and 1% of the population as rare. Researchers estimate there are at least 16 million Chinese with rare diseases, though data to support specific numbers is scarce.

The publication of the rare disease list was long anticipated as part of a major overhaul of the Chinese healthcare system by the current government. Prior to publication, there was no rare disease list or approved orphan drugs.