Newly Established List of Rare Diseases in China May Hasten Orphan Drug Approvals

China is readying its first national list of rare diseases as part of a larger effort to improve diagnosis and accelerate drug approvals for these conditions.

China has the largest population in the world, which makes it likely that it also has the largest incidence of rare diseases in the world, but the prevalence of these conditions is largely unknown. Although it is estimated that nearly 16 million Chinese citizens have rare diseases, the actual number may be much higher – with many of the conditions going undiagnosed, according to experts who spoke with Reuters. Patients also have little financial support because public insurance policies do not offer reimbursement for treating these costly diseases. Although China recently extended its national reimbursable drug list for the first time since 2009, the list mainly contains “essential medicines” rather than options for treating rare diseases.

Compiling a list of rare diseases (those that affect <200,000 individuals) could lead to policies that help accelerate the drug-approval process (similar to the effect of the Orphan Drug Act of 1983 in the United States, which led to the approval of more than 600 “orphan drugs”).

“Here in China, the state has good intentions, but there is no detailed regulation, no preferential policies, and no tax breaks [for orphan drugs],” Li Dingguo, chairman of the Shanghai Rare Disease Prevention and Treatment Fund, told Reuters. “Because China lacks these medical safeguards, we see lots of patients with rare diseases just waiting to die.”

The draft list includes more than 100 diseases and will serve as a basis for policy and the starting point for discussions about better diagnostics and more support for patients and families affected by these conditions. The Chinese health ministry is seeking public opinion on the list and may publish the list by the end of the year.

Source: Reuters, May 19, 2017.