Earlier this week, you may not have seen our reporting on the latest move by China for the rare community. So in case you missed it…
With the largest population in the entire world, China is most likely home to the largest population of rare disease patients. The estimation is 16 million, but many are undiagnosed. Not only is there an obvious need for a focus on rare disease there, but quite frankly it’s a missed piece of revenue. Drugs for treating rare diseases are incredibly expensive but perhaps, if there is a huge additional market for rare disease drugs, the cost of them may decrease for the whole world. If China puts an increased focus on treatment, ensures coverage by insurance companies, and ultimately begins to recognize the importance of rare disease healthcare, it could mean a big change for the country’s rare disease community.
And hopefully- that change is almost here.
China just announced that they are working to publish a national list of rare diseases.
So far, they have over 100 diseases documented. While the United States recognizes over 7,000 rare diseases, this is a starting point for the country. The hope is that the list will serve as a resource for government officials, and eventually stimulate policy change.
The reality is that most people currently living with a rare disease in China face one of two very unfortunate circumstances. They either are undiagnosed, because their medical system lacks study in the area of rare disease. Or, they are diagnosed but unable to receive treatment due to financial burdens. The thing is, because these diseases are rare, the drugs to treat them are often outrageously expensive (as I’m sure you know). And that’s for the ones that China actually puts on the market. Most patients are forced to turn to other means to obtain treatment because of inaccessibility to the drugs they need.
One such patient is Hu Yizhuo. He’s an 8-year-old boy fighting tuberous sclerosis complex.
The disease causes benign tumors to grow throughout the body and triggers frequent seizures. He needs Sabril daily (a drug made by Sanofi SA and Pfizer Inc’s Rapamune). The drug is not easily accessible in China due to cost and a long approval process. In order to keep their son alive, Hu’s parents have been smuggling the drug from Turkey, Taiwan, and Hong Kong, or getting it from doctors in China who prescribe it for off-label use.
They say it’s not a choice, but a necessity for their son’s survival.
However, some families aren’t willing or able to obtain treatment that way, and experts in the field understand the moral dilemma. Li Dingguo is chairman of the Shanghai Rare Disease Prevention and Treatment Fund. He says,
“Here in China, the state has good intentions but there is no detailed regulation, no preferential policies, and no tax breaks (for orphan drugs). Because China lacks these medical safeguards, we see lots of patients with rare diseases just waiting to die.”
But the upcoming list of rare diseases has brought newfound hope to Chinese citizens.
Similar to the Orphan Drug Act of 1983 in the United States, the Chinese anticipate that the creation of this list will lead to a higher rate of approval for various medicines. For the medical community, the list could also be a starting ground for analyzing Chinese specific rare diseases.
But most importantly, for families like Hu’s, the list means a greater potential for drug accessibility and insurance coverage for necessary treatments.
It’s the first of many needed steps to aid the rare disease community in China.
Take some time to read more about the development here. And if you’re a patient ready to contribute to change, the Chinese Organization for Rare Disorders (CORD) is looking for speakers to join their 2017 Rare Story Session at the International Conference on Rare Diseases & Orphan Drugs (ICORD). The event is on September 7-10, 2017 and they do have fellowships available to assist patients in traveling to Beijing.
