The Struggles of being a Rare Disease Patient in China

The State of Rare Diseases in China

The Illness Challenge Foundation (ICF) is an organization devoted to easing the burdens that rare disease patients living in China face. They accomplish this goal through social advocacy, industry support, and community service. The organization was founded in February of 2016, inspired by the amyotrophic lateral sclerosis (ALS) Ice Bucket Challenge.

Their 2018 report showcased the dire need for change to the way China supports rare disease patients. This report documented 20 million people living with rare diseases on the country’s mainland, with the average patient paying 80% of their medical bills out of pocket. No rare disease treatment is fully covered by the country. Haemarthrosis, a symptom of hemophilia, receives the most coverage (an 80% rebate). 60% of the patients in the ICF’s report were at first misdiagnosed or experienced a delay in their diagnosis. Even after diagnosis, 489 people were not able to receive treatment at all because of the extreme financial burden it posed to them.

The lack of China’s commitment to rare diseases is evident by the fact that they list only 121 rare diseases on their national register while approximately 7,000 are documented worldwide.

Incidence of “Common” Rare Diseases in China

Worldwide, multiple sclerosis is the most common rare disease; in fact, in many places it isn’t really rare at all. It affects approximately 90 people out of every 100,000. In China however, it’s one of the least common, affecting approximately 2 out of every 100,000.

Narcolepsy has a prevalence of 50 out of every 100,000 worldwide. In China, the rate of narcolepsy diagnoses increased in 2009 after the swine flu epidemic. There is now a presumed connection between the two diagnoses.

Primary biliary cholangitis affects 40 people out of every 100,000 globally. In China, it is more rare, however researchers in China are working to find new therapeutic options for the condition. They are investigating approaches such as traditional Chinese medicine as well as stem cell therapies.

An Absurd Financial Burden

Pan Longfei is a project manager for ICF. A rare disease patient himself, he can speak directly to the burden faced by patients. Pan was diagnosed at 23 with Kallmann syndrome after struggling to know what was wrong for so long. He was also found to have heart disease, epilepsy, and osteoporosis. He receives hormonal injections 10 times every single month, totaling around 1,000 yuan. He explains that his treatment is cheap compared to other rare conditions.

For instance, those with Niemann-Pick disease or Pompe disease may have to pay 2 million yuan each year for their treatment. Therapies for these conditions are not subsidized at all in China.

Other countries, such as Taiwan, have very different policies. For instance, in Taiwan, the government has agreed to cover any treatment for any rare condition if it is available in more than two other countries.

Let’s be clear though: the problem of expensive treatments for rare diseases is not a problem unique to China. There are many nations, including the United States, in which rare patients face an extreme financial burden for lifesaving therapies. It’s clear we need reform across much of the world.

Other Issues with the System

In addition to the extreme financial burden of some rare therapies, there is a severe lack of transparency regarding how to know if your therapy is eligible for reimbursement. Yin Yuanyue was diagnosed with muscular dystrophy at age 7, and she explains she still has no idea how to find out if her treatment is eligible. Her diagnosis and the side effects associated with it also prevented Yin from receiving an education growing up. How? Schools simply refused to take the wheelchair-bound child. At one point, schools in China were allowed to refuse disabled students from enrolling. Thankfully, that is an area where the country has since improved.

Another problem is that in China, new drugs are required to go through a 6 year clinical trial before they can be marketed. Sadly, those living with rapidly progressing diseases often cannot wait that long.

Unique Awareness Efforts

On the 2019 Rare Disease Day, the ICF presented a theatrical performance to contribute to awareness efforts. The play featured 15 people living with rare diseases including Pan and Yin. It was performed at the Tiangiao Performing Arts Centre in Beijing.

This performance focused on overcoming the struggles and adversities that can accompany a rare diagnosis. Thanks to efforts like these, awareness of rare diseases is slowly growing in the country, but it’s certainly not where it needs to be.

A Global Perspective

Across the globe we need to work to reform the policies affecting rare disease patients in order to ensure they are the most beneficial for the people they are supposed to be serving. This analysis of China is just a glimpse into the global issues present surrounding rare diseases.

You can read more about China’s policies surrounding rare diseases here.

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