Pharma DIY

Why rare disease drugs are hard to get in China


Watching 30 year-old Xu Wei in the lab that he built himself, it’s hard to believe he isn’t a trained scientist. In a white coat and sterile gloves, he uses an electronic pipette to measure out a chemical compound he will soon inject into his two year-old son.

But Xu is no chemist: until recently, he made his living as a trader of electrical goods on Alibaba’s Taobao platform, with no formal education since leaving high school.

Then in 2020 his son was diagnosed with a rare and incurable genetic disorder known as Menkes Syndrome, which affects how the body maintains a healthy level of copper.

The prognosis was death before the boy’s third birthday. There was no cure and mitigating treatments including copper histidine injections were not available in the local market.

“Before the pandemic people would go to places like Taiwan to buy histidine but travel restrictions meant that was no longer possible,” Xu told the Beijing News.

So he set out to make the stuff locally, initially by asking drug companies to synthesise the compound before going on to teach himself how to do it when they refused.

Copper histidine is a so-called ‘orphan drug’ – a medicine that is unprofitable for producers to make because it treats a very rare disease. Chinese pharmaceutical companies in particular have avoided orphan treatments, leading to a thriving black market in smuggled drugs from places such as India (see WiC417 for how this topic was explored in the hit film Dying to Survive).

There are over 600 separate treatments for rare diseases on the global market, but only 55 of these drugs are for sale in China, a report from 2019 found.

“Behind every rare disease patient is a family carrying a heavy burden,” quoted the head of the Beijing Medical Association’s Rare Disease Department as lamenting.

In Xu’s case the struggle to save his son started out by trying to persuade Chinese pharma firms to make copper histidine.

When they refused, saying it would take too long to get regulatory approvals, he downloaded academic papers on synthesising the compound and set about translating them from English.

Once he had done that, Xu approached a lab-for-hire firm in Shanghai and oversaw the production of a batch of histidine himself. Then he bought rabbits and injected them with the finished product, before testing it on himself and eventually giving it to his son.

Finally, as money began to run low, he asked the lab technicians to teach him the steps in making the compound so he could buy the equipment and start to produce histidine at his home in Kunming.

“I know it won’t cure him,” Xu told the Beijing News of the homemade remedy. “But maybe it will buy us some time for a cure to be found.”

Xu has also started work on a second copper compound that he hopes will improve his son’s brain function – although his dream of his son living a longer life rests more on the next generation of gene-based therapy.

Asked if he was worried about the legal implications of his work – others have been arrested for importing drugs from overseas – Xu simply pointed out this was his only option.

The story has racked up millions of hits on social media with most of the contributions praising Xu for his commitment and determination.

“What an amazing father,” marvelled one netizen.

Others bemoaned the shortcomings in medical care that have forced Xu to make the histidine for his son. “There is nothing heartwarming about not being able to simply buy the medicine your child needs,” one contributor wrote.

More expert commentary pointed out that Xu’s son would have had a better chance of long term survival if his condition had been diagnosed at birth, when copper supplements have the most impact as a treatment.

Knowledge of rare diseases is relatively weak in China’s health sector, however, and it can take years to get an accurate diagnosis on some of the rarest conditions.

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