Expensive burden: there is scant health coverage for diseases such as epidermolysis bullosa

Li Wenyan was unable to afford medical bills for her seven-month-old daughter’s rare disease, which could cause her heart to fail within months. So she turned to a new source of funding: asking strangers for money on the internet.

“I come from generations of peasants and my family has an annual income of Rmb30,000 [$4,730],” Ms Li wrote in a post on a crowdfunding site. She added that treatment costs were Rmb120,000 a month and with “debts piled high I have no other option,” alongside two emojis showing hands clasped in prayer.

The campaign attracted more than 1,800 donations, raising almost Rmb30,000 — enough for one course of treatment with the drug Myozyme, manufactured by French company Sanofi.

But with thousands of other people vying for money on the platform, the final amount fell short of her Rmb300,000 target. “The second round of fundraising did not come in time,” Ms Li said.

Ms Li’s daughter, Yuxin, was nine months old when she died of Pompe disease, a genetic disorder which causes sugar molecules to build up in muscle tissue including the heart.

A young sufferer of epidermolysis bullosa

China has the world’s largest population of people with rare diseases — numbering between 15m and 20m, according to estimates. China has rolled out state medical insurance for some drugs over the past decade but the vast majority of orphan drugs are not included. Even when they are, there is no guarantee that regional insurers will cover them. Myozyme, a treatment for Pompe, was approved for use in China in 2015 but Ms Li was unable to gain any state reimbursement for the drug.

People trying to pay for a treatment for a rare disease in China face bills that are on average three times their annual individual incomes and nearly two times higher than their household incomes, according to a survey of rare disease sufferers carried out in 2015.

There are about 35,000 Pompe patients in China, according to an association representing sufferers. About 80 per cent of them are adults who face annual medical costs of around Rmb3m compared with annual average incomes in China of about Rmb70,000 a year. It is impossible for sufferers in most parts of China to obtain government insurance contributions for Pompe drugs.

Ji Chongzhe, of Jilin province in the north-east, says his child developed Pompe symptoms at seven months, but local doctors initially thought it was a cold. Only after a genetic test at a hospital in Beijing was the disease identified. Eight hospital stays later, Mr Ji says he has spent his life savings — over Rmb100,000 — on treatment. He earns just Rmb4,000 a month. “After the New Year holiday we will sell our house, but that will only keep us going for half a year. After that if there’s still no medical coverage, we can only wait for death.”

Medhat El-Bialy, head of rare diseases in emerging markets at Sanofi, admits limited insurance is a key barrier to access. With only 38 per cent of orphan drugs available in the US on the market in China “there is catching up to do,” he says.

Wang Cheng’s four-year-old daughter suffers from Gaucher’s disease, a rare genetic disorder that can cause a build-up of fatty substances in certain organs. He estimates that of the approximately 400 people in the country with the condition, 138 have received help from charity programmes while at least 100 patients have no access to medicine at all. Efforts to treat the disease severely strain charity budgets. Treating just six Gaucher’s disease patients in Shanghai costs the Shanghai Rare Disease Prevention and Treatment Fund about Rmb2m a month, according to its chairman Li Dingguo.

Access to healthcare can also depend on residency. Zhou Yingchun’s family has just paid Rmb300,000 for a bone-marrow transplant for their 15-year-old daughter who suffers from epidermolysis bullosa — a condition which causes the skin to blister and tear. As the operation was performed outside the family’s official place of residence, the family had to pay the entire cost themselves.

Huang Rufang, founder of the Chinese Organisation for Rare Disorders, says wider government insurance was the most urgent need for rare disease patients. “Overall the provincial insurers do not do a good job,” he says.

But recent moves by policymakers give some hope. Beijing last year announced it would draw up its first official list of rare diseases which could open the door for wider cost support.

Some wealthier provinces have already added rare disease drugs to their reimbursement lists, he adds, while the China Food and Drug Administration, the top pharmaceutical administrator, has vowed to speed up the process for approving new treatments. “I think the CFDA’s measures have encouraged drug developers,” Mr Huang says.

The drug administrator’s move “creates an opportunity for Pfizer and for other companies to bring innovative drugs to Chinese patients”, says Krishna Padmanabhan, head of rare disease marketing in China for the US company, describing a “huge unmet medical need” in the country.

Chinese companies are also spotting the opportunities. Beijing-based start-up Prosit Sole Biotechnology is developing a treatment for norovirus gastroenteritis in immunocompromised patients, a market it estimates could be worth $500m per year in the US although only half of that amount in China.

The group intends to run trials in the US, a sign of its overseas ambitions. “Getting insurance coverage in China for orphan drugs is very tough, whereas the system in the US and Europe is more mature. That’s why we are focusing on overseas markets,” says Prosit’s founder Liu Hongyu. “Solving the payment problem in China is much tougher than solving the approvals problem.”

Healthcare: how it works in China

Basic healthcare coverage was free for Chinese citizens until the 1980s, after which state-run hospitals began to charge for treatments. China began rolling out universal state-subsidised health insurance around a decade ago. Contributions are means-tested, meaning lower earners pay less. Now, 95 per cent of the population is covered by schemes under which the state will fund part of the cost of medical treatment, compared with 30 per cent in 2012.

However, while state medical coverage in China is broad, it remains shallow. Apart from a few hundred drugs deemed essential, the only treatments eligible for state co-payment are those named on a national drug reimbursement list, which was updated last year to include about 2,500 drugs.

Many life-saving drugs, including those most used to treat rare diseases, are not on this list — and their prices are set by manufacturers following the abolition of official price caps for most drugs in 2015.

Provinces determine the level at which most drugs on the national list are reimbursed, depending on their resources. As a result, the proportion of costs paid by the state can be as low as 30 per cent. Patients generally pay a larger proportion of their medical bills in China’s poorest provinces and in the countryside, even though incomes are also generally lower.

A rare disease charity in China says the typical annual cost of Myozyme, which is used to treat Pompe disease, is Rmb2m ($315,000). Most provinces will not contribute towards the cost of the drug, which means patients or their families will have to fund the entire cost themselves.

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