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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.
李文燕的女儿在7个月大的时候被诊断患有一种罕见病，孩子的心脏可能在几个月内衰竭，但李文燕根本负担不起女儿的治疗费用。于是，她转向一种新的筹钱方式：在网上求助陌生人捐款。
“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.
李文燕在一家筹款网站上发帖写道：“我叫李文燕……世代农民，全家年收入三万。”她还写道，后续治疗费一个月需要12万元人民币，“对于已经债台高驻的我来说，已经没有办法了”，后面还加了两个双手合十祈祷的表情符号。
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.
这场筹款活动吸引了1800多笔捐款，筹集到了近3万元人民币——只够法国赛诺菲(Sanofi)生产的药品Myozyme一个疗程的费用。
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.
但是，鉴于这个平台还有其他成千上万渴望得到捐款的患者，最终筹款总额并未达到30万元人民币的目标。李文燕说：“第二轮筹款没有及时跟上。”
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.
李文燕的女儿雨馨在9个月大的时候死于庞贝氏症(Pompe disease)。这是一种遗传疾病，会引发糖分子在包括心脏在内的肌肉组织中贮积。
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.
中国有世界最庞大的罕见病患者群体，据估计总人数在1500万至2000万之间。过去10年，中国推出了一些药物的国家医保，但绝大多数罕见病用药不在报销范围。即便被纳入了医保药品目录，也不能保证地方医保能给它们报销。2015年，治疗庞贝氏症的药物Myozyme获批在中国使用，但李文燕无法为这种药获得任何国家补偿。
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.
2015年对罕见病患者进行的一项调查显示，在中国，罕见病患者平均一年的治疗费用是他们个人年收入的三倍，是他们家庭收入的近两倍。
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.
代表庞贝氏症患者的一个协会称，中国约有3.5万名庞贝氏症患者。其中约80%是成年人，他们每年的治疗费用约为300万元人民币，相比之下中国的人均收入为每年7万元人民币左右。在中国大部分地区，患者不可能获得政府医保对庞贝氏症药物的部分报销。
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.”
吉林省的齐崇哲说，他的孩子在7个月大的时候出现了庞贝氏症的症状，但当地医生最初以为只是感冒。在北京一家医院进行基因检测之后，才确诊了庞贝氏症。齐崇哲说，住院8次后，治疗费用花掉了他一辈子的积蓄——10多万元人民币。他每月的工资只有4000元人民币。“过完年，我们要把房子卖了，但这只够坚持半年。之后如果还没有医保报销，我们只能等死。”
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.
赛诺菲新兴市场罕见病部门负责人梅德海特?埃尔-巴埃里(Medhat El-Bialy)承认，医保范围有限是获取药物的一个主要障碍。他表示，在美国可获得的罕见病药物中，只有38%在中国市场可获得，“还有很大追赶空间”。
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.
王成4岁的女儿患有高雪氏症(Gaucher's disease)，一种罕见的遗传性疾病，可能导致脂肪物质在某些器官中堆积。他估计，在中国约400名高雪氏症患者中，有138人得到了慈善项目的帮助，而至少有100名患者得不到任何药物。治疗这种疾病的努力令慈善预算面临严重压力。据上海市罕见病防治基金会理事长李定国介绍，仅在上海治疗6名高雪氏症患者，每月就需要该基金会负担约200万元人民币。
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.
能否享受医保还取决于户籍。周迎春一家刚刚花了30万元人民币为患有大疱性表皮松解症(epidermolysis bullosa)的15岁女儿做了骨髓移植手术，这种疾病会导致皮肤出现水疱并破裂。由于手术不是在户籍所在地进行的，这家人不得不自己承担所有费用。
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.
“罕见病发展中心”(CORD)创始人黄如方表示，覆盖范围更广的政府医疗保险是罕见病患者最迫切的需要。“总体而言，省级医保机构做得不好。”
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.
但政策制定者近期的一些举措给罕见病患者带来了一些希望。中国政府去年宣布，将拟定首份罕见病官方目录，该目录可能为扩大报销范围打开大门。
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.
他还表示，一些较富裕的省份已在本省的医保报销目录中新增罕见病用药，而中国国家食品药品监督管理总局(CFDA)已承诺加快审批新药的进程。“我认为国家食药监局的措施鼓舞了药物研发公司。”黄如方说。
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.
辉瑞(Pfizer)罕见病药物中国营销主管克里什纳?帕德马纳班(Krishna Padmanabhan)表示，中国国家食药监总局的举措“为辉瑞及其他公司创造了机会，让它们为中国患者带来创新药物”。他形容中国存在“巨大的未满足医疗需求”。
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.
中国企业也发现了机遇。总部位于北京的初创公司德益阳光生物技术(Prosit Sole Biotechnology)正在研发一种治疗“免疫功能低下患者诺如病毒胃肠炎”(norovirus gastroenteritis in immunocompromised patients)的药物。据该公司估计，该药在美国的市场规模可能达到每年5亿美元，尽管中国的市场规模只有美国一半。
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.”
该公司计划在美国进行试验，这是其海外雄心的一个标志。“在中国，让医疗保险覆盖罕用药非常困难，而在美国和欧洲，保险制度更为成熟。这就是我们专注海外市场的原因，”德益阳光生物技术的创始人刘宏宇说，“解决中国的药费问题比解决审批问题要困难得多。”
Healthcare: how it works in China
医保在中国如何运作
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.
直至上世纪80年代，基本医疗保障对中国公民都是免费的，此后，公立医院开始为治病收费。大约10年前，中国开始推出政府补贴的全民医疗保险。缴费额度取决于收入，这意味着低收入者交得少。如今，95%的人口得到了由国家负担一部分医疗费用的医保计划的覆盖，相比之下2012年这一比例为30%。
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.
然而，虽然中国的国家医保覆盖面很广，但深度仍然不大。除了几百种被认定为基本的药物外，只有国家医保药品目录上的药品可以报销，该目录在去年更新后包括大约2500种药物。
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.
许多救命的药物，包括那些治疗罕见病的药物，都不在这个目录上——而在2015年中国取消大多数药品的官方价格上限后，它们的价格由制造商设定。
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.
各省根据各自财力，决定国家医保药品目录上多数药物的报销水平。其结果是，国家报销的比例可能低至30%。在中国最贫困的省份和农村地区，患者通常需要自行支付更大比例的医疗费用，尽管他们的收入普遍更低。
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.
中国一家罕见病慈善机构表示，Myozyme一年的费用通常为200万元人民币（合31.5万美元）。大多数省份都不会对这种药的费用给予任何补偿，这意味着患者或他们的家人将不得不自行承担全部费用。
