中國(guó)罕見(jiàn)病患者期盼國(guó)家醫(yī)保

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.

李文燕的女兒在7個(gè)月大的時(shí)候被診斷患有一種罕見(jiàn)病，孩子的心臟可能在幾個(gè)月內(nèi)衰竭，但李文燕根本負(fù)擔(dān)不起女兒的治療費(fèi)用。于是，她轉(zhuǎn)向一種新的籌錢(qián)方式：在網(wǎng)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.

李文燕在一家籌款網(wǎng)站上發(fā)帖寫(xiě)道：“我叫李文燕……世代農(nóng)民，全家年收入三萬(wàn)。”她還寫(xiě)道，后續(xù)治療費(fèi)一個(gè)月需要12萬(wàn)元人民幣，“對(duì)于已經(jīng)債臺(tái)高駐的我來(lái)說(shuō)，已經(jīng)沒(méi)有辦法了”，后面還加了兩個(gè)雙手合十祈禱的表情符號(hào)。

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.

這場(chǎng)籌款活動(dòng)吸引了1800多筆捐款，籌集到了近3萬(wàn)元人民幣——只夠法國(guó)賽諾菲(Sanofi)生產(chǎn)的藥品Myozyme一個(gè)療程的費(fèi)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，鑒于這個(gè)平臺(tái)還有其他成千上萬(wàn)渴望得到捐款的患者，最終籌款總額并未達(dá)到30萬(wàn)元人民幣的目標(biāo)。李文燕說(shuō)：“第二輪籌款沒(méi)有及時(shí)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.

李文燕的女兒雨馨在9個(gè)月大的時(shí)候死于龐貝氏癥(Pompe disease)。這是一種遺傳疾病，會(huì)引發(fā)糖分子在包括心臟在內(nèi)的肌肉組織中貯積。

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.

中國(guó)有世界最龐大的罕見(jiàn)病患者群體，據(jù)估計(jì)總?cè)藬?shù)在1500萬(wàn)至2000萬(wàn)之間。過(guò)去10年，中國(guó)推出了一些藥物的國(guó)家醫(yī)保，但絕大多數(shù)罕見(jiàn)病用藥不在報(bào)銷(xiāo)范圍。即便被納入了醫(yī)保藥品目錄，也不能保證地方醫(yī)保能給它們報(bào)銷(xiāo)。2015年，治療龐貝氏癥的藥物Myozyme獲批在中國(guó)使用，但李文燕無(wú)法為這種藥獲得任何國(guó)家補(bǔ)償。

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年對(duì)罕見(jiàn)病患者進(jìn)行的一項(xiàng)調(diào)查顯示，在中國(guó)，罕見(jiàn)病患者平均一年的治療費(fèi)用是他們個(gè)人年收入的三倍，是他們家庭收入的近兩倍。

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.

代表龐貝氏癥患者的一個(gè)協(xié)會(huì)稱(chēng)，中國(guó)約有3.5萬(wàn)名龐貝氏癥患者。其中約80%是成年人，他們每年的治療費(fèi)用約為300萬(wàn)元人民幣，相比之下中國(guó)的人均收入為每年7萬(wàn)元人民幣左右。在中國(guó)大部分地區(qū)，患者不可能獲得政府醫(yī)保對(duì)龐貝氏癥藥物的部分報(bào)銷(xiāo)。

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.”

吉林省的齊崇哲說(shuō)，他的孩子在7個(gè)月大的時(shí)候出現(xiàn)了龐貝氏癥的癥狀，但當(dāng)?shù)蒯t(yī)生最初以為只是感冒。在北京一家醫(yī)院進(jìn)行基因檢測(cè)之后，才確診了龐貝氏癥。齊崇哲說(shuō)，住院8次后，治療費(fèi)用花掉了他一輩子的積蓄——10多萬(wàn)元人民幣。他每月的工資只有4000元人民幣。“過(guò)完年，我們要把房子賣(mài)了，但這只夠堅(jiān)持半年。之后如果還沒(méi)有醫(yī)保報(bào)銷(xiāo)，我們只能等死。”

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.

賽諾菲新興市場(chǎng)罕見(jiàn)病部門(mén)負(fù)責(zé)人梅德海特•埃爾-巴埃里(Medhat El-Bialy)承認(rèn)，醫(yī)保范圍有限是獲取藥物的一個(gè)主要障礙。他表示，在美國(guó)可獲得的罕見(jiàn)病藥物中，只有38%在中國(guó)市場(chǎng)可獲得，“還有很大追趕空間”。

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歲的女兒患有高雪氏癥(Gaucher's disease)，一種罕見(jiàn)的遺傳性疾病，可能導(dǎo)致脂肪物質(zhì)在某些器官中堆積。他估計(jì)，在中國(guó)約400名高雪氏癥患者中，有138人得到了慈善項(xiàng)目的幫助，而至少有100名患者得不到任何藥物。治療這種疾病的努力令慈善預(yù)算面臨嚴(yán)重壓力。據(jù)上海市罕見(jiàn)病防治基金會(huì)理事長(zhǎng)李定國(guó)介紹，僅在上海治療6名高雪氏癥患者，每月就需要該基金會(huì)負(fù)擔(dān)約200萬(wàn)元人民幣。

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.

能否享受醫(yī)保還取決于戶(hù)籍。周迎春一家剛剛花了30萬(wàn)元人民幣為患有大皰性表皮松解癥(epidermolysis bullosa)的15歲女兒做了骨髓移植手術(shù)，這種疾病會(huì)導(dǎo)致皮膚出現(xiàn)水皰并破裂。由于手術(shù)不是在戶(hù)籍所在地進(jìn)行的，這家人不得不自己承擔(dān)所有費(fèi)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.

“罕見(jiàn)病發(fā)展中心”(CORD)創(chuàng)始人黃如方表示，覆蓋范圍更廣的政府醫(yī)療保險(xiǎn)是罕見(jiàn)病患者最迫切的需要。“總體而言，省級(jí)醫(yī)保機(jī)構(gòu)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舉措給罕見(jiàn)病患者帶來(lái)了一些希望。中國(guó)政府去年宣布，將擬定首份罕見(jiàn)病官方目錄，該目錄可能為擴(kuò)大報(bào)銷(xiāo)范圍打開(kāi)大門(mén)。

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.

他還表示，一些較富裕的省份已在本省的醫(yī)保報(bào)銷(xiāo)目錄中新增罕見(jiàn)病用藥，而中國(guó)國(guó)家食品藥品監(jiān)督管理總局(CFDA)已承諾加快審批新藥的進(jìn)程。“我認(rèn)為國(guó)家食藥監(jiān)局的措施鼓舞了藥物研發(fā)公司。”黃如方說(shuō)。

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.

輝瑞(Pfizer)罕見(jiàn)病藥物中國(guó)營(yíng)銷(xiāo)主管克里什納•帕德馬納班(Krishna Padmanabhan)表示，中國(guó)國(guó)家食藥監(jiān)總局的舉措“為輝瑞及其他公司創(chuàng)造了機(jī)會(huì)，讓它們?yōu)橹袊?guó)患者帶來(lái)創(chuàng)新藥物”。他形容中國(guó)存在“巨大的未滿(mǎn)足醫(yī)療需求”。

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.

中國(guó)企業(yè)也發(fā)現(xiàn)了機(jī)遇?？偛课挥诒本┑某鮿?chuàng)公司德益陽(yáng)光生物技術(shù)(Prosit Sole Biotechnology)正在研發(fā)一種治療“免疫功能低下患者諾如病毒胃腸炎”(norovirus gastroenteritis in immunocompromised patients)的藥物。據(jù)該公司估計(jì)，該藥在美國(guó)的市場(chǎng)規(guī)?？赡苓_(dá)到每年5億美元，盡管中國(guó)的市場(chǎng)規(guī)模只有美國(guó)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.”

該公司計(jì)劃在美國(guó)進(jìn)行試驗(yàn)，這是其海外雄心的一個(gè)標(biāo)志。“在中國(guó)，讓醫(yī)療保險(xiǎn)覆蓋罕用藥非常困難，而在美國(guó)和歐洲，保險(xiǎn)制度更為成熟。這就是我們專(zhuān)注海外市場(chǎng)的原因，”德益陽(yáng)光生物技術(shù)的創(chuàng)始人劉宏宇說(shuō)，“解決中國(guó)的藥費(fèi)問(wèn)題比解決審批問(wèn)題要困難得多。”

Healthcare: how it works in China

醫(yī)保在中國(guó)如何運(yùn)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.

直至上世紀(jì)80年代，基本醫(yī)療保障對(duì)中國(guó)公民都是免費(fèi)的，此后，公立醫(yī)院開(kāi)始為治病收費(fèi)。大約10年前，中國(guó)開(kāi)始推出政府補(bǔ)貼的全民醫(yī)療保險(xiǎn)。繳費(fèi)額度取決于收入，這意味著低收入者交得少。如今，95%的人口得到了由國(guó)家負(fù)擔(dān)一部分醫(yī)療費(fèi)用的醫(yī)保計(jì)劃的覆蓋，相比之下2012年這一比例為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，雖然中國(guó)的國(guó)家醫(yī)保覆蓋面很廣，但深度仍然不大。除了幾百種被認(rèn)定為基本的藥物外，只有國(guó)家醫(yī)保藥品目錄上的藥品可以報(bào)銷(xiāo)，該目錄在去年更新后包括大約2500種藥物。

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.

許多救命的藥物，包括那些治療罕見(jiàn)病的藥物，都不在這個(gè)目錄上——而在2015年中國(guó)取消大多數(shù)藥品的官方價(jià)格上限后，它們的價(jià)格由制造商設(shè)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.

各省根據(jù)各自財(cái)力，決定國(guó)家醫(yī)保藥品目錄上多數(shù)藥物的報(bào)銷(xiāo)水平。其結(jié)果是，國(guó)家報(bào)銷(xiāo)的比例可能低至30%。在中國(guó)最貧困的省份和農(nóng)村地區(qū)，患者通常需要自行支付更大比例的醫(yī)療費(fèi)用，盡管他們的收入普遍更低。

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.

中國(guó)一家罕見(jiàn)病慈善機(jī)構(gòu)表示，Myozyme一年的費(fèi)用通常為200萬(wàn)元人民幣(合31.5萬(wàn)美元)。大多數(shù)省份都不會(huì)對(duì)這種藥的費(fèi)用給予任何補(bǔ)償，這意味著患者或他們的家人將不得不自行承擔(dān)全部費(fèi)用。