中國罕見病患者期盼國家醫保

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個月大的時候被診斷患有一種罕見病，孩子的心臟可能在幾個月內衰竭，但李文燕根本負擔不起女兒的治療費用。於是，她轉向一種新的籌錢方式：在網上求助陌生人捐款。

“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站上發帖寫道：“我叫李文燕……世代農民，全家年收入三萬。”她還寫道，後續治療費一個月需要12萬元人民幣，“對於已經債臺高駐的我來說，已經沒有辦法了”，後面還加了兩個雙手合十祈禱的表情符號。

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.

這場籌款活動吸引了1800多筆捐款，籌集到了近3萬元人民幣——只夠法國賽諾菲(Sanofi)生產的藥品Myozyme一個療程的費用。

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，鑑於這個平臺還有其他成千上萬渴望得到捐款的患者，最終籌款總額並未達到30萬元人民幣的目標。李文燕說：“第二輪籌款沒有及時跟上。”

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個月大的時候死於龐貝氏症(Pompe disease)。這是一種遺傳疾病，會引發糖分子在包括心臟在內的肌肉組織中貯積。

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.

中國有世界最龐大的罕見病患者羣體，據估計總人數在1500萬至2000萬之間。過去10年，中國推出了一些藥物的國家醫保，但絕大多數罕見病用藥不在報銷範圍。即便被納入了醫保藥品目錄，也不能保證地方醫保能給它們報銷。2015年，治療龐貝氏症的藥物Myozyme獲批在中國使用，但李文燕無法爲這種藥獲得任何國家補償。

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年對罕見病患者進行的一項調查顯示，在中國，罕見病患者平均一年的治療費用是他們個人年收入的三倍，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.

代表龐貝氏症患者的一個協會稱，中國約有3.5萬名龐貝氏症患者。其中約80%是成年人，他們每年的治療費用約爲300萬元人民幣，相比之下中國的人均收入爲每年7萬元人民幣左右。在中國大部分地區，患者不可能獲得政府醫保對龐貝氏症藥物的部分報銷。

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，他的孩子在7個月大的時候出現了龐貝氏症的症狀，但當地醫生最初以爲只是感冒。在北京一家醫院進行基因檢測之後，才確診了龐貝氏症。齊崇哲說，住院8次後，治療費用花掉了他一輩子的積蓄——10多萬元人民幣。他每月的工資只有4000元人民幣。“過完年，我們要把房子賣了，但這隻夠堅持半年。之後如果還沒有醫保報銷，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.

賽諾菲新興市場罕見病部門負責人梅德海特?埃爾-巴埃裏(Medhat El-Bialy)承認，醫保範圍有限是獲取藥物的一個主要障礙。他表示，在美國可獲得的罕見病藥物中，只有38%在中國市場可獲得，“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症(Gaucher's disease)，一種罕見的遺傳性疾病，可能導致脂肪物質在某些器官中堆積。他估計，在中國約400名高雪氏症患者中，有138人得到了慈善項目的幫助，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算面臨嚴重壓力。據上海市罕見病防治基金會理事長李定國介紹，僅在上海治療6名高雪氏症患者，每月就需要該基金會負擔約200萬元人民幣。

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.

能否享受醫保還取決於戶籍。周迎春一家剛剛花了30萬元人民幣爲患有大皰性表皮鬆解症(epidermolysis bullosa)的15歲女兒做了骨髓移植手術，這種疾病會導致皮膚出現水皰並破裂。由於手術不是在戶籍所在地進行的，這家人不得不自己承擔所有費用。

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.

“罕見病發展中心”(CORD)創始人黃如方表示，覆蓋範圍更廣的政府醫療保險是罕見病患者最迫切的需要。“總體而言，省級醫保機構做得不好。”

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病患者帶來了一些希望。中國政府去年宣佈，將擬定首份罕見病官方目錄，該目錄可能爲擴大報銷範圍打開大門。

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保報銷目錄中新增罕見病用藥，而中國國家食品藥品監督管理總局(CFDA)已承諾加快審批新藥的進程。“我認爲國家食藥監局的措施鼓舞了藥物研發公司。”黃如方說。

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)罕見病藥物中國營銷主管克里什納?帕德馬納班(Krishna Padmanabhan)表示，中國國家食藥監總局的舉措“爲輝瑞及其他公司創造了機會，讓它們爲中國患者帶來創新藥物”。他形容中國存在“巨大的未滿足醫療需求”。

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.

中國企業也發現了機遇。總部位於北京的初創公司德益陽光生物技術(Prosit Sole Biotechnology)正在研發一種治療“免疫功能低下患者諾如病毒胃腸炎”(norovirus gastroenteritis in immunocompromised patients)的藥物。據該公司估計，該藥在美國的市場規模可能達到每年5億美元，儘管中國的市場規模只有美國一半。

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.”

該公司計劃在美國進行試驗，這是其海外雄心的一個標誌。“在中國，讓醫療保險覆蓋罕用藥非常困難，而在美國和歐洲，保險制度更爲成熟。這就是我們專注海外市場的原因，”德益陽光生物技術的創始人劉宏宇說，“解決中國的藥費問題比解決審批問題要困難得多。”

Healthcare: how it works in China

醫保在中國如何運作

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80年代，基本醫療保障對中國公民都是免費的，此後，公立醫院開始爲治病收費。大約10年前，中國開始推出政府補貼的全民醫療保險。繳費額度取決於收入，這意味着低收入者交得少。如今，95%的人口得到了由國家負擔一部分醫療費用的醫保計劃的覆蓋，相比之下2012年這一比例爲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，雖然中國的國家醫保覆蓋面很廣，但深度仍然不大。除了幾百種被認定爲基本的藥物外，只有國家醫保藥品目錄上的藥品可以報銷，該目錄在去年更新後包括大約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，包括那些治療罕見病的藥物，都不在這個目錄上——而在2015年中國取消大多數藥品的官方價格上限後，它們的價格由製造商設定。

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.

各省根據各自財力，決定國家醫保藥品目錄上多數藥物的報銷水平。其結果是，國家報銷的比例可能低至30%。在中國最貧困的省份和農村地區，患者通常需要自行支付更大比例的醫療費用，儘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.

中國一家罕見病慈善機構表示，Myozyme一年的費用通常爲200萬元人民幣（合31.5萬美元）。大多數省份都不會對這種藥的費用給予任何補償，這意味着患者或他們的家人將不得不自行承擔全部費用。