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An expert group recommended on Tuesday that the Food and Drug Administration approve a powerful new drug to protect against heart attacks. If approved, it would be the first in a major new class of medicines in a generation that significantly lower levels of cholesterol, the leading cause of heart disease.
周二，一批专家建议美国食品与药品管理局（Food and Drug Administration，简称FDA）批准一种预防心肌梗死的强效新药。如果获得批准，该药物将成为一个新的重要药物类别中，最近二三十年获批的首例药物。此类药物能大幅降低胆固醇水平，而胆固醇是心脏病的主要诱因。
Dr. Joshua W. Knowles, a Stanford cardiologist, called the medicines “a triumph of the modern genetic revolution.”
斯坦福大学(Stanford)的心脏病学专家约书亚·W·诺尔斯(Joshua W. Knowles)博士称，这些药物是“现代基因革命的一次胜利”。
The idea for such drugs arose from genetic studies about a decade ago and has tantalized cardiologists ever since. Early results of clinical trials raised hopes that the therapies would be critical new additions to the treatment arsenal for those at risk of heart disease, the biggest killer of Americans.
研制这类药物的想法来自于约10年前的基因研究成果，此后一直令心脏病学专家可望而不可即。初步的临床试验结果带来了希望，这些疗法可能为心脏病高危人群的治疗方法提供新的重要补充。心脏病是美国人的头号杀手。
People who have taken them have seen their LDL cholesterol, the so-called bad cholesterol, plunge to remarkably low levels. But definitive evidence of the drugs’ effectiveness in reducing heart attacks and deaths will only come after large clinical trials are completed in 2017.
服用这些药物的人已经发现体内的低密度脂蛋白(LDL)胆固醇显著下降，LDL胆固醇也就是所谓的“坏胆固醇”。不过，这种药物降低心梗和死亡风险的确凿证据，要到2017年大规模临床试验完成后才可能出现。
The panel, in a 13 to 3 vote, recommended the approval of Sanofi and Regeneron Pharmaceuticals’ drug, alirocumab. On Wednesday, the committee will turn to Amgen’s drug, evolocumab. The F.D.A. usually follows the recommendations of its advisory panels, but not always. The agency says that if it approves the drugs based on their effects on cholesterol, the approval will not be rescinded even if trials now underway fail to show the drugs reduce the risk of heart attacks and deaths.
这个专家委员会以13比3的投票结果，建议批准赛诺菲(Sanofi)和瑞泽恩制药公司(Regeneron Pharmaceuticals)的药物alirocumab。周三，这个委员会将开始评估美国安进公司(Amgen)的药物evolocumab。FDA通常会听取咨询委员会的建议，但也并非总是如此。该机构表示，如果根据它们降低胆固醇的效果而批准了这些药物，那么即使目前进行的试验无法证明它们能降低心脏病和死亡风险，批准的决定也不会被撤销。
Once a drug is approved doctors can prescribe it to patients other than those for whom it was intended, although insurers generally will not pay.
一旦某种药物获得批准，医生就可以向该药物目标人群之外的其他患者开出该药物的处方，不过保险公司一般不会买单。
The drugs are injected every two weeks or once a month, depending on the formulation. The companies are asking that they be approved for use in three groups: patients with high levels of LDL cholesterol who cannot lower it enough with statins, the mainstay drug for cholesterol lowering first introduced in the late 1980s; people at very high risk because they have already had a heart attack or have diabetes and cannot get their levels low enough with statins; and people with high levels of LDL who cannot tolerate statins. Doctors often aim for LDL levels of 70 for people at high risk.
这种药物需要每两周或每个月注射一次，取决于具体的处方。这些企业正在要求批准三种人使用该药物：LDL胆固醇水平较高、且用他汀类药物（主流的降胆固醇药物，上世纪80年代末推出）无法充分降低其水平的患者；已经患有心梗或糖尿病、且无法用他汀类药物充分降低胆固醇水平的高危人群；LDL胆固醇水平较高且无法耐受他汀类药物的的人群。医生通常认为，LDL胆固醇水平达到70即为高危人群。
The problem for the expert group was to decide if there was enough evidence to approve the Sanofi drug without waiting for results from the large clinical trials. Those who voted no said drugs should not be approved until clinical trials establish their efficacy, and voiced the worry that people participating in the trials would drop out once the drugs were approved so they could be sure to get the medicine, not a placebo.
专家组面临的问题是，在不等待大型临床试验的结果的情况下，判断目前是否有足够的证据支持批准赛诺菲(Sanofi)。投反对票的人认为，除非临床试验证实了其功效，否则任何药物都不应该被批准。他们还表达了这样的担忧，即一旦药物被批准，参加临床试验的人们就会退出试验，因为这样他们就能够确保自己服用的是药物本身，而不是安慰剂。
“We need clinical outcomes,” said Dr. Peter Wilson of Emory University.
“我们需要临床结果，”埃默里大学(Emory University)的皮特·威尔森(Peter Wilson)博士说。
Some on the panel felt comfortable recommending approval only for a narrow group of people with a genetic condition, heterozygousfamilial hypercholesterolemia, who cannot control their cholesterol with statins alone. Others favored allowing use of the drug by the much larger group of people at high risk of heart disease for whom statins are insufficient.
委员会的一些成员认为，只可以批准存在基因缺陷的一小批患者服用这些药物，即杂合子型家族性高胆固醇血症患者。这类人无法单独依靠他汀类药物控制胆固醇。还有一些成员支持把药物的适用范围扩大到一个大得多的群体，即他汀类药物已经无法满足其需求的心脏病高危人群。
The group’s chairman, Dr. Robert J. Smith of Brown University, argued for broader availability of the drugs. He said he sees patients in his own practice with out of control cholesterol who are at very high risk because, for example, they have already had a heart attack. Two years is a long time for them to wait for clinical trial findings. “I am unwilling to subject patients to that wait,” Dr. Smith said.
委员会主席、布朗大学(Brown University)的罗伯特·J·史密斯(Robert J. Smith)博士呼吁在更大范围内提供这种药物。他说，自己在行医过程中看到，一些胆固醇水平失控的患者处境十分危险，原因是他们可能已经患过心梗。为了临床试验结果再等待两年对他们来说太漫长了。“我不愿意让病人就这么等着，”史密斯博士说。
The companies and many independent cardiologists say they have reason to believe the drugs will perform as expected. Lowering LDL cholesterol has generally been found to protect against heart disease. And, they say, the drugs were designed to mimic mutations in a gene, PCSK9, that protects people from getting heart disease, even if they smoke or have high blood pressure.
这些企业和许多独立心脏病学专家称，他们有理由相信这些药物会产生预期的效果。降低LDL胆固醇被普遍认为能预防心脏病。此外，他们还说，这些药物模仿了PCSK9基因突变，这种基因能防止人们罹患心脏病，哪怕他们吸烟或有高血压。
But the drugs have caused trepidation among insurers and others who would have to pay the bills because the drugs are certain to be expensive — perhaps $10,000 a year — and millions of people are likely to qualify to take them if they are approved for the broader group. Sanofi estimates that 11 million Americans might qualify. Amgen puts the number at eight million.
不过，这些药物已经引发了一些恐慌，比如必须为它们买单的保险公司和其他人，以及该药物获准适用于更大群体的话，上百万名可能有资格服用这些药物的患者。因为这些药物注定昂贵，也许每年要花1万美元。赛诺菲估计，可能有1100万美国人有资格接受该药物。安进公司认为这个数字是800万。
So far, it looks like just about everyone who takes the new drugs responds. LDL cholesterol levels plunge by 40 to 65 percent, even if the starting level was achieved with a statin.
迄今为止，似乎每一名使用了新药的患者都见到了疗效。LDL胆固醇水平下降了40%到65%，即使他们之前一直在服用他汀类药物来控制胆固醇。
Safety studies so far have found the drugs seem to have no more side effects than a placebo but the large clinical trials are needed to get more substantial information.
目前的安全性研究表明，这些药物的副作用似乎和安慰剂差不多，不过，在大型的临床实验之后才能获得更多实质性的信息。
Meanwhile, pharmaceutical companies see the tantalizing prospect of multibillion dollar blockbuster drugs, and are racing to get them to market as rapidly as possible. Sanofi bought an F.D.A.-backed voucher for $67.5 million that gave them an expedited review. The company says the F.D.A. agreed to decide whether to approve their drug by July 24. Amgen, which did not pay, expects an answer by Aug. 27, the company says. Pfizer also has a drug in this new class but it is further behind in development.
与此同时，制药公司已经看到了价值数十亿美元的重磅药物的诱人前景，它们正在竞相以最快的速度将其投放市场。赛诺菲以6750万美元的价格购买了FDA的批文，可以获得快速审核。该公司表示，FDA同意在7月24日之前做出是否批准该药物的决定。安进公司没有花这笔钱，该公司表示，预计将在8月27日之前得到答案。辉瑞公司(Pfizer)也有一款这种类型的药物，但研发进度要更慢。
Then there is the question of cost. The new drugs, like many new cancer drugs, are monoclonal antibodies, produced from living cells at great expense. The companies will not say what they plan to charge. But Dr. William Shrank, chief scientific officer at CVS Health, estimates they will cost $7,000 to $12,000 a year.
还有成本的问题。这些新药就像许多新型抗癌药物一样，是单克隆抗体，它们是以高昂的成本用活体细胞制造的。这些公司不愿透露药品未来的定价。但CVS健康公司(CVS Health)的首席科学官威廉·史兰克(William Shrank)估计，服用这种药物一年将需要7000至1.2万美元。
If drugs were restricted to those with dangerously high cholesterol levels who cannot get their LDL low enough with statins, the cost would be $16 billion, he estimated. If people who are intolerant to statins are included, that would add another $20 billion. If people with a history of heart disease are included, the bill for the drugs rises another $150 billion.
他估计，如果药物仅限于那些胆固醇水平过高且无法利用他汀类药物降低胆固醇水平的人，总成本将达到160亿美元。如果包括无法耐受他汀类药物的人，成本将增加200亿美元。算上有心脏病史的人，成本将再升高1500亿美元。
“Managed pharmacy care, indeed the health care system, has never seen a challenge like this to our resilience in absorbing costs,” Dr. Shrank wrote in Health Affairs.
史兰克在健康事务(Health Affairs)网站上写道：“保健药学管理体系吸收成本的能力，实际上整个卫生体系吸收成本的能力，都没有遇到过这样的严峻挑战。”
