美国国家公共电台 NPR Controversy Continues Over Muscular Dystrophy Drug, Despite FDA Approval

Controversy¹ Continues Over Muscular Dystrophy Drug, Despite FDA Approval

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Patients are getting a bigger voice in medical research because of evolving federal standards. That trend was apparent this week when the Food and Drug Administration provisionally approved a drug even without clear scientific evidence that it works. The drug treats Duchenne muscular dystrophy, a rare and deadly disease that strikes boys and gradually saps their strength. NPR's Richard Harris reports.

RICHARD HARRIS, BYLINE³: Hundreds of patients, supporters and medical researchers gathered in suburban⁴ Washington, D.C., in April when an advisory⁵ committee sat down to pass judgement on a drug called eteplirsen to treat Duchenne muscular dystrophy. Just 12 boys had been studied taking the drug, and just about everybody agreed that the research was deeply flawed. It provided no direct evidence that the drug actually slowed progression of this muscle-wasting disease. But study participant Billy Elsworth stepped up to the microphone that April day with an impassioned plea.

(SOUNDBITE OF ARCHIVED RECORDING)

BILLY ELLSWORTH: I'm going to beat this bloody⁶ disease, but I need your help. So please help me and my friends and do the right thing. FDA, please don't let me die early.

HARRIS: One patient after another described how the drug had helped them even though the flawed studies failed to demonstrate that. After 10 hours of testimony⁷, acrimony and occasional shouts of outrage⁸, the committee voted that the science wasn't there to prove that the drug actually worked.

PAT FURLONG: It was not a warm and fuzzy interaction. We came away really heartbroken in many ways.

FURLONG: Pat Furlong heads the group Parent Project Muscular Dystrophy. Two sons died of the disease, and she is a leading voice for patients. The FDA had turned back three other similar drugs, and the community was concerned that it would happen again. But Furlong held out hope that evolving rules and evolving attitudes about the role of patients would play into the FDA's decision.

FURLONG: We have to look to what is meaningful benefit on the part of the patients, what do the patients value.

HARRIS: In this case, the parents saw real promise in this drug despite the results from the flawed study. The drugs appeared to be safe, so there wasn't much risk. And the research did show that boys on the drug were producing a small amount of a potentially helpful protein called dystrophin, which could signal that the drug provides some benefit. There was a deep and public disagreement among scientists at the FDA about whether to approve the drug. But earlier this week, FDA gave it a provisional thumbs up. Scientist Eric Hoffman sees both sides.

ERIC HOFFMAN: You can look at this as being a potentially damaging precedent⁹. And on the other hand, you can look at it as being a innovative¹⁰ precedent that could bring good things and earlier access to medicines.

HARRIS: Hoffman, who spoke¹¹ via Skype, is at the University of Binghamton's pharmacy¹² school and a CEO of a company developing a drug for muscular dystrophy.

HOFFMAN: It still has to be proven to show benefit, but at least this accelerated approval seems to accelerate that.

HARRIS: Sarepta, which makes eteplirsen, must now follow up with further studies, so the drug could still be pulled from the market. This particular drug addresses just one of more than 20 genetic¹³ mutations that cause muscular dystrophy. This mutation¹⁴ only affects about 1,500 boys in the United States. Sarepta plans to use the same approach now to go after other mutations related to muscular dystrophy and to use those results to comply with the FDA's requirement for more and better studies. In the meantime, Sarepta can now start charging $300,000 per patient per year for eteplirsen. Edward Kaye, interim¹⁵ CEO of Sarepta, acknowledges the unusual circumstances of this provisional approval.

EDWARD KAYE: Yes, there was a lot of pressure, but I think the FDA demonstrated the flexibility¹⁶ that they are allowed under the law. And I think it took a lot of courage for them to do this. It's always easy to take the safe path and say, well, just give us more data.

FURLONG: I think the important aspect of this is maybe a new social contract.

HARRIS: Advocate Pat Furlong says it's important to weigh the values of the patients. And since the drug is apparently¹⁷ safe and possibly useful, they argued to make the drug available while the questions about usefulness and value are fully¹⁸ answered.

FURLONG: And at the end of five years, we can re-evaluate whether or not this really hits its mark in terms of the health care cost and the benefit to patients.

HARRIS: More than a dozen potential drugs for this disease are in the pipeline¹⁹ now, and at the very least, they now have a benchmark against which they can be judged. Richard Harris, NPR News.

1 controversy   

n.争论，辩论，争吵

参考例句：

• That is a fact beyond controversy.那是一个无可争论的事实。
• We ran the risk of becoming the butt of every controversy.我们要冒使自己在所有的纷争中都成为众矢之的的风险。

2 browser   

n.浏览者

参考例句：

• View edits in a web browser.在浏览器中看编辑的效果。
• I think my browser has a list of shareware links.我想在浏览器中会有一系列的共享软件链接。

3 byline   

n.署名；v.署名

参考例句：

• His byline was absent as well.他的署名也不见了。
• We wish to thank the author of this article which carries no byline.我们要感谢这篇文章的那位没有署名的作者。

4 suburban   

adj.城郊的，在郊区的

参考例句：

• Suburban shopping centers were springing up all over America. 效区的商业中心在美国如雨后春笋般地兴起。
• There's a lot of good things about suburban living.郊区生活是有许多优点。

5 advisory   

adj.劝告的，忠告的，顾问的，提供咨询

参考例句：

• I have worked in an advisory capacity with many hospitals.我曾在多家医院做过顾问工作。
• He was appointed to the advisory committee last month.他上个月获任命为顾问委员会委员。

6 bloody   

adj.非常的的；流血的；残忍的；adv.很；vt.血染

参考例句：

• He got a bloody nose in the fight.他在打斗中被打得鼻子流血。
• He is a bloody fool.他是一个十足的笨蛋。

7 testimony   

n.证词；见证，证明

参考例句：

• The testimony given by him is dubious.他所作的证据是可疑的。
• He was called in to bear testimony to what the police officer said.他被传入为警官所说的话作证。

8 outrage   

n.暴行，侮辱，愤怒；vt.凌辱，激怒

参考例句：

• When he heard the news he reacted with a sense of outrage.他得悉此事时义愤填膺。
• We should never forget the outrage committed by the Japanese invaders.我们永远都不应该忘记日本侵略者犯下的暴行。

9 precedent   

n.先例，前例；惯例；adj.在前的，在先的

参考例句：

• Is there a precedent for what you want me to do?你要我做的事有前例可援吗？
• This is a wonderful achievement without precedent in Chinese history.这是中国历史上亘古未有的奇绩。

10 innovative   

adj.革新的，新颖的，富有革新精神的

参考例句：

• Discover an innovative way of marketing.发现一个创新的营销方式。
• He was one of the most creative and innovative engineers of his generation.他是他那代人当中最富创造性与革新精神的工程师之一。

11 spoke   

n.（车轮的）辐条；轮辐；破坏某人的计划；阻挠某人的行动 v.讲，谈（speak的过去式）；说；演说；从某种观点来说

参考例句：

• They sourced the spoke nuts from our company.他们的轮辐螺帽是从我们公司获得的。
• The spokes of a wheel are the bars that connect the outer ring to the centre.辐条是轮子上连接外圈与中心的条棒。

12 pharmacy   

n.药房，药剂学，制药业，配药业，一批备用药品

参考例句：

• She works at the pharmacy.她在药房工作。
• Modern pharmacy has solved the problem of sleeplessness.现代制药学已经解决了失眠问题。

13 genetic   

adj.遗传的，遗传学的

参考例句：

• It's very difficult to treat genetic diseases.遗传性疾病治疗起来很困难。
• Each daughter cell can receive a full complement of the genetic information.每个子细胞可以收到遗传信息的一个完全补偿物。

14 mutation   

n.变化，变异，转变

参考例句：

• People who have this mutation need less sleep than others.有这种突变的人需要的睡眠比其他人少。
• So far the discussion has centered entirely around mutation in the strict sense.到目前为止，严格来讲，讨论完全集中于围绕突变问题上。

15 interim   

adj.暂时的，临时的；n.间歇，过渡期间

参考例句：

• The government is taking interim measures to help those in immediate need.政府正在采取临时措施帮助那些有立即需要的人。
• It may turn out to be an interim technology.这可能只是个过渡技术。

16 flexibility   

n.柔韧性，弹性，（光的）折射性，灵活性

参考例句：

• Her great strength lies in her flexibility.她的优势在于她灵活变通。
• The flexibility of a man's muscles will lessen as he becomes old.人老了肌肉的柔韧性将降低。

17 apparently   

adv.显然地；表面上，似乎

参考例句：

• An apparently blind alley leads suddenly into an open space.山穷水尽，豁然开朗。
• He was apparently much surprised at the news.他对那个消息显然感到十分惊异。

18 fully   

adv.完全地，全部地，彻底地；充分地

参考例句：

• The doctor asked me to breathe in,then to breathe out fully.医生让我先吸气，然后全部呼出。
• They soon became fully integrated into the local community.他们很快就完全融入了当地人的圈子。

19 pipeline   

n.管道，管线

参考例句：

• The pipeline supplies Jordan with 15 per cent of its crude oil.该管道供给约旦15%的原油。
• A single pipeline serves all the houses with water.一条单管路给所有的房子供水。