美国国家公共电台 NPR Controversy Continues Over Muscular Dystrophy Drug, Despite FDA Approval（在线收听）

Controversy 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, BYLINE: Hundreds of patients, supporters and medical researchers gathered in suburban Washington, D.C., in April when an advisory 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.

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BILLY ELLSWORTH: I'm going to beat this bloody 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 testimony, acrimony and occasional shouts of outrage, 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 precedent. And on the other hand, you can look at it as being a innovative precedent that could bring good things and earlier access to medicines.

HARRIS: Hoffman, who spoke via Skype, is at the University of Binghamton's pharmacy 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 genetic mutations that cause muscular dystrophy. This mutation 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, interim 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 flexibility 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 apparently safe and possibly useful, they argued to make the drug available while the questions about usefulness and value are fully 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 pipeline now, and at the very least, they now have a benchmark against which they can be judged. Richard Harris, NPR News.