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【技术产业】药厂CEO强烈申辩FDA拒绝NDA申请无理
Posted by Jacob Goldstein
Last month, specialty pharma shop Pharmacyclics forced its new drug application on the FDA, despite the agency’s initial refusal to accept the application. The move, called “filing over protest,” is quite rare. The company’s CEO, Richard Miller, dropped by our office today to make his case for the drug, and he couldn’t cite a single example of an approved drug that was filed in that way.
The drug, called Xcytrin, was tested on patients who had lung cancer that had spread to the brain. The study featured an unusual design that looked not at conventional measures such as survival or spread of the tumor, but at neurological problems like having trouble thinking or losing the ability to stand up out of a chair and walk across the room. Miller (pictured, left) said that was the result of discussions with the FDA. “They told us they didn’t care what an MRI looked like. What they cared about was clinical benefit.”
But the study failed to meet the standard test of statistical significance — a common measure of a study’s success. To pass that test, the statistical chance that the results were a fluke must be 5% or lower. The Xcytrin study suggested that patients benefited from the drug, but there is a 12% chance that the result was a fluke. Miller argued that the FDA should look beyond statistical significance. “Don’t give us one size fits all,” he said. “When people look at the data, they’ll say, ‘Hey, there was a substantial benefit here.’”
He argued that the drug “offers patients a chance to live the remaining part of their life not incapacitated.” “You’ve got six months to live,” he said. “Do you want to live it in a wheelchair and not be able to recognize your son or daughter, or do you want to live it intact?”
The company expects to learn in the next few months whether the FDA will convene a panel of outside experts to review the data. The agency should rule on the application by the end of the year. [标签:content1][标签:content2]
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作者:admin@医学,生命科学 2011-03-25 15:19
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