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During my years as a Food and Drug Administration official, I learned that the available data on a new drug are seldom as complete and unequivocal as you might like. But getting drugs to patients can be a matter of life or death, so you need to make hard decisions. Fortunately, the regulations offer some flexibility so that the criteria for demonstrating efficacy are more lenient in certain critical situations. One of those pathways is called “accelerated approval.”

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