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This bill authorizes certain targeted clinical trials involving combinations of drugs to treat pediatric cancer. Specifically, the bill modifies requirements relating to molecularly targeted pediatric cancer investigations so as to authorize investigations of new drugs that are used in combination with active ingredients that have already been approved and that (1) have been determined to be the standard of care for treating a pediatric cancer, or (2) have been approved to treat an adult cancer and are directed at molecular targets for pediatric cancer. The Food and Drug Administration must issue guidance and report on the bill's implementation. The Government Accountability Office must report on the effectiveness of the bill's changes with respect to the development of pediatric cancer drugs.
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