The gold standard for the evaluation of new drugs to treat cancer is a randomized clinical trial (RCT) in which the new interventions are proven to provide better outcomes for patients compared with an existing standard of care. When serving as the pivotal trial for regulatory appoval of an investigational cancer drug, such trials are usually designed and powered to show that the experimental intervention is superior to the comparator. When no active treatment options are available, new cancer drugs may alternatively be tested to determine whether they are superior to placebo, with each arm also receiving the best available supportive care.
Gyawali B, Kesselheim AS. US Food and Drug Administration Approval of New Drugs Based on Noninferiority Trials in Oncology: A Dangerous Precedent? JAMA Oncol. 2019;5(5):607–608. doi:10.1001/jamaoncol.2019.0093
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