NDA Approval Under FDCA Section 505(B)(1) Based On Effectiveness Data From One Clinical Trial

For new drug approval, the Food and Drug Administration (FDA) generally requires two or more clinical trials that demonstrate effectiveness. Notwithstanding, the Federal Food, Drug, and Cosmetic Act (FDCA) expressly authorizes FDA to approve drugs based on one trial with “confirmatory” evidence, and the Agency has issued guidance describing when only one trial is sufficient. To evaluate the frequency with which FDA approves new drugs based on only one trial, we researched the relevant statutory background and FDA guidance, and also reviewed new drug and biological product approvals since 1997. Out of 394 products approved from 1998 to 2011, we identified 30 products that were approved based on effectiveness data from one trial.