Guidance for Industry: Clinical Trial Endpoints for the Approval of Cancer Drugs and Biologics

摘要

This guidance is the first in a planned series of cancer endpoint guidances. It provides recommendations to applicants on endpoints for cancer clinical trials submitted to the Food and Drug Administration (FDA) to support effectiveness claims in new drug applications (NDAs), biologics license applications (BLAs), or supplemental applications.2 It also provides background information and discusses general regulatory principles. The endpoints discussed in this guidance are for drugs to treat patients with an existing cancer. This guidance does not address endpoints for drugs to prevent or decrease the incidence of cancer. The FDA is developing guidance on oncology endpoints through a process that includes public workshops and discussions before the FDAs Oncologic Drugs Advisory Committee (ODAC).3 Each subsequent guidance will focus on endpoints for specific cancer types (e.g., lung cancer, colon cancer) to support drug approval or labeling claims. FDAs guidance documents, including this guidance, do not establish legally enforceable responsibilities. Instead, guidances describe the Agencys current thinking on a topic and should be viewed only as recommendations, unless specific regulatory or statutory requirements are cited. The use of the word should in Agency guidances means that something is suggested or recommended, but not required.