Clinical trial endpoints serve to different objectives: In early phase, clinical trials evaluate safety and evidence biological drug activity; for later phase efficacy studies evaluate the clinical benefit.
Food and Drug Administration (FDA) has revised the previous guidance on oncology endpoints published in May 2007. This guidance provides recommendations to applicants on endpoints for cancer clinical trials submitted to the FDA.
In this guidance, the FDA classifies several endpoints in base on type of endpoint and study design. Furthermore, analyses advantages and disadvantages of every endpoint referred.
In addition to the already stablished endpoints, in this revision, the FDA proposes two new endpoints to consider:
- Blood or Body Fluid-Based Biomarkers: Generally, although biomarkers assayed from blood or body fluids have not served as primary endpoints for cancer drug approval, the FDA has accepted blood-based markers as elements of a composite endpoint. This fact has been due to the use of paraprotein levels measured in blood and urine (myeloma) or CA-125 (ovarian cancer), for example.
- Emerging Endpoints: FDA recognizes that owing to advances in science, new endpoints that may be used in drug approvals can be identified. As examples, minimal residual disease (lymphoblastic leukemia) and metastasis-free survival (non-metastasis castration-resistant prostate cancer).