Quality-of-life end points in cancer clinical trials: the U.S. Food and Drug Administration perspective.

TLDR

Quality‑of‑life endpoints are increasingly incorporated into oncology trials, and the FDA’s Oncologic Drugs Advisory Committee now requires a favorable QOL effect for drug approval, especially when survival is not impacted, making QOL more critical than traditional efficacy measures. The FDA aims to evaluate QOL‑based trials by how well they meet stated objectives and encourages collaboration with investigators to integrate QOL instruments into trial design. Protocols must state investigators’ hypotheses, select validated instruments, and include a detailed statistical plan for handling missing data.

Abstract

Increasingly, quality-of-life (QOL) end points are being incorporated into randomized, controlled clinical trials in oncology. The Oncologic Drugs Advisory Committee (U.S. Food and Drug Administration) has recommended that beneficial effects on QOL and/or survival be the basis for approval of new anticancer drugs. Therefore, from a regulatory standpoint, for drugs that do not have an impact on survival, demonstration of a favorable effect on QOL is more important than most other traditional measures used to assess efficacy, such as objective tumor response. Trials incorporating QOL questions will be evaluated on the basis of how well they address the stated objectives. The clinical protocol should delineate investigators' hypotheses and choice of validated instruments and should specify a detailed statistical analysis plan describing strategies for handling missing data. The U.S. Food and Drug Administration welcomes the opportunity to explore with investigators the use of QOL instruments in the design of cancer clinical trials.