Opportunities and challenges of incorporating clinical outcome assessments in brain tumor clinical trials.

Regulatory agencies have progressively emphasized the importance of assessing broader aspects of patient well-being to better define therapeutic gain. As a result, clinical outcome assessments (COAs) are increasingly used to evaluate the impact, both positive and negative, of cancer treatments and in some instances have played a major factor in the regulatory approval of drugs. Challenges remain, however, in the routine incorporation of these measures in cancer clinical trials, particularly in brain tumor studies. Factors unique to brain tumor patients such as cognitive decline and language dysfunction may hamper their successful implementation. Study designs often relegated these outcome measures to exploratory endpoints, further compromising data completion. New strategies are needed to maximize the complementary information that COAs could add to clinical trials alongside more traditional measures such as progression-free and overall survival. The routine incorporation of COAs as either primary or secondary objectives with attention to minimizing missing data should define a novel clinical trial design. We provide a review of the approaches, challenges, and opportunities for incorporating COAs into brain tumor clinical research, providing a perspective for integrating these measures into clinical trials.