FDA Releases Clinical Outcome Assessment Compendium

Promoting the use of patient-focused outcome measurement in drug development has been a major focus of the United States Food and Drug Administration’s (FDA’s) Center for Drug Evaluation and Research (CDER). To continue fostering this goal, the CDER recently published an update of their Clinical Outcome Assessment (COA) Compendium. This compilation was designed to act as a resource regarding COA for a variety of disease types and is meant to serve as a starting point for future clinical trial design, including early stages of drug development. It covers a number of COA types, such as those used in clinical trials to measure patient experiences and those used to support labeling claims. This update adds other COA information: new COAs that have been approved for potential use in future drug development, and the name and approval date for drugs that serve as reference points for COAs. COAs under assessment by the FDA are not included in this resource, as they are considered investigational, but may be added after approval.

High Altitude: This is an important update for clinicians and researchers involved in clinical trial design and development. In recent years there has been a great deal of pressure to incorporate more holistic outcome measures into clinical trials, including standards of patient well-being and quality of life. This reflects an increasing focus on improving both the quantity and quality of life with new cancer treatments. This updated compendium serves as an important reference point helping to guide future COA strategies.

Ground Level: As treatments for cancer become more effective at prolonging life and achieving disease-specific outcomes, it is important for clinicians to evaluate treatments on the basis of their impact on patients, beyond response rates and assessing progression. Incorporation of patient-focused outcomes into clinical trials will enable oncologists to evaluate novel treatments on multiple levels and select treatments that are most reflective of the individual patient’s goals. This will be particularly important in later lines of therapy, when objectives of treatment shift to palliative care rather than prolonging life.