OBJECTIVES: We sought to characterize the differences between FDA-required labeling and recommendations of the National Comprehensive Cancer Network’s (NCCN) Drugs and Biologics Compendium.

METHODS: We reviewed recommendations for all drugs and biologics listed in the NCCN Compendium, excluding: (1) supportive therapies and (2) chemotherapies to quantify the extent to which recommendations vary from FDA-required labeling.

RESULTS: Our final sample consisted of 77 medicines; 28% of all medicines listed in NCCN in October 2017. Recommendations for uses not specifically identified in the FDA-required labeling occurred for 64 (83%). We found 314 total recommendations for unapproved use, for an average of 4.08 per drug studied. In order of prevalence, the compendium included recommendations for: use in other cancer types (45% of medicines; 81 total), drug combinations not listed in the FDA label (43% of medicines; 108 total), disease stages not included in the FDA label (40% of medicines, 80 total) alternative lines of therapy not included in the FDA label (19% of medicines; 17 total), and use in alternate genetic populations or use of testing not included in the FDA label (18% of medicines; 20 total).

CONCLUSIONS: Most oncology medicines studied in the NCCN Drugs & Biologics Compendium are recommended for at least one use beyond those in the product’s FDA-required labeling. Physicians often rely on clinical compendia to help guide treatment decisions, and private payers use compendia to varying degrees to determine coverage. To ensure patients have access to treatments supported by the best current scientific evidence, physicians and payers should continue to utilize a range of compendia and peer-reviewed literature. Policy changes, such as recent FDA guidance that enable more efficient and complete sharing of information about both approved and unapproved uses of FDA-approved medicines have the potential to improve patient access to and clinically appropriate use of beneficial evidence-based treatments.