Prevalence of off-label use of oral oncolytics at a community cancer center

Oral oncolytics are becoming increasingly utilized for cancer treatment, but the frequency of off-label oral oncolytic use is not well described. The extent of off-label oral oncolytic use is a concern because the clinical benefits of such use to patients may not outweigh adverse health outcomes or cost concerns.

Prescription data for January 2011 through November 2013 from the St. Lukes Mountain States Tumor Institute (MSTI) Oral Chemotherapy program (OCP) was retrospectively analyzed. Use was classified as "on-label" if the cancer site, stage, and line of therapy met the FDA-approved indication. All other uses were classified as "off- label." Off-label use was further evaluated by whether it conformed to and was supported by National Comprehensive Cancer Network (NCCN) guideline recommendations.

Twelve hundred and six first-fill oral chemotherapy prescriptions were reviewed, representing 990 unique patients and 44 individual medications. On-label use amounted to 71% and off-label use amounted to 29%. Eighty-eight percent of off-label uses were supported by NCCN guideline recommendations. A total of 3.3% of all prescriptions analyzed were for off-label uses not supported by NCCN guideline recommendations. The top five oral chemotherapies prescribed for off-label uses were capecitabine, temozolomide, lenalidomide, abiraterone, and everolimus.

Oral chemotherapies are more often used on label than off label in current practice at our community cancer center. The majority of off-label use of oral oncolytics in this study was supported by NCCN guideline recommendations.