Off-label immunotherapy prescription: Financial implications for payers and patients.

6 Background: Pembrolizumab (PEM) and nivolumab (NIV) are FDA-approved immunotherapy agents for specific indications. These drugs are extremely costly to the health care system, payers, and ultimately, patients, and little is known about their utilization for non-FDA labeled indications (off-label). Methods: We reviewed claims for PEM and NIV using the focalPoint database by onPoint oncology, which provides access to clearinghouse data for 168 community oncology practices and 2000+ providers throughout the US. Clearinghouse data is unique as it links claims data with remittance (payer response) data. ICD codes representing malignancies other than head and neck cancer, melanoma, non-small cell lung cancer, and claims for 3 indications prior to their FDA-approval were considered off-label. Primary insurance for the patients, as well as patients’ financial responsibility and amounts paid by the insurance were evaluated. Results: A total of 1, 570 patients received PEM or NIV from January 1st 2015 to September 1st 2016 for a cumulative total of 7, 687 distinct claims. Of these patients, 285 (18%) received these agents for off-label indications for a total of 978 distinct claims. The total cost for the insurance for off-label claims was 13. 36 million with the following breakdown: 7. 74 million for 111 patients with commercial insurance, 125 thousand for 5 patients with Medicaid, 3. 85 million for 126 patients with Original Medicare, and 1. 90 million for 43 patients with Medicare Advantage. The patient responsibility for the off-label immunotherapy agents, which included the co-insurance and co-pay, totaled to 1. 06 million or 3, 731 per patient. Patient responsibility for off-label therapy per payer was as follows: 657 per patient for commercial insurance, 0 for Medicaid, 7, 436 for Original Medicare, and 1, 248 for Medicare Advantage. Conclusions: A substantial number of patients received unsupported off-label immunotherapy treatment. The costs of these therapies to the health care system, payers, and patients warrant increased efforts to regulate their use in off-label settings lacking sufficient scientific evidence or while clinical trials are in progress.