Retrospective evaluation of clinical evidence submitted with off-label requests at a community-based oncology infusion center.

Abstract

254 Background: A systematic grading system was created to evaluate literature submitted with off-label requests to the Inova Schar Cancer Institute (ISCI) oncology P&T. Level of evidence (LOE) 1A, 1B, and 1C are granted immediate approval. LOE 2A, 2B, 2C require two members (MD and PharmD) to approve. LOE 3A, 3B, 3C require 3/4 (2MDs, 2PharmDs) members to approve. LOE no benefit (NB) is denied approval. Methods: Identified off-label requests for hematology/oncology patients from December 23, 2016 to January 2, 2019. Primary objective was retrospective evaluation of literature submitted using a systematic grading system through the oncology P&T. Secondary objective was to evaluate financial and clinical outcomes of off-label treatments, including compliance with Quality Oncology Practice Initiative measures. Future aim is to compare LOE and treatment outcomes pre- and post-implementation. Results: There were 73 off-label requests with 126 references submitted as supporting literature. The most frequently requested treatments were nivolumab (21.9%) and pembrolizumab (20.5%). The most common disease state was breast cancer. The LOE for the literature submitted was 1A (10.3%), 1B (23%), 1C (8.7%), 2A (9.5%), 2B (7.9%), 2C (4.8%), 3A (1.6%), 3B (7.1%), 3C (5.6%), and NB (21.4%). Sixty-six treatments were approved and 54 patients received at least one dose of off-label treatment. Thirty off-label requests were made based on molecular tumor board recommendations. Off-label treatment was discontinued for disease progression (29.6%), toxicity (25.9%), or death (14.8%). Three patients completed therapy and 11 received subsequent treatment after discontinuation of the off-label treatment. Average time from last treatment to death was 41.8 days (median 21, range 6-175). Eleven of 55 expired patients received hospice care prior to expiring. Most off-label treatments were approved by third party insurance (55.6%) while 37% were covered by manufacturer provided free drug and 7.4% were paid for by the ISCI Foundation. Conclusions: The standardized grading system for off-label treatments can be implemented to ensure safe and effective use of off-label treatments in oncology patients.