Factors Associated With Off-Label Oncology Prescriptions: The Role of Cost and Financing in a Universal Healthcare System.

Boaz Tadmor,Daniel A. Goldstein,Salomon M. Stemmer,Noa Gordon,Dan Greenberg

doi:10.3389/fphar.2021.754390

Abstract

Purpose: Various solutions have been put forward for prescribing and reimbursing treatments outside their registered indications within universal healthcare systems. However, most off-label oncology prescriptions are not reimbursed by health funds. This study characterized the financing sources of off-label oncology use and the predictors of the decision to forego treatment. Materials and Methods: All 708 off-label oncology requests submitted for approval in a large tertiary cancer center in Israel between 2016 and 2018 were examined for disease and patient sociodemographic characteristics, costs and financing sources, and the factors predicting actual off-label drug administration using multivariate logistic regression analysis. Results: The mean monthly cost of a planned off-label treatment was ILS54,703 (SD = ILS61,487, median = ILS39,928) (approximately US$ 15,500). The main sources of funding were private health insurance (25%) and expanded access pharma company plans (30%). Approximately one third (31%) of the requests did not have a financing source at the time of approval. Of the 708 requests, 583 (or 82%) were filled and treatment was initiated. Predictors for forgoing treatment were the impossibility of out-of-pocket payments or the lack of a financing solution (OR = 0.407; p = 0.005 and OR = 0.400; p < 0.0005). Conclusion: Although off-label recommendations are widespread and institutional approval is often granted, a large proportion of these prescriptions are not filled. In a universal healthcare system, the financing sources for off-label treatments are likely to influence access.

Highlights

Regulatory registration and approval are mandatory for new drugs to be marketed and for public and private health insurance reimbursement
All consecutive off-label requests approved between January 2016 and December 2018 by the Institutional Drug Committee (IDC)
Oncology off-label drug requests were included, since these are reviewed by the IDC and administered in the hospital’s outpatient clinic

Summary

Introduction

Regulatory registration and approval are mandatory for new drugs to be marketed and for public and private health insurance reimbursement. If a drug is unauthorized but used de facto, pharmaceutical companies have no major incentive to expand its registration and marketing authorization This is especially germane to off-patent drugs and to rare indications where Phase III randomized controlled trials may not be feasible or economically viable (ASCO, 2006). With life-threatening and terminal illnesses such as cancer, patients and physicians look to unapproved treatments with limited supporting evidence after standard therapies have been exhausted This “off-evidence” use may benefit patients, based on the reasoning that different cancer indications share the same genetic or molecular characterizations. Off-label drug use may provide real hope for effective treatments that might emerge in the future This is defined as its “option value” (Garrison et al, 2017)

Methods

Results

Conclusion