Background: The off-label use of a drug refers to a use outside the terms of its approval by the Therapeutic Goods Administration (TGA). It is also possible to prescribe unlicensed drugs under the TGA s special access scheme. A high rate of off-label prescribing has previously been reported in cancer. Our study aimed to document the disparity between evidence-based clinical guidelines for anticancer therapy, product approval and funding status of these agents within an academic tertiary/quaternary cancer centre. Methods: All chemotherapy protocols approved for use in our specialist oncology centre were assessed to determine if the drugs were off-label or unlicensed for that indication based on review of their current product information. The Pharmaceutical Benefits Scheme (PBS) funding status for each protocol was subsequently assessed. Results: A total of 448 protocols containing 82 different drugs across 15 tumour groups was identified. Overall, 189 (42.2 ) of protocols were off-label, and three (0.7 ) were unlicensed. This resulted in all 192 protocols being unfunded by the PBS. Of the 189 off-label protocols, 132 (69.9 ) were based on established evidence-based treatment guidelines, and a further 39 (20.6 ) was based on phase II or III clinical trial data. Conclusion: Over 90 of off-label protocols are supported by established treatment guidelines or published peer-reviewed research even though the medications are not approved for that particular use by the TGA. However, these off-label protocols are unfunded by the PBS; this results in a marked inequality of access to appropriate medications for cancer patients across Australia.
Mellor, D., van Koeverden, P. M., Yip, S. W. K., Thakerar, A., Kirsa, S. W., & Michael, M. (2012). Access to anticancer drugs: many evidence-based treatments are off-label and unfunded by the Pharmaceutical Benefits Scheme. Internal Medicine Journal, 42(11), 1224 - 1229. https://doi.org/10.1111/j.1445-5994.2012.02751.x