abstract: Access to anti-cancer drugs: Many evidence-based treatments are off-label and unfunded by the PBS - Australia

Access to anti-cancer drugs: Many evidence-based treatments are off-label and unfunded by the PBS

 ABSTRACT

Background:  The off-label use of a drug refers to a use outside the terms of its approval by the Therapeutic Goods Administration's (TGA). It is also possible to prescribe unlicensed drugs under the Therapeutic Goods Administration's (TGA) Special Access Scheme. A high rate of off-label prescribing has previously been reported in cancer. Our study aimed to document the disparity between clincial evidence-based guidelines for anti-cancer therapy, product approval, and funding status of these agents within an academic tertiary/quaternary cancer centre.

Method:  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 upon 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 were identified. Overall, 189 (42.2%) of protocols were off-label and 3 (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%) were based upon phase II or III clinical trial data.

Discussion:  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.