title: Access to anticancer drugs: many evidence-based treatments are off-label and unfunded by the Pharmaceutical Benefits Scheme.
abstract: 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.
  
  
  CONCLUSIONS
  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.
authors.affiliation: Pharmacy Department, Monash University, Melbourne, Victoria, Australia. dan.mellor@petermac.org
authors.forename: J D
authors.initials: JD
authors.lastname: Mellor
authors.forename: P
authors.initials: P
authors.lastname: Van Koeverden
authors.forename: S W K
authors.initials: SW
authors.lastname: Yip
authors.forename: A
authors.initials: A
authors.lastname: Thakerar
authors.forename: S W
authors.initials: SW
authors.lastname: Kirsa
authors.forename: M
authors.initials: M
authors.lastname: Michael
mesh_terms: D000046:Academic Medical Centers / Q000191:economics / Q000706:statistics & numerical data; D000970:Antineoplastic Agents / Q000191:economics / Q000600:supply & distribution*; D002173:Cancer Care Facilities / Q000191:economics / Q000706:statistics & numerical data; D002985:Clinical Protocols; D017277:Drug Approval; D011307:Drug Prescriptions / Q000706:statistics & numerical data; D004363:Drug Utilization / Q000706:statistics & numerical data; D019317:Evidence-Based Medicine; D005380:Financing, Government; D019983:Guideline Adherence; D006297:Health Services Accessibility / Q000706:statistics & numerical data*; D006801:Humans; D007356:Insurance, Pharmaceutical Services / Q000191:economics; D009369:Neoplasms / Q000188:drug therapy* / Q000191:economics; D056687:Off-Label Use / Q000191:economics / Q000706:statistics & numerical data*; D017410:Practice Guidelines as Topic; D010818:Practice Patterns, Physicians' / Q000706:statistics & numerical data; D012959:Socioeconomic Factors; D062606:Tertiary Care Centers / Q000191:economics / Q000706:statistics & numerical data; D014739:Victoria
chemical_list: D000970:Antineoplastic Agents