title: Is it all about price? Why requests for government subsidy of anticancer drugs were rejected in Australia.
abstract: BACKGROUND
  Australians access anticancer drugs predominantly through the Pharmaceutical Benefits Scheme (PBS).
  
  
  OBJECTIVE
  To determine why the Pharmaceutical Benefits Advisory Committee (PBAC) rejects submissions to list anticancer drugs on the PBS.
  
  
  METHODS
  We reviewed publicly available information about submissions made to the PBAC for PBS listing of anticancer drugs from 2005 to 2014. Submission characteristics, including clinical and economic evidence, PBAC recommendations, and the reasons offered for rejection were recorded. Two reviewers independently categorised the reason for rejection offered by the PBAC. Logistic regression was used to determine submission characteristics associated with rejection.
  
  
  RESULTS
  We identified 213 submissions for 110 unique indications of 60 anticancer drugs. The overall rejection rate was 56% (119/213). Of the 110 indications assessed, 69% (76/110) were rejected at least once. The annual rejection rate ranged from 50 to 73% with little evidence of a trend over time (P = 0.2). Submission characteristics strongly associated with rejection in multivariable analysis included: PBAC judged the clinical evidence to be problematic or uncertain (P < 0.001); PBAC judged the economic evidence to be problematic or uncertain (P < 0.001); and, inactive comparator used (P < 0.001). The most frequent reasons for rejection offered by the PBAC was 'inadequate cost-effectiveness or drug price too high' (75/109, 69%).
  
  
  CONCLUSIONS
  Inadequate cost-effectiveness and PBAC uncertainty about the clinical and economic evidence were the most frequent reasons for rejection. Clarity of information about PBAC deliberations and their reasons for rejection are important for patients and doctors grappling with decisions about the use of expensive unfunded anticancer drugs.
authors.affiliation: NHMRC Clinical Trials Centre, University of Sydney, Sydney, New South Wales, Australia.|Nepean Cancer Care Centre, Nepean Hospital, Sydney, New South Wales, Australia.
authors.forename: Deme J
authors.initials: DJ
authors.lastname: Karikios
authors.affiliation: Sydney School of Public Health, University of Sydney, Sydney, New South Wales, Australia.|Alexion Pharmaceuticals, Sydney, New South Wales, Australia.
authors.forename: Lesley
authors.initials: L
authors.lastname: Chim
authors.affiliation: NHMRC Clinical Trials Centre, University of Sydney, Sydney, New South Wales, Australia.
authors.forename: Andrew
authors.initials: A
authors.lastname: Martin
authors.affiliation: The Crown Princess Mary Cancer Centre, Westmead Hospital, Sydney, New South Wales, Australia.|Sydney Medical School, University of Sydney, Sydney, New South Wales, Australia.
authors.forename: Adnan
authors.initials: A
authors.lastname: Nagrial
authors.affiliation: Sydney School of Public Health, University of Sydney, Sydney, New South Wales, Australia.
authors.forename: Kirsten
authors.initials: K
authors.lastname: Howard
authors.affiliation: Faculty of Social Sciences, University of Wollongong, Wollongong, New South Wales, Australia.
authors.forename: Glenn
authors.initials: G
authors.lastname: Salkeld
authors.affiliation: NHMRC Clinical Trials Centre, University of Sydney, Sydney, New South Wales, Australia.|Concord Cancer Centre, Concord Hospital, Sydney, New South Wales, Australia.|Department of Medical Oncology, Chris O'Brien Lifehouse, Sydney, New South Wales, Australia.
authors.forename: Martin R
authors.initials: MR
authors.lastname: Stockler
mesh_terms: D026683:Advisory Committees; D000970:Antineoplastic Agents / Q000191:economics* / Q000600:supply & distribution; D001315:Australia; D003362:Cost-Benefit Analysis; D017277:Drug Approval; D055317:Evidence-Based Practice; D005380:Financing, Government / Q000191:economics*; D006077:Government Agencies; D006291:Health Policy; D006801:Humans; D007356:Insurance, Pharmaceutical Services / Q000191:economics
chemical_list: D000970:Antineoplastic Agents
keywords: anticancer drugs; cost-effectiveness; decision making; drug subsidisation; health technology assessment