title: Assessment of the Quality of the Clinical Evidence in Submissions to the Australian Pharmaceutical Benefits Advisory Committee: Fit for Purpose?
abstract: BACKGROUND
  Assessments of the comparative clinical (and cost) effectiveness of new medicines are increasingly being used to inform decisions on their reimbursement. Assessments of added clinical benefit are invariably based on evidence generated to support registration.
  
  
  OBJECTIVE
  Our objective was to identify and characterize significant problems relating to the quality of the clinical evidence in submissions to the Australian Pharmaceutical Benefits Advisory Committee (PBAC) seeking subsidy on the Pharmaceutical Benefits Scheme and thus determine whether the evidence presented to the committee was "fit for purpose."
  
  
  METHODS
  We conducted a retrospective analysis of submissions considered by the PBAC between 2005 and 2012 using a published evaluation framework. We developed an additional framework to categorize significant problems in more detail. Significant problems related to the choice of comparator, the unavailability of randomized clinical trial evidence, poor-quality data, a claim of clinical superiority, and a claim of clinical noninferiority.
  
  
  RESULTS
  We identified 261 significant problems in 479 major submissions. There was a significant problem with the sponsor's choice of comparator in 11% of the submissions. The most common significant problem (29%) was the determination of a medicine's comparative performance in the target patient population.
  
  
  CONCLUSIONS
  The supporting clinical evidence is the foundation of a PBAC submission. We found a poor fit for purpose; on average, one in every two major submissions had a significant problem with the supporting evidence. The findings from our study, if confirmed in other jurisdictions, raise important questions regarding what clinical evidence should be generated to support the reimbursement of new medicines.
authors.affiliation: Wonder Drug Consulting Pty. Ltd., Cronulla, NSW, Australia. Electronic address: wonderdrug@optusnet.com.au.
authors.forename: Michael
authors.initials: M
authors.lastname: Wonder
authors.affiliation: Market Access, Asia-Pacific, Zimmer Pty Ltd, Belrose, NSW, Australia.
authors.forename: Sheryl
authors.initials: S
authors.lastname: Dunlop
mesh_terms: D026683:Advisory Committees / Q000191:economics / Q000592:standards*; D001315:Australia; D003362:Cost-Benefit Analysis / Q000592:standards*; D006801:Humans; D007356:Insurance, Pharmaceutical Services / Q000191:economics / Q000592:standards*; D016032:Randomized Controlled Trials as Topic / Q000191:economics / Q000592:standards; D012051:Reimbursement Mechanisms / Q000191:economics / Q000592:standards*; D012189:Retrospective Studies
keywords: decision making; evidence; quality; reimbursement