Are cancer drugs less likely to be recommended for listing by the Pharmaceutical Benefits Advisory Committee in Australia?

BACKGROUND: The hurdle of cost effectiveness for the selection and reimbursement of drugs in Australia limits access to new medicines based on an assessment of cost relative to clinical benefit. Those drugs that are expensive and provide modest benefits will be less likely to receive a government price subsidy. There is concern that the cost-effectiveness hurdle will limit access to new cancer treatments because of their high costs and modest benefits.
OBJECTIVE: To test the hypothesis that Ceteris paribus, cancer drugs are less likely to receive a recommendation for reimbursement on the Pharmaceutical Benefits Scheme (PBS) than non-cancer drugs.
METHODS: We reviewed public summary documents (PSDs) on all major submissions considered by the Pharmaceutical Benefits Advisory Committee (PBAC) from July 2005 to March 2008. Each PSD includes summary information on the clinical, economic and utilization considerations of the PBAC in arriving at a recommendation. A total of 227 PSDs were reviewed, from which 243 PBAC recommendations were identified. Logistic regression was used to determine the effects of drug type (cancer vs non-cancer) and other potentially confounding variables on the outcome of PBS approval versus non-approval.
RESULTS: There were 243 PBAC recommendations in 227 published PSDs: 108 for rejection (44%), 10 deferrals (4%) and 125 (51%) recommendations for listing. Recommendations for listing were made somewhat more often for non-cancer drugs than for cancer drugs: 104/191 (54%) versus 21/52 (40%), respectively; p = 0.07. Based on the results for univariable analyses, there is evidence that four variables have some association (p < 0.25) with PBAC approval, but only type of application, economic modelling and estimated cost to the PBS remained statistically significant at p < 0.05 in the multivariable model. No interaction terms were statistically significant. Cancer drug submissions tend to have a modelled economic evaluation and have a higher cost per QALY than non-cancer drugs (29% vs 15% of cancer and non-cancer drugs, respectively had a reported modelled cost per QALY of more than Australian dollars [$A]45 000; p < 0.001). Submissions that include a modelled economic evaluation and have a higher cost per QALY get approved less often than submissions without an economic modelling (p = 0.04). However, after adjusting for economic modelling, there is no statistical difference between cancer and non-cancer drugs in terms of gaining recommendation for PBS listing.
CONCLUSION: The PBAC applies decision criteria equitably to cancer and non-cancer drugs, in that cancer drugs are neither favoured nor disadvantaged but they are more expensive and target a smaller population than non-cancer drugs. Further debate and research is needed to determine society's willingness to pay for a QALY and whether this differs between drugs for cancer and other indications or for interventions that differ on criteria other than cost effectiveness.