Is it all about price? Why requests for government subsidy of anticancer drugs were rejected in Australia.

BACKGROUND: Australians access anticancer drugs predominantly through the Pharmaceutical Benefits Scheme (PBS). AIM: 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.

Community views on factors affecting medicines resource allocation: cross-sectional survey of 3080 adults in Australia.

Objective The aim of the present study was to determine Australian community views on factors that influence the distribution of health spending in relation to medicines. Methods A cross-sectional web-based survey was performed of 3080 adults aged ≥18 years. Participants were asked to rank, in order of importance, 12 criteria according to which medicines funding decisions may be made. Results Of all respondents, 1213 (39.4%) considered disease severity to be the most important prioritisation criterion for funding a new medicine. This was followed by medicines treating a disease affecting children (13.2%) and medicines for cancer patients (9.1%). Medicines targeting a disease for which there is no alternative treatment available received highest priority from 8.6% of respondents. The remaining eight prioritisation criteria were each assigned a top ranking from 6.6% to 1.7% of respondents. Medicines targeting a disease for which there is no alternative treatment available were ranked least important by 7.7% of respondents, compared with 2.4%, 1.9% and 1.0% for medicines treating severe diseases, diseases affecting children and cancer respectively. 'End-of-life treatments' and 'rare disease therapies' received the least number of highest priority rankings (2.0% and 1.7% respectively). Conclusions These results provide useful information about public preferences for government spending on prescribed medicines. Understanding of public preferences on the funding of new medicines will help the Pharmaceutical Benefits Advisory Committee and government determine circumstances where greater emphasis on equity is required and help inform medicines funding policy that best meets the needs of the Australian population. What is known about this topic? There is increased recognition of the importance of taking into account public preferences in the heath technology assessment (HTA) decision-making process. What does this paper add? The Australian public view the severity of disease to be the most important funding prioritisation criterion for medicines, followed by medicines used to treat children or to treat cancer. What are the implications for practitioners? The general public are capable of giving opinions on distributional preferences. This information can help inform medicines funding policy and ensure that it is consistent with the values of the Australian population.

Societal perspective on access to publicly subsidised medicines: A cross sectional survey of 3080 adults in Australia.

BACKGROUND: Around the world government agencies responsible for the selection and reimbursement of prescribed medicines and other health technologies are considering how best to bring community preferences into their decision making. In particular, community views about the distribution or equity of funding across the population. These official committees and agencies often have access to the best available and latest evidence on clinical effectiveness, safety and cost from large clinical trials and population-based studies. All too often they do not have access to high quality evidence about community views. We therefore, conducted a large and representative population-based survey in Australia to determine what community members think about the factors that do and should influence government spending on prescribed medicines. METHODS: A choice-based survey was designed to elicit the importance of individual criteria when considering the equity of government spending on prescribed medicines. A representative sample of 3080 adult Australians completed the survey by allocating a hypothetical budget to different combinations of money spent on two patient populations. Societal preferences were inferred from absolute majority responses i.e. populations with more than 50% of respondents' allocation for a particular allocation criterion. RESULTS: This study shows that, all else being equal, severity of disease, diseases for which there is no alternative treatment available on the government formulary, diseases that affect patients who are not financially well off, and life-style unrelated diseases are supported by the public as resource allocation criteria. Where 'all else is not equal', participants allocated more resources to the patient population that gained considerable improvement in health and fewer resources to those that gained little improvement in health. This result held under all scenarios except for 'end-of-life treatments'. Responses to cost (and corresponding number of patients treated) trade-off scenarios indicated a significant reduction in the proportion of respondents choosing to divide resources equally and a shift in preference towards devoting resources to the population that were more costly to treat for all criteria with the exception of severity of disease. CONCLUSIONS: The general public have clear views on what's fair in terms of government spending on prescribed medicines. In addition to supporting the application of the 'rule of rescue', important considerations for government spending included the severity of disease being treated, diseases for which there is no alternative treatment available on the government formulary, diseases that affect patients who are not financially well off and life-style unrelated diseases. This study shows that the general public are willing to share their views on what constitutes an equitable allocation of the government's drug budget. The challenge remains to how best to consider those views alongside clinical and economic considerations.

Cost-effectiveness of 12-month treatment with ticagrelor compared with clopidogrel in the management of acute coronary syndromes.

BACKGROUND: The PLATO (Platelet Inhibition and Patient Outcomes) randomized trial (NCT00391872) in patients with acute coronary syndromes (ACS) reported that ticagrelor (in addition to aspirin) reduced the rate of the composite end point of myocardial infarction (MI), stroke, or cardiovascular death compared with clopidogrel (in addition to aspirin) by 16% over 12 months (P < 0.001). No significant difference in the incidence of major bleeding was noted, but ticagrelor was associated with a higher rate of major bleeding not related to coronary artery bypass grafting. OBJECTIVE: By extrapolating the key findings of PLATO, we sought to assess the cost-effectiveness of ticagrelor compared with clopidogrel in the management of ACS in a contemporary Australian setting. METHODS: A Markov model with 4 health states (free from further ACS events, MI, stroke, and death) was developed to simulate the long-term costs and outcomes associated with ACS. Event risks were based on data derived directly from PLATO, and costs and utilities were drawn from published sources. A 10-year time horizon was simulated, and future costs and benefits were discounted at a 5% annual rate. However, treatment with ticagrelor and clopidogrel was only assumed for the first 12 months, with no benefits applied beyond drug cessation. Sensitivity analyses were undertaken based on variations to key data inputs. All costs for resource use applied in the analysis were based on published Australian prices (in 2010/2011 dollars [A$]). RESULTS: Over 10 years, the estimated quality-adjusted life-years lived per-patient were 5.74 and 5.68 for ticagrelor and clopidogrel, respectively. Net costs were A$19,132 for ticagrelor and A$18,428 for clopidogrel. These equated to an incremental cost-effectiveness ratio of A$9031 per quality-adjusted life-year gained for ticagrelor compared with clopidogrel. Sensitivity analyses indicated the result to be robust. CONCLUSIONS: When assessed from the perspective of the Australian health care system, ticagrelor is likely to be cost-effective compared with clopidogrel in preventing downstream morbidity and mortality associated with ACS.

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.