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\u27 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 \u27all else is not equal\u27, 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 \u27end-of-life treatments\u27. 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\u27s fair in terms of government spending on prescribed medicines. In addition to supporting the application of the \u27rule of rescue\u27, 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\u27s drug budget. The challenge remains to how best to consider those views alongside clinical and economic considerations

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. \u27End-of-life treatments\u27 and \u27rare disease therapies\u27 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

Omecamtiv mecarbil in chronic heart failure with reduced ejection fraction, GALACTIC‐HF: baseline characteristics and comparison with contemporary clinical trials

Aims: The safety and efficacy of the novel selective cardiac myosin activator, omecamtiv mecarbil, in patients with heart failure with reduced ejection fraction (HFrEF) is tested in the Global Approach to Lowering Adverse Cardiac outcomes Through Improving Contractility in Heart Failure (GALACTIC‐HF) trial. Here we describe the baseline characteristics of participants in GALACTIC‐HF and how these compare with other contemporary trials. Methods and Results: Adults with established HFrEF, New York Heart Association functional class (NYHA) ≥ II, EF ≤35%, elevated natriuretic peptides and either current hospitalization for HF or history of hospitalization/ emergency department visit for HF within a year were randomized to either placebo or omecamtiv mecarbil (pharmacokinetic‐guided dosing: 25, 37.5 or 50 mg bid). 8256 patients [male (79%), non‐white (22%), mean age 65 years] were enrolled with a mean EF 27%, ischemic etiology in 54%, NYHA II 53% and III/IV 47%, and median NT‐proBNP 1971 pg/mL. HF therapies at baseline were among the most effectively employed in contemporary HF trials. GALACTIC‐HF randomized patients representative of recent HF registries and trials with substantial numbers of patients also having characteristics understudied in previous trials including more from North America (n = 1386), enrolled as inpatients (n = 2084), systolic blood pressure < 100 mmHg (n = 1127), estimated glomerular filtration rate < 30 mL/min/1.73 m2 (n = 528), and treated with sacubitril‐valsartan at baseline (n = 1594). Conclusions: GALACTIC‐HF enrolled a well‐treated, high‐risk population from both inpatient and outpatient settings, which will provide a definitive evaluation of the efficacy and safety of this novel therapy, as well as informing its potential future implementation

The selection and reimbursement of new anti-cancer drugs in Australia: The nature of oncologist and public preferences for decision-making purposes

The studies that form the major part of this thesis examine the views and attitudes of the Pharmaceutical Benefits Advisory Committee (PBAC), Australian oncologists and the general public to gather information on the value perceptions of these stakeholders in order to inform the adequacy of the current evaluative framework for the assessment and valuation of anti-cancer drugs. The decision criteria used for the selection and reimbursement of anti-cancer drugs by the PBAC, pan-Canadian Oncology Drug Review (pCODR) and National Institute of Health and Care Excellence (NICE) were reviewed. A systematic literature review was conducted on the preferences/priorities of patients, the general public and payers for anti-cancer drugs. A literature review on the oncologists’ views and preferences on the benefits, harms, costs and value of anti-cancer drugs was also conducted. An analysis of the PBAC recommendations revealed that the PBAC appears to apply decision criteria equally to anti-cancer and non-cancer drugs in that anti-cancer drugs are neither favoured nor disadvantaged. A web-based survey was conducted to elicit Australian oncologists’ preferences for different treatment attributes of two anti-cancer drugs (bevacizumab and everolimus). A cross sectional survey was conducted to explore societal preferences for government spending on medicines and views on distributional preferences based on 12 allocation criteria relevant to the Australian PBS. Findings arising from the studies presented in this thesis show that the general public are willing and capable of giving opinions on distributional preferences, sharing their views on what constitute an equitable allocation of the government’s drug budget, and that the perceived value of anti-cancer drugs can be influenced by oncologists’ preferences for different treatment attributes. To enable integration of public and patient preferences into health technology assessment (HTA) decisions, further research should focus on defining a strategy to incorporate public perspectives (patients and the general public) into funding decisions of anti-cancer drugs with a view to reconciling the different value perceptions among stakeholders to ensure that the general public/patient values and preferences are reflected in HTA assessment and recommendations

Weighing up the benefits and harms of a new anti-cancer drug: A survey of Australian oncologists

Background: Little is known about the relative importance that oncologists attribute to the benefits and harms of anti-cancer drugs when considering treatment options with their patients. Aim: To quantify the trade-offs made between overall survival, progression-free survival and adverse effects. Methods: A web-based survey elicited importance weights for the benefits and harms of bevacizumab or everolimus. Combining the importance weights with trial-based probabilities produced a score and ranking for each treatment option. Results: A total of 40 responses was received for the bevacizumab scenario and 32 for the everolimus scenario. All respondents regarded overall survival and progression-free survival as the most important attributes - more important than avoiding the potential harms regardless of drugs. Among the potential harms, respondents allocated the highest mean importance weight to gastrointestinal (GI) perforation and rated absolute improvement in overall survival as 1.6 times and 2.3 times as important as avoiding GI perforation in the two versions of the bevacizumab scenario respectively. For the everolimus scenario, stomatitis and pneumonitis were allocated the highest mean importance weights with absolute improvement in overall survival rated as 2.2 times as important as avoiding stomatitis/pneumonitis. All 40 respondents (100%) favoured treatment option with bevacizumab to no bevacizumab based on respondents\u27 determined weights for treatment attributes. The converse was found for everolimus with 22 (69%) of respondents preferring the \u27no everolimus\u27 option. Conclusion: Oncologists\u27 preferences over the benefits and harms of treatment do, when combined with evidence of effect, influence treatment decisions for anti-cancer drugs

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 \u3c 0.25) with PBAC approval, but only type of application, economic modelling and estimated cost to the PBS remained statistically significant at p \u3c 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 \u3c 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 = 0.01). 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 noncancer 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. An erratum to this article is available at http://dx.doi.org/10.2165/11585540-000000000-00000

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 \u3c 0.001); PBAC judged the economic evidence to be problematic or uncertain (P \u3c 0.001); and, inactive comparator used (P \u3c 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

Respondents’ preferences by scenarios: (1) all else being equal and (2) benefit and cost trade-offs.

<p>Respondents’ preferences by scenarios: (1) all else being equal and (2) benefit and cost trade-offs.</p