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Prostate-specific membrane antigen (PSMA) positron emission tomography/computed tomography (PET/CT) has emerged as a promising imaging tool in prostate cancer diagnosis. PSMA PET/CT radiotracers are produced in-house (gallium-68, eg,Hospitals and diagnostic facilities currently conducting PSMA PET/CT in Australia in metropolitan and regional areas completed a survey of PSMA PET/CT throughput, radiotracers involved, and the cost of assets, departmental staffing, consumables, and occupancy. Total costs were estimated using a top-down microcosting approach, involving identifying all relevant cost components and valuing each component for the average patient, and a gross costing approach, involving apportioning cost components at an aggregated level.Data were collected from 8 facilities. The most common radiotracer used wasThis study provides a detailed and accurate estimation of the cost of PSMA PET/CT in Australia. These costs can be used as a benchmark to identify potential efficiencies and help policy makers set the appropriate reimbursement rate for this procedure. The use of data from facilities using different radiotracers in metropolitan and regional areas and with different throughput increases the generalizability of the results, especially in countries with similar health systems.

BACKGROUND AND OBJECTIVES: Prostate-specific membrane antigen (PSMA) positron emission tomography (PET) combined with computed tomography (CT) is a new imaging modality to detect the extra-prostatic spread of prostate cancer. PSMA PET/CT has a higher sensitivity and specificity than conventional imaging (CT ± whole body bone scan [WBBS]). This study conducted a cost-utility analysis of PSMA PET/CT compared with conventional imaging for patients with newly diagnosed, intermediate-risk or high-risk primary prostate cancer. PERSPECTIVE: Australian healthcare perspective. SETTING: Tertiary. METHODS: A decision-analytic Markov model combined data from a variety of sources. The time horizon was 35 years. The sensitivity and specificity of PSMA PET/CT and CT alone were based on meta-analyses and the test accuracy of CT+WBBS was based on a single randomised controlled trial. Health outcomes included cases detected, life-years, and quality-adjusted life-years. Costs related to other diagnostic tests, initial treatment, adverse events, and post-disease progression were included. All costs were reported in 2021 Australian Dollars (A$). RESULTS: The deterministic incremental cost-effectiveness ratio of PSMA PET/CT was estimated to be A $21,147/quality-adjusted life-year gained versus CT+WBBS, and A$36,231/quality-adjusted life-year gained versus CT alone. The results were most sensitive to the time horizon, and the initial treatments received by patients diagnosed with metastatic cancer. The probability of PSMA PET/CT being cost effective was estimated to be 91% versus CT+WBBS and 89% versus CT alone, using a threshold of AU$50,000/quality-adjusted life-year gained. CONCLUSIONS: PSMA PET/CT is likely to be more costly than CT+WBBS or CT alone in Australia; however, it is still likely to be considered cost effective compared with conventional imaging.

Objective The National Institute for Health and Care Excellence (NICE) updated its eligibility criteria for unilateral cochlear implants (UCIs) in 2019. NICE claimed this would not impact the cost-effectiveness results used within its 2009 technology appraisal guidance. This claim is uncertain given changed clinical practice and increased healthcare unit costs. Our objective was to estimate the cost-effectiveness estimates of UCIs in UK adults with severe to profound hearing loss within the contemporary NHS environment. Methods A cost–utility analysis employing a Markov model was undertaken to compare UCIs with hearing aids or no hearing aids for people with severe to profound hearing loss. A clinical pathway was developed to estimate resource use. Health-related quality of life, potential adverse events, device upgrades and device failure were captured. Unit costs were derived mostly from the NHS data. Probabilistic sensitivity analysis further assessed the effect of uncertain model inputs. Results A UCI is likely to be deemed cost-effective when compared to a hearing aid (£11,946/QALY) or no hearing aid (£10,499/QALY). A UCI has an 93.0% and 98.7% likelihood of being cost-effective within the UK adult population when compared to a hearing aid or no hearing aid, respectively. ICERs were mostly sensitive to the proportion of people eligible for cochlear implant, discount rate, surgery and device costs and processor upgrade cost. Conclusion UCIs remain cost-effective despite changes to clinical practice and increased healthcare unit costs. Updating the NICE criteria to provide better access UCIs is projected to increase annual implants in adults and children by 70% and expenditure by £28.6 million within three years. This increased access to UCIs will further improve quality of life of recipients and overall social welfare.

Background Patients with Lynch syndrome (an inherited cancer predisposition syndrome) remain largely underdiagnosed despite clinically and cost-effective testing strategies to detect patients. This is largely due to poor referral rates for high-risk patients for consideration of genetic testing. Targeted approaches to improve the implementation of guidelines and thus uptake rates of genetic testing require the use of limited and valuable healthcare resources. Decision makers must carefully balance the potential health impacts of implementation approaches against the associated costs, similar to when assessing the direct impact of health interventions. This protocol outlines the methods used to conduct an economic evaluation of different implementation approaches aimed at improving referral rates of high-risk patients, including estimating implementation approach costs. Methods A cluster randomised controlled trial (the Hide and Seek Project, HaSP) is underway to compare two different implementation approaches aimed at improving referral rates, and thus detection, of Lynch syndrome among colorectal cancer patients across eight Australian hospital networks. An in-depth process evaluation is being conducted alongside the trial and includes measures to collect comprehensive data on both implementation and intervention costs. These costs, in addition to HaSP outcome data, will be incorporated as inputs into an existing microsimulation model—Policy1-Lynch—to project the downstream economic and health impacts and determine the more cost-effective implementation approach from the Australian healthcare perspective. Discussion The ability to model the impact of different implementation approaches will enable the most efficient way of improving Lynch syndrome detection. The approach used in this study could also be applied to assess other implementation approaches aimed at increasing the uptake of cost-effective health interventions. Trial registration ANZCTR, ACTRN12618001072202. Registered on 27 June 2018.

The Patient-Reported Outcomes Measurement Information System (PROMIS-29) is gaining popularity as healthcare system funders increasingly seek value-based care. However, it is limited in its ability to estimate utilities and thus inform economic evaluations. This study develops the first mapping algorithm for estimating EuroQol 5-Dimension 5-Level (EQ-5D-5L) utilities from PROMIS-29 responses using a large dataset and through extensive comparisons between econometric models.An online survey was conducted to collect responses to PROMIS-29 and EQ-5D-5L from the general Australian population (N = 3013). Direct and indirect mapping methods were explored, including linear regression, Tobit, generalised linear model, censored regression model, beta regression (Betamix), the adjusted limited dependent variable mixture model (ALDVMM) and generalised ordered logit. The most robust model was selected by assessing the performance based on average ten-fold cross-validation geometric mean absolute error and geometric mean squared error, the predicted mean, maximum and minimum utilities, as well as the fitting across the entire distribution.The direct approach using ALDVMM was considered the preferred model based on lowest geometric mean absolute error and geometric mean squared error in cross-validation (0.0882, 0.0299) and its superiority in predicting the actual observed mean, full health states and lower utility extremes. The robustness and precision in prediction across the entire distribution of utilities with ALDVMM suggest it is an accurate and valid mapping algorithm. Moreover, the suggested mapping algorithm outperformed previously published algorithms using Australian data, indicating the validity of this model for economic evaluations.This study developed a robust algorithm to estimate EQ-5D-5L utilities from PROMIS-29. Consistent with the recent literature, the ALDVMM outperformed all other econometric models considered in this study, suggesting that the mixture models have relatively better performance and are an ideal candidate model for mapping.

Objectives While all newborns in Australia are tested for congenital hearing loss through universal newborn hearing screening programs, some children will acquire hearing loss in their first five years of life. Delayed diagnosed or undiagnosed hearing loss in children can have substantial immediate- and long-term consequences. It can significantly reduce school readiness, language and communication development, social and emotional development, and mental health. It can also compromise lifetime educational achievements and employment opportunities and future economic contribution to society through lost productivity. The need for a universal hearing screening program for children entering their first year of primary school has been noted in two separate Australian Government hearing inquiries in the last decade. Sound Scouts is a hearing screening application (app) that tests for hearing loss in children using a tablet or mobile device, supervised by parents at home. It tests for sensorineural or permanent conductive hearing loss and central auditory processing disorder in children. In 2018 the Australian Government funded the roll-out of Sound Scouts to allow up to 600,000 children to test their hearing using Sound Scouts. This study estimated the cost-effectiveness of screening 5-year-old children for hearing loss using Sound Scouts at home, compared with no screening. Design A decision-analytic model was developed to estimate the incremental costs and quality-adjusted life years (QALYs) of administering Sound Scouts over a 20-year time horizon. Testing accuracy was based on comparing Sound Scouts test results to clinical test results while other parameters were based on published data. Costs were estimated from the perspective of the Australian health care system. Univariate and probabilistic sensitivity analyses were undertaken. Results Sound Scouts is estimated to result in an average incremental cost of A$61.02 and an average incremental increase in QALYs of 0.01. This resulted in an incremental cost-effectiveness ratio of A$5392 per QALY gained, which is likely to be considered cost-effective by Australian decision makers. Screening with Sound Scouts was found to have a 96.2 per cent probability of being cost-effective using a threshold of A$60,000 per QALY gained. Conclusions Using Sound Scouts to screen five-year-old children for hearing loss (at home) is likely to be cost-effective. Screening children using Sound Scouts will result in early identification and intervention in childhood hearing loss, thereby reducing early childhood disadvantage through cumulative gains in quality of life, education, and economic outcomes over their lifetime.

OBJECTIVES: Hearing loss is one of the most prevalent congenital disorders among children. Many countries have implemented Universal Newborn Hearing Screening (UNHS) for early diagnosis and treatment of hearing loss. Despite widespread implementation, the value for money of UNHS is unclear due to lack of cost and outcomes data from rigorous study designs. The objective of this research is to conduct a within-study cost-effectiveness analysis of UNHS compared with targeted screening (targeting children with risk factors of hearing loss) from the Australian healthcare system perspective. This evaluation is the first economic evaluation to assess the cost-effectiveness of UNHS compared to targeted screening using real-world data from a natural experiment. DESIGN: The evaluation assumed the Australian healthcare system perspective and considered a time horizon of five years. Utilities were estimated using responses to the Health Utilities Index Mark III. Screening costs were estimated based on the Victorian Infant Hearing Screening Program. Ongoing costs were estimated based on administrative data, while external data sources were used to estimate costs related to hearing services. Missing data were handled using the multiple imputation method. Outcome measures included Quality-adjusted Life Years (QALYs) and four language and communication-related outcomes: Peabody Picture Vocabulary Test, Wechsler Nonverbal scale of ability, Progressive Achievement Test, and comprehensive, expressive, and total language scores based on the Preschool Language Scale. RESULTS: On average, the UNHS cost an extra Australian dollar (A$)22,000 per diagnosed child and was associated with 0.45 more QALYs per diagnosed child compared with targeted screening to 5 years, resulting in an Incremental Cost-Effectiveness Ratio (ICER) of A$48,000 per QALY gained. Higher screening costs in the UNHS arm were the main drivers of the results. The ICERs for language outcomes lay between A$3,900 (for expressive language score) and A$83,500 per one-point improvement in language score (i.e., for Wechsler Nonverbal scale of ability). UNHS had a 69% probability of being more cost-effective compared to targeted screening at a willingness to pay threshold of A$60,000 per QALY gained. ICERs were most sensitive to the screening costs. CONCLUSIONS: The evaluation demonstrated the usefulness of a within-study economic evaluation to understand the value for money of the UNHS program in the Australian context. Findings from this evaluation suggested that screening costs were the key driver of cost-effectiveness results. Most outcomes were not significantly different between UNHS and targeted screening groups. The ICER may be overestimated due to the short follow-up period. Further research is warranted to include long-term resource use and outcome data, late diagnosis, transition and remission between severity levels, and timing of diagnosis and treatment.

Non-preference-based patient-reported outcome measures (PROMs) are popular in health outcomes research. These measures, however, cannot be used to estimate health state utilities, limiting their usefulness for economic evaluations. Mapping PROMs to a multi-attribute utility instrument is one solution. While mapping is commonly conducted using econometric techniques, failing to specify the complex interactions between variables may lead to inaccurate prediction of utilities, resulting in inaccurate estimates of cost-effectiveness and suboptimal funding decisions. These issues can be addressed using machine learning. This paper evaluates the use of machine learning as a mapping tool. We adopt a comprehensive approach to compare six machine learning techniques with eight econometric techniques to map the Patient-Reported Outcomes Measurement Information System Global Health 10 (PROMIS-GH10) to the EuroQol five dimensions (EQ-5D-5L). Using data collected from 2015 Australians, we find the least absolute shrinkage and selection operator (LASSO) model out-performed all machine learning techniques and the adjusted limited dependent variable mixture model (ALDVMM) out-performed all econometric techniques, with the LASSO performing better than ALDVMM. The variable selection feature of LASSO was then used to enhance the performance of the ALDVMM in a hybrid model. Our analysis identifies the potential benefits and challenges of using machine learning techniques for mapping and offers important insights for future research.

© 2019 ISPOR–The Professional Society for Health Economics and Outcomes Research Background: Migraine is a common, chronic, disabling headache disorder. Triptans, used as an acute treatment for migraine, are available via prescription in Australia. An Australian Therapeutic Goods Administration (TGA) committee rejected reclassifying sumatriptan and zolmitriptan from prescription medicine to pharmacist-only between 2005 and 2009, largely on the basis of concerns about patient risk. Nevertheless, pharmacist-only triptans may reduce migraine duration and free up healthcare resources. Objectives: To estimate the cost-effectiveness of reclassifying triptans from prescription-only to pharmacist-only in Australia. Methods: The study design included decision-analytic modeling combining data from various sources. Behavior before and after reclassification was estimated using medical practitioner and patient surveys and also administrative data. Health outcomes included migraine frequency and duration as well as adverse events (AEs) discussed by the TGA committee. Efficacy and AEs were estimated using randomized controlled trials and observational studies. Results: Reclassifying triptans will reduce migraine duration but increase AEs. This will result in 337 quality-adjusted life-years gained at an increased cost of A$5.9 million over 10 years for all Australian adults older than 15 years (19.6 million). The incremental cost-effectiveness ratio was estimated to be A$17 412/quality-adjusted life-year gained. Conclusions: The incremental cost-effectiveness ratio is likely to be considered cost-effective by Australian decision makers. Serotonin syndrome, a key concern of the TGA committee, had little impact on the results. Further research is needed regarding pharmacist-only triptan use by migraineurs currently using over-the-counter medicines and by nonmigraineurs, the efficacy of triptans, and the risk of cardiovascular and cerebrovascular AEs and chronic headaches with triptans.

Initially patients require a prescription to access most new medicines. Some medicines may later be reclassified, allowing patients to access them without a prescription. Currently, Australian Therapeutic Goods Administration guidelines regarding reclassification decisions focus on patient risk rather than on potential benefits to patient health and the healthcare system. We conducted two extensive case studies demonstrating an economic evaluation approach to medicine reclassification in Australia, which were presented at various conferences and to key stakeholders. This article discusses the advantages and challenges of using an economic evaluation approach to inform medicine reclassification decisions. Advantages identified include systematically and transparently synthesising evidence from multiple sources; predicting the overall expected impact of reclassification on health outcomes and costs before it occurs; considering a broader range of risks and benefits; aggregation of health impacts into a single measure (quality-adjusted life years); identification of drivers of uncertainty; insight into the effects of different regulatory decisions; and improved consistency of evidence. Challenges include data availability and quality, estimating behavioural changes, model complexity, the lack of an incremental cost-effectiveness ratio threshold, and funding of economic analyses. We recommend that regulatory decision makers use an economic evaluation approach to help inform reclassification decisions, although economic evaluation results should be considered as part of the broader body of evidence. Ultimately, the use of an economic evaluation approach will contribute to helping decision makers maximise population health outcomes in an efficient way. What is known about the topic? In the past, decisions regarding medicine reclassification have generally been made using a deliberative approach focusing on patient risk. However, there are also potential benefits to patient health and effects on the healthcare system. Increasing awareness of these benefits have led to the development of alternative approaches to decision making, including an economic evaluation approach. What does this paper add? This article discusses the advantages and challenges of using an economic evaluation approach to inform medicine reclassification decisions compared with alternative approaches. What are the implications for practitioners? Economic evaluation results should be considered as part of the broader body of evidence regarding the types of health impacts, the extent of the available evidence, who will be affected, and the role of medical practitioners and pharmacists in mitigating any risks. However, awareness of the advantages and challenges of this approach in advance will help mitigate some of the challenges and increase acceptance of the economic evaluation results by decision makers and stakeholders.

IntroductionWith almost 50% of cases preventable and the Australian National Bowel Cancer Screening Program in place, colorectal cancer (CRC) is a prime candidate for investment to reduce the cancer burden. The challenge is determining effective ways to reduce morbidity and mortality and their implementation through policy and practice. Pathways-Bowel is a multistage programme that aims to identify best-value investment in CRC control by integrating expert and end-user engagement; relevant evidence; modelled interventions to guide future investment; and policy-driven implementation of interventions using evidence-based methods.Methods and analysisPathways-Bowel is an iterative work programme incorporating a calibrated and validated CRC natural history model for Australia (Policy1-Bowel) and assessing the health and cost outcomes and resource use of targeted interventions. Experts help identify and prioritise modelled evaluations of changing trends and interventions and critically assess results to advise on their real-world applicability. Where appropriate the results are used to support public policy change and make the case for optimal investment in specific CRC control interventions. Fourteen high-priority evaluations have been modelled or planned, including evaluations of CRC outcomes from the changing prevalence of modifiable exposures, including smoking and body fatness; potential benefits of daily aspirin intake as chemoprevention; increasing CRC incidence in people aged Pathways-Bowel is a unique, comprehensive approach to evaluating CRC control; no prior body of work has assessed the relative benefits of a variety of interventions across CRC development and progression to produce a list of best-value investments.Ethics and disseminationEthics approval was not required as human participants were not involved. Findings are reported in a series of papers in peer-reviewed journals and presented at fora to engage the community and policymakers.

There are limited economic evaluations comparing coronary artery bypass grafting (CABG) and percutaneous coronary intervention (PCI) for multi-vessel coronary artery disease (MVCAD) in contemporary, routine clinical practice. The aim was to perform a cost-effectiveness analysis comparing CABG and PCI in patients with MVCAD, from the perspective of the Australian public hospital payer, using observational data sources. Clinical data from the Melbourne Interventional Group (MIG) and the Australian and New Zealand Society of Cardiac and Thoracic Surgeons (ANZSCTS) registries were analysed for 1022 CABG (treatment) and 978 PCI (comparator) procedures performed between June 2009 and December 2013. Clinical records were linked to same-hospital admissions and national death index (NDI) data. The incremental cost-effectiveness ratios (ICERs) per major adverse cardiac and cerebrovascular event (MACCE) avoided were evaluated. The propensity score bin bootstrap (PSBB) approach was used to validate base-case results. At mean follow-up of 2.7 years, CABG compared with PCI was associated with increased costs and greater all-cause mortality, but a significantly lower rate of MACCE. An ICER of $55,255 (Australian dollars)/MACCE avoided was observed for the overall cohort. The ICER varied across comparisons against bare metal stents (ICER $25,815/MACCE avoided), all drug-eluting stents (DES) ($56,861), second-generation DES ($42,925), and third-generation of DES ($88,535). Moderate-to-low ICERs were apparent for high-risk subgroups, including those with chronic kidney disease ($62,299), diabetes ($42,819), history of myocardial infarction ($30,431), left main coronary artery disease ($38,864), and heart failure ($36,966). At early follow-up, high-risk subgroups had lower ICERs than the overall cohort when CABG was compared with PCI. A personalised, multidisciplinary approach to treatment of patients may enhance cost containment, as well as improving clinical outcomes following revascularisation strategies.

The Australian Federal Government’s National Medicines Policy aims to create positive health outcomes for all Australians by providing appropriate access to medicines. Underpinning the policy is the Pharmaceutical Benefits Scheme (PBS), which subsidises the cost of medicines. However, like many healthcare systems around the world, governments are struggling with the challenge of how to manage escalating healthcare expenditure. The landscape is dotted with competing agendas: increased demand for healthcare due to population growth and ageing; patient preferences over treatment options; improved technologies and medicines; the need for pharmaceutical companies to recoup research and manufacturing costs—all have been seen as culprits behind a rising national healthcare bill. Further, spending a dollar on one form of healthcare means not spending a dollar on another. In other words, providing healthcare has an opportunity cost. Increasingly, decision-makers are using economic evaluations as a tool to balance these agenda trade-offs. Only through balance can we ensure that healthcare continues to provide value for money and the system remains sustainable. This chapter explains how the PBS works—its decision-making bodies and processes, how economic evaluation is key to recommending which new medicines are listed on the PBS, and the industry’s perspective on the PBS. The case study focuses on the human papillomavirus (HPV) vaccine to highlight the competing resource demands, business interests, and societal needs that influenced its listing on the PBS. The case study also considers how making the vaccine available through the National Immunisation Programme has contributed to making Australia’s healthcare system more sustainable.

Background Coronary artery disease (CAD) remains a major public health problem in Australia and globally. A variety of imaging techniques allow for both anatomical and functional assessment of CAD and selection of the optimal investigation pathway is challenging. Cardiovascular magnetic resonance (CMR) is not widely used in Australia, partly due to perceived cost and lack of Federal Government reimbursement compared to the alternative techniques. The aim of this study was to estimate the cost-effectiveness of different diagnostic strategies in identifying significant CAD in patients with chest pain suggestive of angina using the evidence gathered in the Clinical Evaluation of Magnetic Resonance Imaging in Coronary Heart Disease 2 (CE-MARC trial), analysed from the Australian health care perspective. Methods A decision analytic model coupled with three distinct Markov models allowed eight potential clinical investigation strategies to be considered; combinations of exercise electrocardiogram stress testing (EST), single-photon emission computed tomography (SPECT), stress CMR, and invasive coronary angiography (ICA). Costs were from the Australian health care system in Australian dollars, and outcomes were measured in terms of quality-adjusted life-years. Parameter estimates were derived from the CE-MARC and EUropean trial on Reduction Of cardiac events with Perindopril in patients with stable coronary Artery disease (EUROPA) trials, and from reviews of the published literature. Results The most cost-effective diagnostic strategy, based on a cost-effectiveness threshold of $45,000 to $75,000 per QALY gained, was EST, followed by stress CMR if the EST was positive or inconclusive, followed by ICA if the stress CMR was positive or inconclusive; this held true in the base case and the majority of scenario analyses. Conclusions This economic evaluation shows that an investigative strategy of stress CMR if EST is inconclusive or positive is the most cost-effective approach for diagnosing significant coronary disease in chest pain patients within the Australian health care system.

Background Lynch syndrome (LS) is an inherited, cancer predisposition syndrome associated with an increased risk of colorectal, endometrial and other cancer types. Identifying individuals with LS allows access to cancer risk management strategies proven to reduce cancer incidence and improve survival. However, LS is underdiagnosed and genetic referral rates are poor. Improving LS referral is complex, and requires multisystem behaviour change. Although barriers have been identified, evidence-based strategies to facilitate behaviour change are lacking. The aim of this study is to compare the effectiveness of a theory-based implementation approach against a non-theory based approach for improving detection of LS amongst Australian patients with colorectal cancer (CRC). Methods A two-arm parallel cluster randomised trial design will be used to compare two identical, structured implementation approaches, distinguished only by the use of theory to identify barriers and design targeted intervention strategies, to improve LS referral practices in eight large Australian hospital networks. Each hospital network will be randomly allocated to a trial arm, with stratification by state. A trained healthcare professional will lead the following phases at each site: (1) undertake baseline clinical practice audits, (2) form multidisciplinary Implementation Teams, (3) identify target behaviours for practice change, (4) identify barriers to change, (5) generate intervention strategies, (6) support staff to implement interventions and (7) evaluate the effectiveness of the intervention using post-implementation clinical data. The theoretical and non-theoretical components of each trial arm will be distinguished in phases 4–5. Study outcomes include a LS referral process map for each hospital network, with evaluation of the proportion of patients with risk-appropriate completion of the LS referral pathway within 2 months of CRC resection pre and post implementation. Discussion This trial will determine the more effective approach for improving the detection of LS amongst patients with CRC, whilst also advancing understanding of the impact of theory-based implementation approaches in complex health systems and the feasibility of training healthcare professionals to use them. Insights gained will guide the development of future interventions to improve LS identification on a larger scale and across different contexts, as well as efforts to address the gap between evidence and practice in the rapidly evolving field of genomic research. Trial registration ANZCTR, ACTRN12618001072202. Registered on 27 June 2018. Electronic supplementary material The online version of this article (10.1186/s13063-019-3457-6) contains supplementary material, which is available to authorized users.

Unplanned pregnancies can lead to poorer maternal and child health outcomes. The Australian Therapeutic Goods Administration committee rejected reclassifying a range of oral contraceptive pills (OCPs) from prescription to pharmacist-only medicines in 2015, mainly based on safety concerns. Improving access to OCPs may encourage some women to use contraceptives or switch from other contraceptive methods. However, some adverse events may increase and some women may stop using condoms, increasing their risk of sexually transmitted infections. This study aimed to estimate the cost effectiveness of reclassifying OCPs from prescription to pharmacist-only. Healthcare system. Australian primary care. A Markov model was used to synthesise data from a variety of sources. The model included all Australian women aged 15–49 years (N = 5,644,701). The time horizon was 35 years. Contraceptive use before reclassification was estimated using data from the Household, Income and Labour Dynamics in Australia (HILDA) survey, while survey data informed use after reclassification. Health outcomes included pregnancies, pregnancy outcomes (live birth, miscarriage, stillbirth, ectopic pregnancy and abortion), sexually transmitted infections, adverse events (venous thromboembolism, depression, myocardial infarction and stroke), ovarian cancer cases and quality-adjusted life-years. Costs included those related to general practitioner and specialist consultations, contraceptives and other medicines, pharmacist time, hospitalisations and adverse events. All costs were reported in 2016 Australian Dollars. A 5% discount rate was applied to health outcomes and costs. Reclassifying OCPs resulted in 85.70 million quality-adjusted life-years experienced and costs of $46,910.14 million over 35 years, vs. 85.68 million quality-adjusted life-years experienced and costs of $50,274.95 million with OCPs remaining prescription-only. Thus, reclassifying OCPs was more effective and cost saving. However, a sensitivity analysis found that more research on the probability of pregnancy in women not using contraception and not trying to conceive is needed. Reclassifying OCPs is likely to be considered cost effective by Australian decision makers.

Objective Our study examines the financial cost of lymphedema following a diagnosis of breast cancer and addresses a significant knowledge gap regarding the additional impact of lymphedema on breast cancer survivors. Methods An online national survey was conducted with 361 women who had either breast cancer without lymphedema (BC) (group 1, n = 209) or breast cancer with lymphedema (BC+LE) (group 2, n = 152). Participant recruitment was supported by the Breast Cancer Network Australia and the Australasian Lymphology Association. Results Both breast cancer and lymphedema result in significant out-of-pocket financial costs borne by women. Of patients with BC+LE, 80% indicated that their breast cancer diagnosis had affected them financially compared with 67% in the BC group (P

© 2016, Springer International Publishing Switzerland. Introduction: Estimating the real-world cost-effectiveness of a new drug relies on understanding the differences between clinical trial data (pre-reimbursement) and clinical practice (post-reimbursement). This is important for decision makers when reviewing reimbursement decisions, prices, and considering other drugs for the same condition. Differences can arise from differences in patient characteristics, but also from the availability of new evidence and evolving treatment practices. This paper examines these issues using a case study. Methods: In 2001, the Australian Government funded trastuzumab for the treatment of HER2+ metastatic breast cancer through the Herceptin Program. The administrative arrangements of the Program resulted in rich observational data that captured information about patients treated with trastuzumab between 2001 and 2010 (n = 3830). The dataset included patient characteristics, dispensed medicines, medical service use and date of death. Results: Compared to participants in the clinical trials, patients were older, received more prior chemotherapies and a broader range of co-administered chemotherapies. Treatment practices differed from the clinical trials, but also changed over time. For example, in situ hybridization testing, rather than immunohistochemistry testing, and a three weekly administration schedule, rather than one weekly, were increasingly used. Compared to the clinical trials, patients administered trastuzumab with a concomitant chemotherapy generally experienced longer overall survival (151.3 weeks, 95 % CI: 142.6, 163.4), while those who received trastuzumab as a monotherapy experienced shorter overall survival (94.4 weeks, 95%CI: 86.4, 102.9). These findings may be due to a differing relative treatment effect in clinical practice, but may also be due to a range of other factors. Conclusion: This analysis demonstrates the challenges for decision makers that arise because new evidence and evolving treatment practices create a gap between clinical trial data and real-world clinical practice and outcomes.

IntroductionIn multisite intervention trials, implementation success often varies widely across settings. Process evaluations are crucial to interpreting trial outcomes and understanding contextual factors and causal chains necessary for successful implementation. Lynch syndrome is a hereditary cancer predisposition conferring an increased risk of colorectal, endometrial and other cancer types. Despite systematic screening protocols to identify Lynch syndrome, the condition remains largely underdiagnosed. The Hide and Seek Project (‘HaSP’) is a cluster randomised controlled trial determining the effectiveness of two approaches to improving Lynch syndrome detection at eight Australian hospital networks. To enhance widespread implementation of optimal Lynch syndrome identification, there is a need to understand not only what works, but also why, in what contexts, and at what costs. Here we describe an in-depth investigation of factors influencing successful implementation of procedures evaluated in the HaSP trial.Methods and analysisA mixed-methods, theory-driven process evaluation will be undertaken in parallel to the HaSP trial. Data will include: interviews of Implementation Leads and Lynch syndrome stakeholders, pre–post implementation questionnaires, audio analysis of meetings and focus groups, observation of multidisciplinary team meetings, fidelity checklists and project log analysis. Results will be triangulated and coded, drawing on the Theoretical Domains Framework, Consolidated Framework for Implementation Research and Proctor’s implementation outcomes.Ethics and disseminationUse of a theory-based process evaluation will enhance interpretation and generalisability of HaSP trial findings, and contribute to the implementation research field by furthering understanding of the conditions necessary for implementation success. Ethical approval has been granted and results will be disseminated via publications in peer-reviewed journals and conference presentations. At trial completion, key findings will be fed back to sites to enable refinement of intervention strategies, both in the context of Lynch syndrome and for the possible generalisability of intervention components in other genetic and broader clinical specialties.HaSP trial registration numberAustralian New Zealand Clinical Trials Registry (Identifier: ACTRN12618001072202). Registered 27 June 2018. http://www.ANZCTR.org.au/ACTRN12618001072202.aspx.

BACKGROUND: Permanent childhood hearing loss is one of the most common birth conditions associated with speech and language delay. A hearing screening can result in early detection and intervention for hearing loss. OBJECTIVES: To update and expand previous systematic reviews of economic evaluations of childhood hearing screening strategies, and explore the methodological differences. DATA SOURCES: MEDLINE, Embase, the Cochrane database, National Health Services Economic Evaluation Database (NHS EED), the Health Technology Assessment (HTA) database, and Canadian Agency for Drugs and Technologies in Health’s (CADTH) Grey matters. STUDY ELIGIBILITY CRITERIA, PARTICIPANTS AND INTERVENTIONS: Economic evaluations reporting costs and outcomes for both the intervention and comparator arms related to childhood hearing screening strategies. RESULTS: Thirty evaluations (from 29 articles) were included for review. Several methodological issues were identified, including: few evaluations reported outcomes in terms of quality-adjusted life years (QALYs); none estimated utilities directly from surveying children; none included disutilities and costs associated with adverse events; few included costs and outcomes that differed by severity; few included long-term estimates; none considered acquired hearing loss; some did not present incremental results; and few conducted comprehensive univariate or probabilistic sensitivity analysis. Evaluations published post-2011 were more likely to report QALYs and disability-adjusted life years (DALYs) as outcome measures, include long-term treatment and productivity costs, and present incremental results. LIMITATIONS: We were unable to access the economic models and, although we employed an extensive search strategy, potentially not all relevant economic evaluations were identified. CONCLUSIONS AND IMPLICATIONS: Most economic evaluations concluded that childhood hearing screening is value for money. However, there were significant methodological limitations with the evaluations.

OBJECTIVE To report on the cost-effectiveness of BRECONDA (Breast RECONstruction Decision Aid), a web-based decision aid to facilitate decisions regarding breast reconstruction surgery, with usual care for women with breast cancer. METHODS The economic evaluation was conducted alongside a randomized controlled trial. Women diagnosed with breast cancer or ductal carcinoma in situ and eligible for breast reconstruction following mastectomy were randomized to access BRECONDA for 6 months + usual care (n = 106) or usual care (n = 116) and were assessed at baseline preintervention, and then 1-month and 6-months post-randomization. Decisional conflict, satisfaction with information, decisional regret, and utilities were assessed by using maximum-likelihood linear mixed effects models. Costs included the fixed costs of BRECONDA, health care provider time, and health care resource use. Nonparametric bootstrapping was used to estimate incremental cost-effectiveness ratios. RESULTS BRECONDA resulted in significantly less decisional conflict and greater satisfaction with information over time. Quality-adjusted life years did not differ between participants who received the decision aid compared with usual care. The cost of BRECONDA was estimated to be small (AUD$10) relative to other health care interventions and resulted in decreased health care costs overall (AUD$764). Based on the point estimates, the decision aid was more effective and less costly (dominant) for all measures of effectiveness. It was estimated that the decision aid has an 87% probability of being cost-effective at $60 000 per quality-adjusted life year gained. CONCLUSIONS The BRECONDA web-based intervention designed to facilitate decisions regarding breast reconstruction surgery is likely to be cost-effective compared with usual care for women with breast cancer.

Background: Economic evaluation of mandatory health programmes generally do not consider the utility impact of a loss of consumer choice upon implementation, despite evidence suggesting that consumers do value having the ability to choose. Objectives: The primary aim of this study was to explore whether the utility impact of a loss of consumer choice from implementing mandatory health programmes can be measured using discrete choice experiments (DCEs). Methods: Three case studies were used to test the methodology: fortification of bread-making flour with folate, mandatory influenza vaccination of children, and the banning of trans-fats. Attributes and levels were developed from a review of the literature. An orthogonal, fractional factorial design was used to select the profiles presented to respondents to allow estimation of main effects. Overall, each DCE consisted of 64 profiles which were allocated to four versions of 16 profiles. Each choice task compared two profiles, one being voluntary and the other being mandatory, plus a 'no policy' option, thus each respondent was presented with eight choice tasks. For each choice task, respondents were asked which health policy they most preferred and least preferred. Data was analysed using a mixed logit model with correlated coefficients (200 Halton draws). The compensating variation required for introducing a programme on a mandatory basis (versus achieving the same health impacts with a voluntary programme) that holds utility constant was estimated. Results: Responses were provided by 535 participants (a response rate of 83 %). For the influenza vaccination and folate fortification programmes, the results suggested that some level of compensation may be required for introducing the programme on a mandatory basis. Introducing a mandatory influenza vaccination programme required the highest compensation (Australian dollars [A$] 112.75, 95 % CI -60.89 to 286.39) compared with folate fortification (A$18.05, 95 % CI -3.71 to 39.80). No compensation was required for introducing the trans-fats programme (-A$0.22, 95 % CI -6.24 to 5.80) [year 2010 values]. In addition to the type of mandatory health programme, the compensation required was also found to be dependent on a number of other factors. In particular, the study found an association between the compensation required and stronger libertarian preferences. Conclusions: DCEs can be used to measure the utility impact of a loss of consumer choice. Excluding the utility impact of a loss of consumer choice from an economic evaluation taking a societal perspective may result in a sub-optimal, or incorrect, funding decision. © 2013 Springer International Publishing Switzerland.

Objectives To evaluate the cost effectiveness of a community-delivered consultation aimed at improving infant sleep and maternal well-being. Methods A decision-analytic model was developed that compared the costs and benefits of an infant sleep consultation with usual care. The effectiveness of the consultation was based on clinical evidence, and improvements in maternal quality of life were estimated by mapping the Edinburgh Postnatal Depression Scale scores to published utility scores. Cost effectiveness was calculated as the incremental cost per quality-adjusted life-year gained (QALY). Results The statistically significant improvements in mean Edinburgh Postnatal Depression Scale scores at 4- and 16-month follow-ups were used to estimate the benefit in terms of QALYs. The modeled results demonstrated that the infant sleep consultation is low-cost (A$ 436), more effective in terms of QALYs gained (0.017), and cost-effective. The estimated incremental cost-effectiveness ratio was A$ 4031/QALY gained. The main drivers of the model were the use of early parenting centers and nurse training costs. Conclusions Community-based nurse-delivered infant sleep consultations aid infant sleep, improve maternal quality of life, and are cost-effective compared with usual care and lead to improvements in quality of life through a reduction in postnatal depression.

Published economic evaluations of trastuzumab for the treatment of HER2-positive metastatic breast cancer have arrived at different conclusions regarding the cost-effectiveness of trastuzumab, despite comparative efficacy being demonstrated by a small set of randomised controlled trials (RCTs). This article aims to provide insight into the quality of the evaluations and explore the possible drivers of the conflicting conclusions. A systematic literature review was conducted to identify all published economic evaluations that compared the incremental costs and outcomes of trastuzumab versus a comparator. Fifteen economic evaluations were identified. In the evaluations that estimated efficacy using an RCT, the key drivers of the conclusions regarding cost-effectiveness were: the approach used to estimate overall survival in the control group given crossover to trastuzumab following progression in the trials; the inclusion of treatment beyond progression; inclusion of wastage due to unused vial portions, adverse events, and the cost of HER2 testing. Four evaluations used non-randomised approaches to estimate efficacy, thus introducing the potential for confounding. As a result these evaluations reported relatively optimistic estimates of comparative effectiveness. Finally the evaluations used different thresholds to determine whether treatment with trastuzumab was cost-effective. There were numerous drivers of the different conclusions regarding the cost-effectiveness of trastuzumab, many of which are due to judgements made by the authors when translating data from RCTs. Many of the potential drivers were not identified by the published systematic reviews of economic evaluations and perhaps more remain unidentified because of inconsistent and limited reporting.

Background Lower back pain is a common and costly condition in Australia. This paper aims to conduct an economic evaluation of lumbar artificial intervertebral disc replacement (AIDR) compared with lumbar fusion for the treatment of patients suffering from significant axial back pain and/or radicular (nerve root) pain, secondary to disc degeneration or prolapse, who have failed conservative treatment. Methods A cost-effectiveness approach was used to compare costs and benefits of AIDR to five fusion approaches. Resource use was based on Medicare Benefits Schedule claims data and expert opinion. Effectiveness and re-operation rates were based on published randomized controlled trials. The key clinical outcomes considered were narcotic medication discontinuation, achievement of overall clinical success, achievement of Oswestry Disability Index success and quality-adjusted life-years gained. Results AIDR was estimated to be cost-saving compared with fusion overall ($1600/patient); however, anterior lumbar interbody fusion and posterolateral fusion were less costly by $2155 and $807, respectively. The incremental cost-effectiveness depends on the outcome considered and the comparator. Conclusions AIDR is potentially a cost-saving treatment for lumbar disc degeneration, although longer-term follow-up data are required to substantiate this claim. The incremental cost-effectiveness depends on the outcome considered and the comparator, and further research is required before any firm conclusions can be drawn.

Background. The probability of reimbursement is a key factor in determining whether to proceed with or abandon a product during its development. The purpose of this article is to illustrate how the methods of iterative Bayesian economic evaluation proposed in the literature can be incorporated into the development process of new medical devices, adapting them to face the relative scarcity of data and time that characterizes the process. Methods. A 3-stage economic evaluation was applied: an early phase in which simple methods allow for a quick prioritization of competing products; a mid-stage in which developers synthesize the data into a decision model, identify the parameters for which more information is most valuable, and explore uncertainty; and a late stage, in which all relevant information is synthesized. A retrospective analysis was conducted of the case study of absorbable pins, compared with metallic fixation, in osteotomy to treat hallux valgus. Results. The results from the early analysis suggest absorbable pins to be cost-effective under the beliefs and assumptions applied. The outputs from the models at the mid-stage analyses show the device to be cost-effective with a high probability. Late-stage analysis synthesizes evidence from a randomized controlled trial and informative priors, which are based on previous evidence. It also suggests that absorbable pins are the most cost-effective strategy, although the uncertainty in the model output increased considerably. Conclusions. This example illustrates how the method proposed allows decisions in the product development cycle to be based on the best knowledge that is available at each stage.

Prostate cancer (PCa) is the most commonly diagnosed cancer in Australian males. There are limited data on the long-term health system costs associated with PCa. The aim of this study is to estimate long-term health care costs of PCa.We estimated the health system costs for a cohort of 1873 males diagnosed with PCa between 2000 and 2002, using linked medical, pharmaceutical and hospital data. Treatment was defined by an initial phase, measuring health care costs up to 6 months following diagnosis and a continuing phase (including metastatic treatment), measuring treatments to 9.5 years. Nonparametric models were used to calculate average health care costs by PCa risk groups at diagnosis (low to metastatic) and treatment pathways.Health system costs increased with increasing PCa risk category, from $16 923 (low risk) to $39 101 (metastatic risk group). For men with initial localized risk, costs were $8 454 for the active surveillance treatment pathway, $9621 for external beam radiation therapy/brachytherapy, $19 210 for androgen deprivation therapy, $20 636 for radical prostatectomy, $21 161 for radical prostatectomy + external beam radiation therapy/brachytherapy, $21 388 for any of the treatments previously listed + androgen deprivation therapy, with an additional cost of $55 370 if metastatic treatment was undertaken.Treatment costs are highest during two phases over the natural cycle of the disease, the initial diagnosis phase and the metastatic treatment phase. Both the initial phase costs and low-risk category costs are driven largely by the rates of radical prostatectomy. Our study provides comprehensive long-term estimates of PCa costs.

Pharmaceutical expenditure has increased rapidly across many Organisation for Economic Cooperation and Development (OECD) countries over the past three decades. This growth is an increasing concern for governments and other third-party payers seeking to provide equitable and comprehensive healthcare within sustainable budgets. In order to create headroom for increasing utilisation, and to fund new high-cost therapies, there is an active push to ‘disinvest’ from low-value drugs. The aim of this article is to review how reimbursement policy decision makers have sought to partially or completely disinvest from drugs in a range of OECD countries (UK, France, Canada, Australia and New Zealand) where they are publicly funded or subsidised. We employed a systematic literature search strategy and the incorporation of grey literature known to the authorship team. We canvass key policy instruments from each country to outline key approaches to the identification of candidate drugs for disinvestment assessment (passive approaches vs. more active approaches); methods of disinvestment and value-based purchasing (de-listing, restricting treatment, price or reimbursement rate reductions, encouraging generic prescribing); lessons learnt from the various approaches; the potential role of coverage with evidence development; and the need for careful stakeholder management. Dedicated sections are provided with detailed coverage of policy approaches (with drug examples) from each country. Historically, countries have relied on ‘passive disinvestment’; however, due to (1) the availability of new cost-effectiveness evidence, or (2) ‘leakage’ in drug utilisation, or (3) market failure in terms of price competition, there is an increasing focus towards ‘active disinvestment’. Isolating low-value drugs that would create headroom for innovative new products to enter the market is also motivating disinvestment efforts by multiple parties, including industry. Historically, disinvestment has mainly taken the form of price reductions, especially when market failures are perceived to exist, and restricting treatment to subpopulations, particularly when a drug is no longer considered value for money. There is considerable experimentation internationally in mechanisms for disinvestment and the opportunity for countries to learn from each other. Ongoing evaluation of disinvestment strategies is essential, and ought to be reported in the peer-reviewed literature.

Background The last year of life is one of the most resource-intensive periods for people with cancer. Very little population-based research has been conducted on end-of-life cancer care in the Australian health care setting. The objective of this program is to undertake a series of observational studies examining resource use, costs and quality of end-of-life care in a cohort of elderly cancer decedents using linked, routinely collected data. Methods/Design This study forms part of an ongoing cancer health services research program. The cohorts for the end-of-life research program comprise Australian Government Department of Veterans’ Affairs decedents with full health care entitlements, residing in NSW for the last 18 months of life and dying between 2005 and 2009. We used cancer and death registry data to identify our decedent cohorts and their causes of death. The study population includes 9,862 decedents with a cancer history and 15,483 decedents without a cancer history. The median age at death is 86 and 87 years in the cancer and non-cancer cohorts, respectively. We will examine resource use and associated costs in the last 6 months of life using linked claims data to report on health service use, hospitalizations, emergency department visits and medicines use. We will use best practice methods to examine the nature and extent of resource use, costs and quality of care based on previously published indicators. We will also examine factors associated with these outcomes. Discussion This will be the first Australian research program and among the first internationally to combine routinely collected data from primary care and hospital-based care to examine comprehensively end-of-life care in the elderly. The research program has high translational value, as there is limited evidence about the nature and quality of care in the Australian end-of-life setting. Electronic supplementary material The online version of this article (doi:10.1186/s13012-014-0148-2) contains supplementary material, which is available to authorized users.

The Commonwealth Government of Australia has subsidised access to drugs since 1948 via the Pharmaceutical Benefits Scheme (PBS). Through the PBS, the Commonwealth Government aims to provide affordable, timely and equitable access to necessary medicines at an affordable cost to the Government. The PBS is one of the three pillars of government funding of the Australian health system. The other two pillars are free treatment in public hospitals, funded jointly by the Commonwealth Government and State and Territory governments, and the Medicare Benefits Scheme (MBS) where the Commonwealth Government subsidises consultations with clinicians occurring out of hospital. Pharmaceutical policy today in Australia is complex as a result of multiple reforms implemented over a number of years. These reforms introduced or changed pre-existing mechanisms with the aim to control prices and manage demand, and thus control expenditure, while maintaining equitable access. However some policies conflict and some result in unintentional, and sometimes detrimental, incentives. Section 1 provides some historical background to the PBS and explains the avenues through which patients can access PBS-subsidised drugs. Section 2 discusses PBS expenditure in the context of total expenditure on drugs in Australia, the high rate of growth in PBS expenditure over time, and the key drivers of the high rate of growth. Section 3 describes the role of the Pharmaceutical Benefits Advisory Committee, focussing on the process used to evaluate the drug, the drivers of PBAC decisions, and issues faced when reviewing currently listed drugs. Section 4 lists several mechanisms that can be used to minimise uncertainty and reduce the risk of making an incorrect decision. Finally Section 5 describes the methods used by the Australian Department of Health and Ageing (DoHA) to manage the costs and demand for, and thus affect uptake of, drugs once listed on the PBS.

The Australian government recently introduced mandatory folic acid fortification of bread to reduce the incidence of neural tube defects (NTDs). The economic evaluation of this policy contained a number of limitations. This study aimed to address the limitations and to reconsider the findings. Cost-effectiveness analysis was used to assess the cost and benefits of mandatory versus voluntary folic acid fortification. Outcomes measures were quality-adjusted life-years (QALYs), lifeyears gained (LYG), avoided NTD cases, and additional severe neuropathy cases. Costs considered included industry costs and regulatory costs to the government. It was estimated that mandatory fortification would prevent 31 NTDs, whereas an additional 14 cases of severe neuropathy would be incurred. Overall, 539 LYG and 503 QALYs would be gained per year of mandatory compared with voluntary fortification. Mandatory fortification was cost-effective at A$10,723 per LYG and at A$11,485 per QALY. Probabilistic sensitivity analysis showed that at A$60,000 and A$151,000 per QALY, the probability that mandatory fortification was the most cost-effective strategy was 79% and 85%, respectively. Threshold analysis of loss of consumer choice indicated that with a compensation value above A$1.21 [assuming a willingness to pay (WTP) threshold of A$60,000 per QALY] or A$3.19 (assuming a WTP threshold of A$151,000 per statistical life-year) per capita per year mandatory fortification would not be cost-effective. Mandatory fortification was found to be cost-effective; however, inclusion of the loss of consumer choice can change this result. Even with mandatory fortification, mean folate intake will remain below the recommended NTD preventive level. © 2013 American Society for Nutrition.

Objective: Governments are increasing their focus on mandatory public health programmes following positive economic evaluations of their impact. This review aims to examine whether loss of consumer choice should be included in economic evaluations of mandatory health programmes (MHP). Method: A systematic literature review was conducted to identify economic evaluations of MHP, whether they discuss the impact on consumer choice and any methodological limitations. Results: Overall 39 economic evaluations were identified, of which 10 discussed the loss of consumer choice and 6 attempted to place a value on the loss of consumer choice. Methodological limitations included: measuring the marginal cost of compliance, unavailability of price elasticity estimates, the impact of income effects, double counting health impacts, biased willingness-to-pay responses, and "protest" responses. Overall it was found that the inclusion of the loss of consumer choice rarely impacted on the final outcome of the study. Conclusion: The impact of MHP on the loss of consumer choice has largely been ignored in economic evaluations. Its importance remains uncertain due to its infrequent inclusion and significant methodological limitations. Further research regarding which methodology is best for valuing the loss of consumer choice and whether it is important to the final implementation decision is warranted. © 2010 Elsevier Ireland Ltd.