Cost-Effectiveness of Erythropoietin in Traumatic Brain Injury: A Multinational Trial-Based Economic Analysis.

The EPO-TBI multi-national randomized controlled trial found that erythropoietin (EPO), when compared to placebo, did not affect 6-month neurological outcome, but reduced illness severity-adjusted mortality in patients with traumatic brain injury (TBI), making the cost-effectiveness of EPO in TBI uncertain. The current study uses patient-level data from the EPO-TBI trial to evaluate the cost-effectiveness of EPO in patients with moderate or severe TBI from the healthcare payers' perspective. We addressed the issue of transferability in multi-national trials by estimating costs and effects for specific geographical regions of the study (Australia/New Zealand, Europe, and Saudi Arabia). Unadjusted mean quality-adjusted life-years (QALYs; 95% confidence interval [CI]) at 6 months were 0.027 (0.020-0.034; p < 0.001) higher in the EPO group, with an adjusted QALY increment of 0.014 (0.000-0.028; p = 0.04). Mean unadjusted costs (95% CI) were $US5668 (-9191 to -2144; p = 0.002) lower in the treatment group; controlling for baseline IMPACT-TBI score and regional heterogeneity reduced this difference to $2377 (-12,446 to 7693; p = 0.64). For a willingness-to-pay threshold of $US50,000 per QALY, 71.8% of replications were considered cost-effective. Therefore, we did not find evidence that EPO was significantly cost-effective in the treatment of moderate or severe TBI at 6-month follow-up.

Differential item functioning in quality of life measurement: An analysis using anchoring vignettes.

Systematic differences in the ways that people use and interpret response categories (differential item functioning, DIF) can introduce bias when using self-assessments to compare health or quality of life across heterogeneous groups. This paper reports on an exploratory analysis involving the use of anchoring vignettes to identify DIF in a commonly used measure for assessing health-related quality of life - namely the EQ-5D. Using data from a bespoke (i.e. custom) survey that recruited a representative sample of 4300 respondents from the general Australian population in 2014 and 2015, we find that the assumptions of response consistency (RC) and vignette equivalence (VE) hold in a sub-sample of respondents aged 55-65 years (n = 914), which demonstrates that vignettes can appropriately identify DIF in EQ-5D reporting for this age group. We find that the EQ-5D is indeed subject to DIF, and that failure to account for DIF can lead to conclusions that are misleading when using the instrument to compare health or quality of life across heterogeneous groups. We also provide several important insights in terms of the identifying assumptions of RC and VE. We conclude that the implications of DIF could be of considerable importance, not only for outcomes research, but for funding decisions in healthcare more broadly given the strong reliance on patient-reported outcome measures in economic evaluations for health technology assessment.

Response-Scale Heterogeneity in the EQ-5D.

This paper discusses two types of response-scale heterogeneity, which may impact upon the EQ-5D. Response-scale heterogeneity in reporting occurs when individuals systematically differ in their use of response scales when responding to self-assessments. This type of heterogeneity is widely observed in relation to other self-assessed measures but is often overlooked with regard to the EQ-5D. Analogous to this, preference elicitation involving the EQ-5D could be subject to a similar type of heterogeneity, where variations across respondents may occur in the interpretations of the levels (response categories) being valued. This response-scale heterogeneity in preference elicitation may differ from variations in preferences for health states, which have been observed in the literature. This paper explores what these forms of response-scale heterogeneity may mean for the EQ-5D and the potential implications for researchers who rely on the instrument as a measure of health and quality of life. We identify situations where they are likely to be problematic and present potential avenues for overcoming these issues. Copyright © 2016 John Wiley & Sons, Ltd.

Realising the Value of Linked Data to Health Economic Analyses of Cancer Care: A Case Study of Cancer 2015.

There is a growing appetite for large complex databases that integrate a range of personal, socio-demographic, health, genetic and financial information on individuals. It has been argued that 'Big Data' will provide the necessary catalyst to advance both biomedical research and health economics and outcomes research. However, it is important that we do not succumb to being data rich but information poor. This paper discusses the benefits and challenges of building Big Data, analysing Big Data and making appropriate inferences in order to advance cancer care, using Cancer 2015 (a prospective, longitudinal, genomic cohort study in Victoria, Australia) as a case study. Cancer 2015 has been linked to State and Commonwealth reimbursement databases that have known limitations. This partly reflects the funding arrangements in Australia, a country with both public and private provision, including public funding of private healthcare, and partly the legislative frameworks that govern data linkage. Additionally, linkage is not without time delays and, as such, achieving a contemporaneous database is challenging. Despite these limitations, there is clear value in using linked data and creating Big Data. This paper describes the linked Cancer 2015 dataset, discusses estimation issues given the nature of the data and presents panel regression results that allow us to make possible inferences regarding which patient, disease, genomic and treatment characteristics explain variation in health expenditure.

The effects of reduced copayments on discontinuation and adherence failure to statin medication in Australia.

This paper assesses whether the concession card, which offers discounted out-of-pocket costs for prescription medicines in Australia, affects discontinuation and adherence to statin therapy. The analysis uses data from the Australian Hypertension and Absolute Risk Study (AusHEART), which involves patients aged 55 years and over who visited a GP between April and June 2008. Socioeconomic and clinical information was collected and linked to administrative data on pharmaceutical use. Patients without a concession card were 63% more likely (hazard ratio (HR) 95% confidence interval (CI): 1.14-2.33) to discontinue and 60% (odds ratio (OR) CI: 1.04-2.44) more likely to fail to adhere to therapy compared to concessional patients. Smokers were 2.12 (HR CI: 1.39-3.22) times more likely to discontinue use and 2.23 (OR CI: 1.35-3.71) times more likely to fail to adhere compared to non-smokers. Patients who had recently initiated statin medication were also 2.28 (HR CI: 1.22-4.28) times more likely to discontinue use. In conclusion, higher copayments act as a disincentive for persistent and adherent use of statin medication.

How fair is Medicare? The income-related distribution of Medicare benefits with special focus on chronic care items.

OBJECTIVE: To use patient-level data, clinical information and linked Medicare records to assess the distribution of benefits (rebates) across income groups, including benefits relating to chronic conditions such as the Chronic Disease Dental Scheme (CDDS). DESIGN, SETTING AND PARTICIPANTS: Nationally representative, cluster-stratified survey (the Australian Hypertension and Absolute Risk Study) involving 322 general practitioners who each collected clinical data on 15-20 patients aged≥55,2012s who presented between 1 April 2008 and 30 June 2008 and who consented to have their information linked with Medicare administrative records over 12 months. MAIN OUTCOME MEASURES: Distribution of total out-of-hospital Medicare expenditure quantified using concentration indices and determinants of use calculated by odds ratios. RESULTS: There were 2862 patients in the study. After controlling for need, the concentration index for overall funding was slightly progressive (pro-poor) at -0.008 (95% CI, -0.009 to -0.008). Medicare expenditure on chronic care-related services consistently contributed to progressivity of the overall scheme, particularly services under the CDDS with a need-adjusted concentration index of -0.205 (95% CI, -0.208 to -0.201). Uptake of chronic care items varied by locality and comorbid conditions (there was greater uptake by patients with one or more comorbid conditions). CONCLUSIONS: Chronic care items, particularly dental items, have primarily been used by individuals from lower income households. Uptake of chronic care items contributes to the overall progressivity of Medicare.