Applied Health Economics and Health Policy

Correction to: Economic Evaluations of Childhood Hearing Loss Screening Programmes: A Systematic Review and Critique The third author’s first name should have been spelled “Teresa” rather than “Theresa”.

Reducing Drug Wastage in Pharmaceuticals Dosed by Weight or Body Surface Areas by Optimising Vial Sizes Abstract Background When pharmaceuticals are administered based on patient characteristics (for example weight or body surface area), an amount of product will be unused and must be disposed of. This wastage represents inefficiency and can distort decision making. Methods We present a method for the analysis of optimum fill volumes of pharmaceuticals to minimise wastage across a patient population, using publicly available data. Wastage for patients at each ‘step’ e.g. by kg of bodyweight is calculated, the frequency of each of these steps in the structure of the population is then estimated using the method of moments, with wastage then estimated for each ‘step’ multiplied by its prevalence. Illustrative examples of pembrolizumab and cabazitaxel show how wastage could be reduced using UK population data, whilst simultaneously reducing administrative burden. Results Changing the available vial sizes for pembrolizumab (available as 50 mg/100 mg vials) to 70 mg/100 mg, wastage could be cut from 13.3% to 8.7%. For cabazitaxel (only 60 mg vials available), increasing the fill to 70 mg could reduce wastage from 19.4% to 18.8%, or alternatively, adding a 12.5 mg vial reduce this to 6.5%. A secondary finding is that wastage is higher when the larger vial size is perfectly divisible by the smaller vial size. Conclusions Reductions in wastage have the potential to reduce the cost of manufacturing medicines, which is not necessarily low for novel products. These cost reductions could lead to increased profit (at the same prices), constant profit with a better return rate (at lower prices), or a combination of the two. Most importantly, they would improve the efficiency of the health-care sector, increasing funding available to treat patients.

Vaccination: Is There a Place for Penalties for Non-compliance? Abstract The introduction of punitive measures to control outbreaks of measles in Europe has sparked debate and public protest about the ethical justification of penalties and exclusionary processes for non-immunisation. This article advances an ethics framework related to compulsory vaccination policies, which we use to analyse three case studies: of mandatory policies that are enforced by fines; of policies that require vaccination for the provision of social goods; and of community-led policies in which communities themselves decide how to enforce vaccination compliance. We report on contemporary, ongoing and past measures that have been used to increase vaccine uptake, consider their rationale and the related public responses, elaborate on socio-cultural and contextual influences, and discuss the ethical justification for mandatory vaccination. We argue for a measured approach that protects fundamental human rights to evidence-based information and medical counsel to support health decision making and that simultaneously raises awareness about the role of immunisation in protecting the wider community. We think more emphasis needs to be placed on immunisation as a means of promoting social good, reducing harm and protecting vulnerable groups.

A Payer Perspective of the Hospital Inpatient Additional Care Costs of Antimicrobial Resistance in France: A Matched Case–Control Study Abstract Background and Objective Antimicrobial resistance (AMR) has become one of the biggest threats to global public health given its association with mortality, morbidity and cost of health care. However, little is known on the economic burden of hospitalization attributable to AMR from a public health insurance perspective. We assessed the excess costs to the French public health insurance system attributable to AMR infections in hospitals. Methods Bacterial infectious disease-related hospitalizations were extracted from the National health data information system for all stays occurring in 2015. Bacterial infections, strains, and microbial resistance were identified by specific French ICD-10 codes. Information about health care expenditure, co-morbidities and demographic characteristics (i.e. gender, age) are provided. We used a matched case–control approach to determine the excess of reimbursements paid to stays with AMR compared to stays with an infection without resistance. Cases and controls were matched on gender, age, Charlson comorbidity index, category of infection, infection as principal diagnosis (two classes), microorganism and hospital status. The overall AMR cost was extrapolated to stays with AMR and excluded from the sample (multiple infections), and a second extrapolation was performed to consider stays with unknown resistance status. Results The final sample included 52,921 matched-pairs (98.2% cases). Our results suggest that AMR overall cost reached EUR109.3 million in France with a mean of EUR1103 per stay; extrapolation to the entire database shows that the overall cost could potentially reach EUR287.1 million if all cases would be identified. The mean excess length of hospital stay attributable to AMR was estimated at 1.6 days. Conclusion AMR causes substantial cost burden in France for the public health insurance. Our study confirms the need to reinforce programs to prevent AMR infection and thereby reduce their economic burden.

Economic Evaluations of Childhood Hearing Loss Screening Programmes: A Systematic Review and Critique Abstract 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.

Thopaz+ Portable Digital System for Managing Chest Drains: A NICE Medical Technology Guidance Abstract The Thopaz+ portable digital system was evaluated by the Medical Technologies Advisory Committee (MTAC) of the National Institute for Health and Care Excellence (NICE). The manufacturer, Medela, submitted a case for the adoption of Thopaz+ that was critiqued by Cedar, on behalf of NICE. Due to a lack of clinical evidence submitted by the manufacturer, Cedar carried out its own literature search. Clinical evidence showed that the use of Thopaz+ led to shorter drainage times, a shorter hospital stay, lower rates of chest drain re-insertion and higher patient satisfaction compared to conventional chest drainage when used in patients following pulmonary resection. One comparative study of the use of Thopaz+ in patients with spontaneous pneumothorax was identified and showed shorter drainage times and a shorter length of hospital stay compared to conventional drainage. No economic evidence was submitted by the manufacturer, but a simple decision tree model was included. The model was improved by Cedar and showed a cost saving of £111.33 per patient when Thopaz+ was used instead of conventional chest drainage in patients following pulmonary resection. Cedar also carried out a sub-group analysis of the use of Thopaz+ instead of conventional drainage in patients with pneumothorax where a cost saving of £550.90 was observed. The main cost driver for the model and sub-group analysis was length of stay. The sub-group analysis was based on a single comparative study. However, the MTAC received details of an unpublished audit of Thopaz+ which confirmed its efficacy in treating patients with pneumothorax. Thopaz+ received a positive recommendation in Medical Technologies Guidance 37.

Modelled Economic Evaluation of Nivolumab for the Treatment of Second-Line Advanced or Metastatic Squamous Non-Small-Cell Lung Cancer in Australia Using Both Partition Survival and Markov Models Abstract Objectives To assess the cost-effectiveness of nivolumab for patients with advanced or metastatic squamous non-small-cell lung cancer (NSCLC) progressed on or after platinum-based chemotherapy using a modelled economic evaluation. Methods Both partition survival (PS) and Markov models, comprised of three health states, were adopted to evaluate the cost-effectiveness of nivolumab compared to docetaxel from an Australian healthcare system perspective with a 6-year time horizon. Reconstructed individual patient data (IPD) were derived from published Kaplan–Meier curves from the pivotal trial for overall survival (OS) and progression-free survival (PFS) using a validated algorithm. Best-fitting survival curves were selected to extrapolate the OS, PFS and post-progression survival (PPS) beyond trial duration. Expected costs and health outcomes [i.e. quality-adjusted life year (QALY), and life year (LY)] associated with each of the health states (i.e. PF, PD and dead) were accrued over the time horizon. Both deterministic and probabilistic sensitivity analyses were undertaken. Results Nivolumab was associated with both higher costs and benefits in both PS and Markov models. In particular, from the PS model, nivolumab cost an additional A$198,862/QALY and A$181,623/LY gained. The Markov model showed that nivolumab had an incremental cost-effectiveness ratio (ICER) of A$220,029/QALY and A$193,459/LY, respectively. The sensitivity analyses showed base-case results were sensitive to the extrapolation approach, duration of treatment, cost of nivolumab and time horizon modelled. Conclusions Using an often-quoted willingness-to-pay per QALY threshold in Australia (i.e. A$50,000), the treatment with nivolumab cannot be considered cost-effective. It might be funded publicly by special arrangements given unmet clinical needs for patients.

Using Post-market Utilisation Analysis to Support Medicines Pricing Policy: An Australian Case Study of Aflibercept and Ranibizumab Use Abstract Objectives To describe how post-market utilisation analysis in Australia informs cost-effectiveness assessment and pricing decisions, using aflibercept and ranibizumab as case studies. Methods Pharmaceutical claims were used to identify initiators of aflibercept and ranibizumab in the year after aflibercept-listing (December 2012), and ranibizumab initiators in the year before aflibercept listing. The dispensing rates for each cohort were calculated, and their demographic and clinical characteristics compared using Kruskal–Wallis tests. Results Aflibercept and ranibizumab each accounted for half the age-related macular degeneration market following aflibercept listing. Aflibercept initiators had similar dispensing rates to ranibizumab initiators in the pre- and post-aflibercept era (~ three scripts during the first 90 days, and eight to nine scripts during the following 12 months). All cohorts were similar in terms of their age, sex, residential aged-care status and geographic remoteness, and no differences were observed in their overall co-morbidity scores and history of thromboembolic events. Conclusions Contrary to clinical trial protocols, post-market utilisation research for ranibizumab and aflibercept demonstrates equivalent use in practice in terms of dose frequency, and the demographic and clinical characteristics of initiators. This supports Australia’s decision to pay the same price for each rather than giving a premium to aflibercept. Many other countries are likely overpaying for aflibercept if their utilization patterns are similar to Australia’s, and could benefit from incorporating routine utilisation assessment.

A Transparent Universal Health Coverage Index with Decomposition by Socioeconomic Groups: Application in Asian and African Settings Abstract Background Health and wellbeing as one of the Sustainable Development Goals requires all countries to achieve Universal Health Coverage (UHC). That is, all people must have access to healthcare when needed at an affordable price. While several indices were developed recently to assess UHC status, these indices appeared to be difficult for practitioners to apply without statistical knowledge. Objective This paper presents a transparent and step-by-step practical calculation method of such an index using Excel spreadsheets, applied to some Asian and African countries. We also decompose the contribution of socioeconomic groups to UHC index values. Methods We utilized the well known UHC illustration (three-dimensional box, showing population coverage, service coverage and financial protection) to calculate the UHC index. We also broke down the index into socioeconomic groups. For validation, correlation coefficients between our index and other UHC indices were calculated and the relationship of our index with out-of-pocket (OOP) payments was estimated. Results World Bank data from six Asian and 15 African countries on health-service coverage of people in five socioeconomic quintiles with financial protection were used to calculate our UHC index. Among the Asian countries, indices ranged between 26.0% (Nepal) and 58.7% (Kazakhstan), while in African countries indices ranged between 8.9% (Chad) and 55.3% (Namibia). Decomposition of the UHC index showed a higher contribution to the index by richer socioeconomic groups. The correlation coefficients between our estimated UHC index values and those of others ranged between 0.774 and 0.900. Our index reduced by 1.4% in response to a 1% increase in OOP payments. Conclusions This spreadsheet approach for calculating the UHC index appeared to be useful, where the interrelation of UHC dimensions was easily observed. Decomposition of the index could be useful for policy-makers to identify the subpopulations and health services with need for further interventions towards UHC achievement.

The Association Between Adiposity and Inpatient Hospital Costs in the UK Biobank Cohort Abstract Background High adiposity is associated with higher risks for a variety of adverse health outcomes, including higher rates of age-adjusted mortality and increased morbidity. This has important implications for the management of healthcare systems, since the endocrinal, cardiometabolic and other changes associated with increased adiposity may be associated with substantial healthcare costs. Methods We studied the association between various measures of adiposity and inpatient hospital costs through record linkage between UK Biobank and records of inpatient care in England and Wales. UK Biobank is a large prospective cohort study that aimed to recruit men and women aged between 40 and 69 from 2006 to 2010. We applied generalised linear models to cost per person year to estimate the marginal effect of adiposity, and average adjusted predicted costs of adiposity. Results Valid cost and body mass index (BMI) data from 457,689 participants were available for inferential analysis. Some 54.4% of individuals included in the analysis sample had positive inpatient healthcare costs over the period of follow-up. Median hospital costs per person-year of follow-up were £89, compared to mean costs of £481. Mean BMI overall was 27.4 kg/m2 (standard deviation 4.8). The marginal effect of a unit increase in BMI was £13.61 (99% confidence interval £12.60–£14.63) per person-year of follow up. The marginal effect of a standard deviation increase in BMI was £69.20 (99% confidence interval £64.98–£73.42). The marginal effect of becoming obese was £136.35 (99% confidence interval £124.62–£148.08). Average adjusted predicted inpatient hospital costs increased almost linearly when modelled using continuous measure of adiposity. Sensitivity analysis of different scenarios did not substantially change these conclusions, although there was some evidence of attenuation of the effects of adiposity when controlling for waist-hip ratios, and when individuals who self-reported any pre-existing conditions were excluded from analysis. Conclusions Higher adiposity is associated with higher inpatient hospital costs. Further scrutiny using causal inferential methods is warranted to establish if further public health investments are required to manage the large healthcare costs observationally associated with overweight and obesity.