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A packed issue of Value in Health in which methodological studies predominate. As a set, the methods studies exemplify the broad scope of health economics, including discrete choice experiment (DCE), clinical trials, outcomes research, and productivity losses.
First up, an intriguing study into a novel method to help with attribute selection in DCE design. Attribute selection is an underappreciated problem in many health DCEs, and it is often difficult to land on a final set of attributes. This study explored using a best-worse scaling case 1 survey to guide selection by determining attribute importance. This may be a useful method for conceptually difficult health DCEs with a fairly low burden of adoption.
Time to wade once again into the muddy waters of the EQ-5D-3L vs 5L valuation debate in an international comparison of valuation sets applied to cost-effectiveness analysis. Using valuation sets from 6 countries, the authors compared incremental QALY differences using either 3L or 5L versions in 8 case study cost-effectiveness analyses. In most cases, the QALY gains using the 5L were lower than if the 3L were used instead. In some cases, the difference between 3L and 5L was large, up to an 84% mean reduction across case studies in the case of the Netherlands. The authors recommend further investigation and caution against the rapid adoption of 5L.
A health economics analysis plan (HEAP) sets out a priori how the analytical part of an economic evaluation will be carried out. As with the better-known statistical analysis plan (SAP), it is hoped this kind of declaration can help to improve transparency and reduce bias in the analysis. An expert Delphi consensus survey was used to try to provide recommendations towards standardizing the content of HEAPs. While the 58 items may seem excessive, not all would be required in most cases. Another benefit of using HEAPs in the context of trials is that they can better align with the statisticians’ and triallists’ planning and analysis. Thus, helping avoid misunderstandings and the headaches of incompatible analysis decisions. [CoI disclosure: I was one of the contributors to this study.]
Another methodological study responded to a common criticism of the friction cost approach for valuing productivity losses; filling a vacancy can create a chain of vacancies if an employed person fills the role. This has previously been ignored in friction cost estimates. The authors propose a multiplier that is estimated at a national level to account for the additional frictions of the potential chain of vacancies.
Finally in methodology, a value framework for diagnostic technologies was proposed. The framework aims to be applicable internationally, especially in low and middle-income countries. Both systematic review and qualitative methods with stakeholders (from various countries in the Latin America region) were used to compile a list of 15 criteria.
Three cost-effectiveness analyses are included in this issue. The first compared transcatheter embolization techniques for upper GI bleeding in the US setting. The results show clear dominance of liquid embolization agents compared to the use of coils. Another study looked at a pharmacist-led medication management program for hypertension using data from an observational study for key effectiveness data. The third CEA was on the topic of ultrasound scans in late pregnancy to screen growth restriction and included a value of information analysis. The value of information results offered the most important insights given the high costs of a randomized trial in this setting and the current evidence base.
There are two systematic reviews in the issue. A truly mammoth review, including 480 studies and 537 registered trials, summarised patient-reported outcomes in cancer RCTs. Another review investigated the costs incurred by kidney donors, concluding that donors in North America tend to be under-compensated given the expenses they face. A scoping review explored qualitative studies with children or adolescents supporting quality of life measures. While a large number of studies in these groups were identified, the details of the methods were generally lacking.
Three further studies I have presumptively grouped as data science and applied econometrics. Of most interest to me personally was a real-world evidence (RWE) study of the three main transcatheter aortic valve replacement (TAVR) devices using a national Japanese registry. This seems like a prime topic for RWE because of the lack of head-to-head trials of competing devices. This matter has seriously limited past HTA efforts and health system decision making. There was also an interesting review of immature survival data in NICE submissions for anti-cancer agents. 41% of submission were found to have immature data. Immature data can make the current standard methods for probabilistic sensitivity analysis incomplete and may require greater exploration. This is an issue with strong overlap with other important issues in oncology research regarding the appropriateness of approval based on outcomes that are proxy measures of longer-term survival. An analysis of Canadian trauma centre records aimed to develop indicators of resource use at the hospital level, finding that most of the variation was unexplained by the available variables.
Also included in this issue was a commentary piece on the proposal for the United States to adopt reference pricing. The focus of the piece is on four key administrative barriers to effective reference pricing. There is also some discussion of strategy, which is an inseparable part of any reference pricing policy evaluation, and which may well be decisive in the eventual fate of the policy proposal.
And after all that, I didn’t even make it to reading the ISPOR Critical Appraisal of Systematic Reviews With Costs and Cost-Effectiveness Outcomes Good Practices Task Force Report. Although, given how helpful these best practice guides are, I am sure it will be a useful reference.