Chris Sampson’s journal round-up for 27th January 2020

Every Monday our authors provide a round-up of some of the most recently published peer reviewed articles from the field. We don’t cover everything, or even what’s most important – just a few papers that have interested the author. Visit our Resources page for links to more journals or follow the HealthEconBot. If you’d like to write one of our weekly journal round-ups, get in touch.

A general framework for classifying costing methods for economic evaluation of health care. The European Journal of Health Economics [PubMed] Published 20th January 2020

When it comes to health state valuation and quality of life, I’m always very concerned about the use of precise terminology, and it bugs me when people get things wrong. But when it comes to costing methods, I’m pretty shoddy. So I’m pleased to see this very useful paper, which should help us all to gain some clarity in our reporting of costing studies.

The authors start out by clearly distinguishing between micro-costing and gross-costing in the identification of costs and between top-down and bottom-up valuation of these costs. I’m ashamed to say that I had never properly grasped the four distinct approaches that can be adopted based on these classifications, but the authors make it quite clear. Micro-costing means detailed identification of cost components, while gross-costing considers resource use in aggregate. Top-down methods use expenditure data collected at the organisational level, while bottom-up approaches use patient-level data.

A key problem is that our language – as health economists – is in several respects contradictory to the language used by management accountants. It’s the accountants who are usually preparing the cost information that we might use in analyses, and these data are not normally prepared for the types of analysis that we wish to conduct, so there is a lot that can go awry. Perhaps most important is that financial accounting is not concerned with opportunity costs. The authors provide a kind of glossary of terms that can support translation between the two contexts, as well as a set of examples of the ways in which the two contexts differ. They also point out the importance of different accounting practices in different countries and the ways in which these might necessitate adjustment in costing methods for economic evaluation.

The study includes a narrative review of costing studies in order to demonstrate the sorts of errors in terminology that can arise and the lack of clarity that results. The studies included in the review provide examples of the different approaches to costing, though no study is identified as ‘bottom-up gross-costing’. One of the most useful contributions of the paper is to provide two methodological checklists, one for top-down and one for bottom-up costing studies. If you’re performing, reviewing, or in any way making use of costing studies, this will be a handy reference.

Health state values of deaf British Sign Language (BSL) users in the UK: an application of the BSL version of the EQ-5D-5L. Applied Health Economics and Health Policy [PubMed] Published 16th January 2020

The BSL translation of the EQ-5D is like no other. It is to be used – almost exclusively – by people who have a specific functional health impairment. For me, this raises questions about whether or not we can actually consider it simply a translation of the EQ-5D and compare values with other translations in the way we would any other language. This study uses data collected during the initial development and validation of the EQ-5D-5L BSL translation. The authors compared health state utility values from Deaf people (BSL users) with a general population sample from the Health Survey for England.

As we might expect, the Deaf sample reported a lower mean utility score (0.78) than the general population (0.84). Several other health measures were used in the BSL study. A staggering 43% of the Deaf participants had depression and a lot of the analysis in the paper is directed towards comparing the groups with and without psychological distress. The authors conduct some simple regression analyses to explore what might be the determinants of health state utility values in the Deaf population, with long-standing physical illness having the biggest impact.

I had hoped that the study might be able to tell us a bit more about the usefulness of the BSL version of the EQ-5D-5L, because the EQ-5D has previously been shown to be insensitive to hearing problems. The small sample (<100) can’t tell us a great deal on its own, so it’s a shame that there isn’t some attempt at matching with individuals from the Health Survey for England for the sake of comparison. Using the crosswalk from the EQ-5D-3L to obtain 5L values is also a problem, as it limits the responsiveness of index values. Nevertheless, it’s good to see data relating to this under-represented population.

A welfare-theoretic model consistent with the practice of cost-effectiveness analysis and its implications. Journal of Health Economics [PubMed] Published 11th January 2020

There are plenty of good reasons to deviate from a traditional welfarist approach to cost-benefit analysis in the context of health care, as health economists have debated for decades. But it is nevertheless important to understand the ways in which cost-effectiveness analysis, as we conduct it, deviates from welfarism, and to aim for some kind of consistency in our handling of different issues. This paper attempts to draw together disparate subjects of discussion on the theoretical basis for aspects of cost-effectiveness analysis. The author focuses on issues relating to the inclusion of future (unrelated) costs, to discounting, and to consistency with welfarism, in the conduct of cost-per-QALY analyses. The implications are given consideration with respect to adopting a societal perspective, recognising multiple budget holders, and accounting for distributional impacts.

All of this is based on the description of an intertemporal utility model and a model of medical care investment. The model hinges especially on how we understand consumption to be affected by our ambition to maximise QALYs. For instance, the author argues that, once we consider time preferences in an overall utility function, we don’t need to worry about differential discounting in health and consumption. The various implications of the model are compared to the recommendations of the Second Panel on Cost-Effectiveness in Health and Medicine. In general, the model supports the recommendations of the Panel, where others have been critical. As such, it sets out some of the theoretical basis for those recommendations. It also implies other recommendations, not considered by the Panel. For example, the optimal cost-effectiveness threshold is likely to be higher than GDP per capita.

It’s difficult to judge the validity of the framework from a first read. The paper is dense with theoretical exposition. My first instinct is ‘so what’. One of the great things about the practice of cost-effectiveness analysis in health care is that it isn’t constrained by restrictive theoretical frameworks, and so the very idea of a kind of unified theoretical framework is a bit worrying to me. But my second thought is that this is a valuable paper, as it attempts to gather up several loose threads. Whether or not these can be gathered up within a welfarist framework is debatable, but the exercise is revealing. I suspect this paper will help to trigger further inquiry, which can only be a good thing.

Registered reports: time to radically rethink peer review in health economics. PharmacoEconomics – Open [PubMed] Published 23rd January 2020

As a discipline, health economics isn’t great when it comes to publication practices. We excel in neither the open access culture of medical sciences nor the discussion paper culture of economics proper. In this article, the authors express concern about publication bias, and the fact that health economics journals – and health economists in general – aren’t doing much to combat it. In fairness to the discipline, there isn’t really any evidence that publication bias abounds. But that isn’t really the point. We should be able to prove and ensure that it doesn’t if we want our research to been seen as credible.

One (partial) solution to publication bias is the adoption – by journals – of registered reports. Under such a system, researchers would submit study protocols to journals for peer review. If the journal were satisfied with the methods then they could guarantee to publish the study once the results are in, regardless of how sexy the results may or may not be. The authors of this paper identify the prevalence of studies in major health economics journals that could benefit from registered reports. These would be prospectively designed experimental or quasi-experimental studies. It seems that there are plenty.

I’ve used this blog in the past to propose more transparent research practices and to complain about publication practices in health economics generally, while others have complained about the use of p-values in our discipline. The adoption of registered reports is one tactic that could bring improvements and I hope it will be given proper consideration by those in a position to enact change.

Credits

The potential of the super QALY to reconcile the key contentions in health economics

Economics is largely about trade-offs and compromise. Academics study the former but don’t often engage in the latter. In health economics, as in other fields, a key trade-off is between equity and efficiency. We’ve been studying this for a.very.long.time. Despite this, as Culyer has identified, equity is hardly considered in current health technology assessments. We all agree it should be, but just can’t seem to figure it out. Indeed, ihas been argued that incorporating equity concerns into cost-effectiveness analyses could still be a long time coming.

But let’s be a bit more positive. The elusive `Super QALY’, as it has been described, should come eventually. And when it does, it’ll be great! One of the reasons, I propose here, is that it has the power to reconcile many of the disagreements that currently fuel (hamper?) debate in our field. Hence, the super QALY might just allow us to get on with fussing over minutia issues of economic evaluation.

Trade-offs

There are necessary trade-offs in decisions of resource allocation. These might be described as the ‘positive’ tensions economists deal with; they relate to decisions that must be made, regardless of our values. The equity–efficiency trade-off is the main one here. But there are others. For example, health care interventions have the dual aim of increasing both the quantity and quality of an individual’s life. The QALY attempts to address this. However, the way we value quality of life also incorporates considerations of length of life in so much as ‘death’ is used in the valuation of health states. This is problematic, as has been discussed. Economists haven’t really gotten round to disagreeing about this yet, but there’s plenty else on which we disagree.

Disagreements

These might be described as ‘normative’ tensions. They concern what different economists think should and should not be done; mainly relating to the process of valuing health states. There are welfarists and non-welfarists. There are those who support societal preferences, and those who support capturing patient experience. It should be clear to most that neither side in these debates is wrong. Most health economists acknowledge the value of capturing utility as well as the importance of capabilities. Most will attach some value to society’s preferences and some to those of the individual.

A super-QALY solution

It’s never been completely clear what the ‘extra’ in extra-welfarism (as currently practiced) actually consists. The super QALY will surely formalise this; it could involve some completely non-welfarist notions. The most common idea of the super QALY is one where the current health-related QALY is weighted based on some equity considerations. So, if this is where economic evaluation is heading, we’re likely to end up with an extra step of estimating the equity impact of an intervention. But, while most studies seem to suggest that this might just be an add-on process, I think it would require a realignment of the methods we already use.

Equity analysis

There’s no need for me to reiterate the importance of equity considerations. Plainly we (economists, the public) care about needs, capabilities, opportunities and equality. How we define the equity analysis is incidental. More important is that we get on with doing it and just see what happens. There are lots of measures we could use and different approaches we could take. For arguments sake (and because I quite like it), let’s say the equity analysis is characterised by a ‘minimum capabilities‘ approach. Something similar to Daniels’s normal opportunity range. People could have the normal opportunity range, have fewer opportunities or have more opportunities. We can argue later about where the threshold lies. People below the threshold could be said to be in ‘need’. Again, argue about this later. States could be defined using a capabilities measure; let’s just say the ICECAP-A for now (though I don’t much like it). Here in the world of health economics we like 0-1 scales, so the ICECAP-A could be valued based on these anchors. So, let’s say 1 is the minimum capabilities or normal opportunity range threshold. Zero equates to being dead. Values can drop below zero where opportunity sets represent a state worse that non-existence. For the equity analysis we are not interested in utility or satisfaction, so the valuation would not be by the individual. Values could be elicited from society, possibly. The valuation technique could be a person trade-off, maybe. Or we could let ethicists come up with weightings. This framework, surely, would satisfy the non-welfarists.

Health utility analysis

I see no reason why the estimation of health benefits cannot be utility-based. Utilitarian satisfaction is sufficient if non-welfarist concerns are incorporated in an equity analysis. Personally I believe that whether this is based on experiences or preferences is largely inconsequential and that, in terms of health, most of the differences demonstrated between the 2 are a function of the elicitation methods. Therefore, utility analysis would remain largely unchanged. However, the value of 0 would change. Zero currently represents either being dead or in a health state equivalent to being dead, despite these two things not being of equivalent value to a person. Under the new framework there is no need to incorporate death into the health utility analysis, as it is accounted for in the equity analysis. 0 should represent the worst health state imaginable. There would be no negative values.

Cost-effectiveness analysis

These 2 analyses would then be combined to form a relatively routine cost-effectiveness analysis to address the efficiency of the intervention. The QALY would be calculated in the usual way, but the ‘Q’ would become ‘super’ by being a function of the 2 different outcomes. Tentatively this could be done by multiplying the two values (alternative formulations could be defined by societal values or by ethicists, depending on your wont). Costings would be carried out in the usual manner and a super ICER could be calculated. Furthermore, the net benefit approach could be implemented in the usual way; possibly with separate willingness-to-pay values for each input to the super QALY (indeed, they may be willingness to pay values from different agents). The table below summarises how the approach might accommodate the various tensions in health economics.

Equity analysis Health utility analysis
Equity Effectiveness
Life Morbidity
Non-welfarism Welfarism
Flourishing Satisfaction
Society The individual

All public policies could be subject to an equity analysis in the way set out above. It is in no way health-specific. Each policy field could then us this to weight their usual outcomes measures – preferably utility-based – to estimate the cost-effectiveness of their intervention. At this point the super QALY makes it onto daytime TV and health economists form a new unelected chamber at the Palace of Westminster.

No doubt this explicitly extra-welfarist approach to the super QALY raises more questions than it is currently able to answer, but we need to get on with trying stuff like this. The super QALY has proven elusive to date but, if we do make it, it may solve a lot of our problems. We may find ourselves having to invent new things to argue about.