Paul Mitchell’s journal round-up for 1st January 2018

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.

Does the approach to economic evaluation in health care depend on culture, values and institutional context? European Journal of Health Economics [PubMedPublished 5th December 2017

In last week’s round-up we looked at a paper that attempted to develop guidance for costing across European economic evaluations, even when the guidelines across countries vary as to what should and should not be included in an economic evaluation. Why is it that there is such variation in health economic evaluation methods across countries? Why are economic outcomes like quality-adjusted life years (QALYs) standard practice in some countries yet frowned upon in others? This editorial argues that cultures, values and institutional context play a role in the economic evaluation methodologies applied across countries. It does so by comparing five large European countries in terms of 1. the organisation and governance of the agencies undertaking health technology assessments (HTAs) and economic evaluation, 2. the methods used for economic evaluation, and 3. the use of HTA and economic evaluation in decision making. The authors argue that due to differences in these areas across countries, it is difficult to see how a “one size fits all” economic evaluation framework can be implemented, when health care systems, their regulations and social values towards health care differ. An argument is presented that where greater social value is placed on horizontal equity (equal treatment of equals) over vertical equity (unequal treatment of unequals), the QALY outcome is more likely to be applied in such countries. They argue that of the five largest European countries, the German efficiency frontier model of economic analysis may offer the best off-the-shelf option for countries like the United States who also have similar qualms about the use of QALYs in decision making. However, it may be the case that current economic evaluations lack international application due to other reasons beyond those notable considerations raised in this paper.

Reconciling ethical and economic conceptions of value in health policy using the capabilities approach: a qualitative investigation of Non-Invasive Prenatal Testing. Social Science & Medicine [PubMed] [RePEcPublished 16th November 2017

The capability approach, initially developed by economist and philosopher Amartya Sen, provides an alternative evaluative framework to welfare economics, shifting the focus on individual welfare away from utility and preferences, towards a person’s freedom to do and be valuable things to their life. It has more recently been used as a critique of the current approach to health economic evaluations, specifically what aspects of quality of life are included in the economic outcome, where the current measurement tools used in the generation of QALYs have been argued to have too narrow a focus on health outcomes, with a number of capability measures now developed as alternatives. This study, on the other hand, applies the capability approach to tackle health technologies that pose difficult ethical challenges where standard clinical and economic outcomes used in cost-effectiveness analysis may be in conflict with social values. The authors propose why they think the evaluative framework of the capability approach may be advantageous in such areas, using non-invasive prenatal testing (NIPT), a screening test that analyses cell-free fetal DNA circulating in maternal blood in order to gain information about the fetal genotype, as a case study. The authors propose that adopting a capability evaluative framework in NIPT may account for the enhancement of valuable options available to prospective parents and families, as well as capabilities that may be diminished if NIPT was made routinely available, such as the option of refusing a test as an informed choice. A secondary analysis of qualitative data was conducted on women with experience of NIPT in Canada. Using a constructivist orientation to directed qualitative content analysis, interviews were analysed to see how NIPT related to a pre-existing list of ten Central Human Capabilities developed by philosopher Martha Nussbaum. From the analysis, they found eight of the ten Nussbaum capabilities emerge from the interviewees who were not directly asked to consider capability in the interview. As well as these eight (life; bodily health; bodily integrity; senses, imagination and thought; emotions; practical reason; affiliation; control over one’s environment), a new capability emerged related to care-taking as a result of NIPT, both for potential children and also the impact on existing children. The next challenge for the authors will be trying to formulate their findings into a usable outcome measure for decision-making. However, the analysis undertaken here is a good example of how economists can attempt to tackle the assessment of ethically challenging technologies as a way of dealing with standard economic outcomes that might be considered counter-productive in such evaluations.

Quality of life in a broader perspective: does ASCOT reflect the capability approach? Quality of Life Research [PubMedPublished 14th December 2017

The Adult Social Care Outcomes Toolkit (ASCOT) is a measure developed specifically for the economic assessment of social care interventions in the UK. Although a number of versions of ASCOT have been developed, the most recent version of ASCOT has been argued to be a measure influenced by the capability approach, even though previous versions of the measure were not justified similarly, so it remains to be seen how influential the capability approach is in the composition of this outcome measure. This study attempts to add justification of linking the capability approach with the ASCOT by conducting a literature review on the capability approach to identify key issues of quality of life measurement and how ASCOT deals with these issues. The methods for conducting the literature review are not described in detail in this paper, but the authors state that three primary issues with quality of life measurement in the capability approach literature that emerge from their review are concerned with 1. the measurement of capability, 2. non-reliance on adaptive preferences, and 3. focus on a multidimensional evaluative space. The authors argue that capability measurement is tackled by ASCOT, through the use of “as I want” phraseology at the top level on the ASCOT dimensions. Adaptive preferences are argued to be tackled by the use of general population preferences of different states on ASCOT and the outcome addresses several dimensions of quality of life. I would argue that there is much more to measuring capability beyond these three areas identified by the authors. Although the authors rightly question if the “as I want” phraseology is adequate to measure capability in their conclusion, the other two criteria could equally justify most measures for generating QALYs, so the criteria they use to be a capability measure is set at a very low benchmark. I remain unconvinced about how much of a capability measure ASCOT actually is in practice.

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On the commensurability of efficiency

In this week’s round-up, I highlighted a recent paper in the journal Cambridge Quarterly of Healthcare Ethics. There are some interesting ideas presented regarding the challenge of decision-making at the individual patient level, and in particular a supposed trade-off between achieving efficiency and satisfying health need.

The gist of the argument is that these two ‘values’ are incommensurable in the sense that the comparative value of two choices is ambiguous where the achievement of efficiency and need satisfaction needs to be traded. In the journal round-up, I highlighted 2 criticisms. First, I suggested that efficiency and health need satisfaction are commensurable. Second, I suggested that the paper did not adequately tackle the special nature of microlevel decision-making. The author – Anders Herlitz – was gracious enough to respond to my comments with several tweets.

Here, I’d like to put forth my reasoning on the subject (albeit with an ignorance of the background literature on incommensurability and other matters of ethics).

Consider a machine gun

A machine gun is far more efficient than a pistol, right? Well, maybe. A machine gun can shoot more bullets than a pistol over a sustained period. Likewise, a doctor who can treat 50 patients per day is more efficient than a doctor who can treat 20 patients per day.

However, the premise of this entire discussion, as established by Herlitz, is values. Herlitz introduces efficiency as a value and not as some dispassionate indicator of return on input. When we are considering values – as we necessarily are when we are discussing decision-making and more generally ‘what matters’ – we cannot take the ‘more bullets’ approach to assessing efficiency.

That’s because ‘more bullets’ is not what we mean when we talk about the value of efficiency. The production function is fundamental to our understanding of efficiency as a value. Once values are introduced, it is plain to see that in the context of war (where value is attached to a greater number of deaths) a machine gun may very well be considered more efficient. However, bearing a machine gun is far less efficient than bearing a pistol in a civilian context because we value a situation that results in fewer deaths.

In this analogy, bullets are health care and deaths are (somewhat confusingly, I admit) health improvement. Treating more people is not better because we want to provide more health care, but because we want to improve people’s health (along with some other basket of values).

Efficiency only has value with respect to the outcome in whose terms it is defined, and is therefore always commensurable with that outcome. That is, the production function is an inherent and necessary component of an efficiency to which we attach value.

I believe that Herlitz’s idea of incommensurability could be a useful one. Different outcomes may well be incommensurable in the way described in the paper. But efficiency has no place in this discussion. The incommensurability Herlitz describes in his paper seems to be a simple conflict between utilitarianism and prioritarianism, though I don’t have the wherewithal to pursue that argument so I’ll leave it there!

Microlevel efficiency trade-offs

Having said all that, I do think there could be a special decision-making challenge regarding efficiency at the microlevel. And that might partly explain Herlitz’s suggestion that efficiency is incommensurable with other outcomes.

There could be an incommensurability between values that can be measured in their achievement at the individual level (e.g. health improvement) and values that aren’t measured with individual-level outcomes (e.g. prioritisation of more severe patients). Those two outcomes are incommensurable in the way Herlitz described, but the simple fact that we tend to think about the former as an efficiency argument and the latter as an equity argument is irrelevant. We could think about both in efficiency terms (for example, treating n patients of severity x is more efficient than treating n-1 patients of severity x, or n patients of severity x-1), we just don’t. The difficulty is that this equity argument is meaningless at the individual level because it relies on information about outcomes outside the microlevel. The real challenge at the microlevel, therefore, is to acknowledge scope for efficiency in all outcomes of value. The incommensurability that matters is between microlevel and higher-level assessments of value.

As an aside, I was surprised that the Rule of Rescue did not get a mention in the paper. This is a perfect example of a situation in which arguments that tend to be made on efficiency grounds are thrown out and another value (the duty to save an immediately endangered life) takes over. One doesn’t need to think very hard about how Rule of Rescue decision-making could be framed as efficient.

In short, efficiency is never incommensurable because it is never an end in itself. If you’re concerned with being more efficient for the sake of being more efficient then you are probably not making very efficient decisions.

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Chris Sampson’s journal round-up for 18th December 2017

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.

Individualized glycemic control for U.S. adults with type 2 diabetes: a cost-effectiveness analysis. Annals of Internal Medicine [PubMed] Published 12th December 2017

The nature of diabetes – that it affects a lot of people and is associated with a wide array of physiological characteristics and health impacts – has given rise to recommendations for individualisation of care. This paper evaluates individualisation of glycemic control targets. Specifically, the individualised programme allocated people to one of 3 HbA1c targets (<6.5%, <7%, <8%) according to their characteristics, while the comparator was based on a single fixed target (<7%). The researchers used a patient-level simulation model. Risk equations developed by the UKPDS study were used to predict diabetes complications and mortality. The baseline population was derived from the NHANES study for 2011-12 and constitutes people who self-reported as having diabetes and who were at least 30 years old at diagnosis (to try and isolate type 2 diabetes). It’s not much of a surprise that the individualised approach dominated uniform intensive control, saving $13,547 on average per patient with a slight improvement in QALY outcomes. But the findings are not all in favour of individualisation. Quality of life improvements due to the benefits of medication were partially counteracted by a slight decrease in life years gained due to a higher rate of (mortality-increasing) complications. The absolute lifetime risk of myocardial infarction was 1.39% higher with individualisation. A key outstanding question is how much the individualisation process would actually cost to get right. Granted, it probably wouldn’t cost as much as the savings estimated in this study, but the difficulty of ensuring adequate data quality to consistently inform individualisation should not be underestimated.

Microlevel prioritizations and incommensurability. Cambridge Quarterly of Healthcare Ethics [PubMed] Published 7th December 2017

This article concerns the ethical challenges of decision-making at the microlevel. For example, decisions may need to be made about allocating resources between 2 or more identifiable patients, perhaps within a particular clinic or amongst an individual clinician’s patients. The author asserts two relevant values: health need satisfaction and efficiency. Health need satisfaction is defined in terms of severity (regardless of capacity to benefit from available treatments), while efficiency is defined in terms of the maximisation of health benefit (subject to the effectiveness of treatment). The author then argues that these two values are incommensurable in the sense that we can have situations in which health need satisfaction is greater (or less) for a given choice over another, while efficiency could be lower (or higher). Thus, it is not always possible to rank choices given two non-cardinally-comparable values. It might not be clear whether it is better to treat patient A or patient B if the implications of doing so are different in terms of need and efficiency. The author then goes on to suggest some solutions to this apparent problem, starting by highlighting the need for decision makers (in this case clinicians) to recognise different decision paths. The first solution is to generate some guidelines that offer complete ordering of possible choices. These might be based on a process of weighting the different values (e.g. health need satisfaction and efficiency). The other ‘solution’ is to leave the decision to medical practitioners, who can create reasons for choices that may be unique to the case at hand. In this case, certain decision paths should be avoided, such as those that would entail discrimination. I have a lot of problems with this assessment of decision-making at the individual level. Mainly, the discussion is undermined by the fact that efficiency and health need satisfaction are entirely commensurable insofar as we care about either of them in relation to prioritisation in health care. We tend to understand both health need satisfaction and opportunity cost (the basis for estimating efficiency) in terms of health outcomes. The essay also fails to clearly identify the uniqueness of the challenge of microlevel decision-making as distinct from the process of creating clinical guidelines. This may call for a follow-up blog post…

EQ-5D: moving from three levels to five. Value in Health Published 6th December 2017

If you work on economic evaluation, the move from using the EQ-5D-3L to the EQ-5D-5L – in terms of the impact on our results – is one of the biggest methodological step changes in recent memory. We all know that the 5L (and associated value set for England) is better than the 3L. Don’t we? So it is perhaps a bit disappointing that the step to the 5L has been so tentative. This editorial articulates the challenge. NICE makes standards. EuroQoL does research. NICE was (relatively) satisfied with the 3L. EuroQoL wasn’t. We have a clash between an inherently (perhaps necessarily) conservative institution and an inherently progressive institution. Hopefully, their interaction will put us on a sustainable path that achieves both methodological consistency and scientific rigour. This editorial also provides us with a DOI-citable account of the saga that includes the development of the 5L value set for England and NICE’s subsequent memorandum.

Current UK practices on health economics analysis plans (HEAPs): are we using heaps of them? PharmacoEconomics [PubMed] Published 6th December 2017

You could get by for years in economic evaluation without even hearing about ‘health economics analysis plans’ (HEAPs). It probably depends on the policies set by the clinical trials unit (CTU) that you’re working with. The idea is that HEAPs are an equivalent standard operating procedure (SOP) to a statistical analysis plan – setting out how the trial data will be analysed before the analysis begins. This could aid transparency and consistency, and prevent dodgy practices. In this study, the researchers sought to find out whether HEAPs are actually being used, and their perceived role in clinical trials research. A survey targeted 46 UK CTUs, asking about the role of health economists in the unit and whether they used HEAP SOPs. Of 28 respondents, 11 reported having an embedded health economics team. A third of CTUs reported always having a HEAP in place. Most said they only used HEAPs ‘sometimes’, and publicly funded trials were said to be more likely to use a HEAP. The majority of respondents agreed it was acceptable to produce the HEAP at any point prior to a lockdown of the data. The findings demonstrate inconsistency in who writes HEAPs and who is perceived to be the audience. I agree with the premise that we need HEAPs. Though I’m not sure what they should look like, except that statistical analysis plans probably should not be used as a template. It would be good if some of these researchers took things a step further and figured out what ought to go into a HEAP, so that we can consistently employ their recommendations. If you’re on the HEALTHECON-ALL mailing list, you’ll know that they’re already on the case.

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