Chris Sampson’s journal round-up for 25th March 2019

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.

How prevalent are implausible EQ-5D-5L health states and how do they affect valuation? A study combining quantitative and qualitative evidence. Value in Health Published 15th March 2019

The EQ-5D-5L is able to describe a lot of different health states (3,125, to be precise), including some that don’t seem likely to ever be observed. For example, it’s difficult to conceive of somebody having extreme problems in pain/discomfort and anxiety/depression while also having no problems with usual activities. Valuation studies exclude these kinds of states because it’s thought that their inclusion could negatively affect the quality of the data. But there isn’t much evidence to help us understand how ‘implausibility’ might affect valuations, or which health states are seen as implausible.

This study is based on an EQ-5D-5L valuation exercise with 890 students in China. The valuation was conducted using the EQ VAS, rather than the standard EuroQol valuation protocol, with up to 197 states being valued by each student. Two weeks after conducting the valuation, participants were asked to indicate (yes or no) whether or not the states were implausible. After that, a small group were invited to participate in a focus group or interview.

No health state was unanimously identified as implausible. Only four states were unanimously rated as not being implausible. 910 of the 3,125 states defined by the EQ-5D-5L were rated implausible by at least half of the people who rated them. States more commonly rated as implausible were of moderate severity overall, but with divergent severities between states (i.e. 5s and 1s together). Overall, implausibility was associated with lower valuations.

Four broad themes arose from the qualitative work, namely i) reasons for implausibility, ii) difficulties in valuing implausible states, iii) strategies for valuing implausible states, and iv) values of implausible states. Some states were considered to have logical conflicts, with some dimensions being seen as mutually inclusive (e.g. walking around is a usual activity). The authors outline the themes and sub-themes, which are a valuable contribution to our understanding of what people think when they complete a valuation study.

This study makes plain the fact that there is a lot of heterogeneity in perceptions of implausibility. But the paper doesn’t fully address the issue of what plausibility actually means. The authors describe it as subjective. I’m not sure about that. For me, it’s an empirical question. If states are observed in practice, they are plausible. We need meaningful valuations of states that are observed, so perhaps the probability of a state being included in a valuation exercise should correspond to the probability of it being observed in reality. The difficulty of valuing a state may relate to plausibility – as this work shows – but that difficulty is a separate issue. Future research on implausible health states should be aligned with research on respondents’ experience of health states. Individuals’ judgments about the plausibility of health states (and the accuracy of those judgments) will depend on individuals’ experience.

An EU-wide approach to HTA: an irrelevant development or an opportunity not to be missed? The European Journal of Health Economics [PubMed] Published 14th March 2019

The use of health technology assessment is now widespread across the EU. The European Commission recently saw an opportunity to rationalise disparate processes and proposed new regulation for cooperation in HTA across EU countries. In particular, the proposal targets cooperation in the assessment of the relative effectiveness of pharmaceuticals and medical devices. A key purpose is to reduce duplication of efforts, but it should also make the basis for national decision-making more consistent.

The authors of this editorial argue that the regulation needs to provide more clarity, in the definition of clinical value, and of the quality of evidence that is acceptable, which vary across EU Member States. There is also a need for the EU to support early dialogue and scientific advice. There is also scope to support the generation and use of real-world evidence. The authors also argue that the challenges for medical device assessment are particularly difficult because many medical device companies cannot – or are not incentivised to – generate sufficient evidence for assessment.

As the final paragraph argues, EU cooperation in HTA isn’t likely to be associated with much in the way of savings. This is because appraisals will still need to be conducted in each country, as well as an assessment of country-specific epidemiology and other features of the population. The main value of cooperation could be in establishing a stronger position for the EU in negotiating in matters of drug design and evidence requirements. Not that we needed any more reasons to stop Brexit.

Patient-centered item selection for a new preference-based generic health status instrument: CS-Base. Value in Health Published 14th March 2019

I do not believe that we need a new generic measure of health. This paper was always going to have a hard time convincing me otherwise…

The premise for this work is that generic preference-based measures of health (such as the EQ-5D) were not developed with patients. True. So the authors set out to create one that is. A key feature of this study is the adoption of a framework that aligns with the multiattribute preference response model, whereby respondents rate their own health state relative to another. This is run through a mobile phone app.

The authors start by extracting candidate items from existing health frameworks and generic measures (which doesn’t seem to be a particularly patient-centred approach) and some domains were excluded for reasons that are not at all clear. 47 domains were included after overlapping candidates were removed. The 47 were classified as physical, mental, social, or ‘meta’. An online survey was conducted by a market research company. 2,256 ‘patients’ (people with diseases or serious complaints) were asked which 9 domains they thought were most important. Why 9? Because the authors figured it was the maximum that could fit on the screen of a mobile phone.

Of the candidate items, 5 were regularly selected in the survey: pain, personal relationships, fatigue, memory, and vision. Mobility and daily activities were also judged important enough to be included. Independence and self-esteem were added as paired domains and hearing was paired with the vision domain. The authors also added anxiety/depression as a pair of domains because they thought it was important. Thus, 12 items were included altogether, of which 6 were parts of pairs. Items were rephrased according to the researchers’ preferences. Each item was given 4 response levels.

It is true to say (as the authors do) that most generic preference-based measures (most notably the EQ-5D) were not developed with direct patient input. The argument goes that this somehow undermines the measure. But there are a) plenty of patient-centred measures for which preference-based values could be created and b) plenty of ways in which existing measures can be made patient-centred post hoc (n.b. our bolt-on study).

Setting aside my scepticism about the need for a new measure, I have a lot of problems with this study and with the resulting CS-Base instrument. The defining feature of its development seems to be arbitrariness. The underlying framework (as far as it is defined) does not seem well-grounded. The selection of items was largely driven by researchers. The wording was entirely driven by the researchers. The measure cannot justifiably be called ‘patient-centred’. It is researcher-centred, even if the researchers were able to refer to a survey of patients. And the whole thing has nothing whatsoever to do with preferences. The measure may prove fantastic at capturing health outcomes, but if it does it will be in spite of the methods used for its development, not because of them. Ironically, that would be a good advert for researcher-centred outcome development.

Proximity to death and health care expenditure increase revisited: a 15-year panel analysis of elderly persons. Health Economics Review [PubMed] [RePEc] Published 11th March 2019

It is widely acknowledged that – on average – people incur a large proportion of their lifetime health care costs in the last few years of their life. But there’s still a question mark over whether it is proximity to death that drives costs or age-related morbidity. The two have very different implications – we want people to be living for longer, but we probably don’t want them to be dying for longer. There’s growing evidence that proximity to death is very important, but it isn’t clear how important – if at all – ageing is. It’s important to understand this, particularly in predicting the impacts of demographic changes.

This study uses Swiss health insurance claims data for around 104,000 people over the age of 60 between 1996 and 2011. Two-part regression models were used to estimate health care expenditures conditional on them being greater than zero. The author analysed both birth cohorts and age classes to look at age-associated drivers of health care expenditure.

As expected, health care expenditures increased with age. The models imply that proximity-to-death has grown in importance over time. For the 1931-35 birth cohort, for example, the proportion of expenditures explained by proximity-to-death rose from 19% to 31%. Expenditures were partly explained by morbidity, and this effect appeared to be relatively constant over time. Thus, proximity to death is not the only determinant of rising expenditures (even if it is an important one). Looking at different age classes over time, there was no clear picture in the trajectory of health care expenditures. For the oldest age groups (76-85), health care expenditures were growing, but for some of the younger groups, costs appeared to be decreasing over time. This study paints a complex picture of health care expenditures, calling for complex policy responses. Part of this could be supporting people to commence palliative care earlier, but there is also a need for more efficient management of chronic illness over the long term.

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Chris Sampson’s journal round-up for 19th November 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.

Valuation of health states considered to be worse than death—an analysis of composite time trade-off data from 5 EQ-5D-5L valuation studies. Value in Health Published 12th November 2018

I have a problem with the idea of health states being ‘worse than dead’, and I’ve banged on about it on this blog. Happily, this new article provides an opportunity for me to continue my campaign. Health state valuation methods estimate how much a person prefers being in a more healthy state. Positive values are easy to understand; 1.0 is twice as good as 0.5. But how about the negative values? Is -1.0 twice as bad as -0.5? How much worse than being dead is that? The purpose of this study is to evaluate whether or not negative EQ-5D-5L values meaningfully discriminate between different health states.

The study uses data from EQ-5D-5L valuation studies conducted in Singapore, the Netherlands, China, Thailand, and Canada. Altogether, more than 5000 people provided valuations of 10 states each. As a simple measure of severity, the authors summed the number of steps from full health in all domains, giving a value from 0 (11111) to 20 (55555). We’d expect this measure of severity of states to correlate strongly with the mean utility values derived from the composite time trade-off (TTO) exercise.

Taking Singapore as an example, the mean of positive values (states better than dead) decreased from 0.89 to 0.21 with increasing severity, which is reassuring. The mean of negative values, on the other hand, ranged from -0.98 to -0.89. Negative values were clustered between -0.5 and -1.0. Results were similar across the other countries. In all except Thailand, observed negative values were indistinguishable from random noise. There was no decreasing trend in mean utility values as severity increased for states worse than dead. A linear mixed model with participant-specific intercepts and an ANOVA model confirmed the findings.

What this means is that we can’t say much about states worse than dead except that they are worse than dead. How much worse doesn’t relate to severity, which is worrying if we’re using these values in trade-offs against states better than dead. Mostly, the authors frame this lack of discriminative ability as a practical problem, rather than anything more fundamental. The discussion section provides some interesting speculation, but my favourite part of the paper is an analogy, which I’ll be quoting in future: “it might be worse to be lost at sea in deep waters than in a pond, but not in any way that truly matters”. Dead is dead is dead.

Determining value in health technology assessment: stay the course or tack away? PharmacoEconomics [PubMed] Published 9th November 2018

The cost-per-QALY approach to value in health care is no stranger to assault. The majority of criticisms are ill-founded special pleading, but, sometimes, reasonable tweaks and alternatives have been proposed. The aim of this paper was to bring together a supergroup of health economists to review and discuss these reasonable alternatives. Specifically, the questions they sought to address were: i) what should health technology assessment achieve, and ii) what should be the approach to value-based pricing?

The paper provides an unstructured overview of a selection of possible adjustments or alternatives to the cost-per-QALY method. We’re very briefly introduced to QALY weighting, efficiency frontiers, and multi-criteria decision analysis. The authors don’t tell us why we ought (or ought not) to adopt these alternatives. I was hoping that the paper would provide tentative answers to the normative questions posed, but it doesn’t do that. It doesn’t even outline the thought processes required to answer them.

The purpose of this paper seems to be to argue that alternative approaches aren’t sufficiently developed to replace the cost-per-QALY approach. But it’s hardly a strong defence. I’m a big fan of the cost-per-QALY as a necessary (if not sufficient) part of decision making in health care, and I agree with the authors that the alternatives are lacking in support. But the lack of conviction in this paper scares me. It’s tempting to make a comparison between the EU and the QALY.

How can we evaluate the cost-effectiveness of health system strengthening? A typology and illustrations. Social Science & Medicine [PubMed] Published 3rd November 2018

Health care is more than the sum of its parts. This is particularly evident in low- and middle-income countries that might lack strong health systems and which therefore can’t benefit from a new intervention in the way a strong system could. Thus, there is value in health system strengthening. But, as the authors of this paper point out, this value can be difficult to identify. The purpose of this study is to provide new methods to model the impact of health system strengthening in order to support investment decisions in this context.

The authors introduce standard cost-effectiveness analysis and economies of scope as relevant pieces of the puzzle. In essence, this paper is trying to marry the two. An intervention is more likely to be cost-effective if it helps to provide economies of scope, either by making use of an underused platform or providing a new platform that would improve the cost-effectiveness of other interventions. The authors provide a typology with three types of health system strengthening: i) investing in platform efficiency, ii) investing in platform capacity, and iii) investing in new platforms. Examples are provided for each. Simple mathematical approaches to evaluating these are described, using scaling factors and disaggregated cost and outcome constraints. Numerical demonstrations show how these approaches can reveal differences in cost-effectiveness that arise through changes in technical efficiency or the opportunity cost linked to health system strengthening.

This paper is written with international development investment decisions in mind, and in particular the challenge of investments that can mostly be characterised as health system strengthening. But it’s easy to see how many – perhaps all – health services are interdependent. If anything, the broader impact of new interventions on health systems should be considered as standard. The methods described in this paper provide a useful framework to tackle these issues, with food for thought for anybody engaged in cost-effectiveness analysis.

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Simon McNamara’s journal round-up for 6th August 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.

Euthanasia, religiosity and the valuation of health states: results from an Irish EQ5D5L valuation study and their implications for anchor values. Health and Quality of Life Outcomes [PubMed] Published 31st July 2018

Do you support euthanasia? Do you think there are health states worse than death? Are you religious? Don’t worry – I am not commandeering this week’s AHE journal round-up just to bombard you with a series of difficult questions. These three questions form the foundation of the first article selected for this week’s round-up.

The paper is based upon the hypothesis that your religiosity (“adherence to religious beliefs”) is likely to impact your support for euthanasia and, subsequently, the likelihood of you valuing severe health states as worse than death. This seems like a logical hypothesis. Religions tend to be anti-euthanasia, and so it appears likely that religious people will have lower levels of support for euthanasia than non-religious people. Equally, if you don’t support the principle of euthanasia, it stands to reason that you are likely to be less willing to choose immediate death over living in a severe health state – something you would need to do for a health state to be considered as being worse than death in a time trade-off (TTO) study.

The authors test this hypothesis using a sub-sample of data (n=160) collected as part of the Irish EQ-5D-5L TTO valuation study. Perhaps unsurprisingly, the authors find evidence in support of the above hypotheses. Those that attend a religious service weekly were more likely to oppose euthanasia than those who attend a few times a year or less, and those who oppose euthanasia were less likely to give “worse than death” responses in the TTO than those that support it.

I found this paper really interesting, as it raises a number of challenging questions. If a society is made up of people with heterogeneous beliefs regarding religion, how should we balance these in the valuation of health? If a society is primarily non-religious is it fair to apply this valuation tariff to the lives of the religious, and vice versa? These certainly aren’t easy questions to answer, but may be worth reflecting on.

E-learning and health inequality aversion: A questionnaire experiment. Health Economics [PubMed] [RePEc] Published 22nd July 2018

Moving on from the cheery topic of euthanasia, what do you think about socioeconomic inequalities in health? In my home country, England, if you are from the poorest quintile of society, you can expect to experience 62 years in full health in your lifetime, whilst if you are from the richest quintile, you can expect to experience 74 years – a gap of 12 years.

In the second paper to be featured in this round-up, Cookson et al. explore the public’s willingness to sacrifice incremental population health gains in order to reduce these inequalities in health – their level of “health inequality aversion”. This is a potentially important area of research, as the vast majority of economic evaluation in health is distributionally-naïve and effectively assumes that members of the public aren’t at all concerned with inequalities in health.

The paper builds on prior work conducted by the authors in this area, in which they noted a high proportion of respondents in health inequality aversion elicitation studies appear to be so averse to inequalities that they violate monotonicity – they choose scenarios that reduce inequalities in health even if these scenarios reduce the health of the rich at no gain to the poor, or they reduce the health of the poor, or they may reduce the health of both groups. The authors hypothesise that these monotonicity violations may be due to incomplete thinking from participants, and suggest that the quality of their thinking could be improved by two e-learning educational interventions. The primary aim of the paper is to test the impact of these interventions in a sample of the UK public (n=60).

The first e-learning intervention was an animated video that described a range of potential positions that a respondent could take (e.g. health maximisation, or maximising the health of the worst off). The second was an interactive spreadsheet-based questionnaire that presented the consequences of the participant’s choices, prior to them confirming their selection. Both interventions are available online.

The authors found that the interactive tool significantly reduced the amount of extreme egalitarian (monotonicity-violating) responses, compared to a non-interactive, paper-based version of the study. Similarly, when the video was watched before completing the paper-based exercise, the number of extreme egalitarian responses reduced. However, when the video was watched before the interactive tool there was no further decrease in extreme egalitarianism. Despite this reduction in extreme egalitarianism, the median levels of inequality aversion remained high, with implied weights of 2.6 and 7.0 for QALY gains granted to someone from the poorest fifth of society, compared to the richest fifth of society for the interactive questionnaire and video groups respectively.

This is an interesting study that provides further evidence of inequality aversion, and raises further concern about the practical dominance of distributionally-naïve approaches to economic evaluation. The public does seem to care about distribution. Furthermore, the paper demonstrates that participant responses to inequality aversion exercises are shaped by the information given to them, and the way that information is presented. I look forward to seeing more studies like this in the future.

A new method for valuing health: directly eliciting personal utility functions. The European Journal of Health Economics [PubMed] [RePEc] Published 20th July 2018

Last, but not least, for this round-up, is a paper by Devlin et al. on a new method for valuing health.

The relative valuation of health states is a pretty important topic for health economists. If we are to quantify the effectiveness, and subsequently cost-effectiveness, of an intervention, we need to understand which health states are better than others, and how much better they are. Traditionally, this is done by asking members of the public to choose between different health profiles featuring differing levels of fulfilment of a range of domains of health, in order to ‘uncover’ the relative importance the respondent places on these domains, and levels. These can then be used in order to generate social tariffs that assign a utility value to a given health state for use in economic evaluation.

The authors point out that, in the modern day, valuation studies can be conducted rapidly, and at scale, online, but at the potential cost of deliberation from participants, and the resultant risk of heuristic dominated decision making. In response to this, the authors propose a new method – direct elicitation of personal utility functions, and pilot its use for the valuation of EQ-5D in a sample of the English public (n=76).

The proposed approach differs from traditional approaches in three key ways. Firstly, instead of simply attempting to infer the relative importance that participants place on differing domains based upon choices between health profiles, the respondents are asked directly about the relative importance they place on differing domains of health, prior to validating these with profile choices. Secondly, the authors place a heavy emphasis on deliberation, and the construction, rather than uncovering, of preferences during the elicitation exercises. Thirdly, a “personal utility function” for each individual is constructed (in effect a personal EQ-5D tariff), and these individual utility functions are subsequently aggregated into a social utility function.

In the pilot, the authors find that the method appears feasible for wider use, albeit with some teething troubles associated with the computer-based tool developed to implement it, and the skills of the interviewers.

This direct method raises an interesting question for health economics – should we be inferring preferences based upon choices that differ in terms of certain attributes, or should we just ask directly about the attributes? This is a tricky question. It is possible that the preferences elicited via these different approaches could result in different preferences – if they do, on what grounds should we choose one or other? This requires a normative judgment, and at present, it appears both are (potentially) as legitimate as each other.

Whilst the authors apply this direct method to the valuation of health, I don’t see why similar approaches couldn’t be applied to any multi-attribute choice experiment. Keep your eyes out for future uses of it in valuation, and perhaps beyond? It will be interesting to see how it develops.

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