Chris Sampson’s journal round-up for 31st December 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.

Perspectives of patients with cancer on the quality-adjusted life year as a measure of value in healthcare. Value in Health Published 29th December 2018

Patients should have the opportunity to understand how decisions are made about which treatments they are and are not allowed to use, given their coverage. This study reports on a survey of cancer patients and survivors, with the aim of identifying patients’ awareness, understanding, and opinions about the QALY as a measure of value.

Participants were recruited from a (presumably US-based) patient advocacy group and 774 mostly well-educated, mostly white, mostly women responded. The online survey asked about cancer status and included a couple of measures of health literacy. Fewer than 7% of participants had ever heard of the QALY – more likely for those with greater health literacy. The survey explained the QALY to the participants and then asked if the concept of the QALY makes sense. Around half said it did and 24% thought that it was a good way to measure value in health care. The researchers report a variety of ‘significant’ differences in tendencies to understand or support the use of QALYs, but I’m not convinced that they’re meaningful because the differences aren’t big and the samples are relatively small.

At the end of the survey, respondents were asked to provide opinions on QALYs and value in health care. 165 people provided responses and these were coded and analysed qualitatively. The researchers identified three themes from this one free-text question: i) measuring value, ii) opinions on QALY, and iii) value in health care and decision making. I’m not sure that they’re meaningful themes that help us to understand patients’ views on QALYs. A significant proportion of respondents rejected the idea of using numbers to quantify value in health care. On the other hand, some suggested that the QALY could be a useful decision aid for patients. There was opposition to ‘external decision makers’ having any involvement in health care decision making. Unless you’re paying for all of your care out of pocket, that’s tough luck. But the most obvious finding from the qualitative analysis is that respondents didn’t understand what QALYs were for. That’s partly because health economists in general need to be better at communicating concepts like the QALY. But I think it’s also in large part because the authors failed to provide a clear explanation. They didn’t even use my lovely Wikipedia graphic. Many of the points made by respondents are entirely irrelevant to the appropriateness of QALYs as they’re used (or in the case of the US, aren’t yet used) in practice. For example, several discussed the use of QALYs in clinical decision making. Patients think that they should maintain autonomy, which is fair enough but has nothing to do with how QALYs are used to assess health technologies.

QALYs are built on the idea of trade-offs. They measure the trade-off between life extension and life improvement. They are used to guide trade-offs between different treatments for different people. But the researchers didn’t explain how or why QALYs are used to make trade-offs, so the elicited views aren’t well-informed.

Measuring multivariate risk preferences in the health domain. Journal of Health Economics Published 27th December 2018

Health preferences research is now a substantial field in itself. But there’s still a lot of work left to be done on understanding risk preferences with respect to health. Gradually, we’re coming round to the idea that people tend to be risk-averse. But risk preferences aren’t (necessarily) so simple. Recent research has proposed that ‘higher order’ preferences such as prudence and temperance play a role. A person exhibiting univariate prudence for longevity would be better able to cope with risk if they are going to live longer. Univariate temperance is characterised by a preference for prospects that disaggregate risk across different possible outcomes. Risk preferences can also be multivariate – across health and wealth, for example – determining the relationship between univariate risk preferences and other attributes. These include correlation aversion, cross-prudence, and cross-temperance. Many articles from the Arthur Attema camp demand a great deal of background knowledge. This paper isn’t an exception, but it does provide a very clear and intuitive description of the various kinds of uni- and multivariate risk preferences that the researchers are considering.

For this study, an experiment was conducted with 98 people, who were asked to make 69 choices, corresponding to 3 choices about each risk preference trait being tested, for both gains and losses. Participants were told that they had €240,000 in wealth and 40 years of life to play with. The number of times that an individual made choices in line with a particular trait was used as an indicator of their strength of preference.

For gains, risk aversion was common for both wealth and longevity, and prudence was a common trait. There was no clear tendency towards temperance. For losses, risk aversion and prudence tended to neutrality. For multivariate risk preferences, a majority of people were correlation averse for gains and correlation seeking for losses. For gains, 76% of choices were compatible with correlation aversion, suggesting that people prefer to disaggregate fixed wealth and health gains. For losses, the opposite was true in 68% of choices. There was evidence for cross-prudence in wealth gains but not longevity gains, suggesting that people prefer health risk if they have higher wealth. For losses, the researchers observed cross-prudence and cross-temperance neutrality. The authors go on to explore associations between different traits.

A key contribution is in understanding how risk preferences differ in the health domain as compared with the monetary domain (which is what most economists study). Conveniently, there are a lot of similarities between risk preferences in the two domains, suggesting that health economists can learn from the wider economics literature. Risk aversion and prudence seem to apply to longevity as well as monetary gains, with a shift to neutrality in losses. The potential implications of these findings are far-reaching, but this is just a small experimental study. More research needed (and anticipated).

Prospective payment systems and discretionary coding—evidence from English mental health providers. Health Economics [PubMed] Published 27th December 2018

If you’ve conducted an economic evaluation in the context of mental health care in England, you’ll have come across mental health care clusters. Patients undergoing mental health care are allocated to one of 20 clusters, classed as either ‘psychotic’, ‘non-psychotic’, or ‘organic’, which forms the basis of an episodic payment model. In 2013/14, these episodes were associated with an average cost of between £975 and £9,354 per day. Doctors determine the clusters and the clusters determine reimbursement. Perverse incentives abound. Or do they?

This study builds on the fact that patients are allocated by clinical teams with guidance from the algorithm-based Mental Health Clustering Tool (MHCT). Clinical teams might exhibit upcoding, whereby patients are allocated to clusters that attract a higher price than that recommended by the MHCT. Data were analysed for 148,471 patients from the Mental Health Services Data Set for 2011-2015. For each patient, their allocated cluster is known, along with a variety of socioeconomic indicators and the HoNoS and SARN instruments, which go into the MHCT algorithm. Mixed-effects logistic regression was used to look at whether individual patients were or were not allocated to the cluster recommended as ‘best fit’ by the MHCT, controlling for patient and provider characteristics. Further to this, multilevel multinomial logit models were used to categorise decisions that don’t match the MHCT as either under- or overcoding.

Average agreement across clusters between the MHCT and clinicians was 36%. In most cases, patients were allocated to a cluster either one step higher or one step lower in terms of the level of need, and there isn’t an obvious tendency to overcode. The authors are able to identify a few ways in which observable provider and patient characteristics influence the tendency to under- or over-cluster patients. For example, providers with higher activity are less likely to deviate from the MHCT best fit recommendation. However, the dominant finding – identified by using median odds ratios for the probability of a mismatch between two random providers – seems to be that unobserved heterogeneity determines variation in behaviour.

The study provides clues about the ways in which providers could manipulate coding to their advantage and identifies the need for further data collection for a proper assessment. But reimbursement wasn’t linked to clustering during the time period of the study, so it remains to be seen how clinicians actually respond to these potentially perverse incentives.

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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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Chris Sampson’s journal round-up for 15th October 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.

Reliability and validity of the contingent valuation method for estimating willingness to pay: a case of in vitro fertilisation. Applied Health Economics and Health Policy [PubMed] Published 13th October 2018

In vitro fertilisation (IVF) is a challenge for standard models of valuation in health economics. Mostly, that’s because, despite it falling within the scope of health care, and despite infertility being a health problem, many of the benefits of IVF can’t be considered health-specific. QALYs can’t really do the job, so there’s arguably a role for cost-benefit analysis, and for using stated preference methods to determine the value of IVF. This study adds to an existing literature studying willingness to pay for IVF, but differs in that it tries to identify willingness to pay (WTP) from the general population. This study is set in Australia, where IVF is part-funded by universal health insurance, so asking the public is arguably the right thing to do.

Three contingent valuation surveys were conducted online with 1,870 people from the general public. The first survey used a starting point bid of $10,000, and then, 10 months later, two more surveys were conducted with starting point bids of $4,000 and $10,000. Each included questions for a 10%, 20%, and 50% success rate. Respondents were asked to adopt an ex-post perspective, assuming that they were infertile and could conceive by IVF. Individuals could respond to starting bids with ‘yes’, ‘no’, ‘not sure’, or ‘I am not willing to pay anything’. WTP for one IVF cycle with a 20% success rate ranged from $6,353 in the $4,000 survey to $11,750 in the first $10,000 survey. WTP for a year of treatment ranged from $18,433 to $28,117. The method was reliable insofar as there were no differences between the first and second $10,000 surveys. WTP values corresponded to the probability of success, providing support for the internal construct validity of the survey. However, the big difference between values derived using the alternative starting point bids indicates a strong anchoring bias. The authors also tested the external criterion validity by comparing the number of respondents willing to pay more than $4,000 for a cycle with a 20% success rate (roughly equivalent to the out of pocket cost in Australia) with the number of people who actually choose to pay for IVF in Australia. Around 63% of respondents were willing to pay at that price, which is close to the estimated 60% in Australia.

This study provides some support for the use of contingent valuation methods in the context of IVF, and for its use in general population samples. But the anchoring effect is worrying and justifies further research to identify appropriate methods to counteract this bias. The exclusion of the “not sure” and “I will not pay anything” responses from the analysis – as ‘non-demanders’ – arguably undermines the ‘societal valuation’ aspect of the estimates.

Pharmaceutical expenditure and gross domestic product: evidence of simultaneous effects using a two‐step instrumental variables strategy. Health Economics [PubMed] Published 10th October 2018

The question of how governments determine spending on medicines is pertinent in the UK right now, as the Pharmaceutical Price Regulation Scheme approaches its renewal date. The current agreement includes a cap on pharmaceutical expenditure. It should go without saying that GDP ought to have some influence on how much public spending is dedicated to medicines. But, when medicines expenditure might also influence GDP, the actual relationship is difficult to estimate. In this paper, the authors seek to identify both effects: the income elasticity of government spending on pharmaceuticals and the effect of that spending on income.

The authors use a variety of data sources from the World Health Organization, World Bank, and International Monetary Fund to construct an unbalanced panel for 136 countries from 1995 to 2006. To get around the challenge of two-way causality, the authors implement a two-step instrumental variable approach. In the first step of the procedure, a model estimates the impact of GDP per capita on government spending on pharmaceuticals. International tourist receipts are used as an instrument that is expected to correlate strongly with GDP per capita, but which is expected to be unrelated to medicines expenditure (except through its correlation with GDP). The model attempts to control for health care expenditure, life expectancy, and other important country-specific variables. In the second step, a reverse causality model is used to assess the impact of pharmaceutical expenditure on GDP per capita, with pharmaceutical expenditure adjusted to partial-out the response to GDP estimated in the first step.

The headline average results are that GDP increases pharmaceutical expenditure and that pharmaceutical expenditure reduces GDP. A 1% increase in GDP per capita increases public pharmaceutical expenditure per capita by 1.4%, suggesting that pharmaceuticals are a luxury good. A 1% increase in public pharmaceutical expenditure is associated with a 0.09% decrease in GDP per capita. But the results are more nuanced than that. The authors outline various sources of heterogeneity. The positive effect of GDP on pharmaceutical expenditure only holds for high-income countries and the negative effect of pharmaceutical expenditure on GDP only holds for low-income countries. Quantile regressions show that income elasticity decreases for higher quantiles of expenditure. GDP only influences pharmaceutical spending in countries classified as ‘free’ on the index of Economic Freedom of the World, and pharmaceutical expenditure only has a negative impact on GDP in countries that are ‘not free’.

I’ve never come across this kind of two-step approach before, so I’m still trying to get my head around whether the methods and the data are adequate. But a series of robustness checks provide some reassurance. In particular, an analysis of intertemporal effects using lagged GDP and lagged pharmaceutical expenditure demonstrates the robustness of the main findings. Arguably, the findings of this study are more important for policymaking in low- and middle-income countries, where pharmaceutical expenditures might have important consequences for GDP. In high-income (and ‘free’) economies that spend a lot on medicines, like the UK, there is probably less at stake. This could be because of effective price regulation and monitoring, and better adherence, ensuring that pharmaceutical expenditure is not wasteful.

Parental health spillover in cost-effectiveness analysis: evidence from self-harming adolescents in England. PharmacoEconomics [PubMed] [RePEc] Published 8th October 2018

Any intervention has the potential for spillover effects, whereby people other than the recipient of care are positively or negatively affected by the consequences of the intervention. Where a child is the recipient of care, it stands to reason that any intervention could affect the well-being of the parents and that these impacts should be considered in economic evaluation. But how should parental spillovers be incorporated? Are parental utilities additive to that of the child patient? Or should a multiplier effect be used with reference to the effect of an intervention on the child’s utility?

The study reports on a trial-based economic evaluation of family therapy for self-harming adolescents aged 11-17. Data collection included EQ-5D-3L for the adolescents and HUI2 for the main caregiver (86% mothers) at baseline, 6-month follow-up, and 12-month follow-up, collected from 731 patient-parent pairs. The authors outline six alternative methods for including parental health spillovers: i) relative health spillover, ii) relative health spillover per treatment arm, iii) absolute health spillover, iv) absolute global health spillover per treatment arm, v) additive accrued health benefits, and vi) household equivalence scales. These differ according to whether parental utility is counted as depending on adolescent’s utility, treatment allocation, the primary outcome of the study, or some combination thereof. But the authors’ primary focus (and the main contribution of this study) is the equivalence scale option. This involves adding together the spillover effects for other members of the household and using alternative weightings depending on the importance of parental utility compared with adolescent utility.

Using Tobit models, controlling for a variety of factors, the authors demonstrate that parental utility is associated with adolescent utility. Then, economic evaluations are conducted using each of the alternative spillover accounting methods. The base case of including only adolescents’ utility delivers an ICER of around £40,453. Employing the alternative methods gives quite different results, with the intervention dominated in two of the cases and an ICER below £30,000 per QALY in others. For the equivalence scale approach, the authors employ several elasticities for spillover utility, ranging from 0 (where parental utility is of equivalent value to adolescent utility and therefore additive) to 1 (where the average health spillover per household member is estimated for each patient). The ICER estimates using the equivalence scale approach ranged from £27,166 to £32,504. Higher elasticity implied lower cumulated QALYs.

The paper’s contribution is methodological, and I wouldn’t read too much into the magnitude of the results. For starters, the use of HUI2 (a measure for children) in adults and the use of EQ-5D-3L (a measure for adults) in the children is somewhat confusing. The authors argue that health gains should only be aggregated at the household level if the QALY gain for the patient is greater or equal to zero, because the purpose of treatment is to benefit the adolescents, not the parents. And they argue in favour of using an equivalence scale approach. By requiring an explicit judgement to set the elasticity within the estimation, the method provides a useful and transparent approach to including parental spillovers.

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