Chris Sampson’s journal round-up for 1st April 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.

Toward a centralized, systematic approach to the identification, appraisal, and use of health state utility values for reimbursement decision making: introducing the Health Utility Book (HUB). Medical Decision Making [PubMed] Published 22nd March 2019

Every data point reported in research should be readily available to us all in a structured knowledge base. Most of us waste most of our time retreading old ground, meaning that we don’t have the time to do the best research possible. One instance of this is in the identification of health state utility values to plug into decision models. Everyone who builds a model in a particular context goes searching for utility values – there is no central source. The authors of this paper are hoping to put an end to that.

The paper starts with an introduction to the importance of health state utility values in cost-effectiveness analysis, which most of us don’t need to read. Of course, the choice of utility values in a model is very important and can dramatically alter estimates of cost-effectiveness. The authors also discuss issues around the identification of utility values and the assessment of their quality and applicability. Then we get into the objectives of the ‘Health Utility Book’, which is designed to tackle these issues.

The Health Utility Book will consist of a registry (I like registries), backed by a systematic approach to the identification and inclusion (registration?) of utility values. The authors plan to develop a quality assessment tool for studies that report utility values, using a Delphi panel method to identify appropriate indicators of quality to be included. The quality assessment tool will be complemented by a tool to assess applicability, which will be developed through interviews with stakeholders involved in the reimbursement process.

In the first place, the Health Utility Book will only compile utility values for cancer, and some of the funding for the project is cancer specific. To survive, the project will need more money from more sources. To be sustainable, the project will need to attract funding indefinitely. Or perhaps it could morph into a crowd-sourced platform. Either way, the Health Utility Book has my support.

A review of attitudes towards the reuse of health data among people in the European Union: the primacy of purpose and the common good. Health Policy Published 21st March 2019

We all agree that data protection is important. We all love the GDPR. Organisations such as the European Council and the OECD are committed to facilitating the availability of health data as a means of improving population health. And yet, there often seem to be barriers to accessing health data, and we occasionally hear stories of patients opposing data sharing (e.g. care.data). Maybe people don’t want researchers to be using their data, and we just need to respect that. Or, more likely, we need to figure out what it is that people are opposed to, and design systems that recognise this.

This study reviews research on attitudes towards the sharing of health data for purposes other than treatment, among people living in the EU, employing a ‘configurative literature synthesis’ (a new one for me). From 5,691 abstracts, 29 studies were included. Most related to the use of health data in research in general, while some focused on registries. A few studies looked at other uses, such as for planning and policy purposes. And most were from the UK.

An overarching theme was a low awareness among the population about the reuse of health data. However, in some studies, a desire to be better informed was observed. In general, views towards the use of health data were positive. But this was conditional on the data being used to serve the common good. This includes such purposes as achieving a better understanding of diseases, improving treatments, or achieving more efficient health care. Participants weren’t so happy with health data reuse if it was seen to conflict with the interests of patients providing the data. Commercialisation is a big concern, including the sale of data and private companies profiting from the data. Employers and insurance companies were also considered a threat to patients’ interests. There were conflicting views about whether it is positive for pharmaceutical companies to have access to health data. A minority of people were against sharing data altogether. Certain types of data are seen as being particularly sensitive, including those relating to mental health or sexual health. In general, people expressed concern about data security and the potential for leaks. The studies also looked at the basis for consent that people would prefer. A majority accepted that their data could be used without consent so long as the data were anonymised. But there were no clear tendencies of preference for the various consent models.

It’s important to remember that – on the whole – patients want their data to be used to further the common good. But support can go awry if the data are used to generate profits for private firms or used in a way that might be perceived to negatively affect patients.

Health-related quality of life in injury patients: the added value of extending the EQ-5D-3L with a cognitive dimension. Quality of Life Research [PubMed] Published 18th March 2019

I’m currently working on a project to develop a cognition ‘bolt-on’ for the EQ-5D. Previous research has demonstrated that a cognition bolt-on could provide additional information to distinguish meaningful differences between health states, and that cognition might be a more important candidate than other bolt-ons. Injury – especially traumatic brain injury – can be associated with cognitive impairments. This study explores the value of a cognition bolt-on in this context.

The authors sought to find out whether cognition is sufficiently independent of other dimensions, whether the impact of cognitive problems is reflected in the EuroQol visual analogue scale (EQ VAS), and how a cognition bolt-on affects the overall explanatory power of the EQ-5D-3L. The data used are from the Dutch Injury Surveillance System, which surveys people who have attended an emergency department with an injury, including EQ-5D-3L. The survey adds a cognitive bolt-on relating to memory and concentration.

Data were available for 16,624 people at baseline, with 5,346 complete responses at 2.5-month follow-up. The cognition item was the least affected, with around 20% reporting any problems (though it’s worth noting that the majority of the cohort had injuries to parts of the body other than the head). The frequency of different responses suggests that cognition is dominant over other dimensions in the sense that severe cognitive problems tend to be observed alongside problems in other dimensions, but not vice versa. The mean EQ VAS for people reporting severe cognitive impairment was 41, compared with a mean of 75 for those reporting no problems. Regression analysis showed that moderate and severe cognitive impairment explained 8.7% and 6.2% of the variance of the EQ VAS. Multivariate analysis suggested that the cognitive dimension added roughly the same explanatory power as any other dimension. This was across the whole sample. Interestingly (or, perhaps, worryingly) when the authors looked at the subset of people with traumatic brain injury, the explanatory power of the cognitive dimension was slightly lower than overall.

There’s enough in this paper to justify further research into the advantages and disadvantages of using a cognition bolt-on. But I would say that. Whether or not the bolt-on descriptors used in this study are meaningful to patients remains an open question.

Developing the role of electronic health records in economic evaluation. The European Journal of Health Economics [PubMed] Published 14th March 2019

One way that we can use patients’ routinely collected data is to support the conduct of economic evaluations. In this commentary, the authors set out some of the ways to make the most of these data and discuss some of the methodological challenges. Large datasets have the advantage of being large. When this is combined with the collection of sociodemographic data, estimates for sub-groups can be produced. The data can also facilitate the capture of outcomes not otherwise available. For example, the impact of bariatric surgery on depression outcomes could be identified beyond the timeframe of a trial. The datasets also have the advantage of being representative, where trials are not. This could mean more accurate estimates of costs and outcomes. But there are things to bear in mind when using the data, such as the fact that coding might not always be very accurate, and coding practices could vary between observations. Missing data are likely to be missing for a reason (i.e. not at random), which creates challenges for the analyst. I had hoped that this paper would discuss novel uses of routinely collected data systems, such as the embedding of economic evaluations within them, rather than simply their use to estimate parameters for a model. But if you’re just getting started with using routine data, I suppose you could do worse than start with this paper.

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Chris Sampson’s journal round-up for 27th 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.

Ethically acceptable compensation for living donations of organs, tissues, and cells: an unexploited potential? Applied Health Economics and Health Policy [PubMed] Published 25th August 2018

Around the world, there are shortages of organs for transplantation. In economics, the debate around the need to increase organ donation can be frustratingly ignorant of ethical and distributional concerns. So it’s refreshing to see this article attempting to square concerns about efficiency and equity. The authors do so by using a ‘spheres of justice’ framework. This is the idea that different social goods should be distributed according to different principles. So, while we might be happy for brocolli and iPhones to be distributed on the basis of free exchange, we might want health to be distributed on the basis of need. The argument can be extended to state that – for a just situation to prevail – certain exchanges between these spheres of justice (e.g. health for iPhones) should never take place. This idea might explain why – as the authors demonstrate with a review of European countries – policy tends not to allow monetary compensation for organ donation.

The paper cleverly sets out to taxonomise monetary and non-monetary reimbursement and compensation with reference to individuals’ incentives and the spheres of justice principles. From this, the authors reach two key conclusions. Firstly, that (monetary) reimbursement of donors’ expenses (e.g. travel costs or lost earnings) is ethically sound as this does not constitute an incentive to donate but rather removes existing disincentives. Secondly, that non-monetary compensation could be deemed ethical.

Three possible forms of non-monetary compensation are discussed: i) prioritisation, ii) free access, and iii) non-health care-related benefits. The first could involve being given priority for receiving organs, or it could extend to the jumping of other health care waiting lists. I think this is more problematic than the authors let on because it asserts that health care should – at least in part – be distributed according to desert rather than need. The second option – free access – could mean access to health care that people would otherwise have to pay for. The third option could involve access to other social goods such as education or housing.

This is an interesting article and an enjoyable read, but I don’t think it provides a complete solution. Maybe I’m just too much of a Marxist, but I think that this – as all other proposals – fails to distribute from each according to ability. That is, we’d still expect non-monetary compensation to incentivise poorer (and on average less healthy) people to donate organs, thus exacerbating health inequality. This is because i) poorer people are more likely to need the non-monetary benefits and ii) we live in a capitalist society in which there is almost nothing that money can’t by and which is strictly non-monetary. Show me a proposal that increases donation rates from those who can most afford to donate them (i.e. the rich and healthy).

Selecting bolt-on dimensions for the EQ-5D: examining their contribution to health-related quality of life. Value in Health Published 18th August 2018

Measures such as the EQ-5D are used to describe health-related quality of life as completely and generically as possible. But there is a trade-off between completeness and the length of the questionnaire. Necessarily, there are parts of the evaluative space that measures will not capture because they are a simplification. If the bit they’re missing is important to your patient group, that’s a problem. You might fancy a bolt-on. But how do we decide which areas of the evaluative space should be more completely included in the measure? Which bolt-ons should be used? This paper seeks to provide means of answering these questions.

The article builds on an earlier piece of work that was included in an earlier journal round-up. In the previous paper, the authors used factor analysis to identify candidate bolt-ons. The goal of this paper is to outline an approach for specifying which of these candidates ought to be used. Using data from the Multi-Instrument Comparison study, the authors fit linear regressions to see how well 37 candidate bolt-on items explain differences in health-related quality of life. The 37 items correspond to six different domains: energy/vitality, satisfaction, relationships, hearing, vision, and speech. In a second test, the authors explored whether the bolt-on candidates could explain differences in health-related quality of life associated with six chronic conditions. Health-related quality of life is defined according to a visual analogue scale, which notably does not correspond to that used in the EQ-5D but rather uses a broader measure of physical, mental, and social health.

The results suggest that items related to energy/vitality, relationships, and satisfaction explained a significant part of health-related quality of life on top of the existing EQ-5D dimensions. The implication is that these could be good candidates for bolt-ons. The analysis of the different conditions was less clear.

For me, there’s a fundamental problem with this study. It moves the goals posts. Bolt-ons are about improving the extent to which a measure can more accurately represent the evaluative space that it is designed to characterise. In this study, the authors use a broader definition of health-related quality of life that – as far as I can tell – the EQ-5D is not designed to capture. We’re not dealing with bolt-ons, we’re dealing with extensions to facilitate expansions to the evaluative space. Nevertheless, the method could prove useful if combined with a more thorough consideration of the evaluative space.

Sources of health financing and health outcomes: a panel data analysis. Health Economics [PubMed] [RePEc] Published 15th August 2018

There is a growing body of research looking at the impact that health (care) spending has on health outcomes. Usually, these studies don’t explicitly look at who is doing the spending. In this study, the author distinguishes between public and private spending and attempts to identify which type of spending (if either) results in greater health improvements.

The author uses data from the World Bank’s World Development Indicators for 1995-2014. Life expectancy at birth is adopted as the primary health outcome and the key expenditure variables are health expenditure as a share of GDP and private health expenditure as a share of total health expenditure. Controlling for a variety of other variables, including some determinants of health such as income and access to an improved water source, a triple difference analysis is described. The triple difference estimator corresponds to the difference in health outcomes arising from i) differences in the private expenditure level, given ii) differences in total expenditure, over iii) time.

The key finding from the study is that, on average, private expenditure is more effective in increasing life expectancy at birth than public expenditure. The author also looks at government effectiveness, which proves crucial. The finding in favour of private expenditure entirely disappears when only countries with effective government are considered. There is some evidence that public expenditure is more effective in these countries, and this is something that future research should investigate further. For countries with ineffective governments, the implication is that policy should be directed towards increasing overall health care expenditure by increasing private expenditure.

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

An exploratory study on using principal-component analysis and confirmatory factor analysis to identify bolt-on dimensions: the EQ-5D case study. Value in Health Published 14th July 2017

I’m not convinced by the idea of using bolt-on dimensions for multi-attribute utility instruments. A state description with a bolt-on refers to a different evaluative space, and therefore is not comparable with the progenitor, thus undermining its purpose. Maybe this study will persuade me otherwise. The authors analyse data from the Multi Instrument Comparison database, including responses to EQ-5D-5L, SF-6D, HUI3, AQoL 8D and 15D questionnaires, as well as the ICECAP and 3 measures of subjective well-being. Content analysis was used to allocate items from the measures to underlying constructs of health-related quality of life. The sample of 8022 was randomly split, with one half used for principal-component analysis and confirmatory factor analysis, and the other used for validation. This approach looks at the underlying constructs associated with health-related quality of life and the extent to which individual items from the questionnaires influence them. Candidate items for bolt-ons are those items from questionnaires other than the EQ-5D that are important and not otherwise captured by the EQ-5D questions. The principal-component analysis supported a 9-component model: physical functioning, psychological symptoms, satisfaction, pain, relationships, speech/cognition, hearing, energy/sleep and vision. The EQ-5D only covered physical functioning, psychological symptoms and pain. Therefore, items from measures that explain the other 6 components represent bolt-on candidates for the EQ-5D. This study succeeds in its aim. It demonstrates what appears to be a meaningful quantitative approach to identifying items not fully captured by the EQ-5D, which might be added as bolt-ons. But it doesn’t answer the question of which (if any) of these bolt-ons ought to be added, or in what circumstances. That would at least require pre-definition of the evaluative space, which might not correspond to the authors’ chosen model of health-related quality of life. If it does, then these findings would be more persuasive as a reason to do away with the EQ-5D altogether.

Endogenous information, adverse selection, and prevention: implications for genetic testing policy. Journal of Health Economics Published 13th July 2017

If you can afford it, there are all sorts of genetic tests available nowadays. Some of them could provide valuable information about the risk of particular health problems in the future. Therefore, they can be used to guide individuals’ decisions about preventive care. But if the individual’s health care is financed through insurance, that same information could prove costly. It could reinforce that classic asymmetry of information and adverse selection problem. So we need policy that deals with this. This study considers the incentives and insurance market outcomes associated with four policy options: i) mandatory disclosure of test results, ii) voluntary disclosure, iii) insurers knowing the test was taken, but not the results and iv) complete ban on the use of test information by insurers. The authors describe a utility model that incorporates the use of prevention technologies, and available insurance contracts, amongst people who are informed or uninformed (according to whether they have taken a test) and high or low risk (according to test results). This is used to estimate the value of taking a genetic test, which differs under the four different policy options. Under voluntary disclosure, the information from a genetic test always has non-negative value to the individual, who can choose to only tell their insurer if it’s favourable. The analysis shows that, in terms of social welfare, mandatory disclosure is expected to be optimal, while an information ban is dominated by all other options. These findings are in line with previous studies, which were less generalisable according to the authors. In the introduction, the authors state that “ethical issues are beyond the scope of this paper”. That’s kind of a problem. I doubt anybody who supports an information ban does so on the basis that they think it will maximise social welfare in the fashion described in this paper. More likely, they’re worried about the inequities in health that mandatory disclosure could reinforce, about which this study tells us nothing. Still, an information ban seems to be a popular policy, and studies like this indicate that such decisions should be reconsidered in light of their expected impact on social welfare.

Returns to scientific publications for pharmaceutical products in the United States. Health Economics [PubMedPublished 10th July 2017

Publication bias is a big problem. Part of the cause is that pharmaceutical companies have no incentive to publish negative findings for their own products. Though positive findings may be valuable in terms of sales. As usual, it isn’t quite that simple when you really think about it. This study looks at the effect of publications on revenue for 20 branded drugs in 3 markets – statins, rheumatoid arthritis and asthma – using an ‘event-study’ approach. The authors analyse a panel of quarterly US sales data from 2003-2013 alongside publications identified through literature searches and several drug- and market-specific covariates. Effects are estimated using first difference and difference in first difference models. The authors hypothesise that publications should have an important impact on sales in markets with high generic competition, and less in those without or with high branded competition. Essentially, this is what they find. For statins and asthma drugs, where there was some competition, clinical studies in high-impact journals increased sales to the tune of $8 million per publication. For statins, volume was not significantly affected, with mediation through price. In rhematoid arthritis, where competition is limited, the effect on sales was mediated by the effect on volume. Studies published in lower impact journals seemed to have a negative influence. Cost-effectiveness studies were only important in the market with high generic competition, increasing statin sales by $2.2 million on average. I’d imagine that these impacts are something with which firms already have a reasonable grasp. But this study provides value to public policy decision makers. It highlights those situations in which we might expect manufacturers to publish evidence and those in which it might be worthwhile increasing public investment to pick up the slack. It could also help identify where publication bias might be a bigger problem due to the incentives faced by pharmaceutical companies.

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