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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Paul Mitchell’s journal round-up for 6th November 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.

A longitudinal study to assess the frequency and cost of antivascular endothelial therapy, and inequalities in access, in England between 2005 and 2015. BMJ Open [PubMed] Published 22nd October 2017

I am breaking one of my unwritten rules in a journal paper round-up by talking about colleagues’ work, but I feel it is too important not to provide a summary for a number of reasons. The study highlights the problems faced by regional healthcare purchasers in England when implementing national guideline recommendations on the cost-effectiveness of new treatments. The paper focuses on anti-vascular endothelial growth factor (anti-VEGF) medicines in particular, with two drugs, ranibizumab and aflibercept, offered to patients with a range of eye conditions, costing £550-800 per injection. Another drug, bevacizumab, that is closely related to ranibizumab and performs similarly in trials, could be provided at a fraction of the cost (£50-100 per injection), but it is currently unlicensed for eye conditions in the UK. This study investigates how the regional areas in England have coped with trying to provide the recommended drugs using administrative data from Hospital Episode Statistics in England between 2005-2015 by tracking their use since they have been recommended for a number of different eye conditions over the past decade. In 2014/15 the cost of these two new drugs for treating eye conditions alone was estimated at £447 million nationally. The distribution of where these drugs are provided is not equal, varying widely across regions after controlling for socio-demographics, suggesting an inequality of access associated with the introduction of these high-cost drugs over the past decade at a time of relatively low growth in national health spending. Although there are limitations associated with using data not intended for research purposes, the study shows how the most can be made from data routinely collected for non-research purposes. On a public policy level, it raises questions over the provision of such high-cost drugs, for which the authors state the NHS are currently paying more for than US insurers. Although it is important to be careful when comparing to unlicensed drugs, the authors point to clear evidence in the paper as to why their comparison is a reasonable one in this scenario, with a large opportunity cost associated with not including this option in national guidelines. If national recommendations continue to insist that such drugs be provided, clearer guidance is also required on how to disinvest from existing services at a regional level to reduce further examples of inequality in access in the future.

In search of a common currency: a comparison of seven EQ-5D-5L value sets. Health Economics [PubMed] Published 24th October 2017

For those of us out there who like a good valuation study, you will need to set yourself aside a good piece of time to work your way through this one. The new EQ-5D-5L measure of health status, with a primary purpose of generating quality-adjusted life years (QALYs) for economic evaluations, is now starting to have valuation studies emerging from different countries, whereby the relative importance of each of the measure dimensions and levels are quantified based on general population preferences. This study offers the first comparison of value sets across seven countries: 3 Western European (England, Netherlands, Spain), 1 North American (Canada), 1 South American (Uruguay), and two East Asian (Japan and South Korea). The authors in this paper aim to describe methodological differences between the seven value sets, compare the relative importance of dimensions, level decrements and scale length (i.e. quality/quantity trade-offs for QALYs), as well as developing a common (Western) currency across four of the value sets. In brief summary, there does appear to be similar trends across the three Western European countries: level decrements from levels 3 to 4 have the largest value, followed by levels 1 to 2. There is also a pattern in these three countries’ dimensions, whereby the two “symptom” dimensions (i.e. pain/discomfort, anxiety/depression) have equal importance to the other three “functioning” dimensions (i.e. mobility, self-care and usual activities). There are also clear differences with the other four value sets. Canada, although it also has the highest level decrements between levels 3 and 4 (49%), unusually has equal decrements for the remainder (17% x 3). For the other three countries, greater weight is attached to the three functioning dimensions relative to the two symptom dimensions. Although South Korea also has the greatest level decrements between level 3 and 4, it was greatest between level 4 and level 5 in Uruguay and levels 1 and 2 in Japan. Although the authors give a number of plausible reasons as to why these differences may occur, less justification is given in the choice of the four value sets they offer as a common currency, beyond the need to have a value set for countries that do not have one already. The most in-common value sets were the three Western European countries, so a Western European value set may have been more appropriate if the criterion was to have comparable values across countries. If the aim was really for a more international common currency, there are issues with the exclusion of non-Western countries’ value sets from their common currency version. Surely differences across cultures should be reflected in a common currency if they are apparent in different cultures and settings. A common currency should also have a better spread of regions geographically, with no country from Africa, the Middle East, Central and South Asia represented in this study, as well as no lower- and middle-income countries. Though this final criticism is out of the control of the authors based on current data availability.

Quantifying the relationship between capability and health in older people: can’t map, won’t map. Medical Decision Making [PubMed] Published 23rd October 2017

The EQ-5D is one of many ways quality of life can be measured within economic evaluations. A more recent way based on Amartya Sen’s capability approach has attempted to develop outcome measures that move beyond health-related aspects of quality of life captured by EQ-5D and similar measures used in the generation of QALYs. This study examines the relationship between the EQ-5D and the ICECAP-O capability measure in three different patient populations included in the Medical Crises in Older People programme in England. The authors propose a reasonable hypothesis that health could be considered a conversion factor for a person’s broader capability set, and so it is plausible to test how well the EQ-5D-3L dimension values and overall score can map onto the ICECAP-O overall score. Through numerous regressions performed, the strongest relationship between the two measures in this sample was an R-squared of 0.35. Interestingly, the dimensions on the EQ-5D that had a significant relationship with the ICECAP-O score were a mix of dimensions with a focus on functioning (i.e. self-care, usual activities) and symptoms (anxiety/depression), so overall capability on ICECAP-O appears to be related, at least to a small degree, to both health components of EQ-5D discussed in this round-up’s previous paper. The authors suggest it provides further evidence of the complementary data provided by EQ-5D and ICECAP-O, but the causal relationship, as the authors suggest, between both measures remains under-researched. Longitudinal data analysis would provide a more definitive answer to the question of how much interaction there is between these two measures and their dimensions as health and capability changes over time in response to different treatments and care provision.

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Chris Sampson’s journal round-up for 9th October 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.

Evaluating the relationship between visual acuity and utilities in patients with diabetic macular edema enrolled in intravitreal aflibercept studies. Investigative Ophthalmology & Visual Science [PubMed] Published October 2017

Part of my day job involves the evaluation of a new type of screening programme for diabetic eye disease, including the use of a decision analytic model. Cost-effectiveness models usually need health state utility values for parameters in order to estimate QALYs. There are some interesting challenges in evaluating health-related quality of life in the context of vision loss; does vision in the best eye or worst eye affect quality of life most; do different eye diseases have different impacts independent of sight loss; do generic preference-based measures even work in this context? This study explores some of these questions. It combines baseline and follow-up EQ-5D and VFQ-UI (a condition-specific preference-based measure) responses from 1,320 patients from 4 different studies, along with visual acuity data. OLS and random effects panel models are used to predict utility values dependent on visual acuity and other individual characteristics. Best-seeing eye seems to be a more important determinant than worst-seeing eye, which supports previous studies. But worst-seeing eye is still important, with about a third of the impact of best-seeing eye. So economic evaluations shouldn’t ignore the bilateral nature of eye disease. Visual acuity – in both best- and worst-seeing eye – was more strongly associated with the condition-specific VFQ-UI than with the EQ-5D index, leading to better predictive power, which is not a big surprise. One way to look at this is that the EQ-5D underestimates the impact of visual acuity on utility. An alternative view could be that the VFQ-UI valuation process overestimates the impact of visual acuity on utility. This study is a nice demonstration of the fact that selecting health state utility values for a model-based economic evaluation is not straightforward. Attention needs to be given to the choice of measure (e.g. generic or condition-specific), but also to the way states are defined to allow for accurate utility values to be attached.

Do capability and functioning differ? A study of U.K. survey responses. Health Economics [PubMed] Published 24th September 2017

I like the capability approach in theory, but not in practice. I’ve written before about some of my concerns. One of them is that we don’t know whether capability measures (such as the ICECAP) offer anything beyond semantic nuance. This study sought to address that. A ‘functioning and capability’ instrument was devised, which reworded the ICECAP-A by changing phrases like “I am able to be” to phrases like “I am”, so that each question could have a ‘functioning’ version as well as a ‘capability’ version. Then, both the functioning and capability versions of the domains were presented in tandem. Questionnaires were sent to 1,627 individuals who had participated in another study about spillover effects in meningitis. Respondents (n=1,022) were family members of people experiencing after-effects of meningitis. The analysis focusses on the instances where capabilities and functionings diverge. Across the sample, 34% of respondents reported a difference between capability and functioning on at least one domain. For all domain-level responses, 12% were associated with higher capability than functioning, while 2% reported higher functioning. Some differences were observed between different groups of people. Older people tended to be less likely to report excess capabilities, while those with degree-level education reported greater capabilities. Informal care providers had lower functionings and capabilities but were more likely to report a difference between the two. Women were more likely to report excess capabilities in the ‘attachment’ domain. These differences lead the author to conclude that the wording of the ICECAP measure enables researchers to capture something beyond functioning, and that the choice of a capability measure could lead to different resource allocation decisions. I’m not convinced. The study makes an error that is common in this field; it presupposes that the changes in wording successfully distinguish between capabilities and functionings. This is implemented analytically by dropping from the primary analysis the cases where capabilities exceeded functionings, which are presumed to be illogical. If we don’t accept this presupposition (and we shouldn’t) then the meaning of the findings becomes questionable. The paper does outline most of the limitations of the study, but it doesn’t dedicate much space to alternative explanations. One is to do with the distinction between ‘can’ and ‘could’. If people answer ‘capability’ questions with reference to future possibilities, then the difference could simply be driven by optimism about future functionings. This future-reference problem is most obvious in the ‘achievement and progress’ domain, which incidentally, in this study, was the domain with the greatest probability of showing a discrepancy between capabilities and functionings. Another alternative explanation is that showing someone two slightly different questions coaxes them into making an artificial distinction that they wouldn’t otherwise make. In my previous writing on this, I suggested that two things needed to be identified. The first was to see whether people give different responses with the different wording. This study goes some way towards that, which is a good start. The second was to see whether people value states defined in these ways any differently. Until we have answers to both these questions I will remain sceptical about the implications of the ICECAP’s semantic nuance.

Estimating a constant WTP for a QALY—a mission impossible? The European Journal of Health Economics [PubMed] Published 21st September 2017

The idea of estimating willingness to pay (WTP) for a QALY has fallen out of fashion. It’s a nice idea in principle but, as the title of this paper suggests, it’s not easy to come up with a meaningful answer. One key problem has been that WTP for a QALY is not constant in the number of QALYs being gained – that is, people are willing to pay less (at the margin) for greater QALY gains. But maybe that’s OK. NICE and their counterparts tend not to use a fixed threshold but rather a range: £20,000-£30,000 per QALY, say. So maybe the variability in WTP for a QALY can be reflected in this range. This study explores some of the reasons – including uncertainty – for differences in elicited WTP values for a QALY. A contingent valuation exercise was conducted using a 2014 Internet panel survey of 1,400 Swedish citizens. The survey consisted 21 questions about respondents’ own health, sociodemographics, prioritisation attitudes, WTP for health improvements, and a societal decision-making task. Respondents were randomly assigned to one of five scenarios with different magnitudes and probabilities of health gain, with yes/no responses for five different WTP ‘bids’. The estimated WTP for a QALY – using the UK EQ-5D-3L tariff – was €17,000. But across the different scenarios, the WTP ranged from €10,600 to over a million. Wide confidence intervals abound. The authors’ findings only partially support an assumption of weak scope sensitivity – that more QALYs are worth paying more for – and do not at all support a strong assumption of scope sensitivity that WTP is proportional to QALY gain. This is what is known as scope bias, and this insensitivity to scope also applied to the variability in uncertainty. The authors also found that using different EQ-5D or VAS tariffs to estimate health state values resulted in variable differences in WTP estimates. Consistent relationships between individuals’ characteristics and their WTP were not found, though income and education seemed to be associated with higher willingness to pay across the sample. It isn’t clear what the implications of these findings are, except for the reinforcement of any scepticism you might have about the sociomathematical validity (yes, I’m sticking with that) of the QALY.

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