Chris Sampson’s journal round-up for 5th February 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.

Cost-effectiveness analysis of germ-line BRCA testing in women with breast cancer and cascade testing in family members of mutation carriers. Genetics in Medicine [PubMed] Published 4th January 2018

The idea of testing women for BRCA mutations – faulty genes that can increase the probability and severity of breast and ovarian cancers – periodically makes it into the headlines. That’s not just because of Angelina Jolie. It’s also because it’s a challenging and active area of research with many uncertainties. This new cost-effectiveness analysis evaluates a programme that incorporates cascade testing; testing relatives of mutation carriers. The idea is that this could increase the effectiveness of the programme with a reduced cost-per-identification, as relatives of mutation carriers are more likely to also carry a mutation. The researchers use a cohort-based Markov-style decision analytic model. A programme with three test cohorts – i) women with unilateral breast cancer and a risk prediction score >10%, ii) first-degree relatives, and iii) second-degree relatives – was compared against no testing. A positive result in the original high-risk individual leads to testing in the first- and second-degree relatives, with the number of subsequent tests occurring in the model determined by assumptions about family size. Women who test positive can receive risk-reducing mastectomy and/or bilateral salpingo-oophorectomy (removal of the ovaries). The results are favourable to the BRCA testing programme, at $19,000 (Australian) per QALY for testing affected women only and $15,000 when the cascade testing of family members was included, with high probabilities of cost-effectiveness at $50,000 per QALY. I’m a little confused by the model. The model includes the states ‘BRCA positive’ and ‘Breast cancer’, which clearly are not mutually exclusive. And It isn’t clear how women entering the model with breast cancer go on to enjoy QALY benefits compared to the no-test group. I’m definitely not comfortable with the assumption that there is no disutility associated with risk-reducing surgery. I also can’t see where the cost of identifying the high-risk women in the first place was accounted for. But this is a model, after all. The findings appear to be robust to a variety of sensitivity analyses. Part of the value of testing lies in the information it provides about people beyond the individual patient. Clearly, if we want to evaluate the true value of testing then this needs to be taken into account.

Economic evaluation of direct-acting antivirals for hepatitis C in Norway. PharmacoEconomics Published 2nd February 2018

Direct-acting antivirals (DAAs) are those new drugs that gave NICE a headache a few years back because they were – despite being very effective and high-value – unaffordable. DAAs are essentially curative, which means that they can reduce resource use over a long time horizon. This makes cost-effectiveness analysis in this context challenging. In this new study, the authors conduct an economic evaluation of DAAs compared with the previous class of treatment, in the Norwegian context. Importantly, the researchers sought to take into account the rebates that have been agreed in Norway, which mean that the prices are effectively reduced by up to 50%. There are now lots of different DAAs available. Furthermore, hepatitis C infection corresponds to several different genotypes. This means that there is a need to identify which treatments are most (cost-)effective for which groups of patients; this isn’t simply a matter of A vs B. The authors use a previously developed model that incorporates projections of the disease up to 2030, though the authors extrapolate to a 100-year time horizon. The paper presents cost-effectiveness acceptability frontiers for each of genotypes 1, 2, and 3, clearly demonstrating which medicines are the most likely to be cost-effective at given willingness-to-pay thresholds. For all three genotypes, at least one of the DAA options is most likely to be cost-effective above a threshold of €70,000 per QALY (which is apparently recommended in Norway). The model predicts that if everyone received the most cost-effective strategy then Norway would expect to see around 180 hepatitis C patients in 2030 instead of the 300-400 seen in the last six years. The study also presents the price rebates that would be necessary to make currently sub-optimal medicines cost-effective. The model isn’t that generalisable. It’s very much Norway-specific as it reflects the country’s treatment guidelines. It also only looks at people who inject drugs – a sub-population whose importance can vary a lot from one country to the next. I expect this will be a valuable piece of work for Norway, but it strikes me as odd that “affordability” or “budget impact” aren’t even mentioned in the paper.

Cost-effectiveness of prostate cancer screening: a systematic review of decision-analytical models. BMC Cancer [PubMed] Published 18th January 2018

You may have seen prostate cancer in the headlines last week. Despite the number of people in the UK dying each year from prostate cancer now being greater than the number of people dying from breast cancer, prostate cancer screening remains controversial. This is because over-detection and over-treatment are common and harmful. Plenty of cost-effectiveness studies have been conducted in the context of detecting and treating prostate cancer. But there are various ways of modelling the problem and various specifications of screening programme that can be evaluated. So here we have a systematic review of cost-effectiveness models evaluating prostate-specific antigen (PSA) blood tests as a basis for screening. From a haul of 1010 studies, 10 made it into the review. The studies modelled lots of different scenarios, with alternative screening strategies, PSA thresholds, and treatment pathways. The results are not consistent. Many of the scenarios evaluated in the studies were more costly and less effective than current practice (which tended to be the lack of any formal screening programme). None of the UK-based cost-per-QALY estimates favoured screening. The authors summarise the methodological choices made in each study and consider the extent to which this relates to the pathways being modelled. They also specify the health state utility values used in the models. This will be a very useful reference point for anyone trying their hand at a prostate cancer screening model. Of the ten studies included in the review, four of them found at least one screening programme to be potentially cost-effective. ‘Adaptive screening’ – whereby individuals’ recall to screening was based on their risk – was considered in two studies using patient-level simulations. The authors suggest that cohort-level modelling could be sufficient where screening is not determined by individual risk level. There are also warnings against inappropriate definition of the comparator, which is likely to be opportunistic screening rather than a complete absence of screening. Generally speaking, a lack of good data seems to be part of the explanation for the inconsistency in the findings. It could be some time before we have a clearer understanding of how to implement a cost-effective screening programme for prostate cancer.

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Alastair Canaway’s journal round-up for 29th 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.

Is “end of life” a special case? Connecting Q with survey methods to measure societal support for views on the value of life-extending treatments. Health Economics [PubMed] Published 19th January 2018

Should end-of-life care be treated differently? A question often asked and previously discussed on this blog: findings to date are equivocal. This question is important given NICE’s End-of-Life Guidance for increased QALY thresholds for life-extending interventions, and additionally the Cancer Drugs Fund (CDF). This week’s round-up sees Helen Mason and colleagues attempt to inform the debate around societal support for views of end-of-life care, by trying to determine the degree of support for different views on the value of life-extending treatment. It’s always a treat to see papers grounded in qualitative research in the big health economics journals and this month saw the use of a particularly novel mixed methods approach adding a quantitative element to their previous qualitative findings. They combined the novel (but increasingly recognisable thanks to the Glasgow team) Q methodology with survey techniques to examine the relative strength of views on end-of-life care that they had formulated in a previous Q methodology study. Their previous research had found that there are three prevalent viewpoints on the value of life-extending treatment: 1. ‘a population perspective: value for money, no special cases’, 2. ‘life is precious: valuing life-extension and patient choice’, 3. ‘valuing wider benefits and opportunity cost: the quality of life and death’. This paper used a large Q-based survey design (n=4902) to identify societal support for the three different viewpoints. Viewpoints 1 and 2 were found to be dominant, whilst there was little support for viewpoint 3. The two supported viewpoints are not complimentary: they represent the ethical divide between the utilitarian with a fixed budget (view 1), and the perspective based on entitlement to healthcare (view 2: which implies an expanding healthcare budget in practice). I suspect most health economists will fall into camp number one. In terms of informing decision making, this is very helpful, yet unhelpful: there is no clear answer. It is, however, useful for decision makers in providing evidence to balance the oft-repeated ‘end of life is special’ argument based solely on conjecture, and not evidence (disclosure: I have almost certainly made this argument before). Neither of the dominant viewpoints supports NICE’s End of Life Guidance nor the CDF. Viewpoint 1 suggests end of life interventions should be treated the same as others, whilst viewpoint 2 suggests that treatments should be provided if the patient chooses them; it does not make end of life a special case as this viewpoint believes all treatments should be available if people wish to have them (and we should expand budgets accordingly). Should end of life care be treated differently? Well, it depends on who you ask.

A systematic review and meta-analysis of childhood health utilities. Medical Decision Making [PubMed] Published 7th October 2017

If you’re working on an economic evaluation of an intervention targeting children then you are going to be thankful for this paper. The purpose of the paper was to create a compendium of utility values for childhood conditions. A systematic review was conducted which identified a whopping 26,634 papers after deduplication – sincere sympathy to those who had to do the abstract screening. Following abstract screening, data were extracted for the remaining 272 papers. In total, 3,414 utility values were included when all subgroups were considered – this covered all ICD-10 chapters relevant to child health. When considering only the ‘main study’ samples, 1,191 utility values were recorded and these are helpfully separated by health condition, and methodological characteristics. In short, the authors have successfully built a vast catalogue of child utility values (and distributions) for use in future economic evaluations. They didn’t, however, stop there, they then built on the systematic review results by conducting a meta-analysis to i) estimate health utility decrements for each condition category compared to general population health, and ii) to examine how methodological factors impact child utility values. Interestingly for those conducting research in children, they found that parental proxy values were associated with an overestimation of values. There is a lot to unpack in this paper and a lot of appendices and supplementary materials are included (including the excel database for all 3,414 subsamples of health utilities). I’m sure this will be a valuable resource in future for health economic researchers working in the childhood context. As far as MSc dissertation projects go, this is a very impressive contribution.

Estimating a cost-effectiveness threshold for the Spanish NHS. Health Economics [PubMed] [RePEc] Published 28th December 2017

In the UK, the cost-per-QALY threshold is long-established, although whether it is the ‘correct’ value is fiercely debated. Likewise in Spain, there is a commonly cited threshold value of €30,000 per QALY with a dearth of empirical justification. This paper sought to identify a cost-per-QALY threshold for the Spanish National Health Service (SNHS) by estimating the marginal cost per QALY at which the SNHS currently operates on average. This was achieved by exploiting data on 17 regional health services between the years 2008-2012 when the health budget experienced considerable cuts due to the global economic crisis. This paper uses econometric models based on the provoking work by Claxton et al in the UK (see the full paper if you’re interested in the model specification) to achieve this. Variations between Spanish regions over time allowed the authors to estimate the impact of health spending on outcomes (measured as quality-adjusted life expectancy); this was then translated into a cost-per-QALY value for the SNHS. The headline figures derived from the analysis give a threshold between €22,000 and €25,000 per QALY. This is substantially below the commonly cited threshold of €30,000 per QALY. There are, however (as to be expected) various limitations acknowledged by the authors, which means we should not take this threshold as set in stone. However, unlike the status quo, there is empirical evidence backing this threshold and it should stimulate further research and discussion about whether such a change should be implemented.

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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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