David Mott’s journal round-up for 16th September 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.

Opening the ‘black box’: an overview of methods to investigate the decision‑making process in choice‑based surveys. The Patient [PubMed] Published 5th September 2019

Choice-based surveys using methods such as discrete choice experiments (DCEs) and best-worst scaling (BWS) exercises are increasingly being used in health to understand people’s preferences. A lot of time and energy is spent on analysing the data that come out from these surveys but increasingly there is an interest in better understanding respondents’ decision-making processes. Whilst many will be aware of ‘think aloud’ interviews (often used for piloting), other methods may be less familiar as they’re not applied frequently in health. That’s where this fascinating paper by Dan Rigby and colleagues comes in. It provides an overview of five different methods of what they call ‘pre-choice process analysis’ of decision-making, describing the application, state of knowledge, and future research opportunities.

Eye-tracking has been used in health recently. It’s intuitive and provides an insight into where the participants’ focus is (or isn’t). The authors explained that one of the ways it has been used is to explore attribute non-attendance (ANA), which essentially occurs when people are ignoring attributes either because they’re irrelevant to them, or simply because it makes the task easier. However, surprisingly, it has been suggested that ‘visual ANA’ (not looking at the attribute) doesn’t always align with ‘stated ANA’ (participants stating that they ignored the attribute) – which raises some interesting questions!

However, the real highlight for me was the overview of the use of brain imaging techniques to explore choices being made in DCEs. One study highlighted by the authors – which was a DCE about eggs and is now at least #2 on my list of the bizarre preference study topics after this oddly specific one on Iberian ham – predicted choices from an initial ‘passive viewing’ using functional magnetic resonance imaging (fMRI). They found that incorporating changes in blood flow (prompted by changes in attribute levels during ‘passive viewing’) into a random utility model accounted for a lot of the variation in willingness to pay for eggs – pretty amazing stuff.

Whilst I’ve highlighted the more unusual methods here, after reading this overview I have to admit that I’m an even bigger advocate for the ‘think aloud’ technique now. Although it may have some limitations, the amount of insight offered combined with its practicality is hard to beat. Though maybe I’m biased because I know that I won’t get my hands on any eye-tracking or brain imaging devices any time soon. In any case, I highly recommend that any researchers conducting preference studies give this paper a read as it’s really well written and will surely be of interest.

Disentangling public preferences for health gains at end-of-life: further evidence of no support of an end-of-life premium. Social Science & Medicine [PubMed] Published 21st June 2019

The end of life (EOL) policy introduced by NICE in 2009 [PDF] has proven controversial. The policy allows treatments that are not cost-effective within the usual range to be considered for approval, provided that certain criteria are met. Specifically, that the treatment targets patients with a short life expectancy (≤24 months), offers a life extension (of ≥3 months) and is for a ‘small patient population’. One of the biggest issues with this policy is that it is unclear whether the general population actually supports the idea of valuing health gains (specifically life extension) at EOL more than other health gains.

Numerous academic studies, usually involving some form of stated preference exercise, have been conducted to test whether the public might support this EOL premium. A recent review by Koonal Shah and colleagues summarised the existing published studies (up to October 2017), highlighting that evidence is extremely mixed. This recently published Danish study, by Lise Desireé Hansen and Trine Kjær, adds to this literature. The authors conducted an incredibly thorough stated preference exercise to test whether quality of life (QOL) gains and life extension (LE) at EOL are valued differently from other similarly sized health gains. Not only that, but the study also explored the effect of perspective on results (social vs individual), the effect of age (18-35 vs. 65+), and impact of initial severity (25% vs. 40% initial QOL) on results.

Overall, they did not find evidence of support for an EOL premium for QOL gains or for LEs (regardless of perspective) but their results do suggest that QOL gains are preferred over LE. In some scenarios, there was slightly more support for EOL in the social perspective variant, relative to the individual perspective – which seems quite intuitive. Both age and initial severity had an impact on results, with respondents preferring to treat the young and those with worse QOL at baseline. One of the most interesting results for me was within their subgroup analyses, which suggested that women and those with a relation to a terminally ill patient had a significantly positive preference for EOL – but only in the social perspective scenarios.

This is a really well-designed study, which covers a lot of different concepts. This probably doesn’t end the debate on NICE’s use of the EOL criteria – not least because the study wasn’t conducted in England and Wales – but it contributes a lot. I’d consider it a must-read for anyone interested in this area.

How should we capture health state utility in dementia? Comparisons of DEMQOL-Proxy-U and of self- and proxy-completed EQ-5D-5L. Value in Health Published 26th August 2019

Capturing quality of life (QOL) in dementia and obtaining health state utilities is incredibly challenging; which is something that I’ve started to really appreciate recently upon getting involved in a EuroQol-funded ‘bolt-ons’ project. The EQ-5D is not always able to detect meaningful changes in cognitive function and condition-specific preference-based measures (PBMs), such as the DEMQOL, may be preferred as a result. However, this isn’t the only challenge because in many cases patients are not in a position to complete the surveys themselves. This means that proxy-reporting is often required, which could be done by either a professional (formal) carer, or a friend or family member (informal carer). Researchers that want to use a PBM in this population therefore have a lot to consider.

This paper compares the performance of the EQ-5D-5L and the DEMQOL-Proxy when completed by care home residents (EQ-5D-5L only), formal carers and informal carers. The impressive dataset that the authors use contains 1,004 care home residents, across up to three waves, and includes a battery of different cognitive and QOL measures. The overall objective was to compare the performance of the EQ-5D-5L and DEMQOL-Proxy, across the three respondent groups, based on 1) construct validity, 2) criterion validity, and 3) responsiveness.

The authors found that self-reported EQ-5D-5L scores were larger and less responsive to changes in the cognitive measures, but better at capturing residents’ self-reported QOL (based on a non-PBM) relative to proxy-reported scores. It is unclear whether this is a case of adaptation as seen in many other patient groups, or if the residents’ cognitive impairments prevent them from reliably assessing their current status. The proxy-reported EQ-5D-5L scores were generally more responsive to changes in the cognitive measures relative to the DEMQOL-Proxy (irrespective of which type of proxy), which the authors note is probably due to the fact that the DEMQOL-Proxy focuses more on the emotional impact of dementia rather than functional impairment.

Overall, this is a really interesting paper, which highlights the challenges well and illustrates that there is value in collecting these data from both patients and proxies. In terms of the PBM comparison, whilst the authors do not explicitly state it, it does seem that the EQ-5D-5L may have a slight upper hand due to its responsiveness, as well as for pragmatic reasons (the DEMQOL-Proxy has >30 questions). Perhaps a cognition ‘bolt-on’ to the EQ-5D-5L might help to improve the situation in future?

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Rita Faria’s journal round-up for 26th August 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.

Vaccine hesitancy and (fake) news: quasi‐experimental evidence from Italy. Health Economics [PubMed] [RePEc] Published 20th August 2019

Has fake news led to fewer children being vaccinated? At least in Italy, the answer seems to be yes.

It’s shocking to read that the WHO has included the reluctance or refusal to vaccinate as one of the 10 threats to global health today. And many of us are asking: why has this happened and what can we do to address it? Vincenzo Carrieri, Leonardo Madio and Francesco Principe help answer this first question. They looked at how fake news affects the take-up of vaccines, assuming that exposure to fake news is proxied by access to broadband and within a difference-in-differences framework. They found that a 10% increase in broadband coverage is associated with a 1.2-1.6% reduction in vaccination rates.

The differences-in-differences method hinges on a court ruling in 2012 that accepted that the MMR vaccine causes autism. Following the ruling, fake news about vaccines spread across the internet. In parallel, broadband coverage increased over time due to a government programme, but it varied by region, depending on the existing infrastructure and geographical conditions. Broadband coverage, by itself, cannot lead to lower vaccination rates. So it makes sense to assume that broadband coverage leads to greater exposure to fake news about vaccines, which in turn leads to lower vaccination rates.

On the other hand, it may be that greater broadband coverage and lower vaccination rates are both caused by something else. The authors wrote a good introduction to justify the model assumptions and show a few robustness checks. Had they had more space, I would have like to read a bit more about the uncertainties around the model assumptions. This is a fantastic paper and good food for thought on the consequences of fake news. Great read!

The cost-effectiveness of one-time birth cohort screening for hepatitis C as part of the National Health Service Health Check programme in England. Value in Health Published 19th August 2019

Jack Williams and colleagues looked at the cost-effectiveness of one-time birth cohort screening for hepatitis C. As hepatitis C is usually asymptomatic before reaching its more advanced stages, people may not be aware that they are infected. Therefore, they may not get tested and treated, even though treatment is effective and cost-effective.

At the level of the individual eligible for testing, the ICERs were between £8k-£31k/QALY, with lower ICERs for younger birth cohorts. The ICERs also depended on the transition probabilities for the progression of the disease, with lower ICERs if progression is faster. Extensive sensitivity and value of information analyses indicate that the key cost-effectiveness drivers are the transition probabilities, probabilities of referral and of treatment post-referral, and the quality of life benefits of being cured.

This is a great example of a good quality applied cost-effectiveness analysis. The model is well justified, the results are thoroughly tested, and the discussion is meticulous. Well done!

NICE, in confidence: an assessment of redaction to obscure confidential information in Single Technology Appraisals by the National Institute for Health and Care Excellence. PharmacoEconomics [PubMed] Published 27th June 2019

NICE walks a fine line between making decisions transparent and protecting confidential information. Confidential information includes commercially sensitive information (e.g. discounts to the price paid by the NHS) and academic-in-confidence information, such as unpublished results of clinical trials. The problem is that the redacted information may preclude readers from understanding NICE decisions.

Ash Bullement and colleagues reviewed NICE appraisals of technologies with an approved price discount. Their goal was to understand the extent of redactions and their consequences on the transparency of NICE decisions. Of the 171 NICE appraisals, 118 had an approved commercial arrangement and 110 had a simple price discount. The type of redacted information varied. Some did not present the ICER, others presented ICERs but not the components of the ICERs, and others did not even present the estimates of life expectancy from the model. Remarkably, the confidential discount could be back-calculated in seven NICE appraisals! The authors also looked at the academic-in-confidence redactions. They found that 68 out of 86 appraisals published before 2018 still had academic-in-confidence information redacted. This made me wonder if NICE has a process to review these redactions and disclose them once the information is in the public domain.

As Ash and colleagues rightly conclude, this review shows that there does not seem to be a consistent process for redaction and disclosure. This is a compelling paper on the practicalities of the NICE process, and with useful reflections for HTA agencies around the world. The message for NICE is that it may be time to review the process to handle sensitive information.

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Alastair Canaway’s journal round-up for 10th June 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.

Analytic considerations in applying a general economic evaluation reference case to gene therapy. Value in Health Published 17th May 2019

For fledgling health economists starting in the world of economic evaluation, the NICE reference case is somewhat of a holy text. If in doubt, check the reference case. The concept of a reference case for economic evaluation has been around since the first US Panel on Cost-Effectiveness in Health and Medicine in 1996 and NICE has routinely used its own reference case for well over a decade. The primary purpose of the reference case is to improve the quality and comparability of economic evaluations by standardising methodological practices. There have been arguments made that the same methods are not appropriate for all medical technologies, particularly those in rare diseases or where no treatment currently exists. The focus of this paper is on gene therapy: a novel method that inserts genetic material into cells (as opposed to a drug/surgery) to treat or prevent disease. In this area there has been significant debate as to the appropriateness of the reference case and whether a new reference case is required in this transformative but expensive area. The purpose of the article was to examine the characteristics of gene therapy and make recommendations on changes to the reference case accordingly.

The paper does an excellent job of unpicking the key components of economic evaluation in relation to gene therapy to examine where weaknesses in current reference cases may lie. Rather than recommend that a new reference case be created, they identify specific areas that should be paid special attention when evaluating gene therapy. Additionally, they produce a three part checklist to help analysts to consider what aspects of their economic evaluation they should consider further. For those about to embark on an economic evaluation of a gene therapy intervention, this paper represents an excellent starting point to guide your methodological choices.

Heterogeneous effects of obesity on mental health: evidence from Mexico. Health Economics [PubMed] [RePEc] Published April 2019

The first line of the ‘summary’ section of this paper caught my eye: “Obesity can spread more easily if it is not perceived negatively”. This stirred up contradictory thoughts. From a public health standpoint we should be doing our utmost to prevent increasing levels of obesity and their related co-morbidities, whilst simultaneously we should be promoting body positivity and well-being for mental health. Is there a tension here? Might promoting body positivity and well-being enable the spread of obesity? This paper doesn’t really answer that question, instead it sought to investigate whether overweight and obesity had differing effects on mental health within different populations groups.

The study is set in Mexico which has the highest rate of obesity in the world with 70% of the population being overweight or obese. Previous research suggests that obesity spreads more easily if not perceived negatively. This paper hypothesises that this effect will be more acute among the poor and middle classes where obesity is more prevalent. The study aimed to reveal the extent of the impact of obesity on well-being whilst controlling for common determinants of well-being by examining the impact of measures of fatness on subjective well-being, allowing for heterogeneous effects across differing groups. The paper focused only on women, who tend to be more affected by excess weight than men (in Mexico at least).

To assess subjective well-being (SWB) the General Health Questionnaire (GHQ) was used whilst weight status was measured using waist to height ratio and additionally an obesity dummy. Data was sourced from the Mexican Family and Life Survey and the baseline sample included over 13,000 women. Various econometric models were employed ranging from OLS to instrumental variable estimations, details of which can be found within the paper.

The results supported the hypothesis. They found that there was a negative effect of fatness on well-being for the rich, whilst there was a positive effect for the poor. This has interesting policy implications: policy attempt to reduce obesity may not work if excess weight is not perceived to be an issue. The findings in this study imply that different policy measures are likely necessary for intervening in the wealthy and the poor in Mexico. The paper offers several explanations as to why this relationship may exist, ranging from the poor having lower returns from healthy time (nod to the Grossman model), to differing labour market penalties from fatness due to different job types for the rich and the poor.

Obviously there are limits to the generalisability of these findings, however it does raise interesting questions about how we should seek to prevent obesity within different elements of society, and the unintended consequences that shifts in attitudes may have.

ICECAP-O, the current state of play: a systematic review of studies reporting the psychometric properties and use of the instrument over the decade since its publication. Quality of Life Research [PubMed] Published June 2019

Those who follow the methodological side of outcome measurement will be familiar with the capability approach, operationalised by the ICECAP suite of measures amongst others. These measures focus on what people are able to do, rather than what they do. It is now 12-13 years since the first ICECAP measure was developed: the ICECAP-O designed for use in older adults. Given the ICECAP measures are now included within the NICE reference case for the economic evaluation of social care, it is a pertinent time to look back over the past decade to assess whether the ICECAP measures are being used and, if so, to what degree and how. This systematic review focusses on the oldest of the ICECAP measures, the ICECAP-O, and examines whether it has been used, and for what purpose as well as summarising the results from psychometric papers.

An appropriate search strategy was deployed within the usual health economic databases, and the PRISMA checklist was used to guide the review. In total 663 papers were identified, of which 51 papers made it through the screening process.

The first 8 years of the ICECAP-O’s life is characterised by an increasing amount of psychometric studies, however in 2014 a reversal occurred. Simultaneously, the number of studies using the ICECAP-O within economic evaluations has slowly increased, surmounting the number examining the psychometric properties, and has increased year-on-year in the three years up to 2018. Overall, the psychometric literature found the ICECAP-O to have good construct validity and generally good content validity with the occasional exception in groups of people with specific medical needs. Although the capability approach has gained prominence, the studies within the review suggest it is still very much seen as a secondary instrument to the EQ-5D and QALY framework, with results typically being brief with little to no discussion or interpretation of the ICECAP-O results.

One of the key limitations to the ICECAP framework to date relates to how economists and decision makers should use the results from the ICECAP instruments. Should capabilities be combined with time (e.g. years in full capability), or should some minimum (sufficient) capability threshold be used? The paper concludes that in the short term, presenting results in terms of ‘years of full capability’ is the best bet, however future research should focus on identifying sufficient capability and establishing monetary thresholds for a year with sufficient capability. Given this, whilst the ICECAP-O has seen increased use over the years, there is still significant work to be done to facilitate decision making and for it to routinely be used as a primary outcome for economic evaluation.

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