Rita Faria’s journal round-up for 18th June 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.

Objectives, budgets, thresholds, and opportunity costs—a health economics approach: an ISPOR Special Task Force report. Value in Health [PubMedPublished 21st February 2018

The economic evaluation world has been discussing cost-effectiveness thresholds for a while. This paper has been out for a few months, but it slipped under my radar. It explains the relationship between the cost-effectiveness threshold, the budget, opportunity costs and willingness to pay for health. My take-home messages are that we should use cost-effectiveness analysis to inform decisions both for publicly funded and privately funded health care systems. Each system has a budget and a way of raising funds for that budget. The cost-effectiveness threshold should be specific for each health care system, in order to reflect its specific opportunity cost. The budget can change for many reasons. The cost-effectiveness threshold should be adjusted to reflect these changes and hence reflect the opportunity cost. For example, taxpayers can increase their willingness to pay for health through increased taxes for the health care system. We are starting to see this in the UK with the calls to raise taxes to increase the NHS budget. It is worth noting that the NICE threshold may not warrant adjustment upwards since research suggests that it does not reflect the opportunity cost. This is a welcome paper on the topic and a must read, particularly if you’re arguing for the use of cost-effectiveness analysis in settings that traditionally were reluctant to embrace it, such as the US.

Basic versus supplementary health insurance: access to care and the role of cost effectiveness. Journal of Health Economics [RePEc] Published 31st May 2018

Using cost-effectiveness analysis to inform coverage decisions not only for the public but also for the privately funded health care is also a feature of this study by Jan Boone. I’ll admit that the equations are well beyond my level of microeconomics, but the text is good at explaining the insights and the intuition. Boone grapples with the question about how the public and private health care systems should choose which technologies to cover. Boone concludes that, when choosing which technologies to cover, the most cost-effective technologies should be prioritised for funding. That the theory matches the practice is reassuring to an economic evaluator like myself! One of the findings is that cost-effective technologies which are very cheap should not be covered. The rationale being that everyone can afford them. The issue for me is that people may decide not to purchase a highly cost-effective technology which is very cheap. As we know from behaviour economics, people are not rational all the time! Boone also concludes that the inclusion of technologies in the universal basic package should consider the prevalence of the conditions in those people at high risk and with low income. The way that I interpreted this is that it is more cost-effective to include technologies for high-risk low-income people in the universal basic package who would not be able to afford these technologies otherwise, than technologies for high-income people who can afford supplementary insurance. I can’t cover here all the findings and the nuances of the theoretical model. Suffice to say that it is an interesting read, even if you avoid the equations like myself.

Surveying the cost effectiveness of the 20 procedures with the largest public health services waiting lists in Ireland: implications for Ireland’s cost-effectiveness threshold. Value in Health Published 11th June 2018

As we are on the topic of cost-effectiveness thresholds, this is a study on the threshold in Ireland. This study sets out to find out if the current cost-effectiveness threshold is too high given the ICERs of the 20 procedures with the largest waiting lists. The idea is that, if the current cost-effectiveness threshold is correct, the procedures with large and long waiting lists would have an ICER of above the cost-effectiveness threshold. If the procedures have a low ICER, the cost-effectiveness threshold may be set too high. I thought that Figure 1 is excellent in conveying the discordance between ICERs and waiting lists. For example, the ICER for extracapsular extraction of crystalline lens is €10,139/QALY and the waiting list has 10,056 people; the ICER for surgical tooth removal is €195,155/QALY and the waiting list is smaller at 833. This study suggests that, similar to many other countries, there are inefficiencies in the way that the Irish health care system prioritises technologies for funding. The limitation of the study is in the ICERs. Ideally, the relevant ICER compares the procedure with the standard care in Ireland whilst on the waiting list (“no procedure” option). But it is nigh impossible to find ICERs that meet this condition for all procedures. The alternative is to assume that the difference in costs and QALYs is generalisable from the source study to Ireland. It was great to see another study on empirical cost-effectiveness thresholds. Looking forward to knowing what the cost-effectiveness threshold should be to accurately reflect opportunity costs.

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Meeting round-up: 7th Meeting of the International Academy of Health Preference Research

The 7th meeting of the International Academy of Health Preference Research (IAHPR) took place in Glasgow on Saturday 4th November 2017. The meeting was chaired by Karin Groothuis-Oudshoorn and Terry Flynn. It was preceded by a Friday afternoon symposium on the econometrics of heterogeneity, which I was unable to attend.

IAHPR is a relatively new organisation, describing itself as an ‘international network of multilingual, multidisciplinary researchers who contribute to the field of health preference research’. To minimise participants’ travel costs, IAHPR meetings are usually scheduled alongside major international conferences such as the meetings of iHEA, EuHEA and AHES (the Australian Health Economics Society). The November meeting took place just before the kick-off of the ISPOR European Congress (a behemoth by comparison). Most, but not all, of the attendees I spoke to, said that they would also be attending the ISPOR Congress.

The meeting was attended by 49 researchers from nine different countries. Nine were from the US, 16 from the UK, and 22 from elsewhere in the EU (sadly, I won’t be able to use the phrase ‘elsewhere in the EU’ for much longer). Understandably, the regional representation of the Glasgow meeting was quite different from that of the (July 2017) Boston meeting, where over 60% of the participants were based in the US.

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In total there were 12 podium presentations (half by student presenters) and about eight posters. Each podium presenter was allocated 12 minutes for their presentation and a further eight minutes for questions and group discussion. The poster authors were given the opportunity to briefly introduce themselves and their research to the group as part of an ‘elevator talks’ session.

Although all of the presentations focused on issues in stated preference research, the range of topics was quite broad, covering preferences between health outcomes, preferences between health services, conceptual and theoretical issues, experimental design approaches, and novel analytical techniques. Most of the studies presented applications of the DCE and best-worst scaling methods. Several presentations examined issues relating to preference heterogeneity and decision heuristics.

A personal highlight was Tabea Schmidt-Ott’s examination of the use of dominance tests to assess rational choice behaviour amongst survey respondents. She reported that such tests were included in a quarter of the health-related DCE studies published in 2015 (including many studies that had been led by IAHPR meeting attendees). Their inclusion had often been used to justify choices about which respondents to exclude from the final samples. Tabea concluded that dominance tests are a weak technique for assessing the rationality of people’s choice behaviour, as the observation of dominated choices can be explained by and accounted for in DCE models.

Overall, the IAHPR meeting was enjoyable and intellectually stimulating. The standard of the presentations and discussions was high, and it was a good forum for learning about the latest advances in stated preference research. It was quite DCE-dominated, so it would have been interesting to have had some representation from researchers who are sceptical about that methodology.

The next meeting will take place in Tasmania, to be chaired by Brendan Mulhern and Richard Norman.

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Chris Sampson’s journal round-up for 25th September 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.

Good practices for real‐world data studies of treatment and/or comparative effectiveness: recommendations from the Joint ISPOR‐ISPE Special Task Force on Real‐World Evidence in Health Care Decision Making. Value in Health Published 15th September 2017

I have an instinctive mistrust of buzzwords. They’re often used to avoid properly defining something, either because it’s too complicated or – worse – because it isn’t worth defining in the first place. For me, ‘real-world evidence’ falls foul. If your evidence isn’t from the real world, then it isn’t evidence at all. But I do like a good old ISPOR Task Force report, so let’s see where this takes us. Real-world evidence (RWE) and its sibling buzzword real-world data (RWD) relate to observational studies and other data not collected in an experimental setting. The purpose of this ISPOR task force (joint with the International Society for Pharmacoepidemiology) was to prepare some guidelines about the conduct of RWE/RWD studies, with a view to improving decision-makers’ confidence in them. Essentially, the hope is to try and create for RWE the kind of ecosystem that exists around RCTs, with procedures for study registration, protocols, and publication: a noble aim. The authors distinguish between 2 types of RWD: ‘Exploratory Treatment Effectiveness Studies’ and ‘Hypothesis Evaluating Treatment Effectiveness Studies’. The idea is that the latter test a priori hypotheses, and these are the focus of this report. Seven recommendations are presented: i) pre-specify the hypotheses, ii) publish a study protocol, iii) publish the study with reference to the protocol, iv) enable replication, v) test hypotheses on a separate dataset than the one used to generate the hypotheses, vi) publically address methodological criticisms, and vii) involve key stakeholders. Fair enough. But these are just good practices for research generally. It isn’t clear how they are in any way specific to RWE. Of course, that was always going to be the case. RWE-specific recommendations would be entirely contingent on whether or not one chose to define a study as using ‘real-world evidence’ (which you shouldn’t, because it’s meaningless). The authors are trying to fit a bag of square pegs into a hole of undefined shape. It isn’t clear to me why retrospective observational studies, prospective observational studies, registry studies, or analyses of routinely collected clinical data should all be treated the same, yet differently to randomised trials. Maybe someone can explain why I’m mistaken, but this report didn’t do it.

Are children rational decision makers when they are asked to value their own health? A contingent valuation study conducted with children and their parents. Health Economics [PubMed] [RePEc] Published 13th September 2017

Obtaining health state utility values for children presents all sorts of interesting practical and theoretical problems, especially if we want to use them in decisions about trade-offs with adults. For this study, the researchers conducted a contingent valuation exercise to elicit children’s (aged 7-19) preferences for reduced risk of asthma attacks in terms of willingness to pay. The study was informed by two preceding studies that sought to identify the best way in which to present health risk and financial information to children. The participating children (n=370) completed questionnaires at school, which asked about socio-demographics, experience of asthma, risk behaviours and altruism. They were reminded (in child-friendly language) about the idea of opportunity cost, and to consider their own budget constraint. Baseline asthma attack risk and 3 risk-reduction scenarios were presented graphically. Two weeks later, the parents completed similar questionnaires. Only 9% of children were unwilling to pay for risk reduction, and most of those said that it was the mayor’s problem! In some senses, the children did a better job than their parents. The authors conducted 3 tests for ‘incorrect’ responses – 14% of adults failed at least one, while only 4% of children did so. Older children demonstrated better scope sensitivity. Of course, children’s willingness to pay was much lower in absolute terms than their parents’, because children have a much smaller budget. As a percentage of the budget, parents were – on average – willing to pay more than children. That seems reassuringly predictable. Boys and fathers were willing to pay more than girls and mothers. Having experience of frequent asthma attacks increased willingness to pay. Interestingly, teenagers were willing to pay less (as a proportion of their budget) than younger children… and so were the teenagers’ parents! Children’s willingness to pay was correlated with that of their own parent’s at the higher risk reductions but not the lowest. This study reports lots of interesting findings and opens up plenty of avenues for future research. But the take-home message is obvious. Kids are smart. We should spend more time asking them what they think.

Journal of Patient-Reported Outcomes: aims and scope. Journal of Patient-Reported Outcomes Published 12th September 2017

Here we have a new journal that warrants a mention. The journal is sponsored by the International Society for Quality of Life Research (ISOQOL), making it a sister journal of Quality of Life Research. One of its Co-Editors-in-Chief is the venerable David Feeny, of HUI fame. They’ll be looking to publish research using PRO(M) data from trials or routine settings, studies of the determinants of PROs, qualitative studies in the development of PROs; anything PRO-related, really. This could be a good journal for more thorough reporting of PRO data that can get squeezed out of a study’s primary outcome paper. Also, “JPRO” is fun to say. The editors don’t mention that the journal is open access, but the website states that it is, so APCs at the ready. ISOQOL members get a discount.

Research and development spending to bring a single cancer drug to market and revenues after approval. JAMA Internal Medicine [PubMed] Published 11th September 2017

We often hear that new drugs are expensive because they’re really expensive to develop. Then we hear about how much money pharmaceutical companies spend on marketing, and we baulk. The problem is, pharmaceutical companies aren’t forthcoming with their accounts, so researchers have to come up with more creative ways to estimate R&D spending. Previous studies have reported divergent estimates. Whether R&D costs ‘justify’ high prices remains an open question. For this study, the authors looked at public data from the US for 10 companies that had only one cancer drug approved by the FDA between 2007 and 2016. Not very representative, perhaps, but useful because it allows for the isolation of the development costs associated with a single drug reaching the market. The median time for drug development was 7.3 years. The most generous estimate of the mean cost of development came in at under a billion dollars; substantially less than some previous estimates. This looks like a bargain; the mean revenue for the 10 companies up to December 2016 was over $6.5 billion. This study may seem a bit back-of-the-envelope in nature. But that doesn’t mean it isn’t accurate. If anything, it begs more confidence than some previous studies because the methods are entirely transparent.

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