Chris Sampson’s journal round-up for 19th 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.

Paying for kidneys? A randomized survey and choice experiment. American Economic Review [RePEc] Published August 2019

This paper starts with a quote from Alvin Roth about ‘repugnant transactions’, of which markets for organs provide a prime example. This idea of ‘repugnant transactions’ has been hijacked by some pop economists to represent the stupid opinions of non-economists. If you ask me, markets for organs aren’t repugnant, they just seem like a very bad idea in terms of both efficiency and equity. But it doesn’t matter what I think; it matters what the people of the United States think.

The authors of this study conducted an online survey with a representative sample of 2,666 Americans. Each respondent was randomised to evaluate one of eight systems compared with the current system. The eight systems differed with respect to i) cash or non-cash compensation of ii) different sizes ($30,000 or $100,000), iii) paid by either a public agency or the organ recipient. Participants made five binary choices that differed according to the gain – in transplants generated – associated with the new system. Half of the participants were also asked to express moral judgements.

Both the system features (e.g. who pays) and the outcomes of the new system influenced people’s choices. Broadly speaking, the results suggest that people aren’t opposed to donors being paid, but are opposed to patients paying. (Remember, we’re talking about the US here!). Around 21% of respondents opposed payment no matter what, 46% were in favour no matter what, and 18% were sensitive to the gain in the number of transplants. A 10% point increase in transplants resulted in a 2.6% point increase in support. Unsurprisingly, individuals’ moral judgements were predictive of the attitudes they expressed, particularly with respect to fairness. The authors describe their results as exhibiting ‘strong polarisation’, which is surely inevitable for questions that involve moral judgement.

Being in AER, this is a long meandering paper with extensive analyses and thoroughly reported results. There’s lots of information and findings that I can’t share here. It’s a valuable study with plenty of food for thought, but I can’t help but think that it is, methodologically, a bit weak. If we want to understand the different views in society, surely some Q methodology would be more useful than a basic online survey. And if we want to elicit stated preferences, surely a discrete choice experiment with a well-thought-out efficient design would give us more meaningful results.

Estimating local need for mental healthcare to inform fair resource allocation in the NHS in England: cross-sectional analysis of national administrative data linked at person level. The British Journal of Psychiatry [PubMed] Published 8th August 2019

The need to fairly (and efficiently) allocate NHS resources across the country played an important part in the birth of health economics in the UK, and resulted in resource allocation formulas. Since 1996 there has been a separate formula for mental health services, which is periodically updated. This study describes the work undertaken for the latest update.

The model is based on predicting service use and total mental health care costs observed in 2015 from predictors in the years 2013-2014, to inform allocations in 2019-2024. Various individual-level data sources available to the NHS were used for 43.7 million people registered with a GP practice and over the age of 20. The cost per patient who used mental health services ranged from £94 to over one million, averaging around £2,000. The predictor variables included individual indicators such as age, sex, ethnicity, physical diagnoses, and household type (e.g. number of adults and kids). The model also used variables observed at the local or GP practice level, such as the proportion of people receiving out-of-work benefits and the distance from the mental health trust. All of this got plugged into a good old OLS regression. From individual-level predictions, the researchers created aggregated indices of need for each clinical commission group (CCG).

A lot went into the model, which explained 99% of the variation in costs between CCGs. A key way in which this model differs from previous versions is that it relies on individual-level indicators rather than those observed at the level of GP practice or CCG. There was a lot of variation in the CCG need indices, ranging from 0.65 for Surrey Heath to 1.62 for Southwark, where 1.00 is the average. You’ll need to check the online appendices for your own CCG’s level of need (Lewisham: 1.52). As one might expect, the researchers observed a strong correlation between a CCG’s need index and the CCG’s area’s level of deprivation. Compared with previous models, this new model indicates a greater allocation of resources to more deprived and older populations.

Measuring, valuing and including forgone childhood education and leisure time costs in economic evaluation: methods, challenges and the way forward. Social Science & Medicine [PubMed] Published 7th August 2019

I’m a ‘societal perspective’ sceptic, not because I don’t care about non-health outcomes (though I do care less) but because I think it’s impossible to capture everything that is of value to society, and that capturing just a few things will introduce a lot of bias and noise. I would also deny that time has any intrinsic value. But I do think we need to do a better job of evaluating interventions for children. So I expected this paper to provide me with a good mix of satisfaction and exasperation.

Health care often involves a loss of leisure or work time, which can constitute an opportunity cost and is regularly included in economic evaluations – usually proxied by wages – for adults. The authors outline the rationale for considering ‘time-related’ opportunity costs in economic evaluations and describe the nature of lost time for children. For adults, the distinction is generally between paid or unpaid work and leisure time. Arguably, this distinction is not applicable to children. Two literature reviews are described. One looked at economic evaluations in the context of children’s health, to see how researchers have valued lost time. The other sought to identify ideas about the value of lost time for children from a broader literature.

The authors do a nice job of outlining how difficult it is to capture non-health-related costs and outcomes in the context of childhood. There is a handful of economic evaluations that have tried to measure and value children’s foregone time. The valuations generally focussed on the costs of childcare rather than the costs to the child, though one looked at the rate of return to education. There wasn’t a lot to go off in the non-health literature, which mostly relates to adults. From what there is, the recommendation is to capture absence from formal education and foregone leisure time. Of course, consideration needs to be given to the importance of lost time and thus the value of capturing it in research. We also need to think about the risk of double counting. When it comes to measurement, we can probably use similar methods as we would for adults, such as diaries. But we need very different approaches to valuation. On this, the authors found very little in the way of good examples to follow. More research needed.

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Chris Sampson’s journal round-up for 12th 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.

Developing open-source models for the US health system: practical experiences and challenges to date with the Open-Source Value Project. PharmacoEconomics [PubMed] Published 7th August 2019

PharmacoEconomics will soon publish a themed issue on transparency in decision modelling (to which I’ve contributed), and this paper – I assume – is one that will feature. At least one output from the Open-Source Value Project has featured in these round-ups before. The purpose of this paper is to describe the experiences of the initiative in developing and releasing two open-source models, one in rheumatoid arthritis and one in lung cancer.

The authors outline the background to the project and its goal to develop credible models that are more tuned-in to stakeholders’ needs. By sharing the R and C++ source code, developing interactive web applications, and providing extensive documentation, the models are intended to be wholly transparent and flexible. The model development process also involves feedback from experts and the public, followed by revision and re-release. It’s a huge undertaking. The paper sets out the key challenges associated with this process, such as enabling stakeholders with different backgrounds to understand technical models and each other. The authors explain how they have addressed such difficulties along the way. The resource implications of this process are also challenging, because the time and expertise required are much greater than for run-of-the-mill decision models. The advantages of the tools used by the project, such as R and GitHub, are explained, and the paper provides some ammunition for the open-source movement. One of the best parts of the paper is the authors’ challenge to those who question open-source modelling on the basis of intellectual property concerns. For example, they state that, “Claiming intellectually property on the implementation of a relatively common modeling approach in Excel or other programming software, such as a partitioned survival model in oncology, seems a bit pointless.” Agreed.

The response to date from the community has been broadly positive, though there has been a lack of engagement from US decision-makers. Despite this, the initiative has managed to secure adequate funding. This paper is a valuable read for anyone involved in open-source modelling or in establishing a collaborative platform for the creation and dissemination of research tools.

Incorporating affordability concerns within cost-effectiveness analysis for health technology assessment. Value in Health Published 30th July 2019

The issue of affordability is proving to be a hard nut to crack for health economists. That’s probably because we’ve spent a very long time conducting incremental cost-effectiveness analyses that pay little or no attention to the budget constraint. This paper sets out to define a framework that finally brings affordability into the fold.

The author sets up an example with a decision-maker that seeks to maximise population health with a fixed budget – read, HTA agency – and the motivating example is new medicines for hepatitis C. The core of the proposal is an alternative decision rule. Rather than simply comparing the incremental cost-effectiveness ratio (ICER) to a fixed threshold, it incorporates a threshold that is a function of the budget impact. At it’s most basic, a bigger budget impact (all else equal) means a greater opportunity cost and thus a lower threshold. The author suggests doing away with the ICER (which is almost impossible to work with) and instead using net health benefits. In this framework, whether or not net health benefit is greater than zero depends on the size of the budget impact at any given ICER. If we accept the core principle that budget impact should be incorporated into the decision rule, it raises two other issues – time and uncertainty – which are also addressed in the paper. The framework moves us beyond the current focus on net present value, which ignores the distribution of costs over time beyond simply discounting future expenditure. Instead, the opportunity cost ‘threshold’ depends on the budget impact in each time period. The description of the framework also addresses uncertainty in budget impact, which requires the estimation of opportunity costs in each iteration of a probabilistic analysis.

The paper is thorough in setting out the calculations needed to implement this framework. If you’re conducting an economic evaluation of a technology that could have a non-marginal (big) budget impact, you should tag this on to your analysis plan. Once researchers start producing these estimates, we’ll be able to understand how important these differences could be for resource allocation decision-making and determine whether the likes of NICE ought to incorporate it into their methods guide.

Did UberX reduce ambulance volume? Health Economics [PubMed] [RePEc] Published 24th June 2019

In London, you can probably – at most times of day – get an Uber quicker than you can get an ambulance. That isn’t necessarily a bad thing, as ambulances aren’t there to provide convenience. But it does raise an interesting question. Could the availability of super-fast, low-cost, low-effort taxi hailing reduce pressure on ambulance services? If so, we might anticipate the effect to be greatest where people have to actually pay for ambulances.

This study combines data on Uber market entry in the US, by state and city, with ambulance rates. Between Q1 2012 and Q4 2015, the proportion of the US population with access to Uber rose from 0% to almost 25%. The authors are also able to distinguish ‘lights and sirens’ ambulance rides from ‘no lights and sirens’ rides. A difference-in-differences model estimates the ambulance rate for a given city by quarter-year. The analysis suggests that there was a significant decline in ambulance rates in the years following Uber’s entry to the market, implying an average of 1.2 fewer ambulance trips per 1,000 population per quarter.

There are some questionable results in here, including the fact that a larger effect was found for the ‘lights and sirens’ ambulance rate, so it’s not entirely clear what’s going on. The authors describe a variety of robustness checks for our consideration. Unfortunately, the discussion of the results is lacking in detail and insight, so readers need to figure it out themselves. I’d be very interested to see a similar analysis in the UK. I suspect that I would be inclined to opt for an Uber over an ambulance in many cases. And I wouldn’t have the usual concern about Uber exploiting its drivers, as I dare say ambulance drivers aren’t treated much better.

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James Altunkaya’s journal round-up for 3rd September 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.

Sensitivity analysis for not-at-random missing data in trial-based cost-effectiveness analysis: a tutorial. PharmacoEconomics [PubMed] [RePEc] Published 20th April 2018

Last month, we highlighted a Bayesian framework for imputing missing data in economic evaluation. The paper dealt with the issue of departure from the ‘Missing at Random’ (MAR) assumption by using a Bayesian approach to specify a plausible missingness model from the results of expert elicitation. This was used to estimate a prior distribution for the unobserved terms in the outcomes model.

For those less comfortable with Bayesian estimation, this month we highlight a tutorial paper from the same authors, outlining an approach to recognise the impact of plausible departures from ‘Missingness at Random’ assumptions on cost-effectiveness results. Given poor adherence to current recommendations for the best practice in handling and reporting missing data, an incremental approach to improving missing data methods in health research may be more realistic. The authors supply accompanying Stata code.

The paper investigates the importance of assuming a degree of ‘informative’ missingness (i.e. ‘Missingness not at Random’) in sensitivity analyses. In a case study, the authors present a range of scenarios which assume a decrement of 5-10% in the quality of life of patients with missing health outcomes, compared to multiple imputation estimates based on observed characteristics under standard ‘Missing at Random’ assumptions. This represents an assumption that, controlling for all observed characteristics used in multiple imputation, those with complete quality of life profiles may have higher quality of life than those with incomplete surveys.

Quality of life decrements were implemented in the control and treatment arm separately, and then jointly, in six scenarios. This aimed to demonstrate the sensitivity of cost-effectiveness judgements to the possibility of a different missingness mechanism in each arm. The authors similarly investigate sensitivity to higher health costs in those with missing data than predicted based on observed characteristics in imputation under ‘Missingness at Random’. Finally, sensitivity to a simultaneous departure from ‘Missingness at Random’ in both health outcomes and health costs is investigated.

The proposed sensitivity analyses provide a useful heuristic to assess what degree of difference between missing and non-missing subjects on unobserved characteristics would be necessary to change cost-effectiveness decisions. The authors admit this framework could appear relatively crude to those comfortable with more advanced missing data approaches such as those outlined in last month’s round-up. However, this approach should appeal to those interested in presenting the magnitude of uncertainty introduced by missing data assumptions, in a way that is easily interpretable to decision makers.

The impact of waiting for intervention on costs and effectiveness: the case of transcatheter aortic valve replacement. The European Journal of Health Economics [PubMed] [RePEc] Published September 2018

This paper appears in print this month and sparked interest as one of comparatively few studies on the cost-effectiveness of waiting lists. Given interest in using constrained optimisation methods in health outcomes research, highlighted in this month’s editorial in Value in Health, there is rightly interest in extending the traditional sphere of economic evaluation from drugs and devices to understanding the trade-offs of investing in a wider range of policy interventions, using a common metric of costs and QALYs. Rachel Meacock’s paper earlier this year did a great job at outlining some of the challenges involved broadening the scope of economic evaluation to more general decisions in health service delivery.

The authors set out to understand the cost-effectiveness of delaying a cardiac treatment (TVAR) using a waiting list of up to 12 months compared to a policy of immediate treatment. The effectiveness of treatment at 3, 6, 9 & 12 months after initial diagnosis, health decrements during waiting, and corresponding health costs during wait time and post-treatment were derived from a small observational study. As treatment is studied in an elderly population, a non-ignorable proportion of patients die whilst waiting for surgery. This translates to lower modelled costs, but also lower quality life years in modelled cohorts where there was any delay from a policy of immediate treatment. The authors conclude that eliminating all waiting time for TVAR would produce population health at a rate of ~€12,500 per QALY gained.

However, based on the modelling presented, the authors lack the ability to make cost-effectiveness judgements of this sort. Waiting lists exist for a reason, chiefly a lack of clinical capacity to treat patients immediately. In taking a decision to treat patients immediately in one disease area, we therefore need some judgement as to whether the health displaced in now untreated patients in another disease area is of greater, less or equal magnitude to that gained by treating TVAR patients immediately. Alternately, modelling should include the cost of acquiring additional clinical capacity (such as theatre space) to treat TVAR patients immediately, so as not to displace other treatments. In such a case, the ICER is likely to be much higher, due to the large cost of new resources needed to reduce waiting times to zero.

Given the data available, a simple improvement to the paper would be to reflect current waiting times (already gathered from observational study) as the ‘standard of care’ arm. As such, the estimated change in quality of life and healthcare resource cost from reducing waiting times to zero from levels observed in current practice could be calculated. This could then be used to calculate the maximum acceptable cost of acquiring additional treatment resources needed to treat patients with no waiting time, given current national willingness-to-pay thresholds.

Admittedly, there remain problems in using the authors’ chosen observational dataset to calculate quality of life and cost outcomes for patients treated at different time periods. Waiting times were prioritised in this ‘real world’ observational study, based on clinical assessment of patients’ treatment need. Thus it is expected that the quality of life lost during a waiting period would be lower for patients treated in the observational study at 12 months, compared to the expected quality of life loss of waiting for the group of patients judged to need immediate treatment. A previous study in cardiac care took on the more manageable task of investigating the cost-effectiveness of different prioritisation strategies for the waiting list, investigating the sensitivity of conclusions to varying a fixed maximum wait-time for the last patient treated.

This study therefore demonstrates some of the difficulties in attempting to make cost-effectiveness judgements about waiting time policy. Given that the cost-effectiveness of reducing waiting times in different disease areas is expected to vary, based on relative importance of waiting for treatment on short and long-term health outcomes and costs, this remains an interesting area for economic evaluation to explore. In the context of the current focus on constrained optimisation techniques across different areas in healthcare (see ISPOR task force), it is likely that extending economic evaluation to evaluate a broader range of decision problems on a common scale will become increasingly important in future.

Understanding and identifying key issues with the involvement of clinicians in the development of decision-analytic model structures: a qualitative study. PharmacoEconomics [PubMed] Published 17th August 2018

This paper gathers evidence from interviews with clinicians and modellers, with the aim to improve the nature of the working relationship between the two fields during model development.

Researchers gathered opinion from a variety of settings, including industry. The main report focusses on evidence from two case studies – one tracking the working relationship between modellers and a single clinical advisor at a UK university, with the second gathering evidence from a UK policy institute – where modellers worked with up to 11 clinical experts per meeting.

Some of the authors’ conclusions are not particularly surprising. Modellers reported difficulty in recruiting clinicians to advise on model structures, and further difficulty in then engaging recruited clinicians to provide relevant advice for the model building process. Specific comments suggested difficulty for some clinical advisors in identifying representative patient experiences, instead diverting modellers’ attention towards rare outlier events.

Study responses suggested currently only 1 or 2 clinicians were typically consulted during model development. The authors recommend involving a larger group of clinicians at this stage of the modelling process, with a more varied range of clinical experience (junior as well as senior clinicians, with some geographical variation). This is intended to help ensure clinical pathways modelled are generalizable. The experience of one clinical collaborator involved in the case study based at a UK university, compared to 11 clinicians at the policy institute studied, perhaps may also illustrate a general problem of inadequate compensation for clinical time within the university system. The authors also advocate the availability of some relevant training for clinicians in decision modelling to help enhance the efficiency of participants’ time during model building. Clinicians sampled were supportive of this view – citing the need for further guidance from modellers on the nature of their expected contribution.

This study ties into the general literature regarding structural uncertainty in decision analytic models. In advocating the early contribution of a larger, more diverse group of clinicians in model development, the authors advocate a degree of alignment between clinical involvement during model structuring, and guidelines for eliciting parameter estimates from clinical experts. Similar problems, however, remain for both fields, in recruiting clinical experts from sufficiently diverse backgrounds to provide a valid sample.

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