Ian Cromwell’s journal round-up for 17th February 2020

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 use of health technology assessment have an impact on the utilisation of health care resources? Evidence from two European countries. European Journal of Health Economics [PubMed] Published 5th February 2020

The ostensible purpose of health technology assessment (HTA) is to provide health care decision-makers with the information they need when considering whether to change existing policies. One of the questions I’ve heard muttered sotto voce (and that I will admit to having asked myself in more cynical moments) is whether or not HTAs actually make a difference. We are generating lots of evidence, but does it have any real impact on decision making? Do the complex analyses health economists undertake make any impact on policy?

This paper used data from Catalonia and England to estimate the impact of a positive HTA recommendation from the regulatory bodies – the National Institute for Health and Care Excellence (NICE) in England and a collection of regional approval bodies in Catalonia and Spain – to assess trends in medical usage prior to and following the publication of HTA-guided recommendations for new cancer drugs between 2011 and the end of 2016. Utilization (volume of drugs dispensed) and expenditure were extracted from retrospective records. The authors built a Poisson regression model that allowed them to observe temporal effects of usage before and following a positive recommendation.

The authors noted that a lack of pre-recommendation utilization data made it difficult to compute a model of negative recommendations (which is the more cynical version of the question!), so it is important to recognize that as a limitation of the approach. They also note, however, that it is typically the case in the UK and Catalonia that approvals for new drugs are conditional on a positive recommendation. Spain has a different system in which medicines may still be available even if they are not recommended.

The results of the model are a bit more complex than is easy to fit into a blog post, but the bottom line is that a positive recommendation does produce an increase in utilization. What stuck out to me about the descriptive findings was the consistent presence of a trend toward increased usage happening before the recommendation was published. But the Poisson model found a significant effect of the recommendation even controlling for that temporal trend. The authors helpfully noted that the criteria going into a recommendation are different between England and Spain (cost per QALY in England, clinical effectiveness alone sometimes in Spain), which makes inter-country comparisons challenging.

Health‐related quality of life in oncology drug reimbursement submissions in Canada: a review of submissions to the pan‐Canadian Oncology Drug Review. Cancer [PubMed] Published 1st January 2020

In Canada, newly-developed cancer drugs undergo HTA through the pan-Canadian Oncology Drug Review (pCODR), a program run under the auspices of the Canadian Agency for Drugs and Technologies in Health (CADTH). Unlike NICE in the UK, the results of CADTH’s pCODR recommendations are not binding; they are intended instead to provide provincial decision-makers with expert evidence they can use when deciding whether or not to add drugs to their formulary.

This paper, written by researchers at the Canadian Centre for Applied Research in Cancer Control (ARCC), reviewed the publicly-available reports governing 43 pCODR recommendations between 2015 and 2018. The paper summarizes the findings of the cost-effectiveness analyses generated in each report, including incremental costs and incremental QALYs (incremental cost per QALY being the reference case used by CADTH). The authors also appraised the methods chosen within each submission, both in terms of decision model structure and data inputs.

Interestingly, and perhaps disconcertingly, the paper reports a notable discrepancy between the ICERs reported by the submitting manufacturer and those calculated by CADTH’s Economics Guidance Panel. This appeared to be largely driven by the kind of health-related quality of life (HRQoL) data used to generate the QALYs in each submission. The authors note that the majority (56%) of the submissions provided to pCODR didn’t collect HRQoL data alongside clinical trials, preferring instead to use values published in the literature. In the face of high levels of uncertainty and relatively small incremental benefits (the median change in QALYs was 0.86), it seems crucial to have reliable information about HRQoL for making these kinds of decisions.

Regulatory and advisory agencies like CADTH have a rather weighty responsibility, not only to help decision makers identify which new drugs and technologies the health care system should adopt, but also which ones they should reject. When manufacturers’ submissions rely on inappropriate data with high levels of uncertainty, this task becomes much more difficult. The authors suggest that manufacturers should be collecting their own HRQoL data in clinical trials they fund. After all, if we want HTAs to have an effect on policy-making, we should also make sure they’re having a positive effect.

The cost-effectiveness of limiting federal housing vouchers to use in low-poverty neighborhoods in the United States. Public Health [PubMed] Published January 2020

My undergraduate education was heavily steeped in discussions of the social determinants of health. Another cynical opinion I’ve heard (again sometimes from myself) is that health economics is disproportionately concerned with the adoption of new drugs that have a marginal effect on health, often at the expense of investment in the other non-health-care determinants. This is a particularly persuasive bit of cynicism when you consider cancer drugs like in our previous two examples, where the incremental benefits are typically modest and the costs typically high. That’s why I was especially excited to see this paper published by my friend Dr. Zafar Zafari, applying health economic analysis frameworks to something atypical: housing policy.

The authors evaluated a trial running alongside a program providing housing vouchers to 4600 low-income households. The experimental condition in this case was that the vouchers could only be used in well-off neighbourhoods (i.e., those with a low level of poverty). The authors considered the evidence showing a link between neighbourhood wealth and lowering rates of obesity-related health conditions like diabetes, and used that evidence to construct a Markov decision model to measure incremental cost per QALY over the length of the study (10-15 years). Cohort characteristics, relative clinical effectiveness, and costs of the voucher program were estimated from trial results, with other costs and probabilities derived from the literature.

Compared to the control group (public housing), use of the housing vouchers provided an additional 0.23 QALYs per person, at a lower cost (about $750 less per person). Importantly, these findings were highly robust to parameter uncertainty, with 99% of ICERs falling below a willingness-to-pay threshold of $20,000/QALY (>90% below a WTP threshold of $0/QALY). The model was highly sensitive to the discount rate, which makes sense considering that we would expect, for a chronic condition like diabetes and a distal relationship like housing, that all the incremental health gains would be occurring years after the initial intervention.

There are a lot of things to like about this paper, but the one that stands out to me is the way they’ve framed the question:

We seek to inform the policy debate over the wisdom of spending health dollars on non-health sectors of the economy by defining the trade-off, or ‘opportunity cost’ of such a decision.

The idea that “health funds” should be focussed on “health care” robs us of the opportunity to consider the health impact of interventions in other policy areas. By bringing something like housing explicitly into the realm of cost-per-QALY analysis, the authors invite us all to consider the kinds of trade-offs we make when we relegate our consideration of health only to the kinds of things that happen inside hospitals.

A multidimensional array representation of state-transition model dynamics. Medical Decision Making [PubMed] Published 28th January 2020

I’ve been building models in R for a few years now, and developed a method of my own more or less out of necessity. So I’ve always been impressed with and drawn to the work of the group Decision Analysis in R for Technologies in Health (the amazingly-named DARTH). I’ve had the opportunity to meet a couple of their scientists and have followed their work for a while, and so I was really pleased to see the publication of this paper, hot on the heels of another paper discussing a formalized approach to model construction in R, and timed to coincide with the publication of a step-by-step guidebook on how to build models according to the DARTH recipe.

The DARTH approach (and, as a happy coincidence, mine too) involves tapping into R’s powerful ability to organize data into multidimensional arrays. The paper talks in depth about how R arrays can be used to represent health states, and how to set up and program models of essentially any level of complexity using a set of basic R commands. As a bonus they include publicly-accessible sample code that you can follow along as you read (which is the best way to learn something like this).

The authors argue that the method they propose is ideal for capturing and reflecting “transition rewards” – that is, effects on the cohort that occur during transitions between health states – in addition to “state rewards” (effects that happen as a consequence of being within a state). The key to this Dynamics Array approach is the use of a three-dimensional array to store the transitions, with the third array representing the passage of time. After walking the reader through the theory, the authors present a sample three-state model and show that the new method is fast, efficient, and accurate.

I hope that I have been sufficiently clear that I am a big fan of DARTH and admire their work a great deal. Because there is one big criticism I have to level at them, which is that this paper (and the others I have cited) is not terribly easy to follow. It sort of presumes that you already understand a lot of the topics that are discussed, which I personally do not. And if I, someone who has built many array-based models in R, am having a tough time understanding the explanation of their approach then woe betide anyone else who is reading this paper without a firm grasp of R, decision modelling theory, matrix algebra, and a handful of the other topics required to benefit from this (truly excellent) work.

DARTH is laying down a well-thought-out path to revolutionizing the standard approach to model building, but they can only do that if people start adopting their approach. If I were a grad student hoping to build my first model, this paper would likely intimidate me enough to maybe go back to the default of building it in Excel. As a postdoc with my own way of doing things there is a big opportunity cost of switching, and part of that cost is feeling too dumb to follow the instructions. I know that DARTH has tutorials and courses and workshops to help people get up to speed, but I hope that they also have a plan to translate some of this knowledge into a form that is more accessible for casual coders, non-economists, and other people who need this info but who (like me) might find this format opaque.

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Brendan Collins’s journal round-up for 14th January 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.

Income distribution and health: can polarization explain health outcomes better than inequality? The European Journal of Health Economics [PubMed] Published 4th December 2018

One of my main interests is health inequalities. I thought polarisation was intuitive; I had seen it in the context of the UK and the US employment market; an increase in poorly-paid ‘McJobs’ and an increase in well-paid ‘MacJobs’, with fewer jobs in the middle. But I hadn’t seen polarisation measured in a statistical way.

Traditional measures of population inequalities like Gini or Atkinson index measure the share of income or the ratio of richest to poorest. But polarisation goes a step further and looks whether there are discrete clusters or groups who have similar incomes. The theory goes that having discrete groups increases social alienation, conflict and socioeconomic comparison and increases health inequalities. Now, I get how you can test statistically for discrete income clusters, and there is an evidence base for the relationship between polarisation and social tension. But groups will cluster based on other factors besides income. I feel like it may be taking a leap to assume a statistical finding (income polarisation) will always represent a sociological construct (alienation) but I confess I don’t know the literature behind this.

China is a country with an increasing degree of polarisation as measured by the Duclos, Esteban and Ray (DER) polarisation indices, and this study suggests that it is related to health status. This study looked at trends in BMI and systolic blood pressure from 1991 to 2011 and found both to increase with increased polarisation. I imagine a lot of other social change went on in this time period in China. I think BMI might not be a good candidate for measuring the effect of polarisation, as being poor is associated with malnourishment and low weight as well as obesity. The authors found that social capital (based on increasing family size, community size, and living in the same community for a long time) had a protective effect against the effects of polarisation on health. Whether this study provides more evidence for the socioeconomic comparison or status anxiety theories of health inequalities, I am not sure; it could equally provide evidence for the neo-materialist (i.e. simply not having enough resources for a healthy life) theories – the relative importance will likely differ by country anyway.

Maybe we don’t need to add more measures of inequality to the mix but I am intrigued. I am just starting my journey with polarisation but I think it has promise.

Two-year evaluation of mandatory bundled payments for joint replacement. The New England Journal of Medicine [PubMed] Published 2nd January 2019

Joint replacements are a big cost to western healthcare systems and often delayed or rationed (partly because replacement joints may only have a 10-20 year lifespan on average). In the UK, for instance, joint replacements have been rationed based on factors like BMI or pain levels (in my opinion, often in an arbitrary way to save money).

This paper found that having a bundled payments and penalties model (Comprehensive Care for Joint Replacement; CJR) for optimal care around hip and knee replacements reduced Medicare spending per episode compared to areas that did not pilot the programme. The overall difference was small in absolute terms at $812 against a total cost of around $24,000 per episode. The programme involves the hospital meeting a set of performance measures, and if they can do so at a lower cost, any savings are shared between the hospital and the payer. Cost savings were mainly driven by a reduction in patients being discharged to post-acute care facilities. Rates of complex patients were similar between pilot and control areas – this is important because a lower rate of complex cases in the CJR trial areas might indicate hospitals ‘cherry picking’ easier to treat, less expensive cases. Also, rates of complications were not significantly different between the CJR pilot areas and controls.
This paper suggests that having this kind of bundled payment programme can save money while maintaining quality.

Association of the Hospital Readmissions Reduction Program with mortality among Medicare beneficiaries hospitalized for heart failure, acute myocardial infarction, and pneumonia. JAMA [PubMed] Published 25th December 2018

Nobody likes being in hospital. But sometimes hospitals are the best places for people. This paper looks at possible unintended consequences of a US programme; the Hospital Readmissions Reduction Program (HRRP) where the Centers for Medicare & Medicaid Services (CMS) impose financial penalties (almost $2billion dollars’ worth since 2012) on hospitals with elevated 30-day readmission rates for patients with heart failure, acute myocardial infarction, and pneumonia. This study compared four time periods (no control group) and found that, after the programme was implemented, death rates for people who had been admitted with pneumonia and heart failure increased, with these increased deaths occurring more in people who had not been readmitted to hospital. The analysis controlled for differences in demographics, comorbidities, and calendar month using propensity scores and inverse probability weighting.

The authors are clear that their results do not establish cause and effect but are concerning nonetheless and worthy of more analysis. Incidentally, there is another paper this week in Health Affairs which suggests that the benefits of the programme in reducing readmissions was overstated.

There has been a similar financial incentive in the English NHS where hospitals are subject to the 30-day readmission rule, meaning they are not paid for people who are readmitted as an emergency within 30 days of being discharged. This is shortly to be abolished for 2019/20. I wonder if there has been similar research on whether this also led to unintended consequences in the NHS. Maybe there is a general lesson here about thinking a bit deeper about the potential outcomes of incentives in healthcare markets?

In these last two papers, we have had two examples of financial incentive programmes from Medicare. The CJR, which seems to have worked, has been dampened down from a mandatory to a voluntary programme, while the HRRP, which may not have worked, has been extended.

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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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