Rita Faria’s journal round-up for 28th 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.

Appraising the value of evidence generation activities: an HIV modelling study. BMJ Global Health [PubMed] Published 7th December 2018

How much should we spend on implementing our health care strategy versus getting more information to devise a better strategy? Should we devolve budgets to regions or administer the budget centrally? These are difficult questions and this new paper by Beth Woods et al has a brilliant stab at answering them.

The paper looks at the HIV prevention and treatment policies in Zambia. It starts by finding the most cost-effective strategy and the corresponding budget in each region, given what is currently known about the prevalence of the infection, the effectiveness of interventions, etc. The idea is that the regions receive a cost-effective budget to implement a cost-effective strategy. The issue is that the cost-effective strategy and budget are devised according to what we currently know. In practice, regions might face a situation on the ground which is different from what was expected. Regions might not have enough budget to implement the strategy or might have some leftover.

What if we spend some of the budget to get more information to make a better decision? This paper considers the value of perfect information given the costs of research. Depending on the size of the budget and the cost of research, it may be worthwhile to divert some funds to get more information. But what if we had more flexibility in the budgetary policy? This paper tests 2 more budgetary options: a national hard budget but with the flexibility to transfer funds from under- to overspending regions, and a regional hard budget with a contingency fund.

The results are remarkable. The best budgetary policy is to have a national budget with the flexibility to reallocate funds across regions. This is a fascinating paper, with implications not only for prioritisation and budget setting in LMICs but also for high-income countries. For example, the 2012 Health and Social Care Act broke down PCTs into smaller CCGs and gave them hard budgets. Some CCGs went into deficit, and there are reports that some interventions have been cut back as a result. There are probably many reasons for the deficit, but this paper shows that hard regional budgets clearly have negative consequences.

Health economics methods for public health resource allocation: a qualitative interview study of decision makers from an English local authority. Health Economics, Policy and Law [PubMed] Published 11th January 2019

Our first paper looked at how to use cost-effectiveness to allocate resources between regions and across health care services and research. Emma Frew and Katie Breheny look at how decisions are actually made in practice, but this time in a local authority in England. Another change of the 2012 Health and Social Care Act was to move public health responsibilities from the NHS to local authorities. Local authorities are now given a ring-fenced budget to implement cost-effective interventions that best match their needs. How do they make decisions? Thanks to this paper, we’re about to find out.

This paper is an enjoyable read and quite an eye-opener. It was startling that health economics evidence was not much used in practice. But the barriers that were cited are not insurmountable. And the suggestions by the interviewees were really useful. There were suggestions about how economic evaluations should consider the local context to get a fair picture of the impact of the intervention to services and to the population, and to move beyond the trial into the real world. Equity was mentioned too, as well as broadening the outcomes beyond health. Fortunately, the health economics community is working on many of these issues.

Lastly, there was a clear message to make economic evidence accessible to lay audiences. This is a topic really close to my heart, and something I’d like to help improve. We have to make our work easy to understand and use. Otherwise, it may stay locked away in papers rather than do what we intended it for. Which is, at least in my view, to help inform decisions and to improve people’s lives.

I found this paper reassuring in that there is clearly a need for economic evidence and a desire to use it. Yes, there are some teething issues, but we’re working in the right direction. In sum, the future for health economics is bright!

Survival extrapolation in cancer immunotherapy: a validation-based case study. Value in Health Published 13th December 2018

Often, the cost-effectiveness of cancer drugs hangs in the method to extrapolate overall survival. This is because many cancer drugs receive their marketing authorisation before most patients in the trial have died. Extrapolation is tested extensively in the sensitivity analysis, and this is the subject of many discussions in NICE appraisal committees. Ultimately, at the point of making the decision, the correct method to extrapolate is a known unknown. Only in hindsight can we know for sure what the best choice was.

Ash Bullement and colleagues take advantage of hindsight to know the best method for extrapolation of a clinical trial of an immunotherapy drug. Survival after treatment with immunotherapy drugs is more difficult to predict because some patients can survive for a very long time, while others have much poorer outcomes. They fitted survival models to the 3-year data cut, which was available at the time of the NICE technology appraisal. Then they compared their predictions to the observed survival in the 5-year data cut and to long-term survival trends from registry data. They found that the piecewise model and a mixture-cure model had the best predictions at 5 years.

This is a relevant paper for those of us who work in the technology appraisal world. I have to admit that I can be sceptical of piecewise and mixture-cure models, but they definitely have a role in our toolbox for survival extrapolation. Ideally, we’d have a study like this for all the technology appraisals hanging on the survival extrapolation so that we can take learnings across cancers and classes of drugs. With time, we would get to know more about what works best for which condition or drug. Ultimately, we may be able to get to a stage where we can look at the extrapolation with less inherent uncertainty.

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Meeting round-up: Society for Medical Decision Making 17th Biennial European Conference

The Society for Medical Decision Making (SMDM) held their 17th European Conference between 10th and 12th June at the Stadsgehoorzaal in Leiden, the Netherlands. The meeting was chaired by Anne Stiggelbout and Ewout Steyerberg who, along with Uwe Siebert, welcomed us (early) on Monday morning. Some delegates arrived on Sunday for short courses on a range of topics, from modelling in R and causal inference to the psychology of decision making.

Although based in the US, SMDM holds biennial meetings in Europe which are generally attended by delegates from around the world. Around 300 delegates were in attendance at this meeting, travelling from Toronto to Tehran.

The meeting was ‘Patients Included’ and we were introduced to around 10 patients and caregivers on the first morning. They confidently asked questions and gave comments after the presentations and the plenary, sharing their real-world experience to provide context to findings.

There were five ‘oral abstract’ sessions each comprising six presentations in 15 minute slots (10 minutes long with 5 minutes for audience questions). The sessions covered empirical research relating to physician and patient decision-making, and quantitative valuation and evaluation. Popular applied areas were prostate cancer, breast cancer and precision medicine.

Running in parallel to the oral presentations, workshops were dealing with methodological issues relating to health economics, shared decision-making and psychology.

Four poster sessions, conveniently surrounding the refreshment table, attracted delegates in the morning, breaks and lunch. SMDM provides some of the best poster sessions: posters are always of high quality which means poster sessions are always well attended.

One of the highlights of the meeting was the plenary presentation by Sir David Spiegelhalter who spoke about the challenges of communicating benefits and harms (often probabilities) impartially. Sir David gave examples from the UK’s national breast screening programme to show how presenting information can change people’s interpretation of risk. He also drew on examples of ‘nudges’ which may involve providing information in a persuasive rather than informing way in order to manipulate behaviour. Sir David gave us examples of materials which had been redesigned to improve both patients’ and clinicians’ understanding of the information of benefits and harms. The session concluded with a short video about how Ugandan primary school children have reading comic strips to help interpret information and find facts about the benefits and harms of healthcare interventions.

The European SMDM meeting was thoroughly enjoyable and very interesting. The standard of oral and poster presentations was very high, and the environment was very friendly and conducive to networking.

The next North American meeting is in Montreal (October 2018) and the next European meeting will be in 2020 (location to be confirmed).

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James Lomas’s journal round-up for 21st May 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.

Decision making for healthcare resource allocation: joint v. separate decisions on interacting interventions. Medical Decision Making [PubMed] Published 23rd April 2018

While it may be uncontroversial that including all of the relevant comparators in an economic evaluation is crucial, a careful examination of this statement raises some interesting questions. Which comparators are relevant? For those that are relevant, how crucial is it that they are not excluded? The answer to the first of these questions may seem obvious, that all feasible mutually exclusive interventions should be compared, but this is in fact deceptive. Dakin and Gray highlight inconsistency between guidelines as to what constitutes interventions that are ‘mutually exclusive’ and so try to re-frame the distinction according to whether interventions are ‘incompatible’ – when it is physically impossible to implement both interventions simultaneously – and, if not, whether interventions are ‘interacting’ – where the costs and effects of the simultaneous implementation of A and B do not equal the sum of these parts. What I really like about this paper is that it has a very pragmatic focus. Inspired by policy arrangements, for example single technology appraisals, and the difficulty in capturing all interactions, Dakin and Gray provide a reader-friendly flow diagram to illustrate cases where excluding interacting interventions from a joint evaluation is likely to have a big impact, and furthermore propose a sequencing approach that avoids the major problems in evaluating separately what should be considered jointly. Essentially when we have interacting interventions at different points of the disease pathway, evaluating separately may not be problematic if we start at the end of the pathway and move backwards, similar to the method of backward induction used in sequence problems in game theory. There are additional related questions that I’d like to see these authors turn to next, such as how to include interaction effects between interventions and, in particular, how to evaluate system-wide policies that may interact with a very large number of interventions. This paper makes a great contribution to answering all of these questions by establishing a framework that clearly distinguishes concepts that had previously been subject to muddied thinking.

When cost-effective interventions are unaffordable: integrating cost-effectiveness and budget impact in priority setting for global health programs. PLoS Medicine [PubMed] Published 2nd October 2017

In my opinion, there are many things that health economists shouldn’t try to include when they conduct cost-effectiveness analysis. Affordability is not one of these. This paper is great, because Bilinski et al shine a light on the worldwide phenomenon of interventions being found to be ‘cost-effective’ but not affordable. A particular quote – that it would be financially impossible to implement all interventions that are found to be ‘very cost-effective’ in many low- and middle-income countries – is quite shocking. Bilinski et al compare and contrast cost-effectiveness analysis and budget impact analysis, and argue that there are four key reasons why something could be ‘cost-effective’ but not affordable: 1) judging cost-effectiveness with reference to an inappropriate cost-effectiveness ‘threshold’, 2) adoption of a societal perspective that includes costs not falling upon the payer’s budget, 3) failing to make explicit consideration of the distribution of costs over time and 4) the use of an inappropriate discount rate that may not accurately reflect the borrowing and investment opportunities facing the payer. They then argue that, because of this, cost-effectiveness analysis should be presented along with budget impact analysis so that the decision-maker can base a decision on both analyses. I don’t disagree with this as a pragmatic interim solution, but – by highlighting these four reasons for divergence of results with such important economic consequences – I think that there will be further reaching implications of this paper. To my mind, Bilinski et al essentially serves as a call to arms for researchers to try to come up with frameworks and estimates so that the conduct of cost-effectiveness analysis can be improved in order that paradoxical results are no longer produced, decisions are more usefully informed by cost-effectiveness analysis, and the opportunity costs of large budget impacts are properly evaluated – especially in the context of low- and middle-income countries where the foregone health from poor decisions can be so significant.

Patient cost-sharing, socioeconomic status, and children’s health care utilization. Journal of Health Economics [PubMed] Published 16th April 2018

This paper evaluates a policy using a combination of regression discontinuity design and difference-in-difference methods. Not only does it do that, but it tackles an important policy question using a detailed population-wide dataset (a set of linked datasets, more accurately). As if that weren’t enough, one of the policy reforms was actually implemented as a result of a vote where two politicians ‘accidentally pressed the wrong button’, reducing concerns that the policy may have in some way not been exogenous. Needless to say I found the method employed in this paper to be a pretty convincing identification strategy. The policy question at hand is about whether demand for GP visits for children in the Swedish county of Scania (Skåne) is affected by cost-sharing. Cost-sharing for GP visits has occurred for different age groups over different periods of time, providing the basis for regression discontinuities around the age threshold and treated and control groups over time. Nilsson and Paul find results suggesting that when health care is free of charge doctor visits by children increase by 5-10%. In this context, doctor visits happened subject to telephone triage by a nurse and so in this sense it can be argued that all of these visits would be ‘needed’. Further, Nilsson and Paul find that the sensitivity to price is concentrated in low-income households, and is greater among sickly children. The authors contextualise their results very well and, in addition to that context, I can’t deny that it also particularly resonated with me to read this approaching the 70th birthday of the NHS – a system where cost-sharing has never been implemented for GP visits by children. This paper is clearly also highly relevant to that debate that has surfaced again and again in the UK.

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