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.

Credits

 

Chris Sampson’s journal round-up for 4th 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.

A qualitative investigation of the health economic impacts of bariatric surgery for obesity and implications for improved practice in health economics. Health Economics [PubMed] Published 1st June 2018

Few would question the ‘economic’ nature of the challenge of obesity. Bariatric surgery is widely recommended for severe cases but, in many countries, the supply is not sufficient to satisfy the demand. In this context, this study explores the value of qualitative research in informing economic evaluation. The authors assert that previous economic evaluations have adopted a relatively narrow focus and thus might underestimate the expected value of bariatric surgery. But rather than going and finding data on what they think might be additional dimensions of value, the authors ask patients. Emotional capital, ‘societal’ (i.e. non-health) impacts, and externalities are identified as theories for the types of value that might be derived from bariatric surgery. These theories were used to guide the development of questions and prompts that were used in a series of 10 semi-structured focus groups. Thematic analysis identified the importance of emotional costs and benefits as part of the ‘socioemotional personal journey’ associated with bariatric surgery. Out-of-pocket costs were also identified as being important, with self-funding being a challenge for some respondents. The information seems useful in a variety of ways. It helps us understand the value of bariatric surgery and how individuals make decisions in this context. This information could be used to determine the structure of economic evaluations or the data that are collected and used. The authors suggest that an EQ-5D bolt-on should be developed for ’emotional capital’ but, given that this ‘theory’ was predefined by the authors and does not arise from the qualitative research as being an important dimension of value alongside the existing EQ-5D dimensions, that’s a stretch.

Developing accessible, pictorial versions of health-related quality-of-life instruments suitable for economic evaluation: a report of preliminary studies conducted in Canada and the United Kingdom. PharmacoEconomics – Open [PubMed] Published 25th May 2018

I’ve been telling people about this study for ages (apologies, authors, if that isn’t something you wanted to read!). In my experience, the need for more (cognitively / communicatively) accessible outcome measures is widely recognised by health researchers working in contexts where this is relevant, such as stroke. If people can’t read or understand the text-based descriptors that make up (for example) the EQ-5D, then we need some alternative format. You could develop an entirely new measure. Or, as the work described in this paper set out to do, you could modify existing measures. There are three descriptive systems described in this study: i) a pictorial EQ-5D-3L by the Canadian team, ii) a pictorial EQ-5D-3L by the UK team, and iii) a pictorial EQ-5D-5L by the UK team. Each uses images to represent the different levels of the different dimensions. For example, the mobility dimension might show somebody walking around unaided, walking with aids, or in bed. I’m not going to try and describe what they all look like, so I’ll just encourage you to take a look at the Supplementary Material (click here to download it). All are described as ‘pilot’ instruments and shouldn’t be picked up and used at this stage. Different approaches were used in the development of the measures, and there are differences between the measures in terms of the images selected and the ways in which they’re presented. But each process referred to conventions in aphasia research, used input from clinicians, and consulted people with aphasia and/or their carers. The authors set out several remaining questions and avenues for future research. The most interesting possibility to most readers will be the notion that we could have a ‘generic’ pictorial format for the EQ-5D, which isn’t aphasia-specific. This will require continued development of the pictorial descriptive systems, and ultimately their validation.

QALYs in 2018—advantages and concerns. JAMA [PubMed] Published 24th May 2018

It’s difficult not to feel sorry for the authors of this article – and indeed all US-based purveyors of economic evaluation in health care. With respect to social judgments about the value of health technologies, the US’s proverbial head remains well and truly buried in the sand. This article serves as a primer and an enticement for the use of QALYs. The ‘concerns’ cited relate almost exclusively to decision rules applied to QALYs, rather than the underlying principles of QALYs, presumably because the authors didn’t feel they could ignore the points made by QALY opponents (even if those arguments are vacuous). What it boils down to is this: trade-offs are necessary, and QALYs can be used to promote value in those trade-offs, so unless you offer some meaningful alternative then QALYs are here to stay. Thankfully, the Institute for Clinical and Economic Review (ICER) has recently added some clout to the undeniable good sense of QALYs, so the future is looking a little brighter. Suck it up, America!

The impact of hospital costing methods on cost-effectiveness analysis: a case study. PharmacoEconomics [PubMed] Published 22nd May 2018

Plugging different cost estimates into your cost-effectiveness model could alter the headline results of your evaluation. That might seems obvious, but there are a variety of ways in which the selection of unit costs might be somewhat arbitrary or taken for granted. This study considers three alternative sources of information for hospital-based unit costs for hip fractures in England: (a) spell-level tariffs, (b) finished consultant episode (FCE) reference costs, and (c) spell-level reference costs. Source (b) provides, in theory, a more granular version of (a), describing individual episodes within a person’s hospital stay. Reference costs are estimated on the basis of hospital activity, while tariffs are prices estimated on the basis of historic reference costs. The authors use a previously reported cohort state transition model to evaluate different models of care for hip fracture and explore how the use of the different cost figures affects their results. FCE-level reference costs produced the highest total first-year hospital care costs (£14,440), and spell-level tariffs the lowest (£10,749). The more FCEs within a spell, the greater the discrepancy. This difference in costs affected ICERs, such that the net-benefit-optimising decision would change. The study makes an important point – that selection of unit costs matters. But it isn’t clear why the difference exists. It could just be due to a lack of precision in reference costs in this context (rather than a lack of accuracy, per se), or it could be that reference costs misestimate the true cost of care across the board. Without clear guidance on how to select the most appropriate source of unit costs, these different costing methodologies represent another source of uncertainty in modelling, which analysts should consider and explore.

Credits

Method of the month: Q methodology

Once a month we discuss a particular research method that may be of interest to people working in health economics. We’ll consider widely used key methodologies, as well as more novel approaches. Our reviews are not designed to be comprehensive but provide an introduction to the method, its underlying principles, some applied examples, and where to find out more. If you’d like to write a post for this series, get in touch. This month’s method is Q methodology.

Principles

There are many situations when we might be interested in people’s views, opinions or beliefs about an issue, such as how we allocate health care resources or the type of care we provide to dementia patients. Typically, health economists may think about using qualitative methods or preference elicitation techniques, but Q methodology could be your new method to examine these questions. Q methodology combines qualitative and quantitative techniques which allow us to first identify the range of the views that exist on a topic and then describe in-depth those viewpoints.

Q methodology was conceived as a way to study subjectivity by William Stephenson and is detailed in his 1953 book The Study of Behaviour. A more widely available book by Watts and Stenner (2012) provides a great general introduction to all stages of a Q study and the paper by Baker et al (2006) introduces Q methodology in health economics.

Implementation

There are two main stages in a Q methodology study. In the first stage, participants express their views through the rank-ordering of a set of statements known as the Q sort. The second stage uses factor analysis to identify patterns of similarity between the Q sorts, which can then be described in detail.

Stage 1: Developing the statements and Q sorting

The most important part of any Q study is the development of the statements that your participants will rank-order. The starting point is to identify all of the possible views on your topic. Participants should be able to interpret the statements as opinion rather than facts, for example, “The amount of health care people have had in the past should not influence access to treatments in the future”. The statements can come from a range of sources including interview transcripts, public consultations, academic literature, newspapers and social media. Through a process of eliminating duplicates, merging and deleting similar statements, you want to end up with a smaller set of statements that is representative of the population of views that exist on your topic. Pilot these statements in a small number of Q sorts before finalising and starting your main data collection.

The next thing to consider is from whom you are going to collect Q sorts. Participant sampling in Q methodology is similar to that of qualitative methods where you are looking to identify ‘data rich’ participants. It is not about representativeness according to demographics; instead, you want to include participants who have strong and differing views on your topic. Typically this would be around 30 to 60 people. Once you have selected your sample you can conduct your Q sorts. Here, each of your participants rank-orders the set of statements according to an instruction, for example from ‘most agree to most disagree’ or ‘highest priority to lowest priority’. At the end of each Q sort, a short interview is conducted asking participants to summarise their opinions on the Q sort and give further explanation for the placing of selected statements.

Stage 2: Analysis and interpretation

In the analysis stage, the aim is to identify people who have ranked their statements in a similar way. This involves calculating the correlations between the participants Q sorts (the full ranking of all statements) to form a correlation matrix which is then subject to factor analysis. The software outlined in the next section can help you with this. The factor analysis will produce a number of statistically significant solutions and your role as the analyst is to decide how many factors you retain for interpretation. This will be an iterative process where you consider the internal coherence of each factor: i.e. does the ranking of the statements make sense, does it align with the comments made by the participants following the Q sort as well as statistical considerations like Eigen Values. The factors are idealised Q sorts that are a complete ranking of all statements, essentially representing the way a respondent who had a correlation coefficient of 1 with the factor would have ranked their statements. The final step is to provide a descriptive account of the factors, looking at the positioning of each statement in relation to the other statements and drawing on the post Q sort interviews to support and aid your interpretation.

Software

There are a small number of software packages available to analyse your Q data, most of which are free to use. The most widely used programme is PQMethod. It is a DOS-based programme which often causes nervousness for newcomers due to the old school black screen and the requirement to step away from the mouse, but it is actually easy to navigate when you get going and it produces all of the output you need to interpret your Q sorts. There is the newer (and also free) KenQ that is receiving good reviews and has a more up-to-date web-based navigation, but I must confess I like my old time PQMethod. Details on all of the software and where to access these can be found on the Q methodology website.

Applications

Q methodology studies have been conducted with patient groups and the general public. In patient groups, the aim is often to understand their views on the type of care they receive or options for future care. Examples include the views of young people on the transition from paediatric to adult health care services and the views of dementia patients and their carers on good end of life care. The results of these types of Q studies have been used to inform the design of new interventions or to provide attributes for future preference elicitation studies.

We have also used Q methodology to investigate the views of the general public in a range of European countries on the principles that should underlie health care resource allocation as part of the EuroVaQ project. More recently, Q methodology has been used to identify societal views on the provision of life-extending treatments for people with a terminal illness.  This programme of work highlighted three viewpoints and a connected survey found that there was not one dominant viewpoint. This may help to explain why – after a number of preference elicitation studies in this area – we still cannot provide a definitive answer on whether an end of life premium exists. The survey mentioned in the end of life work refers to the Q2S (Q to survey) approach, which is a linked method to Q methodology… but that is for another blog post!

Credit