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.
Analysis of clinical benefit, harms, and cost-effectiveness of screening women for abdominal aortic aneurysm. The Lancet [PubMed] Published 26th July 2018
This study is an excellent example of the power and flexibility of decision models to help inform decisions on screening policies.
In many countries, screening for abdominal aortic aneurysm is offered to older men but not to women. This is because screening was found to be beneficial and cost-effective, based on evidence from RCTs in older men. In contrast, there is no direct evidence for women. To inform this question, the study team developed a decision model to simulate the benefits and costs of screening women.
This study has many fascinating features. Not only does it simulate the outcomes of expanding the current UK screening policy for men to include women, but also of other policies with different age parameters, diagnostic thresholds and treatment thresholds.
Curiously, the most cost-effective policy for women is not the current UK policy for men. This shows the importance of including the full range of options in the evaluation, rather than just what is done now. Unfortunately, the paper is sparse on detail on how the various policies were devised and if other more cost-effective policies may have been left out.
The key cost-effectiveness driver is the probability of having the disease and its presentation (i.e. the distribution of the aortic diameter), which is quite frequent in cost-effectiveness analysis of diagnostic tests. Neither of these parameters requires an RCT to be estimated. This means that, in principle, we could reduce the uncertainty on which policy to fund by conducting a study on the prevalence of the disease, rather than an RCT on whether a specific policy works.
An exciting aspect is that treatment itself could be better targeted, in particular, that lowering the threshold for treatment could reduce non-intervention rates and operative mortality. The implication is that there may be scope to improve the cost-effectiveness of management, which in turn will leave greater scope for investment in screening. Could this be the next question to be tackled by this remarkable model?
Establishing the value of diagnostic and prognostic tests in health technology assessment. Medical Decision Making [PubMed] Published 13th March 2018
Keeping on the topic of the cost-effectiveness of screening and diagnostic tests, this is a paper on how to evaluate tests in a manner consistent with health technology assessment principles. This paper has been around for a few months, but it’s only now that I’ve had the chance to give it the careful read that such a well thought out paper deserves.
Marta Soares and colleagues lay out an approach to determine the most cost-effective way to use diagnostic and prognostic tests. They start by explaining that the value of the test is mostly in informing better management decisions. This means that the cost-effectiveness of testing necessarily depends on the cost-effectiveness of management.
The paper also spells out that the cost-effectiveness of testing depends on the prevalence of the disease, as we saw in the paper above on screening for abdominal aortic aneurysm. Clearly, the cost-effectiveness of testing depends on the accuracy of the test.
Importantly, the paper highlights that the evaluation should compare all possible ways of using the test. A decision problem with 1 test and 1 treatment yields 6 strategies, of which 3 are relevant: no test and treat all; no test and treat none; test and treat if positive. If the reference test is added, another 3 strategies need to be considered. This shows how complex a cost-effectiveness analysis of a test can quickly become! In my paper with Marta and others, for example, we ended up with 383 testing strategies.
The discussion is excellent, particularly about the limitations of end-to-end studies (which compare testing strategies in terms of their end outcomes e.g. health). End-to-end studies can only compare a limited subset of testing strategies and may not allow for the modelling of the outcomes of strategies beyond those compared in the study. Furthermore, end-to-end studies are likely to be inefficient given the large sample sizes and long follow-up required to detect differences in outcomes. I wholeheartedly agree that primary studies should focus on the prevalence of the disease and the accuracy of the test, leaving the evaluation of the best way to use the test to decision modelling.
And for my third paper for the week, something completely different. But so worth reading! Charles Manski provides an overview of his work on how to use the available evidence to make decisions under uncertainty. It is accompanied by comments from Karl Claxton, Emma McIntosh, and Anirban Basu, together with Manski’s response. The set is a superb read and great food for thought.
Manski starts with the premise that we make decisions about which course of action to take without having full information about what is best; i.e. under uncertainty. This is uncontroversial and well accepted, ever since Arrow’s seminal paper.
Less consensual is Manski’s view that clinicians’ decisions for individual patients may be better than the recommendations of guidelines to the ‘average’ patient because clinicians can take into account more information about the specific individual patient. I would contend that it is unrealistic to expect that clinicians keep pace with new knowledge in medicine given how fast and how much it is generated. Furthermore, clinicians, like all other people, are unlikely to be fully rational in their decision-making process.
Most fascinating was Section 6 on decision theory under uncertainty. Manski focussed on the minimax-regret criterion. I had not heard about these approaches before, so Manski’s explanations were quite the eye-opener.
Manksi concludes by recommending that central health care planners take a portfolio approach to their guidelines (adaptive diversification), coupled with the minimax criterion to update the guidelines as more information emerges (adaptive minimax-regret). Whether the minimax-regret criterion is the best is a question that I will leave to better brains than mine. A more immediate question is how feasible it is to implement this adaptive diversification, particularly in instituting a process in that data are systematically collected and analysed to update the guideline. In his response, Manski suggests that specialists in decision analysis should become members of the multidisciplinary clinical team and to teach decision analysis in Medicine courses. This resonates with my own view that we need to do better in helping people using information to make better decisions.