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.
Weighing clinical evidence using patient preferences: an application of probabilistic multi-criteria decision analysis. PharmacoEconomics [PubMed] Published 10th November 2016
There are at least two ways in which preferences determine the allocation of health care resources (in a country with a HTA agency, at least). One of them we think about a lot; the (societal) valuation of health states as defined by a multi-attribute measure (like the EQ-5D). The other relates to patient preferences that determine whether or not a specific individual (and their physician) will choose to use a particular technology, given its expected clinical outcomes for that individual. A drug may very well make sense at the aggregate level but be a very bad choice for a particular individual when compared with alternatives. It’s right that this process should be deliberative and not solely driven by an algorithm, but it’s also important to maintain transparent and consistent decision making. Multi-criteria decision analysis (MCDA) has been proposed as a means of achieving this, and it can be used to take into account the uncertainty associated with clinical outcomes. In this study the authors present an approach that also incorporates random preference variation along with parameter uncertainty in both preferences and clinical evidence. The model defines a value function and estimates the impact of uncertainty using probabilistic Monte Carlo simulation, which in turn estimates the mean value of each possible treatment in the population. Treatments can therefore be ranked according to patients’ preferences, along with an estimate of the uncertainty associated with this ranking. To demonstrate the utility of the model it is applied to an example for the relative value of HAARTs for HIV, with parameters derived from clinical evaluations and stated preferences studies. It’s nice to see that the authors also provide their R script. One headline finding seems to be that this approach is likely to demonstrate just how much uncertainty is involved that might not previously have been given much attention. It could therefore help steer us towards more valuable research in the future. And it could be used to demonstrate that optimal decisions might change when all sources of uncertainty are considered. Clearly a potential application of this method is in the realm of personalised medicine, which is slowly but inevitably reaching beyond the confines of pharmacogenomics.
Communal sharing and the provision of low-volume high-cost health services: results of a survey. PharmacoEconomics – Open Published 4th November 2016
One of the distributional concerns we might have about the QALY-maximisation approach is its implications for people with rare diseases. Drugs for rare diseases are often expensive (because the marginal cost is likely to be higher) and therefore less cost-effective. There is mixed evidence about whether or not people exhibit a preference for redistributive allocation of QALY-creating resources according to rarity. Of course, the result you get from such studies is dependent on the question you ask. In order to ask the right question it’s important to understand the mechanisms by which people might prefer allocation of additional resources to services for rare diseases. One suggestion in the literature is the preservation of hope. This study presents another, based on the number of people sharing the cost. So imagine a population of 1000 people, and all those people share the cost of health care. For a rare disease, more people will share the cost of the treatment per person treated. So if 10 people have the disease, that’s 100 payers per recipient. If 100 people have the disease then it’s just 10 payers per recipient. The idea is that people prefer a situation in which more people share the cost, and on that basis prefer to allocate resources to rare diseases. A web-based survey was conducted in Australia in which 702 people were asked to divide a budget between a small patient group with a high-cost illness and a large patient group with a low-cost illness. There were also a set of questions in which respondents indicated the importance of 6 possible influences on their decisions. The findings show that people did choose to allocate more funds to the rarer disease, despite the reduced overall health gain. This suggests that people do have a preference for wider cost sharing, which could explain extra weight being given to rare diseases. I think it’s a good idea that deserves more research, but for me there are a few problems with the study. Much of the effect could be explained by people’s non-linear valuations of risk, as the scenario highlighted that the respondents themselves would be at risk of the disease. We also can’t clearly differentiate between an effect due to the rarity of the disease (and associated cost sharing) and an effect due to the severity of the disease.
The challenge of conditional reimbursement: stopping reimbursement can be more difficult than not starting in the first place! Value in Health Published 3rd November 2016
If anything’s going to make me read a paper, it’s an exclamation mark! Conditional reimbursement of technologies that are probably effective but probably not cost-effective can be conducted in a rational way in order to generate research findings and benefit social welfare in the long run. But that can only hold true if those technologies subsequently found (through more research) to be ineffective or too costly are then made unavailable. Otherwise conditional reimbursement agreements will do more harm than good. This study uses discrete choice experiments to compare public (n=1169) and potential policymaker (n=90) values associated with the removal of an available treatment compared with non-reimbursement of a new treatment. The results showed (in addition to some other common findings) that both the public and policymakers preferred reimbursement of an existing treatment over the reimbursement of a new treatment, and were willing to accept an ICER of more than €7,000 higher for an existing treatment. Though the DCE found it to be a significant determinant, 60% of policymakers reported that they thought that reimbursement status was unimportant, so there may be some cognitive dissonance going on there. The most obvious (and probably most likely) explanation for the observed preference for currently reimbursed treatments is loss aversion. But it could also be that people recognise real costs associated with ending reimbursement that are not reflected in either the QALY estimates or the costs to the health system. Whatever the explanation, HTA agencies need to bear this in mind when using conditional reimbursement agreements.
Head-to-head comparison of health-state values derived by a probabilistic choice model and scores on a visual analogue scale. The European Journal of Health Economics [PubMed] Published 2nd November 2016
I’ve always had a fondness for a good old VAS as a direct measure of health state (dare we say utility) values, despite the limitations of the approach. This study compares discrete choices for EQ-5D-5L states with VAS valuations – thus comparing indirect and direct health state valuations – in Canada, the USA, England and The Netherlands (n=1775). Each respondent had to make a forced choice between two EQ-5D-5L health states and then assess both states on a single VAS. Ten different pairs were completed by each respondent. The two different approaches correlated strongly within and across countries, as we might expect. And pairs of EQ-5D-5L states that were valued relatively low or high in the discrete choice model were also valued accordingly in the VAS. But the relationship between the two approaches was non-linear in that values differed more at the ends of the scale, with poor health states valued more differently in the choice model and good health states valued more differently on the VAS. This probably just reflects some of the biases observed in the use of VAS that are already well-documented, particularly context bias and end-state aversion. This study clearly suggests (though does not by itself prove) that discrete choice models are a better choice for health state valuation… but the VAS ain’t dead yet.