The Social Value of a Qaly

August 15 2008

THE SOCIAL VALUE OF A QALY: COMMENTS TO THE FINAL REPORT FROM THE SVQ TEAM.

By senior researcher Erik Nord, The Norwegian Institute of Public Health, Oslo. [email protected]

General

The background for this paper is that I have been invited to a dissemination day on 4th September 2008 at the Wellcome Trust in London where the above project and a related one will be presented.

The following comments refer to that part of the SVQ-report which has to do with incorporating concerns for other aspects than QALY gains in decisions about priority setting in the NHS. I do not at this time address the issue of a monetary value of a QALY.

On the whole, the SVQ-report is a valuable contribution to clarifying the strength of concerns in the UK for other aspects than QALY gains.

My main comment is about an ethical concern in health care which the report only barely evokes. It is the egalitarian view that ‘people should be allowed to realize their potential for health and that the right to treatment should not depend so much on whether their maximum achievable level of functioning and/or length of life is large or moderate (or perhaps even modest)’. Judging from evidence in various countries - including the UK (Dolan and Cookson, 1998) – the concern may very well be of considerable importance and could seriously limit the role health care politicians would want to assign to cost-per-QALY ratios in priority setting across patient groups. I am not implying that the SVQ team is unfamiliar with this possible way of thinking. I am only saying that the issue for some reason has received very little attention in the project so far (I document this in detail below) and that this may be a major omission.

In addition I have a minor comment regarding terminology and some more substantial comments regarding the methods by which the research team tries to estimate societal concerns for age and severity.

Realisation of potential

The idea

Consider two patient groups with about the same level of functional impairment and suffering, i.e. the same score on a time trade-off based scale of quality of life (e.g. EQ-5D). For the sake of the argument I will use people with severe asthma and people in need of hip replacement as an example. While the latter group can be restored to very nearly normal functioning through surgery, the best one can do for the former group is to reduce their symptoms and improve their everyday functioning considerably by means of medication, but still leave them with a substantial handicap. Does it follow that a modern European welfare state with a national health service should be less willing to pay for asthma treatments than for hip replacements? Or is it possible that the two groups should be given equal priority on the grounds that they have equal entitlement to realise their health potential, which in both cases is substantial?

I stress that the realisation of potential view is not the same as saying that everybody has the right to the best possible treatment no matter how much it costs. The view is that at equal treatment costs, it should not be held against some groups that they happen to have lesser capacity to benefit than others (either because of the nature of their health problem combined with the status of medical technology in their disease area or due to characteristics of themselves) as long as also their benefit is substantial and significant to them. Clearly, the view constitutes a fundamentally different basis for priority setting in health care than does the QALY maximisation paradigm.

Some economists may respond that not to give priority to condition/treatment pairs with the better outcomes runs counter to people’s long term self interests behind a veil of ignorance (and is thus irrational). However, it is conceivable, an to some extent indicated in recent research, that the concern described above, which is one in terms of fairness, has a counterpart in the way in which patient groups with different health potentials personally value the best outcomes each of them can obtain (Nord et al, 2008).

The evidence

As far as I can understand, the realisation of potential view is not entirely alien to current NICE guidelines, in which absence of alternative treatment options is included in the list of possible arguments for stretching the willingness to pay for a QALY in a given area. One possible interpretation of this policy is that if highly effective treatment is not possible, then an inferior, best possible treatment gets additional value. Doing the best possible is the flip side of the coin of realising potential.

In the medical ethics literature, the realisation of potential view was argued two decades ago as one of the two most salient concerns for distributive fairness in health care (the other being concerns for the worse off), see for instance Daniels, 1985, 1993.

At about the same time, Norway – for once an early bird – established explicit guidelines for priority setting in its national health service (Norwegian Commission for Priority Setting in Health Care, 1987). There was broad agreement on two main ethical principles. One was that, given significant treatment effect (effect above a certain minimum), severity is the most important criterion for priority setting. The other was that everybody should have the same possibility to become as well as they can, i.e. realize their health potential. Maximising overall health gains when allocating resources across patient groups was a quite remote idea in these guidelines. In 1997 the guidelines were updated (Second Norwegian Commission for Priority Setting in Health Care, 1997). The new commission suggested that compared to the guidelines of 1987, ‘some’ more emphasis should be placed on cost-effectiveness considerations. While this was a notable move, it did not replace the basic thinking of the 1987 guidelines. In 2007, the Norwegian Director for Health expressed an explicit concern that conventional cost- effectiveness analysis favours those with a greater capacity to benefit and particularly is to the disadvantage of severely ill people with moderate potentials for improvement (Larsen, 2008).

Concerns for realising potentials seem to be strong also in Germany. In 2007, the Institute for Quality and Economizing in Health Care (IQWIG) commissioned an international expert panel of economists and medical doctors to outline a method for determining ceiling prices for new medical technologies. The panel suggested a method by which the cost-effectiveness of a technology may be judged relative to existing technologies in the therapeutical area in question (Caro et al, 2008). This was in line with a view held by IQWIG that current within- area performance may be a reasonable basis for determining the cost-effectiveness threshold which a new technology should be able to meet. Differences in cost-effectiveness performances across therapeutic areas and patient groups was thus viewed as quite acceptable by IQWIG. IQWIG’s position was recently endorsed in a public statement from the German Ministry of Health. There seems to be widespread and high level political rejection in Germany of economic evaluation methods that imply that patient groups in which treatment is highly effective and resource use therefore (cet.par.) highly cost-effective should have priority over patient groups where treatment outcomes are more moderate (although still substantial).

Apart from these clear manifestations of concerns for realising potentials in two North- European welfare states’ actual policy making, there are a number of preference studies in various countries that indicate fairly wide popular support for this kind of thinking.

In a preference study among 150 politically elected members of regional health boards in Norway, 72 % took the view of ‘equal access for people with different potentials’ rather than the benefit maximising view (Nord, 1993a, summarised in English in Nord, 1999). A similar result was obtained in a preference study including person trade-off questions in a convenience sample of staff at the Norwegian Institute of Public Health (Nord, 1993b).

In an Australian survey (Nord and Richardson et al, 1995), 53 % of a general population sample felt that among patients who are suffering equally, some priority should be given to those who can be helped most from treatment, while 47 % felt that those who can become a little better should have the same priority as those who can become much better. Altogether this looks like support for fairly light emphasis on capacity to benefit.

In a large qualitative study in the York Health Region in the UK, Dolan and Cookson (1998) observed reluctance to discriminate between groups with different expected treatment outcomes in terms of gained life years when the gain of the least benefiting group surpassed some 10 years. Their general conclusion was that ’50 years after the creation of the British National Health Service, people still seem to believe that there should be priority accorded to those in urgent need of medical attention. Whilst capacity to benefit does matter, it is a secondary consideration.’

Abellan-Perpinan and Pinto-Prades (1999) picked up on the Norwegian studies and came to the conclusion that in a Spanish convenience sample the size of the benefit was of importance, but less so than assumed in the conventional QALY approach. The result may be seen as similar to the ones in Australia and the UK.

In a study in the US, Ubel, Richardson and Pinto Prades (1999) found that subjects would give some priority to a program that restored people with one type of life threatening illness to full health compared to a program that saved the lives of people with another type of life threatening illness but left them in a state of paraplegia. On the other hand they attached equal importance to saving the lives of people with pre-existing paraplegia as compared to people in normal health.

In sum, the view that people have the same right to realise their potential for health whether this is big or small has been clearly argued by medical ethicists, is held quite strongly by politicians in both Germany and Norway and seems to be felt by a majority of people in countries where the issue has been studied empirically. Whether people in the UK feel the same way, is not clear, although the study by Dolan and Cookson suggests that they do. My point is that in the SVQ project so far, the issue has only barely been addressed. In the following I document this.

Absence of the issue in the SVQ report

First, there is in the report an introductory major reference to a preceding literature review by Dolan and colleagues (2005). The review briefly mentions some preference studies in which the importance people assign to effect size was examined (including some of the above mentioned references). While the review overall is useful, it is very brief on this particular issue, offers no assessment of the various studies cited and has no presentation of the underlying ethical and political reasoning (and policy documents) that could have led to a greater appreciation among health economists of why realisation of potential may be an important concern.

Second, in the SVQ report itself, the issue is not mentioned at all in the first wave of qualitative data collection, which was based on open ended discussions in focus groups. This is understandable, given the observation in the report itself than subjects had difficulties with coming up with thoughts about aspects beyond the most obvious ones (like quality of life, length of life and costs).

Third, in the second wave of focus groups, subjects were asked to rank a selection of issues according to how important they considered them to be for priority setting. Realisation of potential was not among the selected candidates. The closest one was ‘whether there is no other treatment available’. Interestingly, this was ranked as number two after severity, which altogether yields a picture of values not unlike the one drawn in Norway in 1987 (see above).

Fourth, the additional Q-sort study in the second wave had 46 statements about which subjects were asked to express agreement or disagreement. Most of these have to do with age, personal responsibility and having dependents, some with productivity and social class. A few have to do with severity (albeit somewhat indirectly, see next section). Only two or three statements have to do with the importance of effect size (31 and 33 and perhaps 41). Statement 31 allows subjects to disagree with QALY-maximisation, but not to indicate why. Statement 33 only says that ‘it’s important that people get something’, which is much less than stating the realisation of potential principle. Statement 41 may be said to allow subjects to downplay the importance of effect size indirectly in that it allows them to place ‘need’ above ‘anything else’, but the implication is not clear. Not one statement puts the realisation of potential issue directly and squarely to the subjects, for instance as follows: A.’People have a right to realise their potential for health. It should not be held against some groups that they happen to have a condition in which the treatment potential is smaller than in other groups as long as also their potential benefit is substantial and significant to them’. B. ‘Patient groups who can be restored to full health should be given priority over patient groups who can be helped significantly, but not restored to full health (all else equal). C. ‘Patient groups who can be given 20 more life years should have priority over patient groups who can be given 10 more life years (all else equal).’

Fifth, in the discrete choice study, two of the attributes refer to the size of the health gain (gain in LE and gain in QoL). Judging by the report, but there was no reference to the various levels of gain as the ‘maximum obtainable’, ie the result of realising the potential of the patients in question. Subjects were simply asked to compare gains of different size, for instance a QoL improvement of 20 % with one of 40 %. Presumably it makes a big difference for valuation if a 20 % gain (a) is the result of choosing a second best treatment option or (b) doing the best possible for that patient group. In my view, the phrasing of the pairwise comparisons is such that differences in effect size were made very clear while the potentially countervailing issue of realising potentials was absent in the stimulus. The conclusion that ‘increasing QALYs are always preferred’ (p.29) may therefore be incorrect if understood also as a description of the subjects’ preferences for priority setting among groups with different potentials for health.

The above point applies equally to the so called ‘matching’ study. The focus is on differences in effect sizes, with no mentioning of possible differences in potentials.

I conclude that while concerns for realising people’s potentials for health are clearly argued in the medical ethics literature, are manifest in official health policy in countries like Norway and Germany, and have much support in a number of studies of population preferences for priority setting - including the UK, the issue is virtually completely overlooked in the SVQ report. In my view this is a serious shortcoming. I believe the issue deserves to be addressed explicitly and in depth in future work. In the extreme, it could suggest a change of paradigm in NICE evaluations.

Other comments

Terminology

Although I am biased, I wonder why the person trade-off study is called ‘matching’ rather than ‘person trade-off’. It seems to me that the standard gamble, the time trade-off and the person trade-off are all matching techniques (equivalence techniques). One of them used to be called ‘equivalence in numbers (of people, as opposed to years or likelihood)’. Now the standard name is ‘person trade-off’. ‘Matching’ is less precise and less known.

Furthermore, it seems to me that in this study, ‘age weights’ refers to improvements in quality of life, not length of life. If this is correct, it should be made clear. Dealing with severity in the Q-sort study

I am not convinced that the statements in the Q-sort study really pinpoint the severity issue. Statement 36 is not bad, but I miss something like the following: ‘If a group of moderately disabled people and a group of severely disabled people both can be given an improvement which they appreciate equally much, the severely disabled group should have priority.’(Alternatively: ‘the two groups should have equal priority’).

Framing in diagrammatic questions

Ideally, discrete choices and person trade-off judgements should be based on ethical reflection and arguments. Take for instance concerns for severity. Ideally, subjects are first asked to think at a principled level, for instance by addressing statements of the kind I propose in the preceding paragraph. Having clarified their principled view, subjects are invited to express the strength of it in more precise terms. For instance: ‘Here are two programs that differ with respect to severity. You have stated that you think severity is important. Can you indicate how important? Program A deals with more severely ill people than does program B. If A helps 100 people, how many would B have to help for you to find the two programs equally worthy of funding?’

This is not what seems to have happened in the diagrammatic approach in the SVQ study, see page 24, second paragraph, as well as the diagrams. The whole focus seems to be on lines, areas and numbers, without any highlighting of what the underlying ethical issue of each diagram is, as though the task is a mathematical one rather than a philosophical, ethical and political one. In addition, I think one of the elements of information stands out visually, namely the area indicating effect size. It is a bit as if one of several pieces of information in a verbal list was in bold and the others not. Altogether, I am not convinced that responses to these diagrammatic stimuli capture subjects deeper feelings about the importance of age and severity. This might explain why the clear result on severity in the ranking study does not bear out in the discrete choice study and the results on severity are inconsistent across the two weighting studies. It might also explain why the researchers find that quality of life improvements are thought to be more valuable in young people than old people. That is going a lot further than saying that young people have stronger claims on life extension than old people.

In the concluding paragraph on severity on page 62 the deviating finding of Dolan and Tsuchyia in 2005 is referred to as ‘unexplained’ and related to the finding in the discrete choice study in the present report. A reference to an attempt at explaining that deviating finding would have been in place (Nord, 2006, see also the author’s reply in the same issue of Health Economics).

Note that this section on the diagrammatic method may be read as a general critique of main stream conjoint analysis (of which both the discrete choice diagrams and the person trade-off diagrams are special cases): Stimuli for introductory principled reflection are generally skipped and such reflection is thus likely to be forced to the background by explicit (sometimes complex) numerical and geometric stimuli. These concerns about the method of course also pertain to how I think future investigations into the issue of realisation of potential ought to be conducted.

The status of popular preferences

One of the findings in this study is that ordinary people are neither very reflective nor very articulate when it comes to discussing ethical issues in priority setting, cfr the report from the first wave of focus groups. This is a well known problem. Many non-economists, and particularly philosophers (e.g. Norman Daniels and Dan Hausman), are therefore sceptical too placing too much emphasis on preferences measurements in the general public. They want to see sound reasoning and arguments, both of which are mostly lacking in popular expressions of preferences on complex issues.

I believe that population preferences are highly relevant to decision making. But I also believe it is wrong to equate ‘social values’ with ‘popular preferences’. There are at least two other important sources for determining social value, both of which are associated with much more reflection than popular preferences. One is the medical ethics literature, where positions are taken on the basis of extremely careful argumentation and analysis. The other is documents from politically representative panels of knowledgeable and thoughtful people commissioned by authorities to give advice on complex ethical issues (like the Norwegian priority setting report from 1987). Arguably, there is at least as much to be learned about values for policy making in such documents as in measurements of popular preferences. Yet, in the literature lists in both the SVQ report and in the preceding review by Dolan et al (2005), medical ethics literature and political perspective documents are completely absent and thus seem to have been completely disregarded as potentially helpful indicators of ‘social values’. I believe this is an unfortunate tradition in health economics. I believe it could be useful for NICE if an official government commission of highly competent doctors, nurses, patients’ representatives, ethicists and economists suggested criteria and guidelines for valuing health gains, taking into account evidence on population preferences, but not only that. Bearing in mind existing official policy in nearby countries like Norway and Germany, and the earlier research by Dolan and Cookson in the UK, I suspect the result could be quite different from ‘QALY-maximisation with small adjustments for other concerns’.

References

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