1. Why should patient/carer experience be treated as evidence?
This is the key question. On the face of it, the experience that patients have in living with a particular condition and with its (attempted) treatments is important in understanding: the nature of the condition; the impact it has on the lives of those with the condition and those around them; the potential for treatments to assist with the condition and the impact it has on life; and the reality of the impact of new treatments for the condition [19,20,21,22,23,24,25]. The experience of patients in these areas is a crucial resource for decision-makers on priority-setting committees if they are to properly understand the condition and the treatment.
Beyond the instrumental value of patient experience, the literature has not engaged with the questions of why and how these experiences should feature in the process as evidence alongside the other forms of evidence, such as that from scientific and economic analyses. This section lays out the justification for its inclusion.
The claim that patient experience should be gathered and presented as part of the evidence considered in the decision-making process depends on the arguments about the nature of the decision and the justification of the process. The decision-making process should be established in such a way that the best (and fairest) decisions can be made in difficult circumstances.
One might assume that the fairest way to distribute scarce resources is to hold a lottery since this gives each person an equal chance at receiving scarce resources. While each person is equally likely to have her needs met, it will quickly become apparent that the lottery does not meet other substantive values that are widely held: it does not account for how sick a person is, i.e. how great her need is, it does not consider how effective the treatments are or how much each one costs, some may even object that it does not consider who wins the lottery and that some features, like age, matter. The lottery thought experiment shows that there are substantive considerations, other values, that matter in addition to the fairness of the process by which winners and losers are chosen. However, while there may be general agreement on the sorts of substantive values that matter (need, effectiveness, etc.) there is not agreement on how much each one matters. The pragmatic approach to the theoretical positions of wanting both an equal process and taking into account disagreement on substantive principles is to propose a process that treats participants fairly but also allows for deliberation and debate on how to weight different kinds of values. Four key features of this argument justify a process that includes equal consideration and the use of evidence, including patient evidence, are as follows:
There is reasonable disagreement about what is the right decision for funding a treatment in a range of cases. This feature of the resource allocation context provides the grounding for the process: it is in the light of this disagreement that the process can be considered to be a fair method of making a decision.
The initial problem of making just decisions about healthcare resources—the problem of allocating these resources in the fairest way—is noticeably defined by a distinct kind of disagreement: people can reasonably disagree about what the right course of action is [26,27,28,29,30]. Some people will judge that the right decision is to fund a particular treatment because those who are likely to benefit from it are in desperate need of help. Others will think that the costs, combined with doubts about the likelihood of benefit, are such that resources should not be allocated to this treatment.
This disagreement is reasonable because it can and does occur between individuals who satisfy a number of important characteristics. Reasonable disagreement is disagreement between individuals who (i) understand that a decision needs to be made, (ii) understand that others are likely to disagree, (iii) understand that there are a range of relevant criteria to be considered and to which others may appeal in the defence of their view, (iv) are able to accept that others may value the criteria in different ways for a given decision and (v) recognise that the processes involved in coming to a decision centre on reasons and reasoning.
The problem of making decisions when there is reasonable disagreement is pervasive in modern, democratic societies that recognize that citizens will hold different views of what is morally right, and this is often the case for making determinations about shared or public goods, like healthcare. If people cannot agree on the right way to distribute healthcare resources, then a fair process leads to decisions that follow a procedural standard rather than applying a specific moral, substantive view to each one.
All people should be given an equal opportunity at having the treatments for their condition being funded, which is achieved by following a fair process and giving equal consideration of relevant criteria from available evidence in the decision-making process.
Because people can reasonably disagree about the way in which healthcare resources should be allocated and so whether particular treatments should be funded, a process which enables all parties to have a fair hearing for their cause is one plausible system for helping to ensure that justice is done. “Equal opportunity” here means that each person or patient group has equal access to the fair process, equal opportunity to present their case and evidence, and the case should be judged on its particular merits and on the basis of all the available evidence. Each case equally follows the process even if the same outcome does not result. This is different from a lottery that gives each person an equal chance, which raises other concerns discussed above, and instead emphasizes the procedural consideration of substantive values. Equal opportunity is connected to the idea, familiar from the work of John Rawls, of justice as fairness . It also underpins the accountability for reasonableness model develop by Daniels and Sabin [27, 28]. Whilst there may be other systems for ensuring justice in resource allocation, this is the one that has been adopted in the literature and by several healthcare committees, such as those in the UK. In our view it is also the most robust and defensible [26, 31,32,33,34,35].
An ‘equal opportunity at having their treatment funded’ is a claim about the content of the decision-making and how the decision-making process is constructed. It means that the various stakeholders’ perspectives and reasoning should be included in the decision-making process. This must include patients and carers with the condition in question and also those with an interest in the treatment or who will be responsible for its delivery. Importantly, in each case, an equal opportunity refers to what evidence and reasons are considered as a part of the decision-making process and how they are considered.
Equal consideration and so equal opportunity requires that all relevant reasons are considered fully and in the light of all of the relevant evidence.
Giving and considering reasons for and against funding is the key element of the fair process [27,28,29, 36,37,38]. A decision is made reasonable by the way it is justified, i.e. the reasons that are given for it. Part of this justification will depend on the evidence that supports the reason for a decision. An articulation of why a decision has been made should include reference to the evidence that supports that reason. This justification may also refer to other considerations and evidence that were not strong enough to support the alternative decision. A process like this is similar to a judge hearing a case: if the judge were pre-decided on the facts and outcome, then the fair legal process would be upended. A fair process relies on the openness of reasoning and consideration of all available evidence. Courts limit what evidence is admissible, which is similar to the concept of considering relevant, appropriate evidence, including patient experience as evidence: in Part B, we discuss the standards for patient evidence and suggest the methodological rigour for it such that it can be included in decision making.
Ultimately there are only two sources of genuine disagreement within a system that is characterised by reasonable disagreement.Footnote 3 First, people may disagree about the value of a particular consideration—about how much weight it should have in this particular case. So, individuals may disagree about how important cost-effectiveness is in a particular case, particularly when compared to patients’ need. Second, people may disagree about the strength of the evidence in support of a particular consideration—about the degree to which the evidence shows that a consideration is in play. So individuals can disagree about whether we have good evidence of patients’ need or of the clinical effectiveness of a treatment. Both will clearly affect the reasoning behind the decision and the decision itself.Footnote 4
Both of these sources of disagreement are intractable in this context. The process is not designed to resolve this problem but to enable a fair decision in the context of it.
If all relevant reasons for and against a particular funding decision are to be considered fully and fairly, a full range of evidence in support of those reasons must be also considered.
Patient and carer experience of the nature of the condition, its effects on their lives, and the effects of the treatment is relevant to the decision-making and so should be formally included within it. This experience, like all other forms of evidence, should feature in the process as evidence in support of the criteria used in the process. Just as health economic evidence about costs and clinical effectiveness can support reasoning about cost-effectiveness, so too, evidence about what it is like to live with a condition can support reasoning about need: the nature of the condition, the effect of the condition on lives of sufferers and their carers, and the way in which the treatment might play a role in their lives. In short, this experience can be important evidence for decision-making and, given our interest for reasons of fairness in the inclusion of the full range of evidence, the experiences of patients and carers should be included.
2. How should patient/carer experiences as evidence, function in decision-making?
It is one thing to justify that these experiences ought to be a part of the decision-making process as evidence but it is another to give an account of how, in general terms, this evidence might play a role in those decisions.
The patient and public involvement literature is replete with examples of the way information provided by patients can bring context and depth to other sources of data collected in clinical research studies. Such context and depth is seen to provide a fuller account of these alternative data sets and to assist in maximising the relevance of such data sets when translated from ‘bench to bedside’. However, this literature invariably fails to make reference either to the role that this added value might play in the actual processes of decision-making or how patient experiences more generally (and not about clinical research) should feature in funding decisions . In developing the argument in the section above, we have relied on the idea that there is a range of relevant criteria that should be taken into consideration in the process of making fair resource allocation decisions. These criteria represent the range of ethically relevant reasons that decision-makers may have for deciding to fund or not fund a particular treatment [27,28,29, 36, 38]. For example, clinical effectiveness clearly matters in the decision about funding; if a treatment had no effect, this would arguably give decision-makers an overwhelming reason not to fund it . Cost-effectiveness, as a measure of value for money, is another criterion that we can readily understand to be a relevant reason for or against funding. If a treatment is not cost-effective, i.e. not good value for money, it is reasonable not to fund. Finally, it is easy to see that patient need is a criterion that is relevant to the decision: if patients are not in need, say because the symptom of the condition ameliorated by the treatment is of little concern, there would be little reason to fund the treatment. Of course in cases where the patients’ need is extreme, this need provides a very strong reason to fund a treatment that promises to meet it.
These three examples illustrate the way in which the criteria function to provide reasons for particular decisions. Clearly in actual cases the criteria and so the reasons pull in different directions: perhaps most often, patient need gives decision-makers reason to fund but cost-effectiveness provides a reason not to fund. The process described above requires that each of the criteria are treated and considered equally. Most importantly, it means that decision-makers should not approach the decision-making task with a prejudged value attached to any of the criteria. They should not be predisposed to value one more highly than another: to do so would be to fail to treat all stakeholders equally in the decision-making process. We assume the equal stance of stakeholders since these decisions occur in the context of allocating healthcare resources to those who are participants in the system, either as insurance-premium or tax payers.
Evidence functions in a definite way to provide support for reasons. So evidence of patient need provides a reason to fund a treatment whereas evidence of a lack of need provides a reason not to fund. Similarly, evidence of clinical effectiveness provides a reason to fund whereas evidence of no effect provides a reason not to fund. In each of these cases the evidence does not necessarily provide an over-riding reason—what gives us the strongest or an over-riding reason will depend on the other reasons, their relative strength, and the judgement of the decision-maker. Also, clearly, lack of evidence gives no reason either way.Footnote 5
So far the argument has shown that fairness requires equal opportunity and that equal opportunity requires equal consideration of relevant criteria. Equal consideration of relevant criteria requires attention to the full range of reasons for and against funding a treatment. Proper attention to all these reasons is possible only when adequate evidence is provided in support of each.
In the examples above, we have mentioned only patient need, clinical effectiveness and cost-effectiveness as relevant reasons. As we will see below, there are a number of others that should be included. We will also discuss how the decision-making process and criteria used function as a range of relevant reasons that ought to be considered.
Which kinds of criteria are those for which patient experience can provide evidence?
When making decisions about new treatments, committees use criteria such as the nature of the condition, the impact of the new treatment, the cost and cost-effectiveness of the treatment, and the treatment’s impact beyond direct health benefits.Footnote 6
The idea is that each of these criteria is to be considered by the committee in order to make a decision about funding. In this way, these criteria function as the relevant considerations that play a central role in making the process of decision-making a fair one—it is because these criteria capture the possible range of relevant reasons that the process is able to be fair and procedurally just. Following from the arguments above then, evidence which supports each of these criteria is also crucial in making the process a fair one.
It is important, therefore, to articulate clearly how accounts of patient and carer experience connect to the decision criteria. There are three ways in which patient and carer experience can function as evidence in support of these criteria—in each case the experience of patients and carers is importantly placed to support or weaken the role the criteria play in the decision-making reasoning. Three of the six criteria are most relevant here: (a) the nature of the condition, (b) the impact of the treatment, and (c) the impact of the treatment beyond direct health benefits. The other criteria depend heavily on knowledge of the costs associated with providing the treatment. Patients and carers are not well placed to contribute to these criteria.
The nature of the condition and its effects on the lives of patients and their carers.
As we have suggested, patients and carers can provide important evidence about the needs that they experience living with the condition [21, 41, 42]. The evidence that they can provide about the course of their lives gives substance to the decision-makers’ understanding of the nature of the condition. This criterion can then be supported in reasoning about the decision: ‘Because we now know more about what it is like to live with this condition, we can see more clearly the nature of the need that these patients have.’ More specifically, patient evidence can provide accounts of:
the lives of patients, family and carers.
what is of value in those respective lives.
the perceived lack/deficiencies in the lives of patients, family and carers.
the ways in which these deficiencies could be met (articulated in terms that do not rely directly on the effectiveness of the treatment).
In each case the evidence provided is able to support reasoning about the needs of patients and carers, which informs a decision.
Patients’ and carers’ experience of the condition and its effects on their lives means they can contextualise the clinical evidence that is provided to the committee.
Patients’ and carers’ knowledge and experience of the condition enable them to provide comment on the significance and relevance of the outcome measures used in research, the relevance of the research to the lives of patients with the condition, and the relevance of both clinical research and quality of life assessments. In this way patients’ and carers’ experiences provide evidence about the value of the research that has been conducted on the treatment under consideration [22, 43,44,45,46,47,48,49,50]. Patients’ and carers’ experience of the condition and its effects on their lives means they can contextualise the purported effects of the treatment.
An account of the experience of the treatment and its affects on the lives of patients and carers adds important context to the findings of clinical research [22, 46, 47, 50]. These are properly understood as ‘raw’ experiences that do not make claims attributing causal responsibility but which can sit alongside clinical effectiveness data. Qualitative evidence from patient experience cannot prove effectiveness, but it can give context and inform feasibility and acceptability of clinical research. For example, in a case where there is strong evidence of clinical effect, patients’ experience of the treatment may strengthen the value of that evidence by adding qualitative evidence around the effects documented in the research. Conversely, in the same case, patients’ experience may undermine actual value of the demonstrated clinical effect.