An empirical ethics study of the coherence of NICE technology appraisal policy and its implications for moral justification.

BACKGROUND: As the UK's main healthcare priority-setter, the National Institute for Health and Care Excellence (NICE) has good reason to want to demonstrate that its decisions are morally justified. In doing so, it has tended to rely on the moral plausibility of its principle of cost-effectiveness and the assertion that it has adopted a fair procedure. But neither approach provides wholly satisfactory grounds for morally defending NICE's decisions. In this study we adopt a complementary approach, based on the proposition that a priority-setter's claim to moral justification can be assessed, in part, based on the coherence of its approach and that the reliability of any such claim is undermined by the presence of dissonance within its moral system. This study is the first to empirically assess the coherence of NICE's formal approach and in doing so to generate evidence-based conclusions about the extent to which this approach is morally justified. METHODS: The study is grounded in the theory, methods and standards of empirical bioethics. Twenty NICE policy documents were coded to identify and classify the normative commitments contained within NICE technology appraisal policy as of 31 December 2021. Coherence was systematically assessed by attempting to bring these commitments into narrow reflective equilibrium (NRE) and by identifying sources of dissonance. FINDINGS: Much of NICE policy rests on coherent values that provide a strong foundation for morally justified decision-making. However, NICE's formal approach also contains several instances of dissonance which undermine coherence and prevent NRE from being fully established. Dissonance arises primarily from four sources: i) NICE's specification of the principle of cost-effectiveness; ii) its approach to prioritising the needs of particular groups; iii) its conception of reasonableness in the context of uncertainty, and iv) its concern for innovation as an independent value. CONCLUSION: At the time of analysis, the level of coherence across NICE policy provides reason to question the extent to which its formal approach to technology appraisal is morally justified. Some thoughts are offered on why, given these findings, NICE has been able to maintain its legitimacy as a healthcare priority-setter and on what could be done to enhance coherence.

The ethical canary: narrow reflective equilibrium as a source of moral justification in healthcare priority-setting.

Healthcare priority-setting institutions have good reason to want to demonstrate that their decisions are morally justified-and those who contribute to and use the health service have good reason to hope for the same. However, finding a moral basis on which to evaluate healthcare priority-setting is difficult. Substantive approaches are vulnerable to reasonable disagreement about the appropriate grounds for allocating resources, while procedural approaches may be indeterminate and insufficient to ensure a just distribution. In this paper, we set out a complementary, coherence-based approach to the evaluation of healthcare priority-setting. Drawing on Rawls, we argue that an institutional priority-setter's claim to moral justification can be assessed, in part, based on the extent to which its many normative commitments are mutually supportive and free from dissonance; that is, on the ability to establish narrow reflective equilibrium across the normative content of a priority-setter's policy and practice. While we do not suggest that the establishment of such equilibrium is sufficient for moral justification, we argue that failure to do so might-like the proverbial canary in the coalmine-act as a generalised warning that something is awry. We offer a theoretical argument in support of this view and briefly outline a practical method for systematically examining coherence across priority-setting policy and practice.

We need to talk about values: a proposed framework for the articulation of normative reasoning in health technology assessment.

It is acknowledged that health technology assessment (HTA) is an inherently value-based activity that makes use of normative reasoning alongside empirical evidence. But the language used to conceptualise and articulate HTA's normative aspects is demonstrably unnuanced, imprecise, and inconsistently employed, undermining transparency and preventing proper scrutiny of the rationales on which decisions are based. This paper - developed through a cross-disciplinary collaboration of 24 researchers with expertise in healthcare priority-setting - seeks to address this problem by offering a clear definition of key terms and distinguishing between the types of normative commitment invoked during HTA, thus providing a novel conceptual framework for the articulation of reasoning. Through application to a hypothetical case, it is illustrated how this framework can operate as a practical tool through which HTA practitioners and policymakers can enhance the transparency and coherence of their decision-making, while enabling others to hold them more easily to account. The framework is offered as a starting point for further discussion amongst those with a desire to enhance the legitimacy and fairness of HTA by facilitating practical public reasoning, in which decisions are made on behalf of the public, in public view, through a chain of reasoning that withstands ethical scrutiny.

The normative grounds for NICE decision-making: a narrative cross-disciplinary review of empirical studies.

The National Institute for Health and Care Excellence (NICE) is the UK's primary health care priority-setter, responsible for advising the National Health Service on its adoption of health technologies. The normative basis for NICE's advice has long been the subject of public and academic interest, but the existing literature does not include any comprehensive summary of the factors observed to have substantively shaped NICE's recommendations. The current review addresses this gap by bringing together 29 studies that have explored NICE decision-making from different disciplinary perspectives, using a range of quantitative and qualitative methods. It finds that although cost-effectiveness has historically played a central role in NICE decision-making, 10 other factors (uncertainty, budget impact, clinical need, innovation, rarity, age, cause of disease, wider societal impacts, stakeholder influence and process factors) are also demonstrably influential and interact with one another in ways that are not well understood. The review also highlights an over-representation in the literature of appraisals conducted prior to 2009, according to methods that have since been superseded. It suggests that this may present a misleading view of the importance of allocative efficiency to NICE's current approach and illustrates the need for further up-to-date research into the normative grounds for NICE's decisions.

The ethics of aggregation in cost-effectiveness analysis or, "on books, bookshelves, and budget impact".

In deciding how to allocate resources, healthcare priority-setters are increasingly paying attention to an intervention's budget impact alongside its cost-effectiveness. Some argue that approaches that use budget impact as a substantive consideration unfairly disadvantage individuals who belong to large patient groups. Others reject such claims of "numerical discrimination" on the grounds that consideration of the full budget impact of an intervention's adoption is necessary to properly estimate opportunity cost. This paper summarizes this debate and advances a new argument against modifying the cost-effectiveness threshold used for decision-making based on a technology's anticipated budget impact. In making this argument, the paper sets out how the apparent link between budget impact and opportunity cost is largely broken if the effects of a technology's adoption are disaggregated, while highlighting that the theoretical aggregation of effects during cost-effectiveness analysis likely only poorly reflects the operation of the health system in practice. As such, it identifies a need for healthcare priority-setters to be cognizant of the ethical implications associated with aggregating the effects of a technology's adoption for the purpose of decision-making. Throughout the paper, these arguments are illustrated with reference to a "bookshelf" analogy borrowed from previous work.

Does NICE apply the rule of rescue in its approach to highly specialised technologies?

The National Institute for Health and Care Excellence (NICE), the UK's main healthcare priority-setting body, recently reaffirmed a longstanding claim that in recommending technologies to the National Health Service it cannot apply the 'rule of rescue'. This paper explores this claim by identifying key characteristics of the rule and establishing to what extent these are also features of NICE's approach to evaluating ultra-orphan drugs through its highly specialised technologies (HST) programme. It argues that although NICE in all likelihood does not act because of the rule in prioritising these drugs, its actions in relation to HSTs are nevertheless in accordance with the rule and are not explained by the full articulation of any alternative set of rationales. That is, though NICE implies that its approach to HSTs is not motivated by the rule of rescue, it is not explicit about what else might justify this approach given NICE's general concern with overall population need and value for money. As such, given NICE's reliance on notions of procedural justice and its commitment to making the reasons for its priority-setting decisions public, the paper concludes that NICE's claim to reject the rule is unhelpful and that NICE does not currently meet its own definition of a fair and transparent decision-maker.

Justice, Transparency and the Guiding Principles of the UK's National Institute for Health and Care Excellence.

The National Institute for Health and Care Excellence (NICE) is the UK's primary healthcare priority-setting body, responsible for advising the National Health Service in England on which technologies to fund and which to reject. Until recently, the normative approach underlying this advice was described in a 2008 document entitled 'Social value judgements: Principles for the development of NICE guidance' (SVJ). In January 2020, however, NICE replaced SVJ with a new articulation of its guiding principles. Given the significant evolution of NICE's methods between 2008 and 2020, this study examines whether this new document ('Principles') offers a transparent account of NICE's current normative approach. It finds that it does not, deriving much of its content directly from SVJ and failing to fully acknowledge or explain how and why NICE's approach has since changed. In particular, Principles is found to offer a largely procedural account of NICE decision-making, despite evidence of the increasing reliance of NICE's methods on substantive decision-rules and 'modifiers' that cannot be justified in purely procedural terms. Thus, while Principles tells NICE's stakeholders much about how the organisation goes about the process of decision-making, it tells them little about the substantive grounds on which its decisions are now based. It is therefore argued that Principles does not offer a transparent account of NICE's normative approach (either alone, or alongside other documents) and that, given NICE's reliance on transparency as a requirement of procedural justice, NICE does not in this respect satisfy its own specification of a just decision-maker.

Exorcising the positivist ghost in the priority-setting machine: NICE and the demise of the 'social value judgement'.

The National Institute for Health and Care Excellence (NICE), the UK's primary health care priority-setting body, has traditionally described its decisions as being informed by 'social value judgements' about how resources should be allocated across society. This paper traces the intellectual history of this term and suggests that, in NICE's adoption of the idea of the 'social value judgement', we are hearing the echoes of welfare economics at a particular stage of its development, when logical positivism provided the basis for thinking about public policy choice. As such, it is argued that the term offers an overly simplistic conceptualisation of NICE's normative approach and contributes to a situation in which NICE finds itself without the necessary language fully and accurately to articulate its basis for decision-making. It is suggested that the notion of practical public reasoning, based on reflection about coherent principles of action, might provide a better characterisation of the enterprise in which NICE is, or hopes to be, engaged.

NICE and Fair? Health Technology Assessment Policy Under the UK's National Institute for Health and Care Excellence, 1999-2018.

The UK's National Institute for Health and Care Excellence (NICE) is responsible for conducting health technology assessment (HTA) on behalf of the National Health Service (NHS). In seeking to justify its recommendations to the NHS about which technologies to fund, NICE claims to adopt two complementary ethical frameworks, one procedural-accountability for reasonableness (AfR)-and one substantive-an 'ethics of opportunity costs' (EOC) that rests primarily on the notion of allocative efficiency. This study is the first to empirically examine normative changes to NICE's approach and to analyse whether these enhance or diminish the fairness of its decision-making, as judged against these frameworks. It finds that increasing formalisation of NICE's approach and a weakening of the burden of proof laid on technologies undergoing HTA have together undermined its commitment to EOC. This implies a loss of allocative efficiency and a shift in the balance of how the interests of different NHS users are served, in favour of those who benefit directly from NICE's recommendations. These changes also weaken NICE's commitment to AfR by diminishing the publicity of its decision-making and by encouraging the adoption of rationales that cannot easily be shown to meet the relevance condition. This signals a need for either substantial reform of NICE's approach, or more accurate communication of the ethical reasoning on which it is based. The study also highlights the need for further empirical work to explore the impact of these policy changes on NICE's practice of HTA and to better understand how and why they have come about.

Affordability and Non-Perfectionism in Moral Action.

One rationale policy-makers sometimes give for declining to fund a service or intervention is on the grounds that it would be 'unaffordable', which is to say, that the total cost of providing the service or intervention for all eligible recipients would exceed the budget limit. But does the mere fact that a service or intervention is unaffordable present a reason not to fund it? Thus far, the philosophical literature has remained largely silent on this issue. However, in this article, we consider this kind of thinking in depth. Albeit with certain important caveats, we argue that the use of affordability criteria in matters of public financing commits what Parfit might have called a 'mistake in moral mathematics'. First, it fails to abide by what we term a principle of 'non-perfectionism' in moral action: the mere fact that it is practically impossible for you to do all the good that you have reason to do does not present a reason not to do whatever good you can do. And second, when used as a means of arbitrating between which services to fund, affordability criteria can lead to a kind of 'numerical discrimination'. Various attendant issues around fairness and lotteries are also discussed.

Innovation as a value in healthcare priority-setting: the UK experience.

All healthcare systems operate with limited resources and therefore need to set priorities for allocating resources across a population. Trade-offs between maximising health and promoting health equity are inevitable in this process. In this paper, we use the UK's National Institute for Health and Care Excellence (NICE) as an example to examine how efforts to promote healthcare innovation in the priority-setting process can complicate these trade-offs. Drawing on NICE guidance, health technology assessment reports and relevant policy documents, we analyse under what conditions NICE recommends the National Health Service fund technologies of an "innovative nature", even when these technologies do not satisfy NICE's cost-effectiveness criteria. Our findings fail to assuage pre-existing concerns that NICE's approach to appraising innovative technologies curtails its goals to promote health and health equity. They also reveal a lack of transparency and accountability regarding NICE's treatment of innovative technologies, as well as raising additional concerns about equity. We conclude that further research needs to evaluate how NICE can promote health and health equity alongside healthcare innovation and draw some general lessons for healthcare priority-setting bodies like NICE.