EXECUTIVE SUMMARY

  Increasing challenges to NICE decisions may be understood as instances of the "legitimacy problem" which arises when access to healthcare resources is explicitly restricted. NICE has sought to address this problem through commitment to due process and, in respect of the "social value" dimension of its work, through development of deliberative mechanisms. However, there remains a need to engage in a comprehensive debate about rationing in the NHS in order to secure public acceptance of the authority of limit-setting bodies such as NICE.

  1.  The author is Senior Lecturer in the School of Law at the University of Bristol. He has published a number of articles on the National Institute for Health and Clinical Excellence in scholarly journals in the UK and worldwide and has written more widely on the relationship between the rationing of healthcare resources and public law.

  2.  This memorandum will seek to address the first question in the Committee's terms of reference: that is, "why NICE's decisions are increasingly being challenged" and will seek to outline proposals to diminish the frequency of such challenges. It also pertains to the issue of why public confidence in the Institute may be lacking.

  3.  The controversy which has been generated by NICE's work—especially (but not exclusively) its technology appraisal role—may in large part be traced to the perception that the Institute functions to "ration" care within the NHS. This view is, in some respects, misleading. Strictly speaking, the remit of the Institute is to consider clinical and cost-effectiveness of treatments and services and not to make judgements on affordability, the latter remaining the responsibility of government. Moreover, it has been demonstrated that technology appraisals have resulted in an additional cumulative cost of £800 million p.a, or approximately 1% of all expenditure upon the NHS.[172]

  4.  Nonetheless, as observed by the Committee in its previous report on NICE, the imposition of an obligation of mandatory funding attached to technology appraisal guidance has resulted in a blurring of the distinction between cost-effectiveness and affordability, such that the Institute may be regarded as "essentially making decisions about how money is spent in the NHS".[173] This concatenation of the two concepts is exacerbated by a lack of public understanding of issues of health economics, such that the public regard the application of health economic metrics (such as QALYs) by NICE as being indiscriminately "about money". The confusion also creates a space within which patient groups and drug companies may seek to advance their interests through the media and other channels. Here, invocation of the negative discourse of "rationing" serves as a rallying cry for the construction of a community of opposition to the NICE preliminary or final appraisal.[174] The view that NICE is a "rationing" body is accordingly widespread.[175]

  5.  The perceived status of NICE as a "rationing" body renders it open to contestation on grounds of legitimacy. In understanding this issue, the work of Norman Daniels and James Sabin is highly instructive.[176] These authors have identified a "legitimacy problem", which may be defined by means of the question: why should patients or clinicians accept the authority of a particular priority-setting body to make moral decisions which limit access to healthcare and thus adversely affect individual well-being? This problem arises because there is no consensus as to the ethical principles which might underpin decisions which function to restrict access to healthcare. In view of this moral disagreement, "suspicion, distrust and even resistance [will] often greet efforts to set limits on access to medical services".[177] Hence, a stakeholder who is disappointed at the outcome of a NICE appraisal will invoke an ethical principle (such as the "rule of rescue") which conflicts with that which is applied by NICE (which may often exhibit utilitarian characteristics). The incommensurability of these positions is likely to be further exacerbated by media coverage, with particular emphasis being placed upon the notion of "putting a price on life" as human interest collides with "bureaucratic" decision-making. The conflict may be played out in a variety of fora, including the Institute's own internal appeals process, the political arena, or by means of legal challenge.

  6.  The "legitimacy problem" has become acute because of the shift from implicit forms of rationing, in which allocative decisions were concealed behind a veil of clinical judgement, to explicit strategies, in which the financial implications of provision of particular services and treatments to a local or national population are visibly exposed to public view. The work of NICE in conducting technology appraisals is an illustration of this broader trend. The shift to explicit allocative strategies may be comprehended as an attempt by government to exercise more systematic control over healthcare expenditure, grounded in a commitment to deployment of the techniques of "evidence-based medicine". This is intended to eliminate inefficacious and inefficient treatments and to reduce arbitrary variations (especially of a geographical nature) in access to services. However, it is widely acknowledged that explicitness generates instability both at an individual level and, more broadly, within the health system, as a consequence of the "ability of small groups to evoke public sympathy and support in contesting government decision-making . . . those who care deeply but are denied access will inevitably challenge the explicit judgement through the mass media and other ways, undermining support for purchasing decisions".[178] According to the "legitimacy problem" thesis, this instability will manifest itself in a challenge to the moral authority of a body like NICE to make decisions which, in effect, limit access to care. The growing frequency of these challenges may simply be explained by the increasing visibility of the Institute and public and media awareness of the implications of its decisions, especially as it appraises treatments for a variety of diseases.

  7.  The "legitimacy problem" thesis enables us not only to understand why decisions reached by NICE are likely to be subject to regular challenge, but also offers a prescription for addressing the problem. Straightforwardly, NICE needs to make a convincing claim to legitimacy in order to minimise the conflict which its work will inevitably generate.

  8.  As the author has argued elsewhere,[179] analysis of the legitimacy of an administrative institution will tend to make reference to a limited and agreed set of values, comprising legislative mandate, accountability and control, efficiency, expertise and due process. Claims by NICE to the realisation of the first three values are likely to be unpersuasive. The vesting of wide discretionary powers in the Institute makes it difficult to argue that it is acting as a mere "transmission belt" for instructions provided by elected representatives in Parliament. Similarly, the relative independence of the Institute from direct ministerial control renders accountability mechanisms indistinct and attenuated. As regards efficiency, the consideration of the cost-effectiveness of treatments and services is central to the Institute's work. But it is clear that this, in itself, is insufficient to ground legitimacy in view of the absence of a societal consensus that utilitarian principles are the correct basis for judgements on resource allocation in healthcare.

  9.  At first sight, the claim to legitimacy through expertise would appear to be the Institute's strongest suit. In particular, those sitting upon the Technology Appraisal Committee, seen as the Institute's key decision-making body, are expected to draw upon their experience and judgement in reaching conclusions as to whether a technology is to be recommended for use upon the NHS. The composition of this Committee, with membership drawn from the NHS, patient and carer organisations, health-related academic disciplines and the pharmaceutical and medical device industries, attests to a commitment to a technocratic form of decision-making. This is underlined by the assertion of the Institute's Chairman and Chief Executive that "the scientific value judgements made by NICE remain, ultimately, those developed and enunciated by the knowledge, experience and expertise of the board members and its independent advisory bodies".[180] Further, the methodology adopted by the Committee, which is based upon the techniques of health technology assessment, and the consequent dominance of the "technical" discourses of health economics and biomedicine within the Committee's decisional processes, support the claim to legitimacy through the exercise of expert judgement.

  10.  However, as the Institute itself recognises, its claim to legitimacy through expertise in respect of "scientific value judgements" does not, in itself, provide a basis for the assertion of moral authority with regard to the dimension of its work which involves "social value judgements", defined as those which "relate to society rather than to basic or clinical science: they take account of the ethical principles, culture and aspirations that should underpin the nature and extent of the care provided by the NHS".[181] If, as argued previously, NICE is engaged in rationing of healthcare resources (or perceived as such), it is readily apparent that its decision-making will carry such a social value component since, as has been noted, priority-setting is an "inescapably political process".[182] The consequence is that a claim to legitimacy in respect of this aspect of the Institute's work must make reference to a value other than exercise of technical expertise.

  11.  It is therefore important to consider the extent to which NICE can successfully claim legitimacy on the basis of its compliance with principles of due process. Here, the framework of "accountability for reasonableness" which is developed in the work of Daniels and Sabin offers a well-regarded model for the attainment of legitimacy through procedural justice within this public policy context. The model establishes four conditions with which bodies making decisions which have the effect of limiting access to healthcare resources should comply. These are:

(a)  publicity: decisions and their rationales should be publicly accessible;

(b)  relevance: rationales should be based upon evidence, appeals and principles which fair-minded persons can accept as relevant to the problem of meeting the varied health needs of the population under resource constraints;

(c)  revision and appeals: opportunities must exist for revision and improvement of decisions in light of new evidence and arguments, and there must be mechanisms for challenge to decisions;

(d)  enforcement: voluntary or public regulation of the process to ensure that the preceding conditions are met.

  12.  Significantly, NICE has committed itself to a process of decision-making which seeks to comply with "accountability for reasonableness", and has been praised for its success in this regard.[183] Why, then, are the Institute's decisions increasingly subject to challenge? One answer might be that, in the context of allocative decision-making in healthcare, the "due process" claim to legitimacy (as captured in the "accountability for reasonableness" model) is also problematic. It is certainly the case that the Daniels and Sabin thesis has been subject to criticism,[184] but as NICE has acknowledged, it is not clear that any viable substitute theory exists.[185] In the light of this conclusion, it might be tempting to label the "legitimacy problem" as too intractable in the healthcare context, and to simply abandon any attempt at resolution. However, this would surely be an unsatisfactory response.

  13.  An alternative would be to argue that, notwithstanding the attempts made to give effect to "accountability for reasonableness", NICE decision-making remains deficient when set alongside the Daniels and Sabin model. In this regard, it is important to appreciate that the four conditions outlined above are not intended to serve as ends in themselves. Rather, they perform an "educative" function in building public understanding as to the need for the rationing of healthcare resources and the criteria which should underpin such decision-making. "Social learning" of this nature may in itself engender legitimacy, as the provision of reasons for decisions builds public confidence in, and acceptance of, the decision-making process. However, Daniels and Sabin's central claim is that compliance with the conditions of "accountability for reasonableness" acts as "connective tissue" to democracy more broadly, in so far as enhanced public understanding enriches debate and empowers the public to deliberate in a more comprehensive, informed and focused manner upon the rationing of healthcare within a range of democratic institutions and processes.[186] In this manner, the "accountability for reasonableness" framework feeds into theories of deliberative democracy, which contend that a process of communication and reasoned argumentation among citizens who are free from coercion and self-interest can contribute to the legitimacy of decisions made under conditions of scarcity, as is the case within health systems.

  14.  Once again, NICE has recognised the applicability of this theoretical work to its functions. It has sought to give practical realisation to deliberation by establishing the Citizens" Council—described in an internal document as "a unique experiment in deliberative democracy for the NHS and seemingly for almost any healthcare system in the world"—to assist in identifying the social values on questions of rationing which are held by the British public.[187] The Council possesses a number of deliberative characteristics, notably its composition as a representative cross-section of the British population, and its mode of operation, which centres upon the hearing and weighing of evidence and debate and discussion within plenary and small group sessions.

  15.  However, a recent evaluation of the Council has reached a cautious conclusion as to its deliberative qualities.[188] Moreover, even if the Council is regarded as deliberative in character, it is arguable that its role within the Institute's decision-making processes is insufficient to provide a basis for the legitimacy of the social value dimension of NICE"s work. It meets only twice a year for six days in total and the topics which it discusses are formulated by the Institute. Most crucially, it has no direct input into the technology appraisal process and its recommendations are not binding. It is therefore more accurate to view the Citizens' Council as fulfilling an informational role: it "feeds data" (in this case on public values) to the Institute, but it is the experts on the NICE board and advisory bodies who are the ultimate decision-makers. To this extent, the Institute's claim to legitimacy through its commitment to due process of a deliberative type is, it is submitted, unpersuasive.

  16.  An obvious means of addressing this deficiency in legitimacy, and hence of rendering NICE"s decision-making more widely acceptable, would be to strengthen the "join" of the Citizens' Council to the main technology appraisal work of the Institute. This could be achieved by giving the Council a standing "consultee" status on all technology appraisals, thereby enabling it to submit evidence directly upon the social value implications of recommending, or restricting the availability of, given technologies. A reform of this nature would, however, entail far greater demands on the time of Council members, especially if the valuable work which the Council currently undertakes on broad ethical topics, such as the relevance of age to NICE decisions on availability of treatments and the role of the "rule of rescue", were to be continued. For this reason, it might only be feasible if membership of the Council were to be increased to ensure a pool of available deliberants whenever required which would, in turn, carry resource implications. Moreover, more direct involvement of the Council would be likely to increase the length of the technology appraisal process, which is already a source of significant criticism of the Institute. Finally, and perhaps most crucially, even the enhanced role for the Council outlined in this paragraph might be insufficient to stem challenges to legitimacy, since the ultimate decision on access to treatments would continue to reside with those professing technical expertise. This remains technocratic decision-making, not deliberative democracy.

  17.  A more radical solution is therefore needed. If, as argued here, a process of public deliberation is the key to resolving any problem of legitimacy from which NICE suffers, and thus minimising the prospect of challenges to its decisions, it would appear to be imperative to engage in a full public debate upon the scope of coverage which can appropriately and affordably be provided by the NHS. Such a debate will foster public understanding of the inevitability of limitations on access to healthcare and of the complexity of the ethical choices which must be made. Although consensus is likely to remain elusive in view of the persistence of moral disagreement in this field, the greater awareness of the need for rationing and appreciation of the rationality and weight of countervailing ethical principles which should emerge from the process of deliberation will serve to engender broader acceptance of decision-making of the type in which NICE is engaged.

  18.  The work of NICE has served to place questions of the rationing of healthcare more firmly on the public agenda, and in that respect has prepared the ground for evolution of such a debate. Yet there remains an unwillingness to take the necessary further step of acknowledging the true nature of the task which it undertakes. This is reflected in NICE's disavowal of any role in respect of affordability—notwithstanding that the practical impact of a negative appraisal is almost always to restrict or exclude access to a particular treatment—and, more broadly, in its rejection of the term "rationing" to describe its work. In the latter respect, the Institute is merely following in the tradition of numerous politicians over the course of several decades, who have sought to diminish the inevitable electoral unpopularity which would attend deployment of the word, preferring instead the more neutral (and rational) terminology of "priority-setting".

  19.  Ultimately, it is the function of government, not of NICE, to initiate such a public debate (albeit that, once such a debate has commenced, the Institute is in a unique position to contribute both from a scientific and ethical perspective). However, an opportunity may exist in the context of current calls for the establishment of a constitution to enshrine the core values of the NHS.[189] Any debate upon such a document must inevitably engage with the issue of the comprehensiveness of the service which is provided. Of course, embarking upon an open and honest public debate upon the need for and criteria which should underpin the rationing of healthcare may cause short-term pain for the government in the form of criticism that it is "putting money before lives". Nevertheless, this author would contend that this is a pill worth swallowing if the longer-term consequence is to secure the legitimacy of bodies such as NICE which must play a central role in ensuring the affordability, as well as the efficacy, of publicly-funded healthcare services.

Dr Keith Syrett

University of Bristol

March 2007

173   Health Committee, National Institute for Clinical Excellence, Second Report HC 515-I (2001-02) at para.106. Back

174   See eg Pfizer Press Release, "Sad day for Patients with Alzheimer's Disease" (11 October 2006), available at http://www.pfizer.co.uk/template2.asp?pageid=379. Back

175   See eg the Daily Mail's response to the Press Notice announcing this inquiry: "Drug rationing watchdog put in the dock by MPs" (3 February 2007); and further "NHS must pay for fat children to get surgery", The Observer (19 November 2006), "Find the Balance", The Times (27 May 2006). For academic analyses along similar lines, see R. Smith, "The Failings of NICE" (2000) 321 BMJ 1363; A. Maynard, K. Bloor and N. Freemantle, "Challenges for the National Institute for Clinical Excellence" (2004) 329 BMJ 227. Back

176   See especially Setting Limits Fairly (New York: OUP, 2002). Back

177   N. Daniels, "Accountability for reasonableness in private and public health insurance" in A. Coulter and C. Ham (eds.), The Global Challenge of Health Care Rationing (Buckingham: Open University Press, 2000) at 89. Back

178   D. Mechanic, "Dilemmas in rationing health care services: the case for implicit rationing" (1995) 310 BMJ 1655 at 1658. Back

179   K. Syrett, "NICE Work? Rationing, Review and the "Legitimacy Problem in the new NHS" (2002) 10 Medical Law Review 1. Back

180   M. Rawlins and A. Culyer, "National Institute for Clinical Excellence and its Value Judgements" (2004) 329 BMJ 224 at 226. Back

181   NICE, Social Value Judgements: Principles for the Development of NICE Guidance (London: NICE, 2005) at para.1.1 Back

182   R. Klein, "Puzzling Out Priorities" (1998) 317 BMJ 959 at 959. Back

183   See eg N. Daniels, "Accountability for Reasonableness" (2000) 321 BMJ 1300; C. Ham and G. Robert (eds.), Reasonable Rationing (Buckingham: Open University Press, 2003), Chapter. Back

184   See eg A. Hasman and S. Holm, "Accountability for Reasonableness: Opening the Black Box of Process" (2005) 13 Health Care Analysis 261; T. Beauchamp and J. Childress, Principles of Biomedical Ethics (Oxford: Oxford University Press, 5th edn., 2001) at 258. Back

185   NICE, above n.10 at para.2.2.5. Back

186   N. Daniels and J. Sabin, "`The Ethics of Accountability in Managed Care Reform" (1998) 17 Health Affairs 50 at 61. Back

187   Cited in C. Davies et al, Opening the Box: Evaluating the Citizens" Council of NICE (Milton Keynes: Open University, 2005) at 43; and see further, M. Rawlins, "Pharmacopolitics and Deliberative Democracy" (2005) 5 Clinical Medicine at 473-475. Back

188   Davies et al, above n.16. Back

189   See A Burnham, "A health constitutional", Progress, September 2006. Back