BACKGROUND

  This submission was prepared by Dr Kiran C R Patel[153] on behalf of the South Asian Health Foundation (SAHF). The SAHF aims to promote improvements in the quality of, and access to, healthcare and health promotion in South Asians. The organisation achieves this by three main mechanisms:

—  Promotion of high quality scholarship and research.

—  Health promotion and education at a grassroots level to communities.

—  Health advocacy via interaction and advisory input into organisations and processes such as those at NICE.

  Further information about the organisation may be obtained via the website www.sahf.org.uk and annual reports submitted to the charities commission.

EXECUTIVE SUMMARY

—  A minority of NICE's decisions are challenged and even fewer are upheld after appeal.

—  There is an understandable incentive for pharmaceutical companies to appeal NICE's decisions and the relative lack of constraints in embarking on an appeal make this a feasible strategy within the current appraisal structure.

—  Allowing NICE to define the maximum acceptable price for a technology would allow implementation of the Office of Fair Trading Report recommendations and might resolve the issue of exploitation of its appeals process.

—  Independent reports indicate that NICE functions successfully within the field of Health Technology Appraisal.

—  Broader support and recognition of NICE's valuable role within the NHS would counter some adverse negative publicity that NICE receives.

—  NICE has played a valuable role in recognising the needs of south Asian patients in the UK.

Why NICE's decisions are increasingly being challenged

"What this shows is not that NICE is in trouble but that it is doing its job. It was set up to ensure that treatments available on the NHS provide value for money. Decisions to restrict drug treatments are hugely emotive to patients and clinicians. Controversy is inevitable."

Fiona Godlee, Editor, British Medical Journal, 2006

"It has become lamentably commonplace for decisions made by the UK's National Institute for Health and Clinical Excellence (NICE) to be greeted with public outrage. But this reaction says less about NICE's decision-making processes—which are commendably rigorous—than about the gulf between patient expectations of the UK's tax-funded health system, and understanding about the necessity for rational spending."

"If the Government really wants to extend choice within the NHS, as it has pledged, it should launch a debate about the health-financing framework necessary to support this philosophy. But its first obligation should be to show vocal support for NICE as the best mechanism to ensure equity in the UK's current health system."

Richard Horton, Editor, The Lancet, 2006

NICE's Decisions: How Many Are Taken to Appeal and How Many Are Actually Upheld?

  1.  The fact that NICE is engaged with ensuring justifiable expenditure within the NHS on drugs and therapeutic interventions inevitably attracts criticism. NICE's 117 recommendations in the period 2000-05 have been evenly distributed across the four possible outcome decisions: "No" (19%), "yes" (23%), "yes with major restrictions" (32%) and "yes with minor restrictions" (26%). Of the negative recommendations, almost two thirds were on the grounds of insufficient evidence, the remainder due to unacceptable cost- effectiveness.

  2.  NICE's 86 guidances between 2000 and 2005 have been subject to 25 appeals (29%). Fifteen were dismissed. Of the 10 appeals that were upheld, five resulted in relatively minor changes in the wording of the guidance. Only five decisions were referred back to the appraisal committee for further appraisal—approximately 6%.

  3.  Given the relatively high proportion of decisions referred (29%) but the low proportion upheld and re-evaluated (6%), the following factors are relevant to understanding why NICE's decisions are challenged without substantial basis in the majority of cases:

—  the current role of NICE;

—  the structure of NICE's processes;

—  the role of health care professionals and their representative organisations;

—  the role of patient interest groups;

—  the role of the pharmaceutical industry; and

—  the role of the media.

(a)  The Current Role of NICE

  4.  If NICE's decisions did not attract criticism the Health Select Committee would be justified in asking why NICE was ineffective and whether it represented a justifiable appropriation of public funds. By virtue of the roles and responsibilities NICE undertakes, it is both appropriate and anticipated that the decisions it delivers as well as the processes by which it arrives at these decisions are open to criticism. The defined remit of the organisation, to address challenging issues and justify often difficult and emotionally fuelling decisions, represents a justifiable appropriation of public funds.

  5.  Resource allocation is inherently controversial—all needs cannot be met and the public expectation is that healthcare should be universally and comprehensively available to all. The UK population is ageing, healthcare costs are rising and expenditure on drugs represents the largest component of this increase in costs (2). Consequently, bodies such as NICE will increasingly find themselves in the invidious position of regulating the availability of healthcare technologies while the relative availability of resources decreases.

  6.  In conclusion: The long-term trend for allocative decision making will be perceived and recognised as increasingly restrictive in nature but necessary to allow the functioning of a viable NHS.

(b)  The Structure of NICE's Processes

  7.  Through its numerous interactions with NICE, SAHF would make the following observations regarding NICE's processes:

—  Equity of voice accorded to stakeholders ("open-ness").

—  A high standard of transparency.

—  Consequent accountability resulting from the above.

—  An appeals system that may be freely used by third parties to further self-interest over the public good.

  8.  Objective analysis from the World Health Organisation has evaluated NICE's methods and processes and declared them to be sound while a recent independent report from the Office of Fair Trading similarly reported positively and proceeded to further recommend an extension of its powers to undertake drug pricing.

  9.  Allowing NICE this mandate to define the maximum acceptable price for a technology would allow implementation of the Office of Fair Trading Report recommendations whilst being appropriately mindful of R&D considerations for industry.

  10.  We believe that NICE's current process is subject to overuse as a result of four issues:

(1)  the lack of adequate safeguards to prevent exploitation of the appeals process;

(2)  the requirement for an appeal process;

(3)  the requirement of fiscal constraints within the NHS; and

(4)  the high costs of R&D for medical therapies and interventions.

  11.  There is no service user cost to pharmaceutical companies when challenging NICE despite significant potential commercial gains in potential market share for drugs that might offer little extra value for money to the health service. It is not unexpected that pharmaceutical companies have acquired increasing confidence in NICE's process and their apprehension in routinely employing the appeals procedure may have diminished. It is natural that organisations and lobby groups are likely to appeal decisions they do not like.

  12.  Conclusion: Objective reports give reason for confidence in NICE's processes. The NICE appeals process is highly vulnerable to overuse by parties with commercial or vested interests. Safeguards to protect NICE and henceforth the health economy should be introduced.

(c)  The Role of Health Care Professionals and their Representative Organisations

  13.  NICE consults widely when making its decisions and it employs multidisciplinary panels that include patients and specialists to ensure a balanced view. To understand how healthcare professional groups may perceive NICE one must consider the situation before the inception of NICE. Pre-NICE, clinical care was guided by professional groups, who were often poorly integrated into the mainstream NHS and took little account of the overall fiscal consequences of their recommendations beyond a single specialty focus. Industry supports many of these professional groups they are potentially susceptible to bias and if not, perceived to be susceptible to bias.

  14.  It is difficult to reconcile these historical approaches with that of a body such as NICE, which must operate transparently, equitably and consider the needs of the NHS as a whole. This situation reflects a challenge for professional groups to create transparency with respect to their interactions with the pharmaceutical industry. A previous Health Select Committee, which examined the relationship between healthcare professionals and the pharmaceutical industry, recommended that a register of interests be instituted—there has been no move to implement this recommendation to date.

  15.  Conclusion: The establishment of NICE has resulted in an increasingly democratic, patient centred and quality focussed system, with attention to health economics which is vital to the NHS today. Ongoing deficiencies in the transparent declarations of interaction of professional groups with pharmaceutical companies results in unrepresentative engagement from some stakeholders.

(d)  The Role of Patient Interest Groups

  16.  As an organisation that represents the interests of south Asian patients in the UK, SAHF have a vested interest in this particular area of healthcare provision to ethnic groups and reduction of health inequalities. Organisations and charities with other specific interests will naturally have a similar bias to toward their own interests.

  17.  As a frequent stakeholder within NICE guidance and guidelines SAHF has recognised that that our work in promoting the healthcare issues faced by south Asian patients should not disadvantage other groups and/or be perceived as detrimental to the welfare of people in general.

  18.  SAHF has sought to provide assurance that our structure and function are not contaminated by financial ties that might be seen to compromise its decision-making or policies. SAHF functions at a level of transparency akin to NICE. Single-issue groups are not remitted to balance their demands with the needs of the population as a whole. This lobbying and stakeholder representation is both expected and appropriate for special interest groups. NICE is highly commendable for providing this voice, even to minority stakeholders.

  19.  The experience of SAHF is that NICE has provided an equitable, transparent framework to address issues pertinent to south Asian health in the UK. Previous guidelines from professional groups have frequently neglected south Asian health and we are definitely of the view that that NICE's existence has allowed a "voice" for marginalised groups that would not previously be heard over the demands of much more powerful voices that represent high profile disease areas. SAHF has never challenged a decision of NICE.

  20.  Conclusion: SAHF acknowledges that NICE has provided a valuable conduit to address issues surrounding south Asian health, a conduit that before the inception of NICE never existed.

(e)  The Role of the Pharmaceutical Industry

  21.  The "industry" naturally has a clear purpose with respect to NICE (1) to ensure its products receive recommendation for NHS use and that such approval allows as widespread a dissemination as possible. To expect otherwise would be unhealthy for the economic aspects of industry. These interests must be balanced with the moral responsibility of ensuring appropriate and safe treatments reach patients.

  22.  With drug development costs of approximately £800 million and the cost of challenging NICE's decision within an appeal being comparatively small, it is inevitable that a reasonable and appropriate strategy for the pharmaceutical industry is to challenge unfavourable decisions from NICE. The commercial factors that can motivate the promotion of some drugs beyond their optimum use have been described in the recent Office of Fair Trading Review of the PPRS Scheme. The appeals launched against NICE's decisions represent another facet of a structural design limitation that understandably requires companies to seek maximum competitive advantage within a marketplace.

  23.  Conclusion: The current weakness in NICE's appeals process means that it is structurally vulnerable to excessive exploitation.

(e)  The Role of the Media

  24.  The Health Select Committee will undoubtedly be aware of the role played by the media in denigrating public service organisations. NICE has relatively little resources allocated to marketing and promotion. It has an invidious role yet valuable function in maintaining the viability of the health service.

  25.  Medical trade publications, particularly those that are "free" but largely funded by industry advertising frequently attempt to confront NICE and report divisive headlines.

  26.  Subscription press coverage has been increasingly favourable to NICE. The BMJ and also the Lancet have recently acknowledged the valuable role that NICE now plays in the UK health economy. Both of these publications are recognised as internationally respected publications yet also have significant industry based revenue from advertising.

  27.  The lay press eg newspapers focus almost entirely on highlighting NICE's restrictive recommendations despite the fact that the majority of NICE's recommendations result in some form of approval. Consequently, NICE is losing public support due to inadequate representation to the public whom it serves NICE's perception should not judged solely according to the popularity it enjoys as a result of the number of positive recommendations it makes, but the appropriateness of its decisions in relation to its remit (value for money) should also be a benchmark.

  28.  Conclusion: NICE should receive an appropriate budget for disseminating its recommendations within the wider health economy and public.

Whether public confidence in the Institute is waning, and if so why

  29.  There are no objective measures that indicate the level of public confidence in NICE. All submissions to this point are likely to represent opinion.

  30.  Public confidence ie the view of the integrity with which NICE makes decisions regardless of whether a particular party finds them agreeable or not should be separated from public perception.

  31.  Public perception is influenced by media coverage that is also influenced by emotive patient vignettes and professional groups that may seek to distort the priorities of the NHS. Many people will probably be unaware of the intricacies of the health service and organisations such as NICE. For some people who are aware of NICE it may be when unfavourable press coverage is relayed to them, often presenting a distorted view of NICEs objectives.

  32.  Conclusion: NICE may be experiencing adverse publicity which requires its aims and objectives to better represented and supported by the health service.

NICE's evaluation process, and whether any particular groups are disadvantaged by the process

  33.  As an organisation that represents the health needs of the UK south Asian population we feel that NICE has an exhaustive stakeholder process that the "voice" of ethnic minority representation to be heard. Conversely, some expert groups may feel their influence has been moderated.

  34.  Conclusion: We believe that NICE has "levelled the playing field" no longer is decision making and resource allocation a case of "he who shouts loudest" but based upon evidence.

The speed of publishing guidance

  35.  We believe that speed at which NICE produces its guidance is a favourable reflection of the demand it is subject to and its capacity to deliver guidance. However speed of decision making could improve, as we also understand the frustration that a decision making process which may take months if not years, can cause. Such lengthy processes sometimes mean guidance is out dated as soon as it is published and therefore the NHS may not be practising at the frontiers of medical advance and delivering such guidance may be cost ineffective per se.

  36.  Conclusion: We believe that the speed at which guidance is produced is usually appropriate but sometimes frustrating for service users. This statement is tempered by the realisation that there was no guidance at all before NICE's introduction. The recommendations of the previous Health Select Committee that NICE receive additional funding have yet to be implemented. Only further investment and periodic secondment of panel members to NICE can expedite the guideline development process.

The appeal system

  37.  We refer to the comments we have previously made regarding NICE's appeal system under the related section "why NICE's decisions are increasingly being challenged".

  38.  Conclusion: The current weakness in NICE's appeals process means that it may be excessively subject to exploitation and potentially to abuse.

Comparison with the work of the Scottish Intercollegiate Guidelines Network (SIGN)

  39.  NICE presides over England, Northern Ireland and Wales, a population of approximately 55 million. SIGN, presides over Scotland only which has a population of five million people. This ten-fold difference precludes equivalence between the two organisations.

  40.  The most important difference between NICE and the SIGN is that NICE undertakes technology appraisals and incorporates health economic analyses which are not undertaken by SIGN (although SIGN is increasingly referencing health economics in its deliberations). Without health economic analysis we believe that NICE's decisions would be unacceptable to the pharmaceutical industry in view of the size of the UK pharmaceutical market and potential criticisms that NICE did not promote the true value of drugs because it was not integrating cost-effectiveness analyses. Such criticisms have arisen in the United States toward evidence review bodies which fail to incorporate formal health economic analysis.

  41.  Conclusion: Given the size of the market for therapeutic interventions in England and Wales, along with the wide range of stakeholders who must be consulted we believe that NICE's processes are commensurate with its function. Indeed, we believe that NICE's decisions would be subject to challenge considerably more frequently if an objective health economic framework did not underpin them.

The implementation of NICE guidance, both technology appraisals and clinical guidelines

  42.  Technology Appraisals: We believe that NICE should be further supported in the implementation of its technology appraisals by appropriate mandating of its recommendations within the reporting structure of the health service. There is no formally monitored scheme for monitoring the adherence of hospitals or PCT's with NICE Technology Guidance that has an appropriate priority. Many groups, including the ABPI amongst others have called for guidance to be made mandatory—we would agree . A study in 2004 suggested that implementation of NICE guidance was variable and more likely to be adopted when there is strong professional support, a stable and convincing evidence base, and no increased or unfunded costs. Further and more detailed research from 2005 covering 28 appraisals suggests the situation is improving: 12 appraisals were implemented fully, 12 were incompletely implemented, and four over-implemented).

  43.  Guidelines: Within the current budget, NICE appears to have insufficient resources to promote its guidelines at local health service level. One solution, might be to introduce requirements for PCT's to provide or commission local NICE implementation Units at a regional level. An enquiry by the Audit Commission supports the need for improvements at local health economy level, in particular, ascribing lack of implementation to poor financial management at local NHS trust level.

  44.  Conclusion: NICE is inadequately resourced to ensure the implementation of its output and structural health service reform that targets often poor financial planning and organisation within local acute and primary care trusts. A more robustly defined interaction with industry is essential to serve the interests of patients and the health economy.

Dr Kiran Patel

Chairman of Trustees

South Asian Health Foundation

March 2007