NICE and the public health

Although NICE was established in 1999 [1], its origins go back to two earlier government ‘white papers’[2, 3]. These outlined plans to establish, within the National Health Service (NHS), two new bodies: the National Institute for Clinical Excellence (NICE) to set clinical standards, and the Commission for Health Improvement (CHI) to monitor the performance of NHS organizations in relation to their responsibilities for clinical governance.

Failure to incorporate (or ‘translate’) the results of clinical research into routine care has become a global problem. As a consequence there are:

• inappropriate variations in clinical practice

• inconsistent access to treatments of established clinical and cost effectiveness (so-called ‘postcode prescribing’)

• failure to adopt new practices of proven clinical and cost effectiveness

• failure to discontinue old practices of proven clinical or cost ineffectiveness.

The apparent global underachievement, by health professionals, is not due to perversity but to three interrelated problems. First, the clinical community is exposed to an explosion of knowledge about best clinical practice, to the extent that it has now become impossible for a single individual to keep up-to-date. Second, the essential infrastructure needed to support high-quality care (particularly, but not exclusively, in the NHS) has too often been unavailable. And, third, health professionals face apparently irreconcilable tensions between quality and efficiency, equity and choice, and demand and resource. The creation of NICE was the British solution to resolving this difficulty.

The Institute was formed, in 1999, with one fundamental aim [1]: ‘To provide health professionals in England and Wales with advice on securing the highest attainable standards of care for National Health Service patients’. Scotland, with its devolved administration, has established its own arrangements although there are close, and effective, interactions with the Institute. Northern Ireland adopts NICE guidance but does not, as yet, have a formal relationship with the Institute.

NICE guidance

The Institute produces three forms of NICE guidance [4]:

• technology appraisals,

• clinical guidelines, and

• interventional procedures.

This review of the Institute's programmes and progress is concerned, exclusively, with the Institute's appraisals and guidelines programmes. The interventional procedures programme [5] is concerned with advising the NHS, including NHS Scotland, on the safety and efficacy (but not cost effectiveness) of invasive diagnostic and therapeutic techniques and is not considered in this review.

Technology appraisals

In this programme [6], the Institute offers guidance on the appropriate use of individual, or groups of, health technologies based on their clinical and cost effectiveness. The technologies encompass:

• pharmaceuticals

• medical devices

• surgical (and other) procedures

• diagnostic methods

• health promotion techniques.

NICE published its first full technology appraisals in April 2000 [7, 8]; and, by the end of March 2004, had produced and disseminated a total of 73 appraisals involving nearly 200 individual products (Table 1). This table shows that 51 out of 76 (67%) appraisals involved pharmaceuticals. It also indicates that although, overall, complete rejection of a technology (or class of technologies) for use in the NHS has been unusual, routine use (for all the licensed, or accepted, indications) has been recommended in only 29 (38%) of instances.

Table 1.

Completed appraisals (as of 31 March 2004)

Technology	Total appraisalsa (no. of products)	Routine useb	Selective usec	Research onlyd	No use
Pharmaceuticals	51 (115)	23	25	1	2
Devices	10 (>67)	3	6	1	0
Diagnostics	3 (9)	1	1	1	0
Procedures	11 (>10)	1	8	2	0
Health promotion	1 (1)	1	0	0	0
Total	76 (>202)	29	40	5	2

^(a)Total published technology appraisals;

^(b)Recommended for routine use;

^(c)Recommended for use is only some patients;

^(d)Recommended for use only as a formal fluid.

Clinical guidelines

NICE has adopted the Institute of Medicine's definition [9] of clinical guidelines: ‘Systematically developed statements to assist practitioner and patient decisions about appropriate health care for specific clinical circumstances’.

This emphasizes three critical features. First, guidelines are (in so far as is possible) evidence-based and developed from an exhaustive review of the relevant literature. Second, they ‘inform’– but cannot ‘mandate’– clinical practice since, at best, they can cover only about 80% of patients with a particular condition. Health professionals must therefore exercise their clinical judgement in recognizing, and managing, the remainder. Third, guidelines are directed at both health professionals and patients. This is deliberate. NICE believes that patients (and their families) have a right to know how they should be treated and what alternatives are available. The Institute also seeks to encourage patients to have easy access to NICE's guidelines because of the impact they can have on their implementation.

NICE's guidelines are developed, on behalf of the Institute, by its seven National Collaborating Centres. These are each composed of consortia of relevant Royal Colleges and specialist societies and are supported, financially by the Institute. Whilst NICE determines the approach to be taken in the construction of a particular guideline, and reserves the right (never exercised) to decline publication, responsibility for the content rests with the NCC and the guideline development group.

At its inception, NICE inherited a guideline programme that had been commissioned by the Department of Health in the mid 1990s. The Institute agreed to oversee their completion and NICE's guideline output, in Table 2, shows both ‘inherited’ and ‘commissioned’ programmes. The Institute, through its NCCs, currently has over 50 guidelines planned or in development. This, as far as I am aware, is the largest guideline development programme of any healthcare system in the world.

Table 2.

Completed clinical guidelines

Subject area	Total	Inherited	Original
Nursing	3	1	2
Cardiology	2	1	1
Pregnancy	3	2	1
Diabetes	6	6	0
Cancer	3	3	0
Mental health	2	0	2
Surgery	2	0	2
Neurology	1	0	1
Respiratory	1	0	1
Women's health	1	0	1
Total	24	13	11

Developing NICE guidance

All NICE guidance shares common features.

Comprehensive evidence base

NICE's technology appraisals and clinical guidelines are based on a full systematic review of the available evidence. Although this is time-consuming and resource intensive, NICE believes that such an approach is an indispensable prerequisite to developing robust guidance that has the confidence of health professionals, patients and the public.

Expert clinical and patient input

All NICE guidance is strongly influenced by the views and judgements of expert clinicians, patients, their carers or their representative organizations. The need for expert clinical advice is obvious, but the involvement of patients is equally important. Patients, themselves, can offer a perspective about individual diseases and their treatments that transcends the knowledge and experience of even the most expert clinicians. This applies particularly to the design and interpretation of disease-specific measures of quality of life which have, too often, been constructed without patient involvement.

The involvement of expert clinicians and patients in developing NICE guidance may create tensions, particularly over judgements about cost effectiveness. The Institute (see below) has a responsibility to all patients seeking care from the NHS. It has to remember that recommending a cost-ineffective treatment for one group of patients will deprive many others of cost-effective care. For patient organizations devoted to single diseases this can be difficult to accept; but it is one that NICE must never forget.

Involvement of manufacturers

The Institute actively seeks evidence from those who provide the NHS with manufactured technologies (i.e. the diagnostic, devices and pharmaceutical industries) in both its appraisals and guidelines programmes. The industries have both a right, and an obligation (as part of their ethical responsibilities) to provide NICE with all the relevant data in their possession.

Transparency of process

The processes by which NICE develops its various forms of guidance are clearly described [6, 10]. The relevant documentation is freely available on the Institute's website apart from data submitted as ‘commercial-in-confidence’ usually (but not invariably) by manufacturers.

NICE guidance attempts to explain the reasons for the Institute's advice and the interpretations that its advisory bodies have placed on the available data. It is not easy, however, for the nuances behind decisions (especially in those instances where difficult judgements about cost effectiveness have had to be made) to be easily described in prose. I believe that NICE must eventually allow public access to meetings of its advisory bodies in the same way that the public can (and does) attend meetings of the Institute's Board. Indeed, the process has started: appeals against decisions of the Appraisal Committee will, later this year, start being held in public.

Allowing public access to the meetings of national scientific advisory committees, although common in the USA (such as the Food and Drug Administration), is almost unknown in the UK. It could be intimidating for younger, less experienced, and less confident committee members. There is also the danger (although US experience contradicts this) that full, critical discussion might be inhibited. My experience of the Institute's public Board meetings does not support this view and I believe that allowing stakeholders (including journalists and the public) to attend meetings, and hear the discussion in its entirety, is the only way that the Institute will achieve complete transparency.

Independent advisory committees

All NICE guidance is developed by the independent members of the Institute's advisory committees and guideline development groups. They are drawn from the NHS, academia, the industries and patient–carer organizations. They are as independent a group of individuals as one could imagine! Whilst the Board retains powers to override their advice, it has never done so. NICE guidance is free from inappropriate influence.

Regular review

NICE guidance is ephemeral and needs to be revised as further evidence emerges. Guidance is therefore always accompanied by a review date, although there are provisions for an earlier review if there is a major change to the evidence base. The need to review NICE guidance has workload implications for the Institute and plans have been made for this.

Economic evaluation

Every country is confronted by the unpalatable fact that the resources available for the provision of healthcare are finite. Even the USA, with an annual per capita expenditure on healthcare more than 500 times greater than that of countries in sub-Saharan Africa, has difficulties in meeting the health needs of all its citizens.

The appropriate allocation of available resources for healthcare is, moreover, rendered even more difficult by the existence of two competing principles. Classical health economics has largely been concerned with maximizing health gain, for the population as a whole, for the expenditure of a given amount of resource. This approach to resource allocation – efficiency – is essentially based on utilitarianism and seeks to secure ‘the greatest good for the greatest number’. This concept, however, can sit uncomfortably with the desire to ensure fairness – equity – in order that the interests and needs of minorities (such as those with rare diseases) can also be met.

Straddling across these two competing goals is the universal recognition of what is (confusingly) described by economists as ‘opportunity costs’. This reflects the obvious fact that money cannot be spent twice: and that if considerable sums are devoted to the treatment of a small number of people with (say) a rare disease there will be many others who will be denied less expensive and more cost-effective treatments.

NICE's approach to economic evaluation depends on whether one treatment, or clinical management strategy, is equivalent or superior to another.

Equivalence

Where two treatments are equivalent in clinical effectiveness, then it is merely a matter of recommending the less expensive. This approach is known as ‘cost minimization’ and (leaving aside the difficulties in determining clinical equivalence) is generally uncontroversial.

Superiority

Where one treatment is superior to another, the Institute has to decide whether the increased cost gives value-for-money in terms of improved health. This is estimated as the ‘incremental cost effectiveness ratio’ (ICER).

The Institute would prefer to express ICERs in terms of cost per quality-adjusted life year (cost per QALY) [6] since this allows different technologies, used for different purposes, to be compared. In some instances, particularly with certain anticancer drugs, this has not been possible and ICERs have, instead, been expressed as cost per life year gained.

NICE has eschewed the concept of an ICER ‘threshold’ above which a technology would invariably be deemed cost ineffective. Apart from the difficulty in defining such a threshold, it would mean that efficiency took priority over equity. ICERs below the region of £15 000–20 000/QALY have, by custom and practice, been regarded by UK health economists as being cost effective. The Institute requires special reasons for regarding ICERs above the range of £25 000–35 000/QALY as cost effective. Such reasons include [6]:

• the degree of uncertainty surrounding the estimates of the ICER(s)

• the particular features of the condition

• the particular features of the population using the technology

• the innovative nature of the technology

• the wider societal costs and benefits

• reference to previous appraisals.

The phrases ‘particular features of the condition’ and ‘the population using the technology’ incorporate matters that include the availability and clinical effectiveness of other interventions for the condition, particular public health issues (e.g. communicable diseases), and special considerations of equity [11]. Case-by-case decisions of this nature are, ultimately, judgements.

The next steps

NICE has been in existence for only 5 years and is now beginning to make its mark on the NHS. Much, though, still needs to be done.

Earlier appraisals

Many of NICE's early appraisals concerned technologies (particularly pharmaceuticals) that had been marketed for some years but which had been the subject of ‘postcode prescribing’. Examples included the atypical antipsychotics, drugs for the treatment of Alzheimer's disease, and taxanes for breast cancer.

In future, the Institute's appraisals programme will include more recent technologies. There is no reason in principle why appraisals of all new products that offer significant diagnostic or therapeutic benefits should not be produced within 2–3 months of their launch. For a pharmaceuticals, this means starting the appraisal during the regulatory process; and completing it once the authorized indications have been decided. Indeed, NICE's appraisals programme already includes such products.

Expanded guidelines programme

I have made no secret of the fact that, in the long term, I believe that NICE's fullest contribution to improving health will be brought about by the development and implementation of its clinical guidelines. To achieve this, I also believe that NICE's ultimate goal must be to provide the NHS with a suite of up-to-date clinical guidelines covering the major causes of morbidity and mortality. This will take time. Expertise in developing guidelines that take account of both clinical and cost effectiveness is limited, though rapidly growing as a consequence of our burgeoning programme. The capacity of the NHS to implement NICE's guidelines is also finite. Nevertheless, over the next few years the Institute's guidelines programme will expand as resources (both human and financial) become available.

Additional programmes

NICE's role is to provide the NHS with the clinical standards that patients can expect to receive from the service. There are several areas into which it would be logical for the Institute to extend its influence.

The NHS currently spends over £120 million per annum on so-called ‘borderline substances’ which, though not pharmaceuticals, can nevertheless be prescribed. Most borderline substances are medical foods such as gluten-free products. There is a need to evaluate their clinical and cost effectiveness and provide physicians and dieticians with advice on appropriate indications.

There is a strong case for the Institute to take over responsibility for providing advice on immunization and vaccination. First, it would ensure consistency in decisions about cost effectiveness. Second, the distinctions between immunization and conventional pharmacotherapy are rapidly becoming obsolete. Examples include drugs to prevent communicable diseases (such as oseltamivir in the prevention of influenza) and therapeutic vaccines (such as palivizumab to prevent respiratory syncytial virus infections). The inclusion of advice on immunization and vaccination, within the Institute's portfolio, would guarantee a coherent strategy for their rational use.

NICE already has responsibility for advising on the use of screening technologies (such as liquid-based cytology for preparing cervical smears) [12]. It would be a logical step for the Institute to advise on the application of screening techniques more generally. I have no doubt that, in due course, NICE's responsibilities will be extended to encompass those currently undertaken by the National Screening Committee.

Research and development

NICE has two particular interests in research and development.

The Institute's guidelines and appraisals programmes reveal gaps in knowledge requiring further research (usually clinical trials). The research recommendations emanating from these forms of NICE guidance are already having an impact on the NHS's Health Technology Assessment programme and I expect this interaction to become increasingly important.

NICE also has its own research needs to underpin the methodologies it uses in developing NICE guidance. Examples include empirical research into the value (within the UK) of a QALY, how cost effectiveness might most reliably be incorporated into guidelines, and what ‘weightings’ might be given to measures of ‘utility’ in cost-effectiveness analyses. These requirements are currently being met through the NHS's Methodology Research programme but the Institute, itself, is committing some of its own resources to this venture.

Implementation

When NICE was originally conceived [3], implementation of its guidance was not seen as part of its business. Nevertheless, unless NICE guidance is implemented, and has a positive effect on public health, its existence is pointless.

Evidence about the uptake and adoption of NICE guidance is patchy and has been variably interpreted. The National Audit Office's findings [13] about the hip prostheses used in the NHS can be viewed as a minor triumph (87% conform to NICE's standard) or a major public scandal (13% do not). Nevertheless, the implementation of NICE guidance appears to be uneven.

NICE, itself, is in no position to promote its guidance to clinicians individually. This must be the responsibility of local health communities and encouraged by the Healthcase Commission's clinical governance reviews. The Institute does, however, have a significant role to play. It must:

• ensure that its guidance topics are ones that meet public health needs

• develop its guidance in as inclusive, and transparent, a manner as is possible

• make certain that its guidance is implementable

• disseminate its guidance to those who are responsible for its implementation, and in a form that is clear and unambiguous.

There are additional measures, too, that NICE is currently considering. NICE might provided NHS managers with implementation ‘tool-kits’ for each piece of guidance. The Institute could also develop ‘templates’ to help Primary Care Trusts, as well as Hospital Trusts, in constructing their own care protocols that take account of local circumstances and available skills.

Conclusions

Despite some early scepticism [14], NICE is here to stay. A recent opinion poll [15] showed that the Institute has an ‘awareness factor’ of 27% amongst a stratified random sample of the adult British public. The same poll has found that 72% of those aware of NICE believe the Institute to be doing a ‘good’ or ‘very good’ job.

NICE has also aroused considerable international interest and several EU member states have either already established, or are in the process of establishing, their own versions. Whilst such accolades are flattering, those most intimately involved with the Institute are aware of two facts: that NICE is only as good as the quality of its guidance; and that its sole purpose is to serve the interests of the British people.