NICE – the National Institute for Clinical Excellence – was established in 1999 by the UK’s Department of Health as an independent body free from political (or other) vested interests. Its remit was to provide advice (“guidance”) to enable health professionals, working in the National Health Service (NHS), to achieve the highest attainable standards of care for their patients.
NICE’s original remit was limited to undertaking “technology appraisals” and to develop “clinical guidelines”. Over the subsequent 15 years, however, its role has been expanded to include other forms of guidance (Table 1). The Institute’s core business, however, remains the production and publication of technology appraisals and clinical guidelines.
These provide advice on the clinical and cost effectiveness of predominantly pharmaceutical products. Although, in the early years, it gave advice on the use of devices and diagnostics these have now been subsumed into other guidance programmes (Table 1).
Table 1 NICE’s guidance programmes
|1999||Technology appraisals||Clinical and cost effectiveness of (mainly) new pharmaceuticals and medical devices|
|1999||Clinical guidelines||Clinical and cost effectiveness of the management of individual conditions|
|2002||Interventional procedures||Efficacy and safety of (mainly) new interventional procedures|
|2005||Public health guidance||Clinical and cost effectiveness of public health measures|
|2009||MedTech guidance||Effectiveness of the use of cost saving devices|
|2009||Diagnostic guidance||Clinical and cost effectiveness of (mainly) new diagnostic techniques|
|2013||Social care guidance||Effectiveness and cost effectiveness of approaches to social care|
|2014||Highly specialised technologies||Clinical and cost effectiveness of technologies for the treatment of very rare (ultra orphan) diseases|
Two undertakes two types of technology appraisals. In a single technology appraisal the Institute compares the clinical and cost effectiveness of a single product with current best practice. Current best practice includes other products with the same licensed indications or – where there is no licensed therapy – with best supportive care. In a multiple technology appraisal NICE compares products within a single therapeutic class to determine whether one is more clinical and cost effective than others. In both instances, and unlike most licensing bodies such as the EU’s European Medicines Agency or the US’s Food and Drugs Administration, NICE is concerned with products’ comparative – rather than absolute – effectiveness. In doing so it takes account of a products efficacy and safety.
Cost effectiveness is examined using the technique of “cost utility analysis” which has two components. A product’s additional (or incremental) clinical benefit is compared with one or more comparators (as discussed above). The benefit is estimated from the improved quality of life attributable to the use of the product, multiplied by the number of years for which it is enjoyed, and expressed as the increased quality adjusted life year (the QALY) gained. The costs obviously include the acquisition costs of the product but also the costs of administration as well as any savings that are accrued by, for example, by reduced costs of treating common adverse effects (such as bone marrow failure with many anti-cancer drugs) or regular therapeutic monitoring (such as with the new oral anticoagulants).
A product’s incremental costs, divided by the incremental benefits, yields the “incremental cost effectiveness ratio” (the so-called ICER) with units of cost (£) per QALY gained. NICE considers that products with ICERs of less than £20,000 per QALY gained can usually be regarded as cost effective for use in the NHS. ICERs in excess of £30,000 per QALY gained would, generally, be considered to be cost ineffective by NICE although, under special circumstances (such as with products for very rare diseases), the Institute may be prepared to accept higher costs per QALY gained.
Although the Institute assembles the evidence decisions about clinical and cost effectiveness are made by the independent advisory committees (the Technology Appraisal Committees). Membership of these committees is drawn from NHS and Universities as well as experts in health economics and biostatistics. NICE applies a strict code of practice for members that ensure there are no inappropriate conflicts of interest.
When NICE considers that a product is cost effective for use in the NHS there is a legal obligation on the service to make it available where the patient’s physician considers it to be appropriate. This has largely eliminated the so-called “postcode lottery” whereby, prior to the formation of NICE, different health authorities made different decisions about the availability of marketed medicines.
NICE’s clinical guidelines aim to provide health professionals with advice on the management of specific conditions. Recently published clinical guidelines have, for example, included:
- Management of children with Type 1 and 2 diabetes
- Management of adults with Type 2 diabetes
- Management of anaemia in patients with chronic renal failure
- Diagnosis and management of drug allergy in adults
NICE’s clinical guidelines are developed by “guideline development groups” (GDGs). Membership encompasses both specialist and generalist healthcare professionals as well as two patients (or “service users” as they prefer to be called). As with the Technology Appraisal Committees, the Institute applies a strict conflict of interest policy for members of GDGs.
The guideline development process starts by scoping out the topic and defining the questions that the proposed guideline should seek to answer. The staff of the guideline centre then undertakes a series of full systematic reviews of the published literature as defined by the guideline’s scope. A single guideline may involve anything between 10 and 20 systematic reviews! The staff then summarise the results of each of the reviews and present them to the GDG. The GDG then prepares a draft full guideline – often running to several pages – that includes details of the evidence considered as well as the basis for its recommendations. Such a document would be impossible for use in everyday clinical practice so the GDG also prepares a short version also in draft form.
The full draft guideline is then widely circulated so that interested parties (including the relevant life sciences industries, professional organisations and patient advocacy groups) can provide comments on the content of the guideline. Up to 2,000 comments may be received for each draft guideline and the GDG is required to review each comment and respond appropriately. NICE’s Guidance Executive Committee then reviews the final draft guideline to ensure that the Institute’s processes have been appropriately applied. The guideline is published on NICE’s website and – for topics of broad interest – a summary may be published in the BMJ or – for more specialist subjects – in one of its associated journals.
There is no obligation for healthcare professionals to use NICE’s clinical guidelines on all their patients with a particular condition. Indeed, it would be impossible to construct a guideline covering all the interactions between healthcare professionals and the patients under their care. Nevertheless, NICE’s clinical guidelines are now widely used within the NHS for two reasons. First, although it has never been tested in the Courts, legal authorities indicate that appropriate adherence to NICE’s guidelines are likely to be an effective defence against claims for malpractice. Second, NICE has earned and retained professional confidence in the quality of NICE’s evidence-based clinical guidelines.
The uptake and use of NICE’s clinical guidelines is correspondingly ubiquitous. For example, it was estimated in 2005 that 25,000 hospitalised patients died each year from preventable pulmonary emboli. In 2010 NICE published a guideline on the prevention of venous thromboembolic disease in hospitalised patients. A major component was the advice that all inpatients should undergo a risk assessment for venous thromboembolism and, where appropriate, be offered prophylaxis. At the time of its publication, surveys showed that less than 40% of patients underwent a risk assessment for venous thromboembolism. Two years later, more than 95% of patients underwent venous thromboembolism risk assessment with prescribing data showing a corresponding increase in the use of anticoagulants. Estimates suggest that the annual mortality from pulmonary emboli has fallen by 7000 per annum.
Another guideline, published in 2008, advised against the routine use, by dentists, of antimicrobial chemoprophylaxis, for patients at risk of infective endocarditis. Although controversial in some quarters, dentists largely accepted the advice and prescribing fell by over 80% during the next 12 months with no excess increase in the incidence of bacterial endocarditis.
It its early years, NICE was viewed with some suspicion by healthcare professionals. Over the 16 years of its existence it has earned and retained the confidence of the professions and the public. It has achieved this for five reasons:
- All NICE guidance is scientifically robust based on systematic reviews of the appropriate literature.
- NICE is fully transparent about what it does and how it does it.
- NICE is inclusive and ensures that all stakeholders “have their say” even if the cannot always “have their way”
- The construction of all NICE guidance is independent of vested interests be they commercial, political or reputational.
- All NICE guidance is “contestable” by publication in draft form to give stakeholders the opportunity to comment.
Dr Richard Smith the editor of the BMJ, who was not well-disposed to NICE in the Institute’s early period, wrote some years later: NICE may prove to be one of Britain's greatest cultural exports, along with Shakespeare, Newtonian physics, the Beatles, Harry Potter, and the Teletubbies.