PM Society Annual Lecture

The future of healthcare and the role of NICE

The PM Society's Annual Lecture, held at the Café Royal on 13 May, was presented by the Chairman of NICE, Professor Sir Michael Rawlins, who focused on the global challenges in providing healthcare services and the role that NICE fulfils in ensuring equitable healthcare in a rapidly changing world.

Global challenges
The challenges faced in providing health services are not unique to the UK, but are common to all healthcare systems. Sir Michael began by outlining these challenges, which include:

An ageing population
Technological advances (not just in pharmaceuticals but in prevention, early diagnosis, minimally invasive procedures, and innovative biopharmaceuticals)
Inappropriate variation in the quality of care (which occurs even in the US)
Underinvestment in public health
Resource constraints
Public expectations, which are now far higher than 50 years ago. Healthcare is now regarded as a right and patients expect high standards.

Other challenges include addressing smoking, alcohol consumption, other substance misuse, overeating, lack of exercise, and sexual activity, particularly in young people; the UK has the highest rates of teenage pregnancy anywhere in Europe.

Healthcare expenditure in the developed world varies from about $500 US per capita per year in Turkey to over $6000 US per capita per year in the US - twice what we spend in this country - though it should be remembered that 20% of the US population does not have access to good healthcare at all. Of course, there is a finite amount of money to go round and increased investment in healthcare would mean shifting resources from other areas, such as education or transport. Failing that, more money has to be raised by income tax.

The solutions
These challenges have been addressed around the world by improving efficiency by re-engineering services (which is just as unpopular in the US as it is here in the UK), disinvestment, and robust commissioning. Value-for-money from new activities has been ensured by technology appraisals, and improved quality of care can be delivered via guidelines.

Effective public health programmes are also important, though only a limited amount of money is available to spend on public health. Sir Michael noted that even when something is discovered that will improve public health we frequently fail to implement it, and cited the example of folic acid in pregnancy; the first trial was published in 1991, yet in 2008 we are still thinking about doing something about it and 400 babies continue to be born each year with spina bifida as a result.

Where NICE fits
NICE was set up in 1999, originally to undertake technology appraisals and produce clinical guidelines. In 2002 interventional procedures were added to the portfolio and in 2005 NICE was also given broad responsibility for public health (transport, education services, etc).

NICE's purpose now is to provide health professionals with advice on securing the highest attainable standards of clinical care for NHS patients and promoting and sustaining the public health. In doing so, NICE is required by law to take into account effectiveness and cost effectiveness.

Output is in the form of NICE guidance. This takes five forms. In the clinical area these are:

Technology appraisals
Clinical guidelines
Interventional procedures.

And in the public health area they are:

Interventions (e.g. needle exchange schemes)
Programmes (e.g. multifaceted activities regarding alcohol intake, etc)

Underpinning these principles is the premise that all NICE guidances should be robust, transparent, inclusive, and independent (of Government or any vested interest). Guidances should also be timely, with not be too big a gap between the introduction of a new drug and when the guidance is issued - if the guidance is too late everyone loses out.

NICE is very much a 'virtual' institute and aims to harness enthusiasm across the country. Appraisal committees rely on the input of eight technology assessment groups around the country, as well as input from the health professions, universities, patients and patient organisations, manufacturers and the public. At any one time, around 2500 people are involved in developing NICE guidance. This is one of the strengths of its organisational structure.

Technology appraisals
Technology appraisals cover pharmaceuticals, devices, and diagnostic methods and other procedures. By law, NICE are required assess both clinical and cost effectiveness. So far, NICE have published over 300 appraisals covering both treatments and conditions. The vast majority of these have been for pharmaceuticals (232), the rest covering devices, procedures and diagnostics.

Sir Michael stressed that NICE very rarely says no to a drug, device or diagnostic method. Only 6% are rejected; full use is approved in about 30% of cases; and 60% are granted restricted use. Very occasionally a drug is approved for research use only.

Clinical guidelines
Clinical guidelines are defined as systematically devised statements to assist practitioner and patient decisions about appropriate healthcare for specific clinical circumstances. NICE guidelines differ from all others in that they cover both clinical and cost effectiveness.

A clinical guideline represents a massive piece of work, and so far around 70 have been published. The process often involves systematically assessing around 30 different questions and takes about two years to complete. Sir Michael also pointed out that when around 120 guidelines have been completed the process needs to start over again and the initial areas reviewed so that the guidelines do not become static.

Economic evaluation
There are three overarching principles to NICE economic evaluation:

The economic perspective – covering the NHS and personal social services only (NICE are not empowered to look wider issues, e.g. the impact on employment, because such a broad perspective would advantage the economically active and disadvantage the economically inactive, e.g. the elderly).
Cost effectiveness – this does not mean affordability or budgetary impact. Something may be cost effective but not affordable.
Balance between efficiency (utilitarianism) and fairness (egalitarianism) – it is sometimes difficult to resolve the inherent tension between these two.

With regard to value for money, there are three approaches: to look at cost minimisation; cost effectiveness analysis – which is a simple approach but one which makes it very difficult to compare one treatment for one condition with a treatment for another condition; and NICE's preferred approach, which is cost utility analysis.

A cost utility analysis expresses the gain in health for a particular treatment or procedure and assesses the costs and savings to the NHS (both direct and indirect). It also assesses the benefits, i.e. the improvements (or change) in health-related quality of life (utility) and the amount of time for which that benefit is enjoyed.

The major problem, in Sir Michael's view, is distinguishing the cost effective form the cost ineffective. One way is to measure the probability of a treatment or procedure being rejected against the cost per QALY (quality adjusted life year) or LYG (life year gained). Unfortunately there is no empirical basis for this. It is also inflexible and implies that efficiency has an absolute priority over equity, as well as being anti-competitive.

NICE therefore looks instead at the question by using a case-by-case approach. This is based on the degree of uncertainty of the ICER (incremental cost effectiveness ratio), the innovative nature of the technology, and wider societal interest (for example, the investment in CJD to preserve the health of future generations), and taking into account previous appraisals and special features of the specific condition.

And rather than having a strict threshold for cost ineffectiveness, NICE has a curvilinear approach, with costs between £20,000 and £30,000 per QUALY being increasingly likely to be rejected on the grounds of cost ineffectiveness.

Costs beyond this are increasingly likely to be rejected, though this is not a certainty, and NICE has gone as far as agreeing to costs of £48,000. These difficult decisions have to be made because, if expenditure on relatively modestly effective treatments is not limited, other patients with other conditions, many of whom do not have a powerful voice, will be deprived of cost effective treatments. NICE aims to be fair to everyone who uses the NHS.

Making these decisions requires difficult social judgements. Issues may include age (children versus the elderly), religious beliefs, socioeconomic status, and disability (an area where modest improvements can confer huge individual benefits). NICE has a Citizens Council which meets twice a year to address these tricky questions. With regard to these social value judgements, NICE guidance is based in part on contemporary bioethics, partly on legal requirements, and partly on the advice from the Citizens Council, which is culturally context specific to the UK.

What next?
NICE will continue to work in the areas in which it is already engaged, as well as a number of additional areas, which include:

Commissioning guides
Scientific advice services
Expanded appraisals programme
Enhanced guidelines programme
NHS evidence – a single pool for the NHS to access all the relevant evidence
Immunisation and screening
Service delivery guidance – what services should be available in a community to deliver high quality care, something which has already been done for cancer.

Talking points
Asked about patient evidence, Sir Michael noted that, in future, NICE must capture patient's perceptions of benefit better than it has done in the past. With regard to uptake of NICE guidance, Sir Michael conceded that this is variable, but that Healthcare Commission figures show that guidance is adhered to by 89% of PCTs. In Sir Michael's view, what is needed is for a patient organisation to take a PCT to court for failing to provide a NICE approved drug. Uptake of guidelines is much more patchy because in any area the levels of engagement vary initially, and it takes time for guidelines to be taken on board. With regard to audit, Sir Michael stated that there is no mechanism in place for auditing guidelines, though the Healthcare Commission does look at uptake in some cases.

Sir Michael also noted that it has been suggested that industry might assist by lending marketeer for a year - a number of companies were enthusiastic about the idea. However the Partners Council felt that this would prejudice the independence of NICE. As to recruiting advisors, this is done mostly by advertising but NICE do also invite people to apply.

Asked about a possible change of Government, Sir Michael stated that both the Liberal Democrats and the Tories have indicated that they would continue to fund NICE. Indeed, governments all over the world are increasingly finding that they need the services a body such as NICE.

Sir Michael Rawlins has been chairman of the National Institute of Health & Clinical Excellence (NICE) since its formation in 1999. He is also chairman of the Advisory Council on the Misuse of Drugs (since 1998). He is an Honorary Professor at the London School of Hygiene and Tropical Medicine, University of London, and Emeritus Professor at the University of Newcastle upon Tyne. He was the Ruth and Lionel Jacobson Professor of Clinical Pharmacology at the University of Newcastle upon Tyne from 1973 to 2006. At the same time he held the position of consultant physician and consultant clinical pharmacologist to the Newcastle Hospitals NHS Trust. He was vice-chairman (1987?92) and chairman (1993?98) of the Committee on Safety of Medicines.

Find out more at www.nice.org.uk

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