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| Agency overview | |
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| Headquarters | London, United Kingdom |
| Website | www |
The National Institute for Health and Care Excellence (NICE) is an executive non-departmental public body of the Department of Health in the United Kingdom, which publishes guidelines in four areas:
- the use of health technologies within the National Health Service (NHS) (such as the use of new and existing medicines, treatments and procedures)
- clinical practice (guidance on the appropriate treatment and care of people with specific diseases and conditions)
- guidance for public sector workers on health promotion and ill-health avoidance
- guidance for social care services and users.
These appraisals are based primarily on evaluations of efficacy and cost-effectiveness in various circumstances.
It serves both the English NHS and the Welsh NHS. It was set up as the National Institute for Clinical Excellence in 1999, and on 1 April 2005 joined with the Health Development Agency to become the new National Institute for Health and Clinical Excellence (still abbreviated as NICE). Following the Health and Social Care Act 2012,
NICE was renamed the National Institute for Health and Care Excellence
on 1 April 2013 reflecting its new responsibilities for social care, and
changed from a special health authority to an Executive Non-Departmental Public Body (NDPB).
NICE was established in an attempt to end the so-called postcode lottery
of healthcare in England and Wales, where treatments that were
available depended upon the NHS Health Authority area in which the
patient happened to live, but it has since acquired a high reputation
internationally as a role model for the development of clinical
guidelines. One aspect of this is the explicit determination of
cost–benefit boundaries for certain technologies that it assesses.
NICE also plays an important role in pioneering technology assessment
in other healthcare systems through NICE International, established in
May 2008 to help cultivate links with foreign governments.
Policy history
The notion of an Institute to determine the clinical effectiveness of interventions first emerged at the end of John Major's Conservative Government as moves elsewhere were being made to set professionally agreed standards for clinical care. In 1996, the UK National Screening Committee (NSC) had been established by Sir Kenneth Calman and Muir Gray (now Sir Muir Gray) by the Policy Team led by Dr Tim Riley and latterly Sir Charles Nightingale for the Department of Health.
The NSC aimed to ensure that evidence-based medicine informed policy
making on what national screening programmes were approved for funding
and what quality assurance mechanisms should be in place. This was a
timely action as screening quality in breast cancer screening services
came under question at Exeter in 1997 and followed in the wake of the 1995 Calman-Hine Report.
The idea of what was originally termed a National Institute for
Clinical Excellence took root when Labour came to power in 1997. Frank Dobson
became Secretary of State and was supported by a team of Ministers keen
on introducing clinical and health outcome measures to achieve
improvements in the quality and delivery of care. The team included Alan Milburn, Baroness Margaret Jay, and Tessa Jowell.
The name and mission was agreed in a meeting between the Ministerial
team, Dr Tim Riley and Dr Felicity Harvey shortly after the election and
it was agreed that NICE should be described in the first policy white paper, The New NHS: Modern, Dependable 1997.
Riley led the team that developed the policy for NICE and which managed
the legislation through Parliament in addition to formalising the new
institute as a Special Health Authority. Riley joined Sir Michael Rawlins (the then recently appointed Chair of NICE) at the Health Select Committee
in February 1999 where questions were raised as to whether NICE was
just a means to "ration" healthcare. Sir Michael Rawlins presented a
compelling case that positioned NICE as a standards setting body first
and foremost.
However, the reality was that although NICE was principally aimed at
aligning professional standards through clinical guidelines and audit,
the acceptability of drugs, devices and technological interventions in
defining those standards, could not be ignored and so the concept of a
"fourth hurdle" for drugs accessing the NHS market was invoked. This
controversial policy shift meant that NICE was critical for decisions on
drug reimbursement. Indeed, the first drug appraisal by NICE was on the
drug Relenza which was turned down amidst criticisms from Glaxo-Wellcome that the appraisal had been fast tracked.
Later, this policy development whereby the criteria for decision
making, the role of costs, and the degree to which decisions of NICE and
the secretary of state would be binding on clinicians was analysed by Andrew Dillon, Trevor Gibbs, Tim Riley, and Trevor A. Sheldon.
Technology appraisals
Since January 2005, the NHS in England and Wales has been legally obliged to provide funding for medicines and treatments recommended by NICE's technology appraisal board.
This was at least in part as a result of well-publicised postcode
lottery anomalies in which certain less-common treatments were funded in
some parts of the UK but not in others due to local decision making in
the NHS.
Before an appraisal, the Advisory Committee on Topic Selection
(ACTS) draws up a list of potential topics of clinical significance for
appraisal. The Secretary of State for Health or the Welsh Assembly
must then refer any technology so that the appraisal process can be
formally initiated. Once this has been done NICE works with the
Department of Health to draw up the scope of the appraisal.
NICE then invites consultee and commentator organisations to take
part in the appraisal. A consultee organisation would include patient
groups, organisations representing health care professionals and the
manufacturers of the product undergoing appraisal. Consultees submit
evidence during the appraisal and comment on the appraisal documents.
Commentator organisations include the manufacturers of products to which
the product undergoing appraisal is being compared. They comment on the
documents that have been submitted and drawn up but do not actually
submit information themselves.
An independent academic centre then draws together and analyses
all of the published information on the technology under appraisal and
prepares an assessment report. This can be commented on by the
Consultees and Commentators. Comments are then taken into account and
changes made to the assessment report to produce an evaluation report.
An independent Appraisal Committee then looks at the evaluation report,
hears spoken testimony from clinical experts, patient groups and carers.
They take their testimony into account and draw up a document known as
the 'appraisal consultation document'. This is sent to all consultees
and commentators who are then able to make further comments. Once these
comments have been taken into account the final document is drawn up
called the 'final appraisal determination'. This is submitted to NICE
for approval.
The process aims to be fully independent of government and
lobbying power, basing decisions fully on clinical and
cost-effectiveness. There have been concerns that lobbying
by pharmaceutical companies to mobilise media attention and influence
public opinion are attempts to influence the decision-making process. A fast-track assessment system has been introduced to reach decisions where there is most pressure for a conclusion.
Clinical guidelines
NICE
carries out assessments of the most appropriate treatment regimes for
different diseases. This must take into account both desired medical
outcomes (i.e. the best possible result for the patient) and also
economic arguments regarding differing treatments.
NICE has set up several National Collaborating Centres bringing
together expertise from the royal medical colleges, professional bodies
and patient/carer organisations which draw up the guidelines. The
centres are the National Collaborating Centre for Cancer, the National Clinical Guideline Centre, the National Collaborating Centre for Women and Children´s Health, and the National Collaborating Centre for Mental Health.
The National Collaborating Centre appoints a Guideline
Development Group whose job it is to work on the development of the
clinical guideline. This group consists of medical professionals,
representatives of patient and carer groups and technical experts. They
work together to assess the evidence for the guideline topic (e.g. clinical trials
of competing products) before preparing a draft guideline. There are
then two consultation periods in which stakeholder organisations are
able to comment on the draft guideline. After the second consultation
period, an independent Guideline Review Panel reviews the guideline and
stakeholder comments and ensures that these comments have been taken
into account. The Guideline Development Group then finalises the
recommendations and the National Collaboration Centre produces the final
guideline. This is submitted to NICE to formally approve the guideline
and issue the guidance to the NHS. To date NICE has produced more than 200 different guidelines.
In October 2014 Andy Burnham
said that a Labour government could reduce variation in access to drugs
and procedures by making it mandatory for commissioners to follow NICE
clinical guidelines. "We need to look at how you strengthen NICE. Where
they have said something is effective and affordable, on what basis does
a local commissioner withhold that from somebody? I’m not comfortable
with that. I don’t support that."
NICE has a service called Clinical Knowledge Summaries (CKS)
which provides primary care practitioners with a readily accessible
summary of the current evidence base and practical guidance.
Social care guidance
Under the Health and Social Care Act 2012, NICE was given responsibility for developing guidance and quality standards for social care,
using an evidence-based model. This is being delivered by the NICE
Collaborating Centre for Social Care (NCCSC), which is hosted by the Social Care Institute for Excellence (SCIE) and 4 partner organisations - Research in Practice, Research in Practice for Adults, Personal Social Services Research Unit and the EPPI-Centre.
NICE receives referrals for social care guidance from the Department of Health and the Department for Education,
and commission the guidance from the NCCSC. NICE, along with the NCCSC,
carries out a scoping exercise with a scoping group and with input from
key stakeholders, at both a workshop and a public consultation, to
ensure the guidance to be produced is focused and achievable. A chairperson
and members of the Guidance Development Group are appointed, and pose
review questions which will enable systematic evidence reviews to take
place, thus delivering the guidance and subsequent recommendations.
Service user and carer involvement takes place throughout, as well as
public consultation on the draft guidance.
The Guidance Development Group then finalises the recommendations
and the NCCSC produces the final guideline. This is submitted to NICE
for formal approval and publication. The entire process from pre-scoping
to publication takes approximately 24 months. The guidance is then
available to NICE standing committees to develop a quality standard on
the topic. The quality standard is developed using the guidance and
other accredited sources, to produce high-level concise statements that
can be used for quality improvement by social care providers and
commissioners, as well as setting out what service users and carers can
expect of high quality social care services.
The NCCSC is unique within NICE, in that it is the only
collaborating centre to have responsibility for the adoption and
dissemination support for guidance and quality standards in the social care arena.
Drawing on the expertise of SCIE and their partners within the sector,
each of the guidance products and quality standards have a needs
assessment carried out to determine the requirements for tools to help
embed the guidance and quality standards within the sector. These can
include tailored versions of guidance for specific audiences, costing
and commissioning tools and even training and learning packages.
As of August 2013, NICE and the NCCSC had scheduled guidance delivery for five topics: domiciliary care,
older adults with long-term conditions, transition between health and
social care settings, transition from children's to adults' services and
child abuse and neglect.
Cost–effectiveness
As
with any system financing health care, the NHS has a limited budget and
a vast number of potential spending options. Choices must be made as to
how this limited budget is spent. Economic evaluations are carried out within a health technology assessment framework to compare the cost-effectiveness of alternative activities and to consider the opportunity cost associated with their decisions.
By choosing to spend the finite NHS budget upon those treatment options
that provide the most efficient results, society can ensure it does not
lose out on possible health gains through spending on inefficient
treatments and neglecting those that are more efficient.
NICE attempts to assess the cost–effectiveness of potential
expenditures within the NHS to assess whether or not they represent
'better value' for money than treatments that would be neglected if the
expenditure took place. It assesses the cost–effectiveness of new
treatments by analysing the cost and benefit of the proposed treatment
relative to the next best treatment that is currently in use.
Quality-adjusted life years
NICE guidance supports the use of quality-adjusted life years
(QALY) as the primary outcome for quantifying the expected health
benefits associated with a given treatment regime. By comparing the
present value (see discounting)
of expected QALY flows with and without treatment, or relative to
another treatment, the net/relative health benefit derived from such a
treatment can be derived. When combined with the relative cost of
treatment, this information can be used to estimate an incremental cost-effectiveness ratio (ICER), which is considered in relation to NICE's threshold willingness-to-pay value.
As a guideline rule, NICE accepts as cost-effective those
interventions with an incremental cost-effectiveness ratio of less than
£20,000 per QALY and that there should be increasingly strong reasons
for accepting as cost-effective interventions with an incremental
cost-effectiveness ratio of over a threshold of £30,000 per QALY.
Over the years, there has been great controversy as to what value
this threshold should be set at. Initially, there was no fixed number.
But the appraisal teams created a consensus amount of about £30,000.
However, in November 2008 Alan Johnson, the then Secretary of State, announced that for end-of-life cancer drugs the threshold could be increased above £30,000.
The first drug to go through the new process was Lenalidomide, whose ICER was £43,800.
Cost per quality-adjusted life year gained
The following example from NICE explains the QALY principle and the application of the cost per QALY calculation.
A patient has a life-threatening condition and is expected to
live on average for 1 year receiving the current best treatment which
costs the NHS £3,000. A new drug becomes available that will extend the
life of the patient by three months and improve his or her quality of
life, but the new treatment will cost the NHS more than three times as
much at £10,000. Patients score their perceived quality of life on a
scale from 0 to 1 with 0 being worst possible health and 1 being best
possible health. On the standard treatment, quality of life is rated
with a score of 0.4 but it improves to 0.6 with the new treatment.
Patients on the new treatment on average live an extra 3 months, so 1.25
years in total. The quality of life gained is the product of life span and quality rating with the new treatment less the same calculation for the old treatment, i.e. (1.25 x 0.6) less (1.0 x 0.4)
= 0.35 QALY. The marginal cost of the new treatment to deliver this
extra gain is £7,000 so the cost per quality life year gained is
£7000/0.35 or £20,000. This is within the £20,000-£30,000 that is
suggested by NICE to be the limit for drugs to be cost-effective.
If the patient was expected to live only one month extra and
instead of three then NICE would issue a recommendation not to fund. The
patient's Primary Care Trust
could still decide to fund the new treatment, but if not, the patient
would then have two choices. He or she could opt to take the free NHS
standard treatment, or he or she may decide to pay out of pocket to
obtain the benefit of the new treatment from a different health care
provider. If the person has a private health insurance policy the person
could check to see whether the private insurance provider will fund the
new treatment. About 8% of the population has some private health
insurance from an employer or trade association and 2% pay from their
own resources.
Basis of recommendations
Theoretically,
it might be possible to draw up a table of all possible treatments
sorted by increasing the cost per quality-adjusted life year gained.
Those treatments with lowest cost per quality-adjusted life year gained
would appear at the top of the table and deliver the most benefit per
value spent and would be easiest to justify funding for. Those where the
delivered benefit is low and the cost is high would appear at the
bottom of the list. Decision makers would, theoretically, work down the
table, adopting services that are the most cost effective. The point at
which the NHS budget is exhausted would reveal the shadow price,
the threshold lying between the CQG gained of the last service that is
funded and that of the next most cost effective service that is not
funded.
In practice this exercise is not done, but an assumed shadow
price has been used by NICE for many years in its assessments to
determine which treatments the NHS should and should not fund. NICE
states that for drugs the cost per QALY should not normally exceed
£30,000 but that there is not a hard threshold, though research has shown that any threshold is "somewhat higher" than being in the range £35,000 - £40,000.
The House of Commons Health Select Committee,
in its report on NICE, stated in 2008 that "the (...) cost-per-QALY it
uses to decide whether a treatment is cost-effective is of serious
concern. The threshold it employs is not based on empirical research and
is not directly related to the NHS budget, nor is it at the same level
as that used by Primary Care Trusts (PCTs) in providing treatments not
assessed by NICE, which tends to be lower. Some witnesses, including
patient organisations and pharmaceutical companies, thought NICE should
be more generous in the cost per QALY threshold it uses, and should
approve more products. On the other hand, some PCTs struggle to
implement NICE guidance at the current threshold and other witnesses
argued that a lower level should be used. However, there are many
uncertainties about the thresholds used by PCTs." It went on to
recommend that "an independent body should determine the threshold used
when making judgements of the value of drugs to the NHS."
Criticism
The
work that NICE is involved in attracts the attention of many groups,
including doctors, the pharmaceutical industry, and patients. NICE is
often associated with controversy, because the need to make decisions at
a national level can conflict with what is (or is believed to be) in
the best interests of an individual patient.
Approved cancer drugs and treatments such as radiotherapy and chemotherapy
are funded by the NHS without any financial contribution being taken
from the patient. Where NICE has approved a treatment, the NHS must fund
it. But not all treatments have been assessed by NICE and these
treatments are usually dependent on local NHS decision making. In the
case of cancer the Cancer Drugs Fund was set up in 2011 after complaints about NICE decisions on new and expensive cancer drugs with limited benefits. Treatment for fertility problems are approved but not always funded by clinical commissioning groups and they may cap the number of rounds.
NICE has been criticised for being too slow to reach decisions. On one occasion, the Royal National Institute of Blind People said it was outraged over its delayed decision for further guidance regarding two drugs for macular degeneration
that are already approved for use in the NHS. However the Department of
Health said that it had 'made it clear to PCTs that funding for
treatments should not be withheld simply because guidance from NICE is
unavailable'.
Some of the more controversial NICE decisions have concerned donepezil, galantamine, rivastigmine (review) and memantine for the treatment of Alzheimer's disease and bevacizumab, sorafenib, sunitinib and temsirolimus for renal cell carcinoma.
All these are drugs with a high cost per treatment and NICE has either
rejected or restricted their use in the NHS on the grounds that they are
not cost-effective.
A Conservative shadow minister once criticized NICE for spending
more on communications than assessments. In its defence, NICE said the
majority of its communications budget was spent informing doctors about
which drugs had been approved and new guidelines for treatments and that
the actual cost of assessing new drugs for the NHS includes money spent
on NICE's behalf by the Department of Health. When these were added to
NICE's own costs, the total cost of the technology appraisal programme
far outstrips the cost of NICE communications.
A report from the University of York Centre for Health Economics written by Karl Claxton
in February 2015 suggested that the maximum threshold, currently around
£30,000 a year, for judging a medicine cost-effective should be more
than halved. They found that any intervention costing more than £13,000
per Quality-adjusted life year risked causing more harm than good by denying cost effective treatment to other patients.
The institute's approach to the introduction of new oral therapy for Hepatitis C has been criticised. Sofosbuvir was approved in 2015. It costs about £30,000 for 12 weeks treatment. NHS England
established 22 Operational Delivery Networks to roll out delivery and
proposes to fund 10,000 courses of treatment in 2016-17. Each has been
given a “run rate” of how many patients they are allowed to treat. This
is the NHS’ single biggest new treatment investment this year. In the North East London network patients with cirrhosis or fibrosis go to the front of the queue and three new patients at the Grahame Hayton Unit at the Royal London Hospital start treatment each month. Those without such complications may faced considerable delays before they start treatment.
