INTRODUCTION

  1.  Prior to the establishment of NICE in 1999, it was widely accepted that there were unacceptable levels of variation in access to specific treatments. The Department identified a number of factors that contributed to these variations, including;

—  the absence of clear standards of care for the NHS;

—  the lack of a coherent approach to the assessment of good practice and what works best; and

—  slow and inconsistent uptake of effective new treatments. [1]

  2.  NICE was established, as part of a wider quality improvement strategy, to help minimise inequity of access to healthcare by addressing variations in practice, to be a national source of robust clinical guidance and to speed up the uptake of cost-effective new medicines and other interventions in the NHS.

  3.  NICE has evolved considerably since the 2001 Health Committee inquiry. It has developed an international reputation and consolidated its position as a widely supported source of robust guidance on care pathways and the clinical and cost effectiveness of different interventions. NICE guidance has helped to secure faster and more consistent access for patients to important new treatments. The Department considers this a major achievement on which the Institute and the thousands of individuals who have contributed to its work should be congratulated.

  4.  NICE's remit has been expanded since the previous Health Committee and now has a number of new and developing areas of work in addition to its technology appraisals and clinical guidelines. NICE now issues guidance on the safety and efficacy of interventional procedures and, since taking on the work of the Health Development Agency in April 2005, NICE has developed a public health work programme and issued its first pieces of public health guidance.

  5.  NICE has a high degree of operational independence from the Department of Health and is responsible for developing its methodology and guidance independently. The Department sets NICE's budget, holds it to account for delivery of its business plan and refers topics to NICE for its work programmes.

Why NICE's Decisions are Increasingly being Challenged, Whether Public Confidence in the Institute is Waning and, If So, Why?

  6.  The Department does not accept the premise that NICE appraisals are increasingly being challenged and the proportion of appeals being appealed has not increased.

  7.  The Department is aware that a high proportion of "negative" technology appraisals are appealed against, but this should not be seen as an indictment of NICE's processes. It is not surprising that negative appraisals are challenged as considerable revenues are at stake for the pharmaceutical companies concerned and appraisals inevitably attract a high level of interest from patient representative groups. NICE's work is both complex and controversial, and the appeal process is an important guarantee of quality and objectivity in NICE's appraisal activity. We welcome the changes NICE has made since 2001 to improve the transparency of the appeal process, specifically by hearing appeals in public.

  8.  The number of appeals that have been made against NICE guidance should also be looked at in the context of the number of appraisals that NICE has carried out. As at the end of February 2007, NICE has published 119 technology appraisals and, of these, 39 have been the subject of an appeal. NICE has published a further 45 clinical guidelines and 4 pieces of public health guidance.

Confidence in NICE

  9.  The Department fully supports NICE's role in issuing independent advice based on an objective assessment of the evidence, is confident in the Institute's ability to deliver its work programme to a high standard and believes there remains a high level of respect for and confidence in NICE more broadly.

  10.  Media and public attention has focussed on NICE's more controversial technology appraisals. It is important to recognise, however, that much of NICE's work, whether technology appraisals, clinical guidelines or emerging public health guidance, is widely welcomed by patients, professionals and other stakeholders. NICE also has significant international standing and is widely regarded as a world leader in its field. NICE's appraisal methodology has been commended by the World Health Organisation, and its publications have a large international audience.

  11.  Given NICE's role in ensuring the clinically and cost-effective use of finite public resources, it is inevitable that it will sometimes produce guidance that is seen as unfavourable by some stakeholders, including patient groups. Clearly, in some instances, this will lead to criticism from sources with a particular interest in that treatment, but we note that where such criticisms are expressed they are often tempered with a statement of overall support for NICE and its work.

  12.  NICE faces a challenge in explaining decisions based on complex health economics to a wider audience. We welcome the efforts that NICE has made to ensure that the reasons for controversial decisions are properly understood, but this will continue to be an area where a sustained communication effort is required.

  13.  It is important that NICE's processes remain transparent to stakeholders and that the rationale for its conclusions is clear. Since 2001, NICE has taken steps to improve the openness of its appraisal process by, for example, holding appeals in public and publishing decisions and considerations on the NICE website. NICE has also significantly improved the design and accessibility of its website to make information easier to find, in particular, from a service user perspective.

  14.  Recent changes to the topic selection process have also been made to further increase the openness and accessibility of the process to stakeholders.

NICE's Evaluation Process—and Whether any Particular Groups are Disadvantaged by the Process

  15.  The Department is not aware that any particular groups are disadvantaged by NICE's methodology.

  16.  The appraisal process and methodology is developed with the involvement of stakeholders and, following a full consultation, revised documentation was published in 2004. NICE will be carrying out a further scheduled review of its technology appraisal methodology during 2007, and this too will be subject to a public consultation phase.

  17.  The Department considers it right that NICE's process and methodology is the subject of continued development and debate and welcomes the open and consultative approach NICE takes to the development of its work.

The Speed of Publishing Guidance

Single Technology Appraisal process

  18.  In collaboration with the Department, NICE has developed a Single Technology Appraisal (STA) process, which is designed to speed up the issuing of guidance to the NHS, without compromising the quality and robustness of NICE's guidance.

  19.  The STA process is used where a single product is being appraised against the standard treatment for use in a single indication, and where there is a manageable evidence base. A Multiple Technology Appraisal (MTA) process is retained for more complex appraisals where multiple treatments are being assessed together. The target timescale for publishing guidance carried out as STAs is 12 months from the date the topic was referred. This compares with 24 months for MTAs.

  20.  The STA process was announced in September 2005. One of the first products to be appraised under the STA process was Herceptin for early-stage HER2-positive breast cancer, for which guidance was published in August 2006.

  21.  Most of the appraisals referred to NICE by the Department as part of the 13th wave and all the "minded" referrals for the 14th wave are to be carried out as STAs.

Clinical guidelines and short clinical guidelines

  22.  NICE has reduced the timeline for the production of full clinical guidelines and, following the success of STAs, NICE and the Department of Health are also developing a Short Clinical Guidelines Programme. It is proposed that NICE will develop two short clinical guidelines a year, and the target timescale for producing a short clinical guideline will be 9 to 11 months, which compares with 24 months for a full clinical guideline.

The Appeal System

  23.  Since the previous Health Committee inquiry, changes have been made to NICE's appeal system to improve the transparency of the process.

  24.  The appeal system is an essential element of the appraisal process, and it is clearly important that appeals are carried out in an open and transparent way. To this end, NICE now conducts its appeals in public and the Appeal Panel's conclusions and considerations are published on its website. The Appeal Panel is chaired by a non-executive member of NICE, who will have had no prior involvement in the appraisal in question, and its membership includes independent industry and lay representatives.

  25.  The Department believes that the appeal process is fit for purpose but notes that, as with other aspects of NICE's work, it will continue to develop. We understand that there will be an opportunity for stakeholders to comment on the appeal process during NICE's methodology review and subsequently the review of its appraisal process.

  26.  There have been 17 occasions when aspects of appeals have been upheld and the Appraisal Committee has been asked to reconsider its advice.

Comparison With the Work of The Scottish Inter-Collegiate Guidelines Network (SIGN)

Introduction

  27.  The development of guidance for the NHS in Scotland is a matter for the devolved administration. It is an inevitable consequence of devolution that, in some cases, countries will have different bodies performing similar roles.

  28.  Whereas NICE issues guidance on both individual treatments (technology appraisals) and pathways of care (clinical guidelines), these two types of guidance are developed by two different organisations in Scotland:

—  The Scottish Medicines Consortium (SMC) is responsible for issuing advice on specific drugs to the NHS in Scotland.

—  SIGN is responsible for developing clinical guidelines, which provide recommendations on the management of clinical conditions.

  29.  The Department believes it is important to note that there are a number of important differences between the appraisal processes of the SMC and NICE. For example, the NICE process involves a more extensive consultation than that undertaken by the SMC. NICE's STA process has the potential to produce guidance to a similar timescale to the SMC.

  30.  There are also a number of key differences between NICE's clinical guidelines process and SIGN's. In particular, NICE consults more thoroughly with stakeholders during the development and NICE's clinical guidelines consider both clinical and cost effectiveness issues, whereas SIGN focuses on clinical effectiveness.

Status of SMC and SIGN guidance in England

  31.  NICE liaises with SIGN to review topics to minimise duplication of effort between the two organisations and the Department has drawn attention to the SMC as a potential source of information where guidance from NICE is not available.

  32.  However, the advice produced by the SMC and SIGN is not equivalent to the guidance produced by NICE. The Department does not therefore believe that there is a strong case for giving SMC or SIGN advice formal status in England.

  33.  The Department appreciates that there is a public expectation that there will be coordination and information-sharing between the guidance producing bodies in different parts of the UK, but the devolved nature of responsibility for NHS policy means that it is unrealistic and inappropriate to expect that such guidance should be identical in every case. We note that NICE appraisals are adopted automatically in Wales, and may supersede some SMC guidance in Scotland.

The Implementation of NICE Guidance—Both Technology Appraisals and Clinical Guidelines—Which Guidance is Acted On, Which Is Not and the Reasons for This

Statutory position of NICE guidance

  34.  NICE technology appraisals are covered by a three month funding direction, which means that, where NICE has recommended a treatment, PCTs are under a statutory obligation to provide funding to make the intervention normally available within three months of guidance being issued. The direction may be amended or waived where it is felt the NHS will need longer to implement the recommendations eg staff training requirements.

  35.  The three month funding direction does not apply to clinical guidelines and public health guidance. It is appropriate for there to be a developmental approach to the implementation of these guidance products that recognises their more complex nature and allows NHS organisations to approach implementation in the most appropriate way.

  36.  There are a number of other non-statutory levers and incentives which support the implementation of NICE guidance.

  37.  NICE guidance is included in "Standards for Better Health"[2] published by the Department of Health. The standards fall into two categories:

(i)  core standards—which set out the minimum level of service patients and service users have a right to expect. The core standards include adherence to NICE's technology appraisals and interventional procedures guidance; and

(ii)  developmental standards—which signal the direction of travel and provide a framework for NHS bodies to plan the delivery of services which continue to improve in line with increasing patient expectations. The developmental standards include clinical guidelines and public health guidance.

  38.  It is the responsibility of local NHS organisations to ensure that they are meeting the standards. They are performance managed by the Strategic Health Authorities (SHAs) and organisations' own assessments of compliance are independently validated by the Healthcare Commission, which publishes progress as part of the annual health check.

  39.  NICE liaises with the Department of Health to feed the cost implications of NICE guidance into the Payment by Results tariff, which provides an incentive for PCTs to commission the services and treatments recommended by NICE. However, coverage of NICE guidance is not universal and this area of work is still developing.

  40.  The Department of Health seeks to ensure that wherever possible NICE guidance underpins specific Quality and Outcomes Framework (QOF) indicators and that there is no contradiction between the QOF and NICE guidance. NICE is working with NHS Employers (who negotiate changes to the QOF with the General Practitioners Committee of the BMA) to map QOF indicators onto NICE guidance. This process aims to ensure that QOF indicators are compatible with NICE guidance and that any apparent differences arising from the different purposes of NICE guidance and QOF are explained.

  41.  In 2004, NICE established an Implementation Directorate and now develops and issues a range of new tools to support existing NICE guidance. These tools have been developed in collaboration with the Department and are designed to help NHS commissioners to make funding decisions. They include:

(i)  Implementation and costing templates.

(ii)  Commissioning guides.

(iii)  Evaluation and review of NICE implementation evidence (ERNIE) database.

Other aspects of support for implementation

  42.  NICE is also developing a range of other guidance and tools for the NHS on disinvestment and reducing ineffective procedures, which support its implementation work. This work includes:

(i)  Guidance on treatments of doubtful effectiveness, which may be inappropriate or unnecessary for some or all patients.

(ii)  Recommendation reminders, which identify and more actively promote existing NICE recommendations, many of which have the potential to deliver savings.

Improvements in implementation

  43.  In 2006, Professor Mike Richards, the National Clinical Director for Cancer, published a report on the "Review of NHS Usage of Cancer Drugs Approved by NICE"[3] which showed significant progress in reducing variation in access to NICE approved cancer drugs across the country in the past two years. The report concluded that there has been a 47% increase in use of key cancer drugs since the last assessment in 2004, and that geographic variation in the use of these drugs has decreased.

  44.  Professor Richards' earlier 2004 report[4] on the uptake of NICE approved drugs concluded that, where uptake is slow, this could largely be attributed to factors such as the approaches of individual clinicians rather than availability of resources.

  45.  The Audit Commission[5] and Healthcare Commission[6], [7] have also published reports that have highlighted the positive impact of NICE guidance and that include recommendations that will assist the NHS in improving performance.

Impact on innovation

  46.  The Department believes that NICE is a powerful lever for encouraging the uptake of innovative and cost-effective new drugs and other interventions within the NHS, and that it contributes an additional incentive for such innovation. Measures to support the uptake of NICE guidance are an important strand of the work of the Long-Term Leadership Strategy which is being taken forward in partnership with the UK pharmaceutical industry.

CONCLUSION

  47.  The Department believes that NICE has achieved a tremendous amount since 1999 and that it enjoys a high degree of respect both domestically and internationally. NICE's work is controversial and inevitably attracts comments and criticism, but we need to take a balanced view of NICE's activities and acknowledge the positive difference that NICE's work has made to the care of thousands of patients and in encouraging the NHS to take up innovation. NICE will continue to develop its processes and methods. The Department believes it is right that these issues continue to be the subject of informed debate.

Department of Health

March 2007

2   "Standards for better health", Department of Health, July 2004. Back

3   "Usage of cancer drugs approved by NICE", Department of Health, September 2006. Back

4   "Variations in usage of cancer drugs approved by NICE", Department of Health, June 2004. Back

5   "Managing the financial implications of NICE guidance", Audit Commission, September 2005. Back

6   "The best medicine: the management of medicines in acute and specialist trusts", Healthcare Commission, January 2007. Back

7   "Talking about medicines: the management of medicines in trusts providing mental health services", Healthcare Commission, January 2007. Back