BACKGROUND

  1.  RNIB is the leading UK charity helping blind and partially sighted people. One of our Royal Charter objectives is the prevention of blindness and in this context we are involved in NICE's appraisal of two new powerful drugs for treating wet Age-related Macular Degeneration (AMD), the main cause of registerable blindness in the UK. In addition, we are consultees for the development of guidance on the treatment and management of glaucoma and are members of the PiN (Patients in NICE) group.

  2.  We would like to limit our observations to a number of the questions set out by the Committee in its press notice of 2 February, which reflect our direct involvement with NICE.

EXECUTIVE SUMMARY

Why are NICE's decisions increasingly being challenged?

  3.  As a patient organisation RNIB has had to challenge NICE's decisions because of its failure to take adequate account of the impact of sight loss on patients who have conditions that can be treated.

  4.  In addition, we have challenged NICE's decisions because of the lack of consideration of costs of disease that are outside the NHS and Social Services remit.

NICE's evaluation process

  5.  We feel that the evaluation process is very thorough and reasonably open. However, it is likely that smaller charities representing orphan diseases will find it much more difficult, if not impossible to contribute to any appraisals.

The speed of publishing guidance

  6.  The speed of publishing guidance is a very serious issue because of the uncertainty created from the time when a new treatment is licensed for use in the UK until the time when NICE issues its guidance. Unfortunately, this has led to a post-code lottery with some PCTs providing funding and others not. In areas where funding is not available, having failed in their appeal, patients are then forced to opt for private treatment.

  7.  Against this background it is not surprising that many people are questioning the time it takes for NICE to come to a decision, particularly when the Scottish Medicines Consortium manages to issue guidance on new drugs within three months of their marketing authorisation.

Implementation of NICE guidance

  8.  In a report published in 2005 the National Audit Office found that only 26% of NHS bodies participating in their study regularly undertook horizon scanning to assess the financial impact of forthcoming guidance on their organisation.

  9.  At present neither NICE, nor the NHS are collecting relevant data on implementation. The only available data is that provided by the drug manufacturers. The systematic collection of data by the NHS would facilitate an assessment of gaps in the provision of treatment in different parts of the country.

OUR DETAILED COMMENTS

WHY ARE NICE'S DECISIONS INCREASINGLY BEING CHALLENGED?

  10.  In 2003 RNIB mounted a robust challenge to NICE over its decision to reject the use of photodynamic therapy for the treatment of wet Age-related Macular Degeneration on the NHS. The main reasons for this were:

—  NICE's failure to take adequate account of the impact of sight loss through AMD on patients with the condition.

—  Lack of consideration of costs of disease that are outside the NHS and Social Services remit.

NICE's failure to recognise the impact of sight loss on a person's quality of life

  11.  As a patient organisation RNIB aims to gauge patient views in discussions about the availability of treatments while at the same time using an evidence-based approach to establish our general policy. From a patient perspective, the need to make economic decisions about the availability of treatments is hard to accept. Patients affected by a condition that is treatable feel that they should not be forced to pay for private treatment. In the case of AMD these are often elderly patients who have paid taxes all their lives and expect treatment on the NHS.

  12.  While most people feel that they have right to treatment they also recognise that the NHS does not have unlimited resources and that some treatments need to be prioritised over others. What they do not accept is an assumption that sight loss is not a severe disability, that people can adapt to losing their sight and that therefore treatment is not a priority. We contend that the main tool used by NICE to assess cost effectiveness (QALY values) does not adequately reflect the severe impact sight loss has on a person's quality of life.

  13.  This view is supported by a number of studies. In the case of AMD a literature review commissioned by the AMD Alliance International strongly questions the use of QALY values to measure quality of life in AMD patients. The authors of the review contend that: "The QALY values obtained using time trade-off and standard gamble methods are not measuring quality of life and such measures give no impression of the ways in which AMD impacts on a person's life. There are many reasons why a person may not want to relinquish any years of life in spite of serious visual impairment but this does not imply that they are content with the present situation or that their quality of life would not be much better without their vision problems." [125]In addition the use of QALY values puts people with long-term conditions at a disadvantage over people with life-threatening conditions. Our concern is that drugs that extend life will always achieve higher values even if they do not guarantee a high quality of life.

  14.  The concerns outlined above compel us to call on NICE to review the tools it uses to assess quality of life gains as part of its technology assessments.

Lack of consideration of costs of disease that are outside the NHS and Social Services remit

  15.  NICE appraisals only take account of costs of non-treatment to the NHS and Social Services. In the case of sight loss this ignores a whole range of additional costs to the economy due to the provision of care to blind and partially sighted people. These include benefits, tax allowances, transport, education and training costs, employment services, informal care and productivity loss. [126]As the retirement age is being raised these costs will increase. Not taking them into account is a serious flaw in NICE's decision-making.

EVALUATION PROCESS

  16.  We feel that the evaluation process is very thorough and reasonably open. As a large charity we are fortunate to be able to put the required resources into participating in a consultation. However, it is likely that smaller charities representing orphan diseases will find it much more difficult, if not impossible to contribute to any appraisals. The drawn-out process and the level of technical knowledge required are likely to exclude some patient organisations.

THE SPEED OF PUBLISHING GUIDANCE

  17.  The speed of publishing guidance is a very serious issue because of the uncertainty created from the time when a new treatment is licensed for use in the UK until the time when NICE issues its guidance. The Department of Health has made it clear on numerous occasions that absence of NICE guidance is not a reason for PCTs to avoid taking decisions on the provision of funding for new treatments. PCTs should decide on the basis of available evidence whether individual patients should receive treatment on the NHS.

  18.  Unfortunately, this has led to a post-code lottery with some PCTs providing funding and others not. PCTs are careful not to use the absence of NICE guidance as the sole excuse for not providing treatment. However, more often than not, exceptionality rules applied in case-by-case appraisals represent a de facto ban and appeals are usually unsuccessful since they need to prove that there have been procedural errors or that new evidence supports treatment.

  19.  Having failed in their appeal patients are then forced to opt for private treatment. In fact, in the case of AMD, they have to take that decision even before a PCT has looked at their case because the "window of opportunity" for treatment is very narrow and they risk losing their sight by the time they have gone through the appeals process.

  20.  Against this background it is not surprising that many people are questioning the time it takes for NICE to come to a decision, particularly when the Scottish Medicines Consortium manages to issue guidance on new drugs within three months of their marketing authoriation.

IMPLEMENTATION OF NICE GUIDANCE

  21.  Implementation of NICE guidance and the introduction of the new treatments, if they are recommended by NICE, is not part of the work of the NICE appraisal committee. However, assisting with introduction and monitoring is within the overall remit of NICE as shown on the NICE web site: "NICE has set up a programme to help support implementation of NICE guidance. The implementation team does not get involved in developing the guideline recommendations but works alongside the guideline developers, the communications team and field based teams to:

—  Ensure intelligent dissemination to the appropriate target audiences.

—  Actively engage with the NHS, local government and the wider community.

—  Work nationally to encourage a supportive environment.

—  Provide tools to support putting NICE guidance into practice.

—  Demonstrate significant cost impacts—either costs or savings at local and national levels.

—  Evaluate uptake of NICE guidance.

—  Share learning.

—  Develop educational material to raise awareness of NICE guidance and encourage people to input into its development".

  22.  When NICE finally approved the use of photodynamic therapy for choroidal neovascularisation (wet AMD), having had it under review for two and a half years, a further nine months delay was imposed by the Department of Health because no groundwork had been done on how to introduce the treatment. Although we do not have data on other drugs, we can assume that this is not a unique occurrence. In a report published in 2005 the National Audit Office found that only 26% of NHS bodies participating in their study regularly undertook horizon scanning to assess the financial impact of forthcoming guidance on their organisation.

  23.  Work is currently ongoing to assess the implications for service configuration and delivery if, as expected, NICE approves the new treatments for wet AMD for use on the NHS. However, we are not confident that this will be sufficient to ensure that all eligible patients presenting for treatment will receive funding during the coming financial year.

  24.  Finally, we strongly welcome NICE's aspiration of evaluating the up-take of its guidance and feel that this should be an ongoing process. The problem is that there does not seem to be any independent way of assessing the level of up-take. At present neither NICE, nor the NHS are collecting relevant data. The only available data is that provided by the drug manufacturers. The systematic collection of data by the NHS would facilitate an assessment of gaps in the provision of treatment in different parts of the country.

CONCLUSION

  25.  As a major patient organisation our responsibility is to ensure that people with sight threatening conditions receive the best possible treatment to prevent avoidable sight loss. We recognise that NICE has an important role to play in assessing the safety and efficacy as well as the cost-effectiveness of new treatments to help end the post-code lottery that has bedevilled the provision of treatments in England and Wales. Unfortunately, NICE is playing this role at a time when the ability of the NHS to meet patients' expectations is increasingly being questioned. With or without NICE guidance PCTs have to make difficult choices partly based on evidence but also increasingly determined by the level of pressure they experience from the media and/or patient organisations.

  26.  Increasingly, the ability to lobby for treatments is becoming a deciding factor. This is not where most people would have imagined the NHS ending up and it raises questions about the way prioritisation in the NHS is being handled. There may therefore be a case for additional Government intervention, or perhaps the abolition of £2 billion worth of unnecessary procedures that the Government has identified in efforts to reduce financial pressures.

  27.  However, outside this wider debate NICE needs to continue its role in a more streamlined manner, with shorter consultation periods, redefining quality of life and cost issues and focusing more strongly on implementation.

Barbara McLaughlan

Campaigns Manager, Eye Health and Social Care

RNIB

March 2007

126   Winyard, S: The cost of sight loss in the UK. RNIB campaign report 23 August 2004. Back