Select Committee on Health Minutes of Evidence

Memorandum by Alzheimer's Society (NC 2)


  1.1  The Alzheimer's Society is pleased to submit this memorandum to the Health Select Committee inquiry into the National Institute for Clinical Excellence (NICE). The Alzheimer's Society is the leading care and research charity for people with dementia and their carers. It was founded in 1979 as the Alzheimer's Disease Society. It provides information and education, support for carers, and quality day and home care. It funds medical and scientific research and campaigns for improved health and social services and greater public understanding of dementia. In 2001 it was Healthcare and Research Charity of the Year and won the IPPR/Guardian award for public involvement in health.

  1.2  Dementia is one of the most significant health and social care challenges of this century. Alzheimer's disease is the most common cause of dementia. There is no known cure for Alzheimer's but the anticholinesterase treatments, donepezil (Aricept), rivastigmine (Exelon) and galantamine (Reminyl) represent the first and only effective pharmaceutical treatments for Alzheimer's to date. The Alzheimer's Society has argued that people with dementia and their carers should have access to these drugs through the NHS since they were first licensed in the UK in 1997. This class of drugs has been subject to rationing on a massive scale in the UK. Although NICE issued guidance that these drugs should be made available on the NHS in January 2001 the Society continues to receive calls from people with dementia, their carers as well as their doctors, who experience difficulties in accessing these treatments.

  1.3  The Alzheimer's Society welcomed the establishment of NICE as set out in the government's white paper A First Class Service.[27] The Society argued at that time that the decisions of NICE would only be effective if they were mandatory. Our experience of the failure of many health authorities to implement NICE guidance on anticholinesterase inhibitors confirmed our fears and we therefore welcome the government's recent announcement (5.12.01) that NICE recommendations must be followed.

  1.4  The Alzheimer's Society supports the principles of NICE. As patients we want clinically effective treatments, as citizens and "owners" of the heath service we all benefit from cost effectiveness. It is important to note that patient groups are not always in favour of treatments. For instance were NICE to undertake a review of neuroleptics in the treatment of dementia the Alzheimer's Society would submit evidence of their harmful side effects, over use and misuse. No one wants to have a treatment that does not work.

  1.5  Our experience of the NICE appraisal process raises a number of questions about the nature and quality of evidence, the processes by which evidence is collected and appraised. Although our evidence contains some strong criticisms of NICE we acknowledge that we participated in one of the earlier appraisals and that changes will subsequently have been made.


  NICE finds itself inevitably operating in the context of changing attitudes to patients and their relationship to health care professionals, and at the centre of a debate about what constitutes clinical evidence and how it should be put into practice[28]. Traditionally research has neglected the patient experience of treatment and this is particularly so in the development of pharmaceuticals where regulatory frameworks and financial imperatives mean that patients are never involved in determining desirable clinical outcomes or appraising effectiveness. Most measures employed by health professionals and researchers do not reflect the views of people affected by the disease.[29]

  2.2  Very recently, and usually post hoc, patient and carer evaluations of health technologies have become available. User-defined outcomes reveal what patients and carers think about a disease, treatment or service provision in terms that are meaningful for their own lives. For example, the outcome measures used in the random controlled trials required for obtaining licensing approval for the new drugs for Alzheimer's demonstrated that a person scored better in cognitive functioning tests. It was our own research that showed that people with dementia and their carers placed a greater value on the drugs' capacity to restore a person's confidence, improve mood and reduce fear and anxiety than on improvements in short-term memory.

  2.3  This kind of evidence is vital if NICE is to come to proper decisions on the basis of a fully rounded view of new health technologies. We welcome the commitment that NICE has given from the start to the user and carer perspective but perceive that NICE has had difficulty in discovering for itself what the patient perspective is and which organisations properly represent that perspective.

  2.4  Initially NICE appeared to believe that evidence from patient organisations would be subjective and anecdotal in contrast to "professional" evidence which would be objective and generalisable. Patient organisations were urged to send in short submissions with the implication that otherwise they would be wasting NICE's time. It has taken the work of the informal grouping Patients Involvement in NICE (PIN) to persuade NICE to recognise that patients are one set of experts able to contribute to the appraisal process and that many patient-led organisations are themselves significant funders and managers of high quality research.

  2.5  In preparing our submission to NICE in early 2000 we decided that we should submit our evidence to NICE in the manner we thought best represented the knowledge and experience of people with dementia and their carers and the expertise of the Alzheimer's Society. Through our research programme we commissioned independent research into the views of people with dementia and their carers. We used the results of this work plus reviews of research into the economic cost of Alzheimer's disease and the impact of the new drugs as the basis of our submission. We also included discussion of the validity of Quality of Life measures as applied to people with dementia. It was our research that showed that qualitative changes in mood appear to be given much greater priority by people with dementia than improvement of short-term memory. This helped to ensure that NICE evaluated the clinical and cost effectiveness of the anticholinesterase treatments using criteria which drew on the priorities of patients and carers.

  2.6  Our submission of evidence, vital though it was to the people we represent, felt like a shot in the dark. We did not know how our evidence would be weighted in relation to other evidence or indeed precisely what other evidence would be sought. How was NICE to evaluate a two point improvement on a cognitive scale alongside responses from our survey of over 2,000 people with dementia and their carers, such as a person with Alzheimer's, aged 58 years of age, who said that the drugs "make me feel more alive - move involved with things" or a carer who reported that the dugs had enabled his wife to "remember how to knit"?

  2.7  The Alzheimer's Society's submission to NICE[30] included the words and views of many people with Alzheimer's as well as their carers. Five years ago, we would not have been in a position to do this. The ability of people with Alzheimer's to articulate clearly what it is like to have the disease is a recent phenomenon. The introduction of the drugs for Alzheimer's has directly enabled people affected by the disease to continue to communicate their views to others. Any new health technology has to be evaluated in terms of the significance which patients and carers place on its benefits. Further clarification of how decisions are made and how different types of evidence are evaluated is necessary. NICE found that the drugs for Alzheimer's were effective. We believe that the Society's submission was an important contribution to the evidence base, but we have no way of confirming this.


  3.1  The decisions that NICE makes can only be as good as the evidence which it receives. Evidence from the pharmaceutical industry or from other health technology manufacturers will be straightforward enough— randomised clinical control trials subject to objective evaluation. The manufacturers' evidence as suggested above is however likely to be narrow in its scope since it is directed to the achievement of a product licence. Other evidence is necessary but what and from whom?

  3.2  NICE commissions from independent university sources a comprehensive and objective review of the evidence. If the quality of this review is inadequate the central core of the decision making process is put at risk. In the case of the appraisal of drugs for Alzheimer's disease the review was carried out by the Wessex Institute for Health Research and Development, University of Southampton.

  3.3  The Wessex Institute report failed to be comprehensive or objective. It contained many assertions unaccompanied by evidence and conclusions not justified by the evidence presented. It was inconsistent in its treatment of different types of evidence citing unpublished and unreferenced observational data as well as properly conducted trials. References in the report were incomplete, some were incorrect, relevant publications were omitted. The report was not independently peer reviewed and made recommendations beyond its brief.

  3.4  NICE appeared to have no mechanism by which the quality of conflicting evidence could be challenged externally. Unasked the Society submitted a critique of the Wessex Institute report. We have no idea if this was noted or even read. We do not know if the Appraisal Committee had come to the same conclusions that we had. In addition, the Society is aware of important evidence that was submitted for the appraisal but was not listed under "sources of evidence" in the final guidance. The Society questioned the apparent omission of these important submissions but NICE was unable to confirm whether or not the appraisal committee had seen these documents from experts in the field.

  3.5  The source of expertise relied on by NICE is also a matter for concern. The list of organisations to be consulted by NICE on the drugs for Alzheimer's disease included the Chartered Society of Physiotherapists (CSP) but did not include the Royal College of Psychiatrists. When we pointed out to NICE that this appeared to be an error (we believed that the Physiotherapists had merely been carried forward from the list for Multiple Sclerosis where they do have relevant expertise) NICE denied it. They justified the inclusion of the CSP on the grounds that they are health professionals. A justification that does not stand up to even cursory scrutiny. While NICE needs to collect evidence from a wide range of bodies it should be from experts with relevant experience of the patient group concerned.

  3.6  NICE has also shown uncertainty in its choice of patient organisations. It seems unable to distinguish between research charities and patient organisations (many of us of course are both). Research charities do have relevant expertise but unless they are user/carer "owned" membership organisations they cannot represent the patient perspective. For instance NICE insisted in treating the Alzheimer's Research Trust, a small specialist research funder based in Cambridge as a "patient organisation." It is not. It has relevant medical and scientific expertise and we welcomed its contribution to the evidence but it is not a membership body and does not claim to speak for patients. Specialist research charities may well have an important contribution to make to NICE appraisals but if NICE is to categorise the bodies which it invites to submit evidence it should do so accurately and consistently.


  4.1  Increasingly the new health technologies which NICE has to appraise will aim to produce improvements in quality of life rather than a cure for a condition. This will particularly be so in the development of new treatments for neurological diseases. It is not surprising therefore that treatments for Multiple Sclerosis, Motor Neurone disease and Alzheimer's disease appraised by NICE have all been controversial.

  4.2  There is considerable difficulty in measuring quality of life in an objective way. Much research on the development of appropriate methodologies is being done but NICE needs to be clear about which are considered satisfactory and why.

  4.3  Particular concern has been expressed about the use of QALYs—Quality Adjusted Life Years as a measure of cost effectiveness by NICE. QALYs have been criticised as utilitarian, inherently weighted against those experiencing illness in later life, insensitive to mental health problems and unable to quantify benefits to the quality of life and health of carers from treatments given to those they care for. Disease specific measures of quality of life need to be used by NICE in conjunction with QALYs and a clear rationale give by NICE to explain how they are used and what weight have been attached to them in the formulation of their conclusions.

    Bosenquet et al estimate that the annual cost of Alzheimer's is £5.4-£5.8 billion.[31] But, the NHS bears relatively little of the cost of this illness. This means that any evaluation of cost-effectiveness that focuses only on the costs and benefits to the NHS would be seriously flawed. Moreover, such an analysis would fail to highlight the perverse incentives that may arise. Many long-term conditions, particularly those that affect older populations, do not lend themselves well to cost-effectiveness studies, particularly where Quality Adjusted Life Years (QALYs) are employed. NICE's evaluation of cost effectiveness needs to be extended to include the costs to social services and to patients and carers.


  5.1  The implementation of NICE guidance on the drugs for dementia has been uneven and inconsistent. The Society has continued to receive calls from people with Alzheimer's and their carers unable to obtain the drugs or who have to pay for them privately. A large variation on spending on these drugs between health authorities persists and if anything this has increased since NICE recommended their use on the NHS in January 2001.

  5.2  The prescription rates of all three drugs have increased substantially. However, data supplied by IMS Health (Appendix 1, Figure 1) shows that those health authorities which were already prescribing the drugs pre-NICE guidance continue to do so, increasing at the same rate (despite starting from a higher base) as those health authorities that had restricted their use in the past. For patients this means an increase in the "postcode lottery", as some health authorities aim to provide the drugs for all people that may benefit while others continue to prescribe at very low levels.

  5.3  Interestingly, prescription rates are not affected by the presence of memory clinics. This is important because some health authorities have argued that they cannot implement the NICE guidance until they have established new memory clinics. Indeed, the evidence from IMS Heath (Table 2) shows a lower prescribing rate in areas with memory clinics. In other areas, health authorities claim that they are implementing the NICE guidance, but continue to restrict access, for example through the operation of waiting lists or by redefining NICE's criteria of those patients who might benefit. It is not clear how far the recent government's announcement on implementation will address these issues.

  5.4  Finally, in some areas of the country in particular, people with Alzheimer's appear to have been denied access to the new drug treatments completely. In particular, the Alzheimer's Society has made known its opposition to one clinical trial in the West Midlands: AD2000. Patients who might benefit from the treatment are still being given the option of entering the trial and are informed that entry means that some patients will receive a placebo. The Society believes the continuation of any trial of these drugs for people in the early to moderate stages of Alzheimer's is unethical.


  The Alzheimer's Society welcomes the creation of NICE as a mechanism for appraising clinical and cost effectiveness of new health technologies. Recommendations and guidance from NICE will only be effective however if the key stakeholders, the patients, the physicians and the manufacturers have confidence in its decisions.

  Issues which need to be addressed are:

    1.   Quality and comprehensiveness of evidence. Particularly the quality assurance of the "independent" reviews of evidence commissioned by NICE.

    2.  Acknowledgement of the expert patient perspective and mechanisms for ensuring patient evidence is considered.

    3.  Transparency of process and openness about the criteria and weighting used in decision-making.

    4.  Consistency in identifying those who should contribute evidence and in how their evidence is treated.

    5.  Poor administration and support for patient groups.

    6.  Equity of access to the NICE process particularly for smaller patient organisations which do not have the resources of larger charities.

  Alleviating the burden and distress of caring for Alzheimer's has important economic, social and humanitarian implications. Anticholinesterase drugs have an important role to play in improving the quality of care and support for both the person with Alzheimer's and their carers.

January 2002

27   Department of Health, June 1998 A First Class Service: Quality in the New NHS, London. Back

28   Cayton H and B Hanley, 2001 "Improving research through consumer involvement" in M Baker and S Kirk (Eds) Research and development for the NHS, Radcliffe Medical Press, Abingdon: 195-207. Back

29   Kelson, M 1999 Patient defined outcomes: a paper prepared on behalf of the Clinical Outcomes Group patient subgroup, College of Health, London. Back

30   Alzheimer's Society, 2000, Appraisal of the drugs for Alzheimer's disease, submission to the national Institute for Clinical Excellence, London available at Back

31   Alzheimer's Society, 2000, Appraisal of the drugs for Alzheimer's disease, submission to the national Institute for Clinical Excellence, London available at Back

previous page contents next page

House of Commons home page Parliament home page House of Lords home page search page enquiries index

© Parliamentary copyright 2002
Prepared 8 July 2002