Select Committee on Health Minutes of Evidence

Memorandum by the Multiple Sclerosis Society (NC 56)


What is MS?

  1.1  MS is a disease of the central nervous system where myelin, which provides electrical insulation and protection to nerves, is attacked and damaged. MS is unpredictable and can cause a wide variety of symptoms such as loss of mobility, pain, fatigue, vision problems, numbness, loss of balance, depression and mood swings. The disease may progress steadily or involve periods of active disease followed by periods of remission.

  1.2  MS is the most common neurological disorder affecting young people. The UK has one of the highest prevalences in the world, with an estimated 85,000 affected by the disease.

What is the MS Society?

  1.3  The MS Society was established in 1953, and is a UK-wide charity dedicated to supporting people who have MS, as well as providing help to those people's families, friends, carers and colleagues. A significant number of our trustees, staff and volunteers either have MS or a personal connection with MS.

  1.4  The Society provides care and support through services which include running a national helpline, producing publications on MS, supplying welfare grants, funding research, providing MS specialist nurses and delivering respite care.

  1.5  We are a democratic organisation of 45,000 individual members. Our annual budget is £25million.


  2.1  The MS Society has been involved with the appraisal of disease-modifying drugs in MS for over two years—twice the "standard" length of technology appraisals. There is no doubt that the appraisal of these drugs has been a challenge for the Institute. The appraisal of drugs for what is a long-term and complex condition has tested the robustness of the Institute's approach, and thrown into sharp relief a number of issues which need to be addressed if the Institute is to meet the agenda set for it in A First Class Service. We are also involved in developing NICE guidelines for MS services.

  2.2  There is a gap between the Institute's rhetoric about openness, transparency and the patient perspective and the actual nature of its structures and practices. This gap undermines the credibility of guidance from the Institute among patients and patient representative organisations.

  2.3  The Institute's approach is dominated by health economics, and is characterised by a heavy reliance on results from randomised controlled trials (RCTs). Such trials are necessarily short term and cannot provide concrete evidence for the drugs which will be used over the long-term. The Institute has not developed a suitable methodology for assessing the cost-effectiveness of long-term interventions.


  3.1  A cultural shift is needed towards a position where the critical nature of the decisions taken by the Institute and the resulting need to command the respect of the public is reflected in its structures, processes and the way in which it conducts its day-to-day business.

  3.2  Appointments to the Institute's Partners Council, Board and Appraisal Committee should be advertised and subject to open recruitment processes.

  3.3  The deliberations of the Appraisal Committee should be opened up by allowing patient representative organisations to observe discussions, and by posting detailed notes of the Committee's meetings on the Institute's website.

  3.4  The current Appeal Panel should be replaced by a lawfully constituted independent body.

  3.5  A public review of the Institute's approach to the use of health economics should be conducted. This should examine, for example, the threshold for cost-effectiveness which has been set by the Institute, the value of relying on RCT evidence, the costs and savings taken into account in appraisals, and the difficulties in assessing cost-effectiveness over the long-term.

  3.6  The Institute should more actively support input from patient representative organisations and make the perspective of users affected by its decisions central to its decision-making processes.


Commanding public respect

  4.1  The Institute's decisions have profound effects on the day-to-day lives of individuals. In the case of MS drugs the issue centres around a reduction in the number and severity of MS relapses. These relapses are a major feature in the lives of those people with MS who suffer them and can involve blindness, incontinence, loss of mobility and cognitive impairment. They have a disabling effect which extends beyond the relapse itself—their unpredictability robs people of certainty, of the ability to plan and often of employment. Any organisation taking such decisions must place a high priority on retaining the respect of the people whose lives are affected by its decisions.

  4.2  There are two ways in which such an organisation can command such respect: by taking decisions in an open and transparent manner, and by the integrity of the structures which support the taking of those decisions.

  4.3  People affected by the Institute's decisions should be able to follow an audit trail of decision-making. It is currently impossible for consultees—let alone members of the general public—to do this. The Institute has indicated that it intends to provide more explanation in its provisional and final appraisal documents of how it has reached a particular decision. If delivered this is a welcome development; but should be accompanied by the prompt publication of notes of meetings of the Appraisal Committee which should include similar levels of explanation (currently, notes are often published months after the meetings and include little flavour of how conclusions have been reached.) In addition to this, patient representative organisations should be able to attend the Appraisal Committee's full discussions of relevant interventions (they are currently required to leave after a presentation and questions). The Institute has advanced two main arguments against the latter suggestion—that its Appraisal Committee may need to discuss issues which are commercially confidential, and that it needs privacy to allow for free discussion. We do not believe that the first obstacle is insurmountable or that the second is appropriate.

  4.4  The very complex nature of the analyses which NICE undertakes is a barrier to real lay understanding of how it reaches its decisions. The second means of commanding respect therefore assumes a greater importance. This is about developing respect for the decision making process and the structures which support it. The public needs to have confidence that the Institute meets the highest standards expected of the modern NHS. The current appointments process for appointments to the Institute's Partners Council, Board and Appraisal Committee does not inspire such confidence, lacking transparency and depending on recommendations from those who are already part of the Institute's apparatus. The inadequacy of this process is thrown into sharp relief by the Institute itself, which has recently announced that appointments to its new Citizen's Council will be advertised and subject to open recruitment procedures. In its response to the Kennedy Report the Institute has suggested that it should no longer need to seek the Secretary of State's approval for most appointments. We do not believe these powers of approval should be removed before the Institute has an appropriate appointments system in place.

  4.5  People affected by the Institute's decisions also need absolute confidence that there is a fair means of appeal if their representative organisations believe that decisions have not been taken in an appropriate way. They cannot at present have such confidence. Legal advice obtained by the Society is that the Institute's appeal mechanism is unlawful. Its Appeal Panel has a shifting membership, but comprises three members of the Institute's Board, one member of its Partners Council and one independent member. The Panel therefore has neither the qualities of independence or impartiality that are required of a public body exercising an appeals function. This objection was made at an appeal hearing held in November on MS drugs, but was overruled by the Institute's Chairman, who was on that occasion also acting as Chairman of the Institute's Appeal Panel. The Institute must establish a properly independent appeals mechanism if it is to maintain the confidence of the public.

The Institute's approach to its work

Involving people

  4.6  The Institute has shown itself consistently unwilling to engage with people who are expert in MS by virtue of having it. Our view is that the Society has a valuable organisational perspective on the appraisal of MS drugs, but that it is complementary to—and not a replacement for—the perspective of people who have MS. We fought an uphill battle to persuade the Appraisal Committee that it should involve people with MS in one of its meetings. Such requests were resolutely denied until the first appeal (in September 2000) criticised the Committee's handling of the patient perspective. The attitude appears to be that "real" people cannot be objective about their conditions and somehow pollute the Institute's scientific approach.

Randomised controlled trials (RCTs) and long term conditions

  4.7  The Institute has taken a very hard line on the acceptability of evidence derived from studies which do not meet the "gold" standard" of RCTs. However, for ethical reasons, RCTs are of relatively short duration—typically two or three years. This presents clear difficulties in assessing the cost-effectiveness of drugs used over longer periods. In essence, by the blunt application of an approach more suited to one-off, curative, interventions, the Institute discriminates against people with long-term conditions where there are interventions that improve quality of life but do not cure. It presumes guilt in relation to cost-effectiveness for long-term interventions and, by setting a requirement for evidence that could only be achieved through trials of a duration that would be unethical, makes it almost impossible to prove innocence. (This approach must be viewed in the context of the proven clinical effectiveness of the MS drugs.) This issue should be discussed openly as part of a review of the Institute's use of health economics. We do not believe that such a debate should wait until the Institute's Quinquennial review.

What is cost-effective and what is not?

  4.8  It is clear from papers presented to the Institute's Annual Public Meeting that £30,000 per Quality Adjusted Life Year (QALY) has effectively become the benchmark of cost-effectiveness in appraisals. There have been no directions from the Department of Health or the National Assembly for Wales that they consider this to be an appropriate test and there has been no public consultation on the issue by the Institute. Instead, the custom and practice of the Appraisal Committee is deemed, of itself, to deliver a test that is reasonable. We do not believe that this is an appropriate way to take a decision on such a fundamental aspect of public policy, and recommend that it is considered in the review mentioned above.

Demands on voluntary organisations

  4.9  Relationships with patient representatives are key to incorporating the views of those ultimately affected by NICE's decisions. However, it is plain that the Institute does not truly understand the nature of the voluntary sector. Voluntary organisations usually carry out a wide range of activities in support of their constituents and there is an opportunity cost attached to involvement in an appraisal, which sees staff time diverted from other activities within an organisation. There is no compensation for this for those organisations which can afford involvement, and no mechanism for enabling the involvement of organisations whose resources are so limited that they cannot become involved. The input to appraisals from voluntary organisations represents a substantial transfer of funds from the charitable to the statutory sector, and this should be recognised. At minimum, the Institute should extend to voluntary organisations involved in technology appraisals the practical support it intends to offer such organisations participating in its guidelines development process.

The culture of the Institute

  4.10  The Kennedy Report makes clear the importance of an appropriate culture within NHS organisations. Attempting to capture the culture of an organisation is difficult, but important. There is an inwardness to the Institute's approach which is in contradiction to current thinking within the NHS and particularly in the thinking contained in the Kennedy Report, with its emphasis on a culture of "respect, honesty and openness". The Institute's response to the Report does not come close to properly addressing these issues.

  4.11  The overriding impression is that the Institute "owns" technology appraisals rather than regarding them as a function shared with other interested parties. This is damaging both to the way the Institute conducts its business and to public confidence in it. A few diverse examples from the Society's experience serve to illustrate NICE's "ownership" of appraisals:

    (i)  When the appraisal of beta interferons was referred back to the Appraisal Committee after the September 2000 appeal we were told that we would be informed of the outcome of that meeting in early January 2001. Instead, on the last working day before Christmas, the Institute announced that it was effectively re-starting the appraisal process. We were given only a few hours notice before this information was put in the public domain. We had made clear on a number of occasions our need for advance notice of this sort of information in order to brief front-line staff and prepare for disseminating information to our members. The cynical timing of this announcement ignored completely the needs of those people with MS who were affected by the decision and who were in many cases devastated by the news.

    (ii)  When the Appraisal Committee met in January 2001 it was told by its Chairman that "stakeholders in the process would not be able to appeal on the basis of the [new health economics] model used as their agreement for that model would be obtained at the start of the process." This was in fact a unilateral decision taken by the Institute—no such agreement that we would waive our rights to appeal was sought or given. It was not until the notes of the January meeting were finally published in June that we became aware of the Chairman's position. Following challenges from several consultees our continuing rights to appeal were eventually acknowledged.

    (iii)  Data from a survey carried out by our colleagues in the MS Trust were used in the new economic modelling which formed the cornerstone of the re-appraisal of the drugs. It was unclear from the formal papers received how this data had been applied. The MS Trust naturally asked for an explanation of how its own data had been used. This request was refused, with the Trust being told that the only way to challenge the use of its own data (or indeed to find out how it had been used) was through the Institute's formal appeal mechanism.

  4.12  The examples above are specific to the appraisal of MS drugs. But the most serious example of how the Institute believes it "owns" the appraisal process is a more general issue—the setting of a £30,000 per QALY threshold for cost-effectiveness behind firmly closed doors. This is an approach which captures the Institute's view of interested parties as supplicants rather than true partners in the appraisal process.

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