Memorandum by the Multiple Sclerosis Society
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 yearstwice
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
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
itselftheir 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 consulteeslet alone members
of the general publicto 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 suggestionthat 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
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
toand not a replacement forthe 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
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 durationtypically
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
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
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"
(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 Instituteno
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 issuethe 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