Evidence submitted by the National Rheumatoid
Arthritis Society (NICE 82)
EXECUTIVE SUMMARY
We are all healthcare professionals, patient
officers of the National Rheumatoid Arthritis Society (NRAS) and
volunteer patient members of NRAS, working in the field of rheumatology.
We all have personal and direct experience of the National Institute
for Health and Clinical Excellence (NICE); as participants in
the NICE Health Technology Appraisal (HTA) and Clinical Guidelines
programmes.
NICE has an important role to play in ensuring
that patients gain access to the medicines they need to treat
or manage their condition, as well as developing and supporting
best clinical practice. Our experience suggests that this role
is increasingly being dominated by economics arguments rather
than an evaluation of the therapeutic benefits (short and long-term),
with innovative and effective licensed medicines not being recommended
by NICE on predominantly cost-effectiveness grounds. The input
and needs of patients (and, to a lesser extent, clinicians) has
been marginalised. This is a particular cause for concern for
us.
For inflammatory arthritis, there is a rapidly
developing armamentarium of novel and highly effective treatments.
There is a lag however, between studies demonstrating safety and
efficacy and the more complex studies required to provide evidence
of real world clinical- and cost-effectiveness. We are concerned
that the existing evaluation process does not take this into account
adequately.
We are concerned about the inequities that persist
between England and Scotland, both in terms of the absolute decisions
being made and in terms of delay in reaching decisions. It is
well documented that the NICE Multiple Technology Appraisal (MTA)
process takes a minimum of 54 weeks in theory, and often much
longer in practice. By contrast, the Scottish Medicines Consortium's
review process takes only 4-6 months. We are hopeful that the
NICE Single Technology Appraisal (STA) process will reduce delays
and that decisions about new, potentially beneficial, treatments
can be made much more quickly.
Whilst a NICE decision is pending, patients
are further disadvantaged as a consequence of the phenomenon known
as "NICE blight". We are seeing many examples where,
in spite of reminders from the Department of Health that Health
Service Circular 1999/176 remains in force, Primary Care Trusts
are refusing to fund a particular drug on the grounds that it
has not yet been endorsed by NICE.
We are particularly concerned by the undue weight
being given to the health economic aspects of NICE HTAs, at the
expense of clinical, therapeutic and quality of life issues, which
are arguably of more importance to patients. The incremental cost
per QALY is increasingly becoming the sole determinant of NICE
recommendations; rather, the cost per QALY should be just one
of the many factors that the Appraisal Committee takes into account
in reaching its conclusions about the use of a particular technology.
Health economic models should not be construed
by the NICE Appraisal Committee as definitive "evidence"
in the same way that data from a well-designed, randomised clinical
trial is evidence. Relying upon the cost per QALY as the preferred
measure of health outcome disadvantages those people needing treatment
for chronic, long-term conditions—such as inflammatory arthritis.
The paradox arises because measures used to derive QALYs tend
to be weighted towards conditions resulting in death or a reduced
life expectancy. In conditions like inflammatory arthritis, there
may be short term gains that have long standing accrual over the
lifetime of the person that are not captured suing existing measures
of utility.
So far as the actual Appraisal Committee meetings
are concerned, the Committee rarely includes anyone with specialist
clinical knowledge of the therapy area or condition under consideration.
Appraisal Committee meetings can be intimidating and confusing
for nominated patient experts. Meetings can be dominated by technical
discussion on the health economic aspects. The anecdotal experience
of the patients who are actually using these medicines is rarely
listened to or, if the decisions are indicative, taken into account.
Patient and clinical experts are required to leave the meeting
before the Appraisal Committee starts to discuss its recommendations.
Overall, we feel that an overly "tokenist"
approach is being taken to patient evidence, in particular, and
the quality of life issues that are key to us.
Finally, we suggest that the process for bringing
a successful appeal is neither fair, independent nor transparent
and needs to be reviewed.
INTRODUCTION
1. The undersigned are all members of a
standing advisory board to the National Rheumatoid Arthritis Society
(NRAS). NRAS provides the secretariat support to the All Party
Parliamentary Group on Inflammatory Arthritis and we act as advisers
to NRAS in respect of its parliamentary work.
2. We welcome the opportunity to make this
short submission to the Health Committee, based upon our collective
personal experience and observations of NICE processes and procedures.
3. We are all healthcare professionals or
patients (patient officers of the charity and volunteer patient
members of NRAS) working in the field of rheumatology and we all
have personal and direct experience of the National Institute
for Health and Clinical Excellence (NICE); as participants in
the NICE Health Technology Appraisal (HTA) and Clinical Guidelines
programmes, as clinicians trying to secure funding to implement
NICE guidance; or as patients. Five members of our advisory board
have contributed to NICE decision-making, either as nominated
clinical or patient experts attending NICE Appraisal Committee
meetings, or as members of Guideline Development Groups.
4. We have experience of participating in
the following NICE technology appraisals and Clinical Guidelines:
— Cox II inhibitors—Osteoarthritis
and rheumatoid arthritis TA027
— Anakinra—Rheumatoid arthritis
TA072
— Etanercept and infliximab—Rheumatoid
arthritis TA036
— Etanercept—Juvenile Idiopathic
Arthritis TA035
— Etanercept and Infliximab—Psoriatic
Arthritis TA104
— Adalimumab, etanercept and infliximab—Ankylosing
spondilitis (appraisal in development)
— Adalimumab, etanercept and infliximab—Rheumatoid
arthritis (appraisal in development)
— Abatacept—Refractory rheumatoid
arthritis (Single Technology Appraisal in development)
— Rituximab—Refractory rheumatoid
arthritis (Single Technology Appraisal in development)
— Osteoarthritis (guideline in development)
— Rheumatoid Arthritis in Adults (guideline
in development)—Ailsa Bosworth has applied to join the Guideline
Development Group
5. In our opinion, NICE has an important
role to play in ensuring that patients gain access to the medicines
they need to treat or manage their condition. Sadly, our experience
suggests that this role is increasingly being sacrificed in favour
of pure economics, with innovative and effective licensed medicines
not being recommended by NICE on predominantly cost-effectiveness
grounds. As a result, the input and needs of patients (and, to
a lesser extent, clinicians) has been marginalised. This is a
particular cause for concern for us.
INEQUITIES ACROSS
COUNTRIES
6. We are concerned about the inequities
that persist between England and Scotland, both in terms of the
absolute decision being made and in terms of the delay in reaching
decisions. For example, in December 2003 the Scottish Medicines
Consortium accepted adalimumab for use in the NHS in Scotland,
soon after it had received a marketing authorisation for the UK.
Over three years later, NICE has not yet issued final guidance
on the use of adalimumab, or completed its review of the other
anti-TNF inhibitor treatments etanercept and infliximab. NICE's
final recommendations are currently the subject of an appeal from
6 different appellants. This is despite evidence for the excellent
clinical efficacy of these therapies in rheumatoid arthritis.
7. Similarly, adalimumab was accepted for
use in NHS Scotland for people with ankylosing spondilitis in
2006. Despite having first added this appraisal to its work programme
three years ago, the NICE Appraisal Committee has not yet been
able to reach a conclusion on whether adalimumab should be recommended
for use in England and Wales for this indication.
8. It is well established and well documented
that the NICE Multiple Technology Appraisal (MTA) process takes
a minimum of 54 weeks in theory, and often much longer in practice.
By contrast, the Scottish Medicines Consortium's review process
takes only 4-6 months. We are hopeful that the NICE Single Technology
Appraisal (STA) process will reduce delay and would urge that
a strictly enforced timetable is attached to this process.
9. Whilst a NICE decision is pending, patients
are disadvantaged as a consequence of the phenomenon known as
"NICE blight". Blight occurs when Primary Care Trusts
decline or refuse funding for a particular drug, on the grounds
that it has not yet been endorsed by NICE. Although Health Service
Circular 1999/176 prohibits Primary Care Trusts from refusing
make a treatment purely on this basis, we are aware of many practical
examples where this is taking place.
10. For example, Addenbrookes Hospital in
Cambridge has refused to fund rituximab therapy, yet this drug
is being made available to patients in Leeds and Plymouth, for
example. Camden Primary Care Trust in London has also refused
to fund rituximab at present, and this is particularly problematic
because Camden is the lead PCT for University College Hospital
(UCLH). This means that any patient referred to UCLH from anywhere
in the country will be bound by Camden's policy. People with rheumatoid
arthritis, who have been successfully treated with rituximab as
part of a long-term clinical trial and are responding well, are
now being taken off treatment pending NICE's decision. In our
view that is unethical.
INCREASING RELIANCE
ON HEALTH
ECONOMIC ASPECTS
11. We are particularly concerned by the
undue weight being given to the health economic aspects of NICE
HTAs, at the expense of clinical, therapeutic and quality of life
issues. Not only are these issues arguably of more importance
to patients, but they also have the potential to impact on the
wider societal costs and benefits of a long-term condition, such
as affecting a person's ability to return to work or their reliance
on the welfare system.
12. It seems to us that the incremental
cost per QALY is starting to become the sole determinant of NICE
recommendations; rather, the cost per QALY should be just one
of the many factors that the Appraisal Committee takes into account
in reaching its conclusions about the use of a particular technology.
13. Health economics is an art, not a science.
It is very easy to manipulate the incremental cost-effective ratios
(ICERs) for a particular technology, simply by changing the inputs
or the assumptions in the model. In that sense, the health economic
analyses undertaken by the academic centres are nothing more than
a series of simulations; they should not be construed by the Appraisal
Committee as definitive "evidence" in the same way that
data from a well-designed, randomised clinical trial is evidence,
unless the model has been validated by an independent third party
to identify the strengths and weaknesses of the assumptions used,
inputs and robustness of model design.
14. As noted previously, cost effectiveness
studies lag behind emerging evidence including some of the industry-funded
safety and efficacy studies and require specific utility measures.
The existing utility measures intended for capturing quality of
life for cost effectiveness in conditions like inflammatory arthritis
are lacking maturity and sophistication rendering QALY measures
flawed. "Disutility" factors which are particularly
pertinent to long-term conditions—such as disability, pain
and morbidity—are not captured, disadvantaging patients
affected by decisions based on these data.
15. As a representative patient organisation,
it is very difficult for us to generate or collate the sort of
evidence which NICE is looking for, particularly for new technologies
where the evidence of clinical and cost effectiveness is limited,
despite evidence of excellent efficacy, and where patient experience
is small.
COMPOSITION OF
THE NICE APPRAISAL
COMMITTEE
16. Again, in our collective experience
of several HTAs, the composition of the Appraisal Committee is
problematic. Firstly, the Committee membership—and the meetings
themselves—can be dominated by the health economists. Secondly,
there is rarely a clinician on the Appraisal Committee with a
specialist's understanding of the therapeutic area under consideration.
When NICE was reviewing the use of etanercept for use in Juvenile
Idiopathic Arthritis, there was no paediatrician sitting on the
Appraisal Committee. This contrasts with the Guideline Development
Groups, which are charged with developing best practice guidance
for the management of a particular condition, and which do include
appropriate clinical expertise.
APPRAISAL COMMITTEE
MEETINGS
17. The Appraisal Committee relies on two
patient and two clinical experts to attend the meetings and give
oral evidence of a particular condition, how the drug performs
in clinical practice and what it is like to live with a particular
condition. Whilst we are very grateful to have the opportunity
to present our case to the Appraisal Committee in person, we cannot
help but feel that a very "tokenist" approach is being
taken to patient evidence, in particular and the quality of life
issues that are key to us.
18. Our experience is that Appraisal Committee
meetings can be intimidating and confusing for nominated patient
experts. The meetings are heavily influenced by technical discussion
on the health economic aspects. As a result the anecdotal experience
of the patients who are actually using these medicines is rarely
listened to or, if the decisions are indicative, taken into account.
19. The inclusion of patient representatives
in NICE's decision-making processes is—at the moment—chiefly
tokenism in our view; the way in which data and results are presented
are challenging—even for healthcare professionals—and
this makes it very hard for patient organisations and representatives
to interpret and respond appropriately. In our view, NICE needs
to provide a greater level of support and guidance to patient
groups if their expertise and experience is to be meaningful and
used appropriately.
20. Patient and clinical experts are required
to leave the meeting before the Appraisal Committee starts to
discuss its recommendations. It is often very difficult to understand
how and why a particular decision has been arrived at, and we
do have concerns about the transparency and fairness of the NICE
decision-making process.
APPEALS PROCESS
21. Finally, when one considers the desirability
of having a NICE process that is fair, independent and transparent,
the anomalies of the appeal process should not be overlooked.
It strikes us as perverse that the Chair of NICE should also review
the merits of whether an appeal should be allowed to proceed,
and then go on to Chair one of the Appeal Panels himself. In essence,
we are required to appeal to the same body of people who made
the original decision. In no sense can that be characterised as
an independent, robust appeals process.
CONCLUSION
22. We would like to thank the Health Committee
for the opportunity to submit our comments and we would be very
pleased to provide the Health Committee with any additional information
or clarification that it might require.
National Rheumatoid Arthritis Society
March 2007
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