Evidence submitted by Archimedes (NICE
68)
EXECUTIVE SUMMARY
1. Although we agree in principle with the
aims of NICE we have concerns with certain aspects of the existing
evaluation process. Our major concerns include:
(a) The one size fits all approach disadvantages
innovative products indicated for diseases with small patient
populations and where survival rates are low;
(b) The lack of dialogue with NICE at an
early stage of clinical development means that the data required
to satisfy a NICE appraisal may not be available;
(c) Expert opinion should be called upon
when necessary to address technical issues which are not well
understood by the appraisal committee;
(d) The time to produce guidance should be
reduced;
(e) The criteria for evaluation of cost effectiveness
have not changed.
All these points are explored further below.
ARCHIMEDES
2. Archimedes is a British pharmaceutical
company, set up in 2004, to develop and bring to market new drugs
for neglected conditions. The company has grown from its three
founders to employ 100 people and specialises in treatments for
patients with cancer and other rare diseases. To date it has invested
almost £40 million through its R & D facility based in
Nottingham.
3. We make reference in our submission to
some of our experiences with a product from our portfolio, Gliadel,
which is currently the subject of two separate NICE appraisal
processes. Gliadel is one of only two treatments for patients
with newly diagnosed high-grade glioma (brain tumours), a condition
which affects around 2000 patients per year, and the current NICE
position is that Gliadel will not be recommended for use in NHS
patients with newly diagnosed high-grade glioma. This review process
commenced in February 2005 and according to NICE is now set to
end in Q1 2008 which has greatly restricted the use of Gliadel
across the UK. We outline below our concerns on the way NICE operated
to reach the decision to date and the wider ramifications for
similar high cost treatments for neglected conditions.
COMMENTARY ON
NICE'S EVALUATION
PROCESS
One size fits all
4. NICE's technology appraisal process adopts
a "one-size-fits-all" approach. While it is right that
the procedure should be rigorous, and desirable that the number
of alternative procedures is minimised, it is:
(a) wasteful of resources to apply the same
extensive and time-consuming process of appraisal to a drug that
may cost the NHS a maximum of £1-2 million a year as is applied
to a "blockbuster" treatment that might cost tens or
even hundreds of millions of pounds;
(b) counterintuitive to apply the same appraisal
methodology to innovative products indicated for rare diseases
with small patient numbers where clinical practicalities restrict
the quantity and type of evaluable data;
(c) unreasonable to expect that innovative
medicines in small therapeutic areas should satisfy the same cost-effectiveness
threshold as those indicated to treat much larger numbers of patients;
(d) the current NICE processes make no allowance
for the above factors. We suggest that below a certain threshold
(perhaps 0.1% of the total budget—currently about £11
million), a quicker and more straightforward appraisal mechanism
should be developed;
(e) cost effective thresholds were set several
years ago and have not been adjusted for inflation or innovation.
Lack of Dialogue
5. The current process allows no dialogue
between NICE and the manufacturer. The products to be reviewed
by NICE are often identified during development and a NICE review
announced. If companies were able to meet with NICE at early stage
in the clinical development process to define the scope of an
appraisal they could ensure that the data which NICE needs is
built into the on-going lengthy and expensive clinical studies.
Equivalent organisations in other European healthcare systems
do consult with manufacturers during their appraisal processes
in order to facilitate the availability of data with which to
support reimbursement. Additionally, there is currently no opportunity
to discuss key issues that arise throughout the process and in
particular as a result of appraisal committee meetings. The only
way in which a consultee can respond through the existing process
is via a written submission and without the benefit of a response
other than through the appraisal documents.
Expert Advice
6. NICE does not always appear to ensure
that it is availing itself of the appropriate expert advice. For
example, at the meeting of the Appraisal Committee held on 22
November 2006 to consider NICE's preliminary view on Gliadel as
one of two brain tumour therapies, no experts on brain tumours
were invited to attend. This occurred despite clear indications
from us and numerous clinical experts in our submissions prior
to this that a major point in the appraisal of our product hinged
upon a technical point requiring specialist knowledge.
Speed of Publishing Guidance
7. The length of time which NICE takes to
appraise new treatments is a serious handicap for companies with
high value innovative products. Often, such "biotech"
products are in therapeutic areas which are not usually being
researched by larger pharmaceutical companies and represent exciting
new medicines. The assessment of Gliadel has now been running
for over 2 years and is scheduled to run for at least another
12 months; a lack of a decision has prevented use of the product
in a number of hospitals over this time period. Only 125 patients
have been treated with Gliadel over two years in England and Wales,
whereas for instance in France 4 times this number of patients
have been treated over the same period.
CONCLUSIONS AND
IMPLICATIONS
8. The UK biotech industry tends to focus
on developing innovative products for conditions with high unmet
medical need and where patient populations are comparatively small.
Very often efforts are concentrated on one product which has been
subject to many years of research and investment. NICE's current
process increases the risk that such products will not be made
available to NHS patients when they have already past all regulatory
approval hurdles. This can also have a significant effect on the
viability of UK biotech companies and the decision of whether
to commercialise such products in the UK.
9. The NICE process could therefore be improved
by:
— Adapting the criteria for affordability
to reflect differences between diseases and options available
for patients;
— Allowing the industry to discuss
product technology appraisals with NICE at an earlier stage in
order that the data required for the appraisal is generated as
part of the clinical development programme;
— Discussing the technology appraisal
at regular intervals to avoid misunderstandings and mistakes such
as the use of experts;
— Updating the parameters used to assess
cost effectiveness including financial thresholds.
Archimedes Pharma Ltd
March 2007
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