Evidence submitted by Amgen (NICE 58)
OVERVIEW
The National Institute for Health and Clinical
Excellence (NICE) was established to ensure "Faster Access
to Modern Medicines". Amgen supports this objective, and
welcomes the contribution the organisation has made towards ensuring
national guidance is published to deliver equitable access to
medicines. Since its formation, the Government and NICE have maintained
dialogue with all stakeholders and collaborated widely to ensure
NICE has evolved into an internationally recognised centre of
excellence for health technology appraisal. However, there are
areas around the organisation's procedures that need reviewing
if NICE is to ensure patients benefit from innovative medicines,
and that the UK continues to be home to innovative research for
serious illnesses. This Inquiry is timely given proposals for
a wider role for NICE as set out in the Cooksey Review of UK health
research funding and Office of Fair Trading report into the Pharmaceutical
Pricing Regulation Scheme. The Health Select Committee's recommendations
and Government response will act as a blueprint for future reforms
of NICE, which must be in place before any wider role is considered.
EXECUTIVE SUMMARY
Britain's entrepreneurial bioscience sector
is a leader in Europe, and bioscience is set to continue transforming
patient health through the development of new, improved and targeted
therapies. To ensure patients have access to these scientific
breakthroughs, and to ensure the UK continues to support innovation,
and be home to a thriving pharmaceuticals and biomedicines sector,
there needs to be changes to the regulations and environment which
currently delay uptake of innovative medicines. With our experience
of NICE, the Scottish Medicines Consortium (SMC) and All Wales
Medicines Strategy Group (AWMSG), Amgen have been able to make
an assessment of the issues and barriers around four key areas:
— Over-reliance on cost/QALY (Quality
Adjusted Life Year) as an assessment of the cost-effectiveness
of medicines compared to current standard practice, especially
considering significant "real world" data often exists.
— Delays in the issuing of guidance,
and continued lack of access to the medicine pending referral
of a technology to NICE, or during the appraisal process.
— The independence of NICE's Appeals
Process
— Barrier to effective implementation
of NICE Guidance
Our experience and perspectives on these shortcomings
are detailed in our recommendations below.
1. About Amgen
Amgen is a biotechnology pioneer established
in 1980. We discover, develop and deliver innovative human therapeutics
which have changed the practice of medicine, helping millions
of people around the world in the fight against cancer, kidney
disease, rheumatoid arthritis, and other serious illnesses. With
a deep and broad pipeline of potential new medicines, Amgen remains
committed to advancing science to dramatically improve people's
lives. In the UK we continue to be an entrepreneurial, science-driven
enterprise dedicated to helping people fight serious illness and
employ 500 people at our development centres in Cambridge and
Uxbridge. In recent years, many of our medicines have undergone
appraisal by NICE, hence we are qualified to comment on and make
recommendations on NICE's process in respect of technology appraisals.
2. NICE's evaluation process, and whether
any particular groups are disadvantaged by the process
2.1 NICE methodology relies heavily on cost/QALY
(Quality Adjusted Life Year), an assessment of the cost-effectiveness
of the new technology compared to current standard practice. This
parameter is only one of several metrics that can be used to assess
value but favours medicines that offer survival benefit rather
than quality of life benefit. As a result, NICE has issued negative
guidance on many medicines even though they offer substantial
quality of life improvements, as opposed to mortality benefits.
Furthermore, NICE appraisals do not give full consideration to
wider healthcare and societal costs outside the narrow definition
of immediate prescribing budgets, for instance reduction in overnight
hospital stays, reduced staff time elsewhere in the patient journey,
or residential care costs.
— Amgen believes a stakeholder group
of Government, NICE, industry and patient groups should undertake
a comprehensive review to propose more efficient methodologies
for evaluating drugs that improve quality of life or more broadly
those drugs for which the QALY is an inappropriate measure. We
recommend the review should include an evaluation of best practices
from other Health Technology Assessments (HTAs) around the world
as well as comparative centres of excellence.
2.2 Amgen believe the industry can be disadvantaged
because the Evidence Review Group (ERG) and NICE Appraisal Committee
have excessively high expectations of the quality and quantity
of clinical and health economic data available that can be generated
by launch. Furthermore, when a medicine is launched into the NHS,
considerable uncertainty exists regarding the cost-effectiveness
of that medicine, and this data can only be gathered once the
medicine is being routinely used in the NHS setting. QALY values
can change over time, as more is learned about the optimum use
of a medicine.
— Amgen believes the NICE Appraisal
Committee needs to fairly assess what evidence they should expect
in their deliberations and factor that appropriately into their
decision making process.
2.3 With the increased research breakthroughs
and targeted therapies, innovative biotechnology medicines will
be increasingly focussed towards sub-sets of patients for whom
no existing therapy exists. Given the small number of appropriate
patients, many of these will have minimal budget impact if routinely
prescribed in eligible patients. The NICE appraisal process requires
considerable resources from manufacturers and consultees alike.
— NICE should only appraise medicines
which have a significant cost burden to the NHS, and for which
significant "real world" data exists. This will ensure
that industry, academic institutions and the voluntary sector
continue to undertake innovative research in difficult to treat
diseases.
3. The speed of publishing guidance
3.1 There are two types of NICE "blight"
operating in the NHS. The first is when NICE announce they will
be reviewing a specific product. Countless examples exist of PCTs
refusing to fund medicines until NICE produce guidance on the
specific product. The second type of "blight" is for
products not selected for appraisal. In these cases, there are
many examples of PCTs refusing to fund treatments as they are
not a priority compared to medicines that have been subject to
NICE appraisal. The Department of Health recently re-issued Good
Practice Guidance on Managing the Introduction of New Healthcare
Interventions, and although this is welcome we believe there are
still unacceptable barriers to prescribing and disadvantaged patients
that require access to new medicines.
— Amgen believes a fundamental review
is required to determine the extent of continued NICE blight,
and that the Department of Health should develop policy mechanisms
to ensure patients are not denied new medicines in the period
NICE is evaluating a medicine or if NICE choose not to review
a medicine.
3.2 With many NICE appraisals there can
be protracted delays in the issuing of guidance. The duration
of the NICE appraisal process has been improved by the introduction
of the Single Technology Appraisal (STA) process; however the
Multiple Technology Appraisal (MTA) remains lengthy and results
in NICE blight. The MTA process is more suited to reviewing established
technologies or topics for disinvestment.
— Amgen believes only the STA process
should be used for new products being introduced into the NHS.
The MTA process should be limited to reviewing products with significant
"real-world" data.
4. The appeal system
4.1 The current appeal system means the
Appeals Panel is comprised entirely of NICE personnel. It is vital
that any appeals system is seen to be independent of NICE to ensure
that the appeal process, is truly objective and distinct from
the Institute.
— To increase the independence and
credibility of the appeal process, Amgen recommends the appeal
process should be separated from NICE and constituted entirely
from non-NICE personnel.
5. The implementation of NICE guidance, both
technology appraisals and clinical guidelines (which guidance
is acted on, which is not and the reasons for this).
5.1 While there is a statutory instrument
requiring the NHS to initiate funding the implementation of NICE
guidance, in reality there is still extensive postcode prescribing
in the NHS. It represents an inefficient use of NHS resources
to have an appraisal system in place that is not fully implemented
by PCTs and trusts.
— Amgen believes adherence to NICE
guidance should be a fundamental part of broader NHS performance
measures such as the GP contract QoF measures, and audit by the
Healthcare Commission. Only by doing this can we ensure NICE guidance
is fully adopted in the NHS.
SUMMARY OF
RECOMMENDATIONS
Amgen believes a stakeholder group of Government,
NICE, industry and patient groups should undertake a comprehensive
review to propose more efficient methodologies for evaluating
drugs that improve quality of life or more broadly those drugs
for which the QALY is an inappropriate measure. We recommend the
review should include an evaluation of best practices from other
Health Technology Assessments (HTAs) around the world as well
as comparative centres of excellence.
Amgen believes the NICE Appraisal Committee
needs to fairly assess what evidence they should expect in their
deliberations and factor that appropriately into their decision
making process.
NICE should only appraise medicines which have
a significant cost burden to the NHS, and for which significant
"real world" data exists. This will ensure that industry,
academic institutions and the voluntary sector continue to undertake
innovative research in difficult to treat diseases.
Amgen believes a fundamental review is required
to determine the extent of continued NICE blight, and that the
Department of Health should develop policy mechanisms to ensure
patients are not denied new medicines in the period NICE is evaluating
a medicine or if NICE choose not to review a medicine.
Amgen believes only the STA process should be
used for new products being introduced into the NHS. The MTA process
should be limited to reviewing products with significant "real-world"
data.
To increase the independence and credibility
of the appeal process, Amgen recommends the appeal process should
be separated from NICE and constituted entirely from non-NICE
personnel.
Amgen believes adherence to NICE guidance should
be a fundamental part of broader NHS performance measures such
as the GP contract QoF measures, and audit by the Healthcare Commission.
Only by doing this can we ensure NICE guidance is fully adopted
in the NHS.
Amgen
March 2007
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