E-DRUG: a NICE start but evidence costs money
---------------------------------------------
[From the BMJ a NICE piece of debate: how much money
will it cost to save money?
http://bmj.com/cgi/content/full/322/7292/943
See also the NICE website at www.nice.org.uk
NN]
BMJ 2001;322:943-944 ( 21 April )
Editorials
Effectiveness, efficiency, and NICE - A NICE start but evidence
costs money
The National Institute for Clinical Excellence (NICE) was
established in England and Wales in 1999 to "provide guidance to
the NHS on the use of selected new and established
technologies."1 NICE synthesises evidence on the effectiveness
and cost of treatments and reaches "a judgment as to whether, on
balance, the intervention can be recommended as a cost-effective
use of NHS resources."1 How has the institute measured up to
these ambitious goals, and what has been learnt about the
demands of an explicit process for assessing health technology?
The institute attracted attention from the international media with
its first judgment that "health professionals should not prescribe
zanamivir (Relenza) during the 1999/2000 influenza season."2 The
additional cost to the NHS would have been about �10m ($15m) for
the benefit of reducing episodes of flu from six days to five.
Although subsequently revised,3 the decision showed that the
institute has teeth and is prepared to bite even home grown drug
companies like GlaxoWellcome (now GlaxoSmithKline). In some
places, such as Australia4 and Ontario, Canada,5 pharmaceutical
companies must prove that their products are cost effective before
they can be reimbursed by the government. Although NICE
operates differently in that it does not automatically assess new
products and provides guidance rather than mandates, it is clear
that products will need to be both effective and provide good value
for money to be recommended for use in the NHS. Unusually,
NICE's remit also includes medical devices and other healthcare
programmes, and its activities are surely being scrutinised by other
healthcare systems. But the evidence on which the analyses of
costs and benefits are based is often incomplete or inappropriate.
The appraisal of hip prostheses, for example, suffered from the
dearth of long term data on revision rates. Similarly, the continuing
deliberations about interferon beta are likely to be constrained by
data from short term trials with outcome measures that are of
limited relevance to decisions about the allocation of resources.
The institute, however, is pragmatic about any shortfalls in
evidence: give the best advice possible using the data available
today, but be prepared to revisit judgments when better data arrive.
For many technologiesparticularly those without a sponsoring
company the onus for generating adequate data will lie with the
NHS health technology assessment programme; its budget may
need to be increased, but using NHS resources to generate
evidence may be money well spent. The institute's appraisals are
likely to have major implications for the drug and medical device
industries because these industries supply much of the information
for appraisal. The need to submit dossiers in support of their
products is making companies think carefully about their research
and development programmes. When products have been
marketed for some time it is possible for companies routinely to
accumulate data, although the data are not typically gathered
within the framework of experimental studies. NICE will, however,
increasingly have to consider products which have yet to reach the
market, so such "real life" data will be lacking. Before launching a
new product, drug companies have in the past focused on
generating evidence for the drug licensing authorities. Such data
are generally of limited value to NICE, so companies are likely to
invest in more pragmatic clinical trials with broader population
bases to collect the data on cost and health outcomes which are
relevant to NICE's decision making. The institute's interest in
finding value for money puts the methods of economic evaluation
under the microscope. Although analytical economic methods have
developed rapidly, they have yet to make a major impact on applied
economic evaluations and on the data submitted to NICE. The
institute's recent publication of more detailed guidance for
economic evaluation provides greater clarity about the institute's
view of best practice.6 The international evidence on the quality of
drug companies' economic evaluations to support reimbursement is
not encouraging. Between 1994 and 1997 a total of 326 evaluations
were submitted to the Australian Department of Health and Aged
Care, and 218 of these had major problems detected by critical
review.7 However, 62% of these problems were caused by the
absence or poor quality of clinical data used in the studies. The
quality of data on effectiveness will probably also be a problem for
NICE. What impact will the institute's guidance have on practice in
the NHS? There will be particular interest in how the NHS reacts to
appraisals that find that a technology benefits patients but
introduces extra costs to the health service, such as coronary
stents and taxanes. The rationale for NICE is, in part, based on the
desire to end the uneven geographical distribution of particular
forms of health care. However, unless funding is earmarked and
made available to health authorities for these interventions, they
can only be offered to patients if the provision of other services
elsewhere in the system is curtailed. Hence, local variations in the
availability of particular services will remain; the appraisal process
will simply shift the unevenness between services. The institute's
role in developing clinical practice guidelines, taken together with
national service frameworks, promises to ameliorate this problem.
It will be necessary to expand the appraisal process, particularly to
identify widely used technologies that are not cost effective, to
release resources for new interventions. In principle, NICE's role in
looking at a wide range of both new and old technologies is
important, although the focus of the latest group of interventions to
be appraised is narrow and concentrates largely on new cancer
drugs.8 The NHS and other healthcare systems that are
collectively funded need transparent decision making about which
types of health care offer value for
money and thus can justifiably be funded; this decision making needs to use
appropriate and explicit methods. There is much to commend in the early stages
of the institute's appraisal process, not least the openness and transparency
it has achieved through its website (www.nice.org.uk). Part of the challenge for
NICE and the assessment of health technology in England and Wales is
economic: gathering, synthesising, and scrutinising data is a valuable exercise
but it is costly. The amount and allocation of research funds should also pass
the test of cost effectiveness. This will depend on whether clinicians and
managers in the NHS take notice of NICE's guidance.
Mark Sculpher, senior research fellow. Michael Drummond, professor. Centre
for Health Economics, University of York, York YO10 5DD Bernie O'Brien,
professor. Department of Clinical Epidemiology and Biostatistics, McMaster
University, Hamilton, Ontario, Canada L8N 3Z5
1. National Institute for Clinical Excellenc1. National Institute for Clinical
Excellence. Appra1. National Institute for Clinical Excellence. Appraisal of new
and existing technologies: interim guidance for manufacturers and sponsors.
London: NICE, 1999. www.nice.org.uk/nice-
web/Embcat.asp?page=oldsite/appraisals/apr_gide. htm (accessed 19 March
2001). 2. National Institute for Clinical Excellence. Zanamivir (Relenza)
guidance from NICE. 12 October 1999. isal of new and existing technologies:
interim guidance for manufacturers and sponsors. London: NICE, 1999.
www.nice.org.uk/nice-web/Embcat.asp?page=oldsite/appraisals/apr_gide. htm
(accessed 19 March 2001). 2. National Institute for Clinical Excellence.
Zanamivir (Relenza) guidance from NICE. 12 October 1999.
www.nice.org.uk/nice-web/Article.asp?a=427&c=153 (accessed 19 March
2001). 3. National Institute for Clinical Excellence. Use of Zanamivir (Relenza)
in the treatment of influenza.
http://www.nice.org.uk/pdf/NiceZANAMIVAR15guidance.pdf (accessed 19
March 2001). 4. Commonwealth of Australia. Guidelines for the pharmaceutical
industry on preparation of submissions to the Pharmaceutical Benefits Advisory
Committee: including major submissions involving economic analyses.
Canberra: Australian Government Publishing Service, 1995. 5. Ministry of
Health. Ontario guidelines for economic analysis of pharmaceutical products.
Ontario: Ministry of Health, 1994. 6. National Institute for Clinical Excellence.
Technology appraisal programme: new guidance documents.
http://www.nice.org.uk/pdf/brdfeb01item6.pdf (accessed 19 March 2001). 7. Hill
SR, Mitchell AS, Henry D. Problems with the interpretation of
pharmacoeconomic analyses: a review of submissions to the Australian
Pharmaceutical Benefits Scheme. JAMA 2000; 283: 2116-2121[Medline]. 8.
National Institute for Clinical Excellence. Technology appraisals work
programme.
http://www.nice.org.uk/pdf/Technology_Appraisals_timetable_Jan_2001.p df
(accessed 19 March 2001)
e. Appra1. National Institute for Clinical Excellence. Appraisal of new and
existing technologies: interim guidance for manufacturers and sponsors.
London: NICE, 1999. www.nice.org.uk/nice-
web/Embcat.asp?page=oldsite/appraisals/apr_gide. htm (accessed 19 March
2001). 2. National Institute for Clinical Excellence. Zanamivir (Relenza)
guidance from NICE. 12 October 1999. isal of new and existing
technologies: interim guidance for manufacturers and sponsors.
London: NICE, 1999. {HYPERLINK "http://www.nice.org.uk/nice-web/Embcat.asp?page=oldsite/appraisals/apr_gide.htm"\}www.nice.org.uk/nice-
web/Embcat.asp?page=oldsite/appraisals/apr_gide.htm (accessed 19
March 2001).
2. National Institute for Clinical Excellence. Zanamivir (Relenza) guidance
from NICE. 12 October 1999. {HYPERLINK "http://www.nice.org.uk/nice-web/Article.asp?a=427&c=153"\}www.nice.org.uk/nice-
web/Article.asp?a=427&c=153 (accessed 19 March 2001). 3. National
Institute for Clinical Excellence. Use of Zanamivir (Relenza) in the
treatment of influenza.
{HYPERLINK "http://www.nice.org.uk/pdf/NiceZANAMIVAR15guidance.pdf"\}http://www.nice.org.uk/pdf/NiceZANAMIVAR15guidance.pdf (accessed
19 March 2001). 4. Commonwealth of Australia. Guidelines for the
pharmaceutical industry on preparation of submissions to the
Pharmaceutical Benefits Advisory Committee: including major
submissions involving economic analyses. Canberra: Australian
Government Publishing Service, 1995. 5. Ministry of Health. Ontario
guidelines for economic analysis of pharmaceutical products. Ontario:
Ministry of Health, 1994. 6. National Institute for Clinical Excellence.
Technology appraisal programme: new guidance documents.
{HYPERLINK "http://www.nice.org.uk/pdf/brdfeb01item6.pdf"\}http://www.nice.org.uk/pdf/brdfeb01item6.pdf (accessed 19 March 2001).
7. Hill SR, Mitchell AS, Henry D. Problems with the interpretation of
pharmacoeconomic analyses: a review of submissions to the Australian
Pharmaceutical Benefits Scheme. JAMA 2000; 283: 2116-2121{HYPERLINK "/cgi/external_ref?access_num=10791503&link_type=MED"}[Medline].
8. National Institute for Clinical Excellence. Technology appraisals work
programme.
{HYPERLINK "http://www.nice.org.uk/pdf/Technology_Appraisals_timetable_Jan_2001.pdf"\}http://www.nice.org.uk/pdf/Technology_Appraisals_timetable_Jan_2001.pdf
(accessed 19 March 2001)
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