British Medical Journal
19 February 2005
Editorial
Neglected diseases
"Priority medicines for Europe and the world" is a wake up call from WHO
Few readers of the BMJ were probably aware or even cared that the
presidency of the EU Council was held by the Dutch government during the
second six months of 2004. Nevertheless, history is likely to remember
the Dutch presidency with gratitude. For in the run up to it, the Dutch
government commissioned the World Health Organization to develop a
research agenda for the European Union that was based on public health
needs for priority medicines.
The commendable report was published last November.[1] Masterminded by
Warren Kaplan and Richard Laing, and using a new approach, it is a work
of scholarship. It covers a wide range of critical issues and makes many
far reaching research proposals for the European Union. But underlying
these extensive data and careful analyses are some chilling
implications. Here are a few.
Firstly, contemporary notions of neglected diseases are far too
circumscribed. They include both rare (orphan) diseases as well as
common disorders (mainly communicable) of developing countries. But the
neglected diseases also include a range of other conditions that are
major burdens in all countries and for which we have few or no effective
remedies. Epidemic influenza, appearing annually, probably causes a
million deaths a year worldwide. But pandemic influenza, appearing every
few decades, has much more devastating consequences. The pandemic of
1918-9, for example, killed an estimated 40-50 million people. Research
into influenza, in relation to its catastrophic potential, is
underfunded.[2] Worse still, we do not even seem able to provide
sufficient vaccine to control epidemic influenza in the most developed
countries. Little confidence exists that we could under-take a global
immunisation programme for an influenza pandemic even if we had
sufficient warning of its antigenic profile unless we can sharpen up on
manufacture and distribution. Other notable examples of neglected
diseases afflicting the world include antibacterial drug resistance,
chronic obstructive pulmonary disease, alcoholic liver disease, and stroke.1
Secondly, the pharmaceutical industry is clearly unable to meet the
needs of people with neglected diseases. This is not a criticism but a
fact, for the pharmaceutical industry is a business and needs to provide
its shareholders with a return on their investment. Many neglected
diseases are unlikely to to do this and investment represents a great
commercial risk. The industry will continue to play a major part in the
discovery and development of drugs; but we need much greater pluralism
in both the funding and discovery of novel treatments.
Examples of this pluralistic approach already exist. Substantial public
funds for basic and clinical research, ultimately aimed at producing new
forms of treatment, are available in the United States and to a lesser
extent in Europe. The research agenda in the WHO report should be a
major feature of the European Union's next research programme. For this
we must rely on the influence of the Dutch presidency. But Europe also
needs to harness the potential of public-private partnerships along the
lines of the Global Alliance for TB Drug Development, or the Hereditary
Disease Foundation in the United States.[3] Public-private partnerships
involve joint investment of resources by bodies including universities,
government supported research organisations, pharmaceutical companies,
venture capitalists, and research based charities. The Hereditary
Disease Foundation's public-private partnerships include, for example,
not only the foundation itself but several pharmaceutical companies and
academic researchers of the Huntington Study Group collectively seeking
an effective treatment for Huntington's disease; and that of the Global
Alliance for TB Drug Development is a joint initiative between the
Rockefeller Foundation, the Bill and Melinda Gates Foundation, WHO, a
group of university based investigators, and several private companies.3
Thirdly, the cost of developing a new drug must be reduced.[4] Unless
savage cuts can be made in the current cost of bringing a new drug to
the market-over $800m (#423m; 605m) and rising at the rate of 10% a
year[5]-drug development will come to a standstill. Half this sum seems
to be consumed by clinical development programmes. But real inroads need
to be made into reducing all regulatory requirements if affordable new
drugs are to be available to consumers.4 Promisingly, the problem has
been acknowledged by both the US Food and Drug Administration and the
European Medicines Agency,[6][7] but international action is needed if
these difficulties are to be resolved. Radical regulatory reform that is
acceptable to the United States, the European Union, and Japan will be
essential to relieve our current woes. This will have to include
reducing the burdens of preclinical studies and adopting innovative
approaches to investigating efficacy and safety in patients.4
Finally, the boundaries between primary and secondary prevention of
disease clearly need to be redrawn. The silos currently occupied on the
one hand by public health specialists and on the other by clinicians
must be eroded. Prevention and control of disease need to go hand in
hand. The transfer of the public health functions of the Health
Development Agency to the National Institute for Clinical Excellence
(NICE) as of April 2005 offers a real opportunity for this to happen-at
least in England.[8]
Michael D Rawlins, professor
Wolfson Unit of Clinical Pharmacology, Medical School, University of
Newcastle, Newcastle upon Tyne NE2 4HH (m.d.rawlins{at}ncl.ac.uk )
Competing interests: MR has been chairman of NICE since its formation in
1999.
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References
1. Kaplan W, Laing R. Priority medicines for Europe and the world.
Geneva: World Health Organization, 2004.
http://mednet3.who.int/prioritymeds/report/index.htm (accessed 5 Nov 2004).
2. Treanor J. Weathering the influenza vaccine crisis. N Engl J Med
2004;351: 2037-40.[Free Full Text]
3. Moses H, Braunwald E, Martin JB, Their SO. Collaborating with
industry-choices for the academic medical centre. N Engl J Med 2002;347:
1371-5.[Free Full Text]
4. Rawlins MD. Cutting the cost of drug development. Nature Rev Drug
Discov 2004;3: 360-4.[CrossRef][ISI][Medline]
5. DiMasi JA, Hansen RW, Grabowski HG. The price of innovation: new
estimates of drug development costs. J Health Econ 2003;22:
151-85.[CrossRef][ISI][Medline]
6. European Medicines Agency. The European Medicines Agency road map to
2010: preparing the ground for the future. London: European Agency for
the Evaluation of Medicinal Products, 2004.
7. Food and Drugs Administration. Challenge and opportunity on the
critical path to new medical products. Department of Health and Human
Services: Washington DC. 2004.
www.fda.gov/oc/initiatives/criticalpath/whitepaper.html (accessed 6th Nov).
8. Department of Health. Report on the reconfiguration of the Department
of Health's arm's length bodies. London: Department of Health, 2004.
www.dh.gov.uk/PublicationsAndStatistics/Publications/PublicationsPolicy
AndGuidance/PublicationsPolicyAndGuidanceArticle/fs/en?CONTENT_ID=408608
1&chk=y4UIfP (accessed 6 Nov 2004).