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Neglected Diseases
The four principles
•
•
•
•
Autonomy
Non-maleficence
Beneficence
Justice
– How can we ensure equitable access to
health care?
The drug pipeline
←
←
←
←
←
←
←
←
Basic science
R&D
Preclinical development
Clinical trials
Registration
Manufacturing
Sales
Distribution
Delivery by health care provider
The R&D gap
14%
12%
10%
8%
6%
4%
2%
0%
Cardiovascular
Cancer
HIV/AIDS
Relative contribution to global
disease burden (DALYs)
Tuberculosis
Malaria
Tropical diseases
Proportion of new drugs,
1975-1999
Trouiller et al., Lancet 2002, 359:2188-94
The R&D gap
in relative terms
140%
120%
100%
equitable level
80%
60%
40%
20%
0%
Cardiovascular
Cancer
HIV/AIDS
Relative contribution to global
disease burden (DALYs)
Tuberculosis
/
Malaria
Tropical diseases
Proportion of new drugs,
1975-1999
Trouiller et al., Lancet 2002, 359:2188-94
The Neglected Diseases
The “big three”:
• HIV/AIDS
• Malaria
• Tuberculosis
“Most neglected” diseases:
• African sleeping sickness
• Dengue fever
• Leishmaniasis
• Schistosomiasis
• Chagas disease
• Leprosy
• Lymphatic filariasis
• Onchocerciasis
Malaria
• The #1 cause of death of children under 5 in
Africa
• Current treatments:
– Resistance problems for all drugs except artemisinin
– Frequent shortages of artemisinin
• Reduced African GNP by 1.3% from 1965 to
1990, for a total reduction of 50%1
1Gallup,
J. and Sachs, J. American Journal of Tropical Medicine and Hygiene, 2001; 64 (1, 2) S., p.90
Tuberculosis
• Current first-line treatment:
– 4-pill regimen for 6 to 9 months
– All 4 drugs developed over 40 years ago
• Extensively drug-resistant strains now emerging
– Some strains virtually untreatable
• In some countries, 4%-7% of GDP is lost to this
single disease (World Bank)
Visceral Leishmaniasis
• Kills at least 40,000/year
• Main treatments require hospitalization for
several weeks
– Serious adverse side effects, IV administration
– Better treatments are prohibitively expensive
• Lack of effective diagnostics: testing is invasive
and requires highly experienced staff
Onchocerciasis (river blindness)
• Parasitic infection, endemic in areas of Africa
and South America
• 2nd leading cause of blindness in the world
– 18 million infected, 250,000 already blind
• Causes disfiguring skin disease
• Shortens life expectancy by up to 15 years
Onchocerciasis (river blindness)
• 1987: Merck discovers a drug that is effective
against the parasite that causes river blindness,
and announces that it will donate as much as is
needed, to all who need it, for as long as needed
• WHO’s Onchocerciasis Control Programme:
–
–
–
–
Up to 18 million infections prevented
1.25 million cured
600,000 cases of blindness prevented
25 million hectares of land made safe for cultivation
and resettlement
– Some countries moving towards elimination
Onchocerciasis
Lancet 2007; 369: 2021-29
“our results suggest that resistant adult parasite
populations, which are not responding as expected
to ivermectin, are emerging.”
Why?
•
•
•
•
•
Market failure
Lack of government funding
Lack of charitable funding
Academic culture
Lack of awareness
Market failure
• Most drug development is done by the private
pharmaceutical industry
• Costs recovered by charging patients or their
governments high prices
• Little or no profit in developing drugs for poor
populations
Government funding
• Governments in high-income countries prioritize
the health of their own population
• CIHR mandate:
“To excel, according to internationally accepted
standards of scientific excellence, in the creation of
new knowledge and its translation into improved
health for Canadians, more effective health services
and products and a strengthened Canadian health
care system.”
Charitable funding
• Residents of high-income countries are more
likely to donate for research on visible, familiar
diseases
–
–
–
–
Canadian Cancer Foundation
Heart and Stroke Foundation of Canada
Canadian Diabetes Association
Canadian Malaria Foundation?
Academic culture
• Universities prioritize research that leads to
publications or which generates wealth for the
institution
– Emphasis on publication leads to plenty of basic
research but very limited translational research
– Wealth-generation encourages research into firstworld problems, and patenting
Academic culture
• From the web site of one of the country’s top
university technology transfer offices:
(identity withheld)
Lack of awareness
• Since it doesn’t affect “us”…
– Little media attention
– No strong lobbying groups
– No political pressure
What can we do?
• Market failure: intervene in the market
–
–
–
–
Subsidize ND research
Prizes
Advance market commitments
Public-private partnerships
• This might be beyond the reach of a university
student…
What can we do?
• Government funding: lobby governments for
increased spending on global health research
• Charitable funding: donate
• Academic culture: push your university to
prioritize worldwide social impact in its research,
as opposed to just financial and academic
benefit
• Raise awareness: talk to decision-makers,
students, the public, the media, everyone!
Questions?
Further reading
•
WHO: Neglected Diseases
www.who.int/neglected_diseases
•
WHO: Special Programme for Research and Training in Tropical Diseases
www.who.int/tdr
•
WHO Report: Priority Medicines for Europe and the World, Chapter 6.9 – Neglected Diseases
mednet3.who.int/prioritymeds/report/index.htm
•
Nature: Outlook: Neglected Diseases
www.nature.com/nature/outlook/neglecteddiseases
•
Malaria R&D Alliance
www.malariaalliance.org
•
Medicines for Malaria Venture
www.mmv.org
•
TB Alliance
www.tballiance.org
•
Drugs for Neglected Diseases Initiative
www.dndi.org
•
Institute for One World Health
www.iowh.org
•
WHO Onchocerciasis Control Programme
www.who.int/blindness/partnerships/onchocerciasis_OCP
•
Merck’s Mectizan donation program
www.merck.com/cr/enabling_access/developing_world/mectizan/
•
Universities Allied for Essential Medicines
www.essentialmedicine.org and www.ubc-uaem.org