Neglected Diseases

The four principles

• Autonomy

• Non-maleficence

• Beneficence

• Justice– How can we ensure equitable access to

health care?

The drug pipeline

← Basic science← Preclinical development← Clinical trials← Registration← Manufacturing← Sales← Distribution← Delivery by health care provider

R&D

The R&D gap

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Cardiovascular Cancer HIV/AIDS Tuberculosis Malaria Tropical diseases

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Cardiovascular Cancer HIV/AIDS Tuberculosis Malaria Tropical diseases

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Cardiovascular Cancer HIV/AIDS Tuberculosis Malaria Tropical diseases

Trouiller et al., Lancet 2002, 359:2188-94

Relative contribution to global disease burden (DALYs)

Proportion of new drugs, 1975-1999

The R&D gap

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Cardiovascular Cancer HIV/AIDS Tuberculosis Malaria Tropical diseases

equitable level

in relative terms

Trouiller et al., Lancet 2002, 359:2188-94

Relative contribution to global disease burden (DALYs)

Proportion of new drugs, 1975-1999/

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 first-

world 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 Diseaseswww.who.int/tdr

• WHO Report: Priority Medicines for Europe and the World, Chapter 6.9 – Neglected Diseasesmednet3.who.int/prioritymeds/report/index.htm

• Nature: Outlook: Neglected Diseaseswww.nature.com/nature/outlook/neglecteddiseases

• Malaria R&D Alliancewww.malariaalliance.org

• Medicines for Malaria Venturewww.mmv.org

• TB Alliancewww.tballiance.org

• Drugs for Neglected Diseases Initiativewww.dndi.org

• Institute for One World Healthwww.iowh.org

• WHO Onchocerciasis Control Programmewww.who.int/blindness/partnerships/onchocerciasis_OCP

• Merck’s Mectizan donation program www.merck.com/cr/enabling_access/developing_world/mectizan/

• Universities Allied for Essential Medicineswww.essentialmedicine.org and www.ubc-uaem.org