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looking ahead
SCIENCE WITH A SOUL

DEVELOPING DRUGS
FOR NEGLECTED DISEASES



source: www.accessmed-msf.org

website: www.dndi.org

neglected diseases
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context: over the last few decades, progress in science has enabled increasingly sophisticated medicines to be developed to cure a wide variety of diseases. Research programmes in both private and public sectors, however, have progressively marginalised some infectious tropical diseases. These ‘neglected diseases’, such as leishmaniasis, trypanosomiasis and Chagas disease, have a devastating impact on the world’s poor – but because they affect only the poor, they do not constitute a market lucrative enough to attract investment in research and development of new drugs.

Currently available treatments for each of these diseases leave a great deal to be desired. It is clear that patients in developing countries afflicted by neglected diseases urgently need new treatments that are safe, efficient, short course, easily administered, reasonably priced and accessible.

what:in the summer of 2003 a new not-for-profit drug research organisation that will harness cutting-edge science to develop medicines for diseases afflicting the world’s poorest people was established in Geneva. Prestigious health and research institutes from Brazil, France, India, Kenya and Malaysia joined Médecins Sans Frontières to launch the Drugs for Neglected Diseases Initiative (DNDi).

DNDi will work in close collaboration with the UNDP/World Bank/WHO Special Programme for Research and Training in Tropical Diseases (WHO/TDR) to achieve its goals. One year after its launch, this organisation is starting clinical trials for two fixed-dose drug combinations against malaria, and counts another seven projects in the pipeline for sleeping sickness, visceral leishmaniasis and Chagas disease.

who: the six organisations involved are the Indian Council of Medical Research, Institut Pasteur (France), the Kenya Medical Research Institute, Médecins Sans Frontières, the Ministry of Health of Malaysia and the Oswaldo Cruz Foundation (Brazil). WHO/TDR will participate in the meetings of the Scientific Advisory Committee of DNDi as an observer to provide expert scientific and technical advice as required.

why: a mere 10% of the world’s health research efforts go into diseases that account for 90% of the global disease burden. “Patients in developing countries are being forced to use drugs with failing efficacy and significant side-effects", said Dr Yves Champey, interim director of DNDi. “They deserve a better deal. DNDi will mobilise scientific innovation to create new medicines for the world’s most neglected patients".

sustainable R&D: DNDi plans to spend around US$250 million over 12 years to develop 6-7 drugs including several drugs already in the pipeline to combat diseases that threaten a combined 350 million people every year. To increase the chance of short and middle-term success, the organisation will develop drugs from existing compounds as well as fund and co-ordinate research to identify and develop new chemical entities.

DNDi will be the first not-for-profit organisation to exclusively focus on the world’s most neglected diseases. Moving away from the traditional public-private partnership structure, it intends to take drug development out of the marketplace by encouraging the public sector to take more responsibility for health. This basic principle is reflected in the composition of its founding partners, four of which are public sector institutions. DNDi’s success will depend not only on government and private donations but also on the contribution of pharmaceutical companies, for instance access to compound libraries, expertise, and R&D facilities.

filling the gap: the network proposes an alternative model of research and development that matches patients’ needs with gaps identified in the drug development process. At the end of its first year, the Drugs for Neglected Diseases Initiative has a balanced portfolio of nine long-, medium-, and short-term projects that fill identified gaps in the drug development pipeline: at the early discovery stage, at the stage before drugs enter clinical development, and at the point where drugs should reach patients but do not.


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