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10 February 2014

Drugs for Neglected Diseases

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This week, in an event hosted by Fundación madrid+d, Raquel González, the Madrid Delegate for Doctors Without Borders, shared with OpenMind a revealing perspective on the advances of medicine and the priorities of medical investigation. With the 21st century well underway, there are 17 diseases classified as neglected. The reason is that

in spite of revolutionary progress in the development of medicines over recent decades, the essential drugs needed to treat many catastrophic diseases affecting the world’s poor people are too expensive, no longer in production, very toxic, or ineffective.

There are 17 diseases that the World Health Organization (WHO) classifies as neglected, because they are not on the priority lists of worldwide bio-medical and pharmaceutical research. While it is estimated that these diseases account for 90% of the global morbidity rate in the planet, affecting over one billion people worldwide, half of them children, they only receive 10% of research funds. The net result is that among the 1,556 new pharmaceutical products approved between 1975 and 2004, only 21 (or 1.3%) had been developed specifically for tropical diseases including malaria and tuberculosis.

To address the situation, the Drugs for Neglected Diseases Initiative (DNDI) was established in 2003 as a independent non-profit organization centered on the needs of patients. Its creation was upon agreement by seven public and private institutions: Doctors Without Borders, the Indian Council on Medical Research, the Kenyan Technical Research Institute, the Ministry of Health of Malaysia, the Pasteur Institute (France), the Oswaldo Cruz (Fiocruz) Foundation of Brazil, and the Special WHO Program for Neglected Diseases Research and Training.

Its objective is to develop medicines especially for people suffering from the most neglected diseases. These diseases affect the poorest people who live in Africa in particular, but also in Asia and Latin America. DNDi now boasts 120 employees in its offices in Switzerland, Brazil, Congo, Kenya, Malaysia, the US, and Japan, with a network of an additional six hundred collaborating people.

Its work therefore consists in detecting the need for treatments and involve public and private institutions–including pharmaceutical labs–to produce and distribute these treatments with the highest quality standards. The organization also promotes the taking place of the investigation and development phases in the affected countries. The objective is to obtain effective drugs that are inexpensive and may be administered easily and quickly in order to promote the uninterrupted use of these treatments. All of the above according to the most exacting quality and security standards. With its partners, DNDi has developed and distributed new and accessible treatments for diseases linked to poverty, including Chagas disease, sleeping sickness, malaria or leishmaniasis, which affect the most vulnerable population groups in the world,

Poverty and disease are closely linked. People without economic resources in developing countries are not only faced with a high number of neglected diseases, they also suffer exclusion from the health system. Neglected diseases imply economic and social costs for people individually, for their families, communities, and countries. In general they are not considered epidemiological emergencies and as a result attract little attention from the media and public sector. In addition, private companies do not consider this group of diseases as a lucrative sector, which constitutes a considerable obstacle when the time comes to invest in research and development (R&D) for diagnostic tools, vaccines, and specific treatments.

It is a health, but also economic, problem. As DNDi Director Bernard Pécoul explains, these illnesses “are one of the important reasons that the 1.4 billion people living under the poverty threshold cannot emerge from marginalization […]; they are the most common infections among the world’s poorest, the main cause of chronic disability and poverty” (1). And this is the vicious circle: a population condemned to perpetual misery is not attractive in the eyes of the market. “They are diseases that lead to death or to affected young people’s inability to work, which impoverishes their countries,” Pécoul says. “Their economic impact is very strong. The affected people cannot sustain a market, so there is no investment from the private sector.”

Still, new partnerships between the private and public sector that are nonprofit and dedicated to pharmaceutical R&D are starting to fill this gap, at least with respect to diseases such as visceral leishmaniasis, African human trypanosomiasis, and Chagas disease.

The DNDi represents an institutional model of good practices, that brings scientific research to the area of development cooperation by way of knowledge management and the dissemination of results for economically marginalized populations suffering from neglected diseases. DNDi is currently developing urgently needed new medicines for the treatment of malaria, leishmaniasis, Chagas disease, and sleeping sickness, all of which have high morbidity and mortality rates. Although the transmission of the most neglected diseases does not discriminate, special care is necessary to address the needs of those who are the most neglected among the neglected: women and children. The initiative has also set for itself the secondary objective of reinforcing local research capacity in countries where the diseases are endemic. A third objective that is equally important consists in social sensitization through testimony regarding the needs of new medicines for neglected diseases. Before it is possible to access patients, treatments for Neglected Diseases that are effective and adapted to the local contexts must be created. The DNDi aims to continually extend the reach of its global network of contacts with respect to the specific objectives linked to neglected disease medicine development. Current contacts include scientists and academics, pharmaceutical companies, NGOs, IGOs, the WHO, Health Ministries, national programs for the control of diseases, and other partnerships for the development of vaccines, medicines, diagnostic means, and financing agencies. All work together with a view to developing better and more adapted treatments. All share the evolution of the effort and all strive to promote northern-southern and southern-southern partnerships as well as technology transfers. The larger the global contacts network to share knowledge concerning neglected diseases, the more likely it will be that capacities are used to achieve the best possible results for patients.

The majority of patients with neglected diseases live in environments with health systems that have not been designed to meet their needs and this turns them into neglected patients. In principle, the DNDi works in close collaboration with the governments of these populations to research and develop new treatments that are adequate to the context in which they will be administered (taking into account for instance the thermal stability, or their oral, rectal, intravenous, or by injection administration) and actively supports efforts to optimize the purchasing and distribution capacity. The DNDi offers its support to help in the optimization of existing purchase and distribution networks developed by the ministries of health, pharmaceutical companies, the WHO, the Global Fund, UNICEF, nonprofit distributors, MSF, and other NGOs. The DNDi also supports and advocates on behalf of new purchase mechanisms being developed such as UNITAID as well as global subsidy programs to oversee the adequate use of improved medicines. Finally the DNDi can contribute to extending the distribution of effective medicines in the Southern region by generating income based on sub-licensing agreements for the sale of medicines in Northern region.

Sleeping sickness, endemic in 36 African countries, causes tens of thousands of deaths every year. Its last epidemic took place at the end of the nineties and affected half a million people. The medicine that was most used in dealing with the disease then, melarsoprol, is so toxic that it can be lethal and its only alternative until recent times was a very expensive medicine that was complicated to administer and that even disappeared from the market as the manufacturer found it to be unprofitable. This has changed thanks to DNDi. Since 2009, a medicine exists that boasts better effectiveness, cost, and logistics. It is the first new drug to deal with the disease in 25 years.

In addition to this new remedy, the DNDi has developed another five that improve upon already existing drugs. Two are medicines for malaria: ASAQ, developed in collaboraton with Sanofi, manufactured in Africa, and distributed in 32 countries, and ASMQ, based on a technology transfered from Brazil to the NDIA. Another two are for leishmaniasis, and the sixth drug is for Chagas disease, with a child dose developed by a public laboratory in Brazil. Objectives for the immediate future are filariasis and child HIV. Another thirty projects are underway, eleven of which concern new medicines.

“We have been able to sign contracts with many pharmaceutical laboratories including in very early stages of the development of pharmaceuticals,” Pécoul indicates. “They know that they are not going to do business but they are projects that receive support from within the company itself, and they also realize that the future market will be in those countries.” Among its partners is the Spanish plant of the GSK Laboratory, which is focused on medicines for neglected diseases.

Neglected tropical diseases affect the poorest ammong the poorest, people “without political or economic power” whose health is also an indicator of our own, Pécoul reminds us. He illustrates the point with a quote from Mahatma Gandhi: “A civilization is judged by how it treats its minorities.”

To access the original version of this article published in Madrid+d, click here.

Raquel González

Madrid Delegate of Médicos sin Fronteras (Doctors without borders)

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