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Filling R&D Gaps Key To Succeeding In NTD Control And Elimination Goals By 2020

January 30, 2012

On the occasion of today’s high-level event in London, ‘Uniting to Combat Neglected Tropical Diseases,’ organized in support of the new World Health Organization (WHO) Neglected Tropical Disease (NTD) 2020 Roadmap, the Drugs for Neglected Diseases initiative (DNDi) welcomes the commitments from various partners and emphasizes that filling the major gaps in research and development (R&D) for new treatment and diagnostic tools is key to effectively support elimination or control of targeted NTDs by 2020.

Since its inception in 2003, DNDi, a not-for-profit R&D organization, has worked to develop and deliver new drugs for NTDs, in particular those with the highest mortality rates: human African trypanosomiasis (sleeping sickness), Chagas disease, and visceral leishmaniasis (kala azar). DNDi was created to address the market and public-policy failures that had led to a virtual standstill in R&D for new drugs to treat neglected diseases. Working collaboratively with public and private partners worldwide, DNDi has developed six new treatments for neglected patients, with several new drug candidates in the R&D pipeline, including two for sleeping sickness (fexinidazole, oxaborole/SCYX-7158) and one for Chagas (E1224) in clinical trials.

‘For some NTDs, especially those with the highest death rates such as sleeping sickness, Chagas disease, and visceral leishmaniasis, disease control or elimination will only be achievable with further commitments to R&D,’ says Dr Bernard Pécoul, Executive Director of DNDi. ‘New drugs and diagnostics are urgently needed to improve patient care, respond to the challenge of drug resistance, and enhance prospects for achieving disease elimination.’

In the case of sleeping sickness, which continues to kill in remote or unstable pockets of sub-Saharan Africa, diagnostic tools are inadequate (painful lumbar punctures must be performed), and while nifurtimox-eflornithine combination therapy is an improved option therapy, it still requires intravenous infusions. For visceral leishmaniasis, prevalent in East Africa, India, and Brazil, invasive diagnostics, long treatment duration (30 days), and drug resistance (up to 65% in India) pose obstacles to control. For Chagas disease, no test of cure exists, and safe, effective drugs have yet to be specifically developed for chronic-stage disease, though over 100 million people in Latin America are at risk.

For the filarial parasitic-worm diseases of onchocerciasis (river blindness) and lymphatic filariasis (elephantiasis), which together infect over 150 million people, the standard treatment of ivermectin, alone or in combination with albendazole, can lead to brain damage or death in people co-infected with loiasis (Loa loa; African eye worm). Moreover, current drugs kill only juvenile and not adult worms, which continue to infect, requiring repeated treatments.

Therefore while continuing to provide existing medicines for NTDs, parallel and greatly enhanced R&D initiatives for new drugs and diagnostics are necessary for disease control or elimination.

Based on DNDi’s experience over the past nine years, there are four key elements necessary to support the WHO NTD Roadmap goals by 2020:

    Boosting innovation and promoting more open sharing of research knowledge: For instance, today it was announced that 11 pharmaceutical companies have committed to provide access to their compound libraries, including data and knowledge about the compounds, to find potential drug candidates for NTDs. Licensing agreements concluded, for example the agreement announced between DNDi and Abbott, ensure patient access to drugs developed from the collaboration in all endemic countries, regardless of income level.
    Forging public and private innovative partnerships for drug development: The collaborations DNDi has established with private partners are essential to developing new treatments. DNDi seeks a balanced mix of partnerships with private and public organizations, including academia, national control programmes, and implementing organizations. Innovative industrial partnerships include Eisai, Sanofi, and Cipla to co-develop new drugs at an affordable price, and a collaboration announced today with Johnson & Johnson, Abbott, and Pfizer to work on a macrofilaricide drug for filarial diseases.
    Ensuring endemic-country involvement and leadership to meet patient needs: Strong leadership from governments, including national control programmes, is essential for defining needs and eventually ensuring treatment access for patients, as well as playing a crucial role in conducting research. DNDi works with partners in endemic countries to strengthen regional clinical research networks, which bring together researchers, clinicians, regulatory agencies, national program managers, and, ideally, patients themselves. These research platforms are vital for ensuring that the R&D process starts and ends with patient needs at the centre.
    Attaining sustainable and diversified funding for R&D: DNDi welcomes the financial commitments from DFID (UK), the Bill & Melinda Gates Foundation, and public funders such as Germany, France, the Netherlands, Spain, and Switzerland, yet additional commitments from other major funders, such as the US Agency for International Development (USAID) and governments of emerging economies, must be secured to support accelerated NTD product development. In addition, new R&D incentives, such as prizes, and innovative financing mechanisms, such as a financial transaction tax (FTT) for health, must be supported.

‘To make a real difference in the lives of the poorest and most neglected populations—to bring the best science to the most neglected —we must go beyond individual success stories and move toward sustainable change, and today’s commitments show that we are headed in a right direction’, says Dr Pécoul. ‘We need both strong coordination of programmes to treat patients today and a new global framework for R&D under the leadership of WHO to ensure R&D priority setting according to needs, secure sustainable financing, and establish an enabling policy environment that guarantees both innovation and access for neglected patients.’

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