Alternative Approaches to New Drug Development: Two R&D Initiatives

Author(s): Philippe Desjeux1, Bernard Pécoul2
Affiliation(s): 1Senior Programme Officer for Disease Control, iOWH, San Francisco, CA, USA, 2Executive Director, Drugs for Neglected Diseases Initiative (DNDi), Geneva, Switzerland
Summary (max 100 words): Philippe Desjeux: The illnesses of invisible people usually stay invisible. This statement is reflective of the limited attempts to develop new treatment regimens for neglected diseases. Most of these diseases are preventable or curable, but often strike poor and marginalized people living in remote rural areas. Development of effective, safe and affordable drugs for neglected diseases is an urgent need. Many of the available drugs are not adequate: they are either toxic, difficult to administer or too expensive. Therefore, investment in drug research and development for neglected diseases is crucial to bridge the gap between the pharmaceutical R&D model and the unique requirements of the global health field, where the traditional market system does not work. Meeting the challenge: The Institute for OneWorld Health (iOWH) is the first non-profit pharmaceutical company in the USA, formed to address the 10/90 gap in health R&D. Our mission is to develop safe, effective and affordable new medicines for people with infectious diseases in the developing world. OneWorld Health’s core competencies lie in pharmaceutical product development. Our in-house teams identify development leads through partnerships with industry and universities. Together, iOWH works to optimize existing drug candidates, complete preclinical and clinical investigation needs, secure quality manufacturing of developed products, and obtain the necessary regulatory approvals to bring the product to the beneficiaries. Concurrently, iOWH collaborates to devise product delivery and access strategies with relevant stakeholders, which include governments, donors, and international NGOs. These Public-Private Partnerships (PPPs) allow for a more rapid development of new drugs by utilizing complementary skills and resources. iOWH’s current pipeline includes programmes for visceral leishmaniasis (VL), malaria, and diarrheal disease. Paromomycin, for the treatment of VL, is currently the most advanced pharmaceutical product for iOWH. After the completion of a Phase III clinical trial in Bihar, India, for paromomycin, iOWH has submitted the dossier for regulatory approval in India. iOWH’s product selection criteria is designed to meet the needs of the poor in the developing world. In addition to customary selection criteria such as scientific merit, probability of success, clinical and regulatory developmental path, iOWH examines the unmet medical needs in the developing world, cost-of-goods, and mechanisms of delivery, including the endemic country’s infrastructure. These criteria allow us to devise products that will be both appropriate for communities with high disease burden and affordable and accessible to the population in a manner which is sustainable. Conclusions: iOWH strives to provide a flexible and innovative vehicle to engage both the pharmaceutical and biotech industries, as well as public health organizations in global health product development. Through partnerships and collaborations, by adhering to the highest ethical standards for clinical research, and by utilizing the scientific and manufacturing capacity of the developing world, OneWorld Health can deliver affordable and effective new medicines where they are needed most.Bernard Pécoul: The majority world shoulders a disproportionate burden of disease and has few drugs with which to respond to this challenge. In 2005, Africa, Asia (excluding China)- Pacific, and Latin America, which housed 63 per cent of the world?s population, had a mere 11.7 per cent share of the world's $602 billion pharmaceutical market. This stark disparity is echoed in the dearth of research funding dedicated to the diseases prevalent in developing regions. Over the past 30 years, only 21 of the 1,556 new chemical entities marketed between 1975 and 2004 were for tropical diseases and tuberculosis. Millions continue to suffer from diseases such as tuberculosis, malaria, leishmaniasis, sleeping sickness, and Chagas disease. Regrettably, these diseases target impoverished populations with immune systems already weakened by hunger and other diseases. If patients are to have any hope of survival they urgently need new, more effective treatments for these diseases, as the few available drugs are compromised by poor efficacy, toxicity, long courses of treatment, parenteral administration and resistance to the parasite.
Meeting challenges: The Drugs for Neglected Diseases Initiative (DNDi), a not-for-profit drug R&D initiative, is seeking to research and develop new drugs for these neglected diseases. Existing treatments for these diseases are often inadequate and ineffective and patients need new medicines urgently. Founded by a group of 5 renowned medical research organizations including the Indian Council for Medical Research, the Oswaldo Cruz Foundation from Brazil, the Kenya Medical Research Institute, the Ministry of Health of Malaysia, and the Pasteur Institute, as well as the WHO’s Special Programme for Research and Training in Tropical Diseases, and Médecins sans Frontières (MSF), DNDi presents an alternative approach to drug development. It facilitates north-south and south-south collaboration, capacity building, and knowledge sharing among researchers, scientists, industry, and governments.
Conclusion (max 400 words): DNDi's current portfolio of 20 projects focuses on discovery and development projects for malaria, leishmaniasis, sleeping sickness, and Chagas disease. Its alternative approach will make new drugs available for the treatment of neglected diseases within the next decade. It is already on the road to success with its two fixed-dose artesunate-based combination therapies scheduled to be delivered to patients by the end of 2006.

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