Drugs for Neglected Diseases <italic>initiative</italic> model of drug development for neglected diseases: current status and future challenges

Drugs for Neglected Diseases initiative’s mission & business model „ Scope of diseases, unmet needs & activities covered by Drugs for Neglected Diseases initiative The Drugs for Neglected Diseases initiative (DNDi) is an international and nongovernmental not-for-profit organization committed to the development of novel and/or improved treatments for neglected diseases. The diseases chosen initially included visceral leishmaniasis (VL), Chagas disease and human African trypanosomiasis (HAT, also commonly known as sleeping sickness). They are caused by different but related flagellate protozoa, known as kinetoplastids. In addition, DNDi selected two fixed-dose antimalaria drug projects in response to a well-defined need [1–3]. These neglected diseases, together with other tropical parasitic and viral infections, such as onchocerciasis, schistosomiasis, lymphatic filariasis, Buruli ulcer and dengue (included in WHO’s official list of 17 neglected tropical diseases [101]), account for substantial morbidity and mortality in endemic countries [4,5]. According to the WHO, these diseases are strongly related to poverty, remain endemic in tropical countries, affect populations with low visibility causing stigma and discrimination, and have an important impact on morbidity and mortality. Neglected tropical diseases are also insufficiently addressed from an R&D perspective. As a consequence there is a lack of safe, effective and field-adapted treatments made available to the patients affected by these pathologies. This is illustrated by the fact that only 16 out of 1393 (1.1%) new chemical entity (NCE) marketed drugs from 1975 to 1999 were developed for neglected diseases including malaria and tuberculosis [6]. Although these diseases account for 11.4% of the global disease burden, the efforts to improve the situation have remained marginal in recent years, with only 21 (1.3%) out of 1556 approved molecules that were specifically developed to address neglected diseases between 1975 and 2004 [7]. This poor performance can be explained by the lack of a profitable market, as well as by the absence of stimulating mechanisms leading to funding and execution of R&D activities to efficiently combat these diseases [102]. Fully aware of these issues based on its field experience, Médecins Sans Frontières (Doctors without Borders) committed its 1999 Nobel Peace Prize funds to develop an alternative R&D model for new drugs for neglected diseases that eventually led to the creation of DNDi in 2003.