Executive summary

People in developing countries, who make up about 80% of the population, only represent about 20% of worldwide medicine sales. For these people, the imbalance between their needs and the availability of medicines is fatal. This report seeks to explore one element of this stark reality: the lack of research and development (R&D) into drugs to treat the diseases of the poor. In 30 years of work, MSF has witnessed first-hand the human impact of the lack of drugs for infectious diseases. Until very recently, patients suffering from sleeping sickness had to undergo painful treatment with an arsenic-based medicine because more effective treatment was unavailable. Yet the disease afflicts up to 500,000 people and threatens another 60 million in Africa. For Chagas disease, which threatens a quarter of the population of Latin America, only children can be treated because no effective drugs exist for adults. The human suffering caused by infectious diseases could be reduced; with billions of dollars dedicated to health R&D it should be possible to develop effective treatments for these diseases. However, the lack of R&D for diseases common in developing countries means that very few new drugs have been brought to market for them.
By Tonm Stoddart/IPG A young patient receives treatment at an MSF sleeping sickness clinic in Omugo, Uganda. This painful disease infects up to 500,000 people and threatens 60 million more, predominantly in sub-Saharan Africa. The drugs available to treat sleeping sickness are archaic, toxic or difficult to administer. © Tom Stoddart/IPG
In 1999, MSF convened an international body of health experts to study the current state of drug R&D for diseases that affect people in the developing world. This independent body, the Drugs for Neglected Diseases (DND) Working Group, has since undertaken an analysis and proposed some recommendations for moving forward. When treatment options don't exist or are inadequate, a disease can be considered "neglected," or even "most neglected" in some cases. The neglect is a result of market failure and public policy failure. Strategies must be developed to address neglected and most neglected diseases specifically. In spring 2001, the 20 top-grossing pharmaceutical companies in the world were surveyed about recent drug development activity. While the survey demonstrated some activity in neglected diseases, it indicated that private sector investment in this field was minimal. None of the responding companies has brought a drug to market in the last five years for any of the most neglected diseases included in the survey. The DND Working Group has also explored the failure of the public sector to take a needs-based approach to managing drug development. Basic research leading to discovery of compounds - and thus potential drugs - has almost always been publicly funded. However, because politicians naturally respond to the needs of their constituencies, and because wealth is concentrated in industrialized countries, research money goes to the diseases primarily affecting these wealthier constituencies. While some government money has been devoted to diseases affecting developing countries, it is a pittance compared with overall spending on drug development. Private philanthropy has in recent years sought to fill in a bit of this gap, but it is not sufficient and cannot and should not take the place of public support. Recent initiatives and policies seeking to redress the R&D imbalance are also outlined here. Public-private partnerships have been successful in mobilizing public and private sector expertise around certain diseases. Yet, to date, none of these provides an adequate strategy for developing drugs for the most neglected diseases. Finally, recommendations for moving forward are presented, among them: that a well-defined and needs-driven research agenda be established at a global level; that governments fulfill their responsibility to become directly and proactively involved in searching for solutions; that funding be increased for research into neglected and most neglected diseases; and that a new not-for-profit initiative be explored as one way to address the shortage of R&D for the most neglected diseases.