The system to encourage pharmaceutical research and development, in operation since the late 20th century, is increasingly showing its limitations. Important needs are unmet and too many drugs are entering the market at sometimes unaffordable prices, despite offering only minor or even no clinical advantages over existing treatments. These drugs are better suited to generating profits in a market protected by industrial property rights, than to meeting the real needs of populations around the world.

Criticism of this system is growing. Alternatives are being devised and tried out. One example is the search for a treatment for human African trypanosomiasis (or sleeping sickness), a disease particularly prevalent in equatorial Africa. Several parties came together to find solutions to the various difficulties encountered in the field. Chief amongst them, the fact that affected patients tend to be poor, to lack health insurance, and to live in areas which do not have healthcare facilities adequately equipped to administer burdensome treatments. In the late 1990s, Doctors Without Borders (Médecins sans Frontières) set up a non-profit organisation to develop treatments for the millions of patients suffering from neglected tropical diseases: the Drugs for Neglected Diseases initiative (DNDi). DNDi, funded primarily by the public sector and some private foundations, focuses on long-term research programmes, mobilising a network that spans the global North-South divide. In collaboration with the World Health Organization (WHO) and some pharmaceutical companies, a more suitable replacement was found for the former, highly toxic, treatment for trypanosomiasis. Initially, evaluation of nifurtimox + eflornithine showed this intravenous combination to be highly effective in patients with severe disease. But DNDi continued the programme, in order to find an alternative suitable for patients who live far from a healthcare facility.

In collaboration with the Swiss Tropical and Public Health Institute, DNDi identified the potential of oral fexinidazole, details > HERE. In collaboration with Sanofi, a company long involved in this disease, DNDi organised the development of this drug, with the aim of providing an affordable treatment.

This sustained initiative has shown that it is possible to develop drugs that satisfy a genuine need, with the sole aim of providing a therapeutic advance, without the promise of exclusive market access or huge profits.

©Prescrire 1 December 2020

Source: "An exemplary initiative" Prescrire International 2020; 29 (221): 283. Free

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