Amy Maxmen in Nature:
First, there was the pitching and rolling in an old Jeep for eight hours. Next came the river crossing in a slender canoe. When Nathalie Strub Wourgaft finally reached her destination, a clinic in the heart of the Democratic Republic of the Congo, she was exhausted. But the real work, she discovered, had just begun. It was July 2010 and the clinic was soon to launch trials of a treatment for sleeping sickness, a deadly tropical disease. Yet it was woefully unprepared. Refrigerators, computers, generators and fuel would all have to be shipped in. Local health workers would have to be trained to collect data using unfamiliar instruments. And contingency plans would be needed in case armed conflict scattered study participants — a very real possibility in this war-weary region. This was a far cry from Wourgaft's former life as a top executive in the pharmaceutical industry, where the hospitals that she commissioned for trials were pristine, well-resourced and easy to reach. But Wourgaft, now medical director for the innovative Drugs for Neglected Diseases initiative (DNDi), was confident that the clinic could handle the work. She was right. With data from this site and others, the DNDi will next year seek approval for a sleeping-sickness tablet, fexinidazole. It would be a massive improvement on existing treatment options: an arduous regimen of intravenous injections, or a 65-year-old arsenic-based drug that can be deadly.
The DNDi is an unlikely success story in the expensive, challenging field of drug development. In just over a decade, the group has earned approval for six treatments, tackling sleeping sickness, malaria, Chagas' disease and a form of leishmaniasis called kala-azar. And it has put another 26 drugs into development. It has done this with US$290 million — about one-quarter of what a typical pharmaceutical company would spend to develop just one drug. The model for its success is the product development partnership (PDP), a style of non-profit organization that became popular in the early 2000s. PDPs keep costs down through collaboration — with universities, governments and the pharmaceutical industry. And because the diseases they target typically affect the world's poorest people, and so are neglected by for-profit companies, the DNDi and groups like it face little competitive pressure. They also have lower hurdles to prove that their drugs vastly improve lives. Now, policymakers are beginning to wonder whether their methods might work more broadly. “For a long time, people thought about R&D as so complicated that it could only be done by the biggest for-profit firms in the world,” says Suerie Moon, a global-health researcher at the Harvard T.H. Chan School of Public Health in Cambridge, Massachusetts, who studied PDPs and joined the DNDi's board of directors in 2011. “I think we are at a point today where we can begin to take lessons from their experience and begin to apply to them non-neglected disease,” she says.