The US National Institutes of Health has moved beyond its traditional role of supporting basic research with a new programme that involves creating an early drug development pipeline for rare and neglected diseases within the NIH.

The federal agency is putting US$24 million into the Therapeutics for Rare and Neglected Diseases (TRND) programme, which will seek out research collaborations with academic scientists working on rare illnesses.

Unusually, the NIH will develop any promising treatments for rare or neglected diseases to the point where they are sufficiently ‘de-risked’ for pharmaceutical companies, disease-oriented foundations or other interested parties to take them forward into clinical trials.

The Institutes’ Office of Rare Diseases Research (ORDR) will oversee the programme and TRND’s laboratory operations will be administered by the National Human Genome Research Institute (NHGRI). The latter operates the NIH Chemical Genomics Center (NCGC), a principal collaborator in the TRND programme, while other areas of the NIH will also pitch in.

A rare disease is classed as one that affects fewer than 200,000 Americans. The NIH estimates there are more than 6,800 rare diseases afflicting over 25 million Americans in total, yet effective pharmacological treatments exist for only around 200 of these illnesses.

Many neglected diseases also lack treatments. Unlike rare diseases, they may be quite common in some parts of the world, especially developing countries where people cannot afford expensive treatments, the NIH points out. Private companies seldom pursue new therapies for these types of illnesses because of the high costs and failure rates involved and the low likelihood of making a profit, it adds.

“The federal government may be the only institution that can take the financial risks needed to jump-start the development of treatments for these diseases, and NIH clearly has the capability to do the work,” commented Dr Raynard Kington, the Institutes’ acting director.

The TRND programme will work closely with disease-specific experts on selected projects, leveraging in-house capabilities to carry out much of the preclinical development work but also contracting out other components as scientific opportunities dictate. The programme will devote some of its efforts to finding new approaches that speed up the drug development process and lower costs.

In contrast to traditional drug development, TRND will publish its failures as well as its successes, “so everyone in the drug development community can learn from them”, observed NCGC director Dr Christopher Austin.