$120 Million for Rare and Neglected Diseases
This investment will also not only benefit Americans, but also people in developing countries, where US rare diseases are often common and the limited treatments are very expensive
The US National Institute of Health (NIH) defines a rare disease as one that affects fewer than 200,000 Americans. However, the NIH Office of Rare Disease Research (ORDR) estimates that between 25 and 30 million people in the USA suffer from one of these diseases. More than 6,800 diseases fall into this classification, but of those, only around 200 are treatable.
This may all be about to change, as the US Congress has just voted the NIH $24 million per year for the next five years ($120 million in total) to start the Therapeutics for Rare and Neglected Diseases program (TRND). This funding will go towards developing pharmaceutical treatments for these rare diseases, and the laboratory work will be conducted by the National Human Genome Research Institute and be co-ordinated by the ORDR, which will also involve various other parts of the NIH in the research including the Chemical Genomics Centre.
The reason that rare disease research has been so neglected is that private companies seldom devote time and resources to it, given the costs involved. If researchers discover a new possible drug target for a disease treatment (and this is the case for all drugs), a pharmaceutical company must spend an average of $10 million over two to four years to get through the pre-clinical phase to clinical trials, and 80 to 90 percent of drugs do not make it that far. In the case of major diseases, the small fraction of successful drugs recoup the investments lost in the failures, however, when it comes to rare diseases, this is not often the case so drug developers spend their money elsewhere.
This is the situation that the NIH’s new funding hopes to rectify. It can by no means solve the entire problem, but it will provide the NIH with the means to take important first steps. Finally, this investment will also not only benefit Americans, but also people in developing countries, where US rare diseases are often common and the limited treatments are very expensive. The work done by the NIH with this money may able to change this, and so benefit those with these diseases suffering all over the world.