The National Institutes of Health has thrown its hat into the drug development ring with the announcement of a new program that will seek to bring drugs that treat rare and neglected diseases onto the market.

"This is a tremendously important initiative for people with rare diseases," Peter Saltonstall, president of the National Organization for Rare Disorders (NORD), said in a statement. "There are nearly 7,000 rare diseases and only about 200 of them have an FDA-approved therapy."

The new NIH program, called Therapeutics for Rare and Neglected Diseases, or TRND, will be fueled by $24 million per year for five years. The aim of the $120 million project, according to acting NIH director Raynard Kington, will be to target diseases and disorders that are ignored by pharmaceutical companies because they afflict a relatively small segment of the population - less than 200,000 people in the US by definition.

"NIH is eager to begin the work to find solutions for millions of our fellow citizens faced with rare or neglected illnesses," said Kington in a statement. "The federal government may be the only institution that can take the financial risks needed to jumpstart the development of treatments for these diseases, and NIH clearly has the scientific capability to do the work."

The program will usher compounds through cost-intensive and failure-prone preclinical development, then contract with biotech and pharma companies to conduct trials in patients. "TRND will develop promising treatments for rare diseases to the point that they are sufficiently 'de-risked' for pharmaceutical companies, disease-oriented foundations, or others, to undertake the necessary clinical trials," said acting National Human Genome Research Institute (NHGRI) director Alan Guttmacher in the NIH statement.

TRND will be housed near the NIH's main campus in Maryland, with the NIH's Office of Rare Diseases Research (ORDR) overseeing the program and NHGRI administering its laboratory operations. TRND will also seek collaborations with external partners including academic researchers, patient advocacy organizations, disease-oriented foundations, and others interested in treatments for particular illnesses, according to NIH.

NORD is one patient advocacy group that hopes it can lend its 25 years of experience working with rare disease patients, doctors, and researchers to the trans-NIH initiative. "We are going to be talking to people at NIH about what we can do to help," a NORD spokesperson told The Scientist. "It's important to have the patient community have a role in this."


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