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NIH To Develop Drug Pipeline For Rare Diseases

by Britt E. Erickson
May 18, 2009 | A version of this story appeared in Volume 87, Issue 21

Credit: Shutterstock
The schistosomiasis parasite is transmitted in water.
Credit: Shutterstock
The schistosomiasis parasite is transmitted in water.

NIH plans to invest $24 million a year for the next five years to jump-start an agency-wide effort called Therapeutics for Rare & Neglected Diseases (TRND). The initiative is expected to create within NIH a pipeline of new treatments for such diseases. The agency defines rare diseases as those that afflict fewer than 200,000 Americans. NIH estimates that there are more than 6,800 rare diseases, but only about 200 of them have effective pharmacologic treatments. Neglected diseases are those that may be common in developing countries where treatments are unaffordable. Private pharmaceutical or biotechnology companies rarely pursue therapies for such diseases because of the high failure rates and the low likelihood of making a profit. "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," Raynard S. Kington, acting director of NIH, said in a statement. NIH would not disclose which rare or neglected diseases it will focus on first but said it plans to complement the work of private industry, not compete with it. One likely candidate is schistosomiasis, a parasitic disease that afflicts many people in developing countries, according to NIH officials. NIH's goal is to eventually transfer licenses for TRND-discovered drugs to private companies for further development, clinical testing, and marketing.


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