NIH Unveils $24M Program To Help Develop Drugs For Rare, Neglected Diseases
The NIH on Wednesday announced the launch of a five-year program aimed at developing drugs for rare and neglected diseases that are often too financially risky for pharmaceutical companies to tackle, the Wall Street Journal reports. To jumpstart the new Therapeutics for Rare and Neglected Disease (TRND) Program, Congress has set aside $24 million for the first year.
Rare diseases are those affecting fewer than 200,000 in the U.S., and neglected diseases, "while rare in the U.S., are often linked to parasites that sicken millions of people who live in tropical parts of the world," the Wall Street Journal writes. The program seeks to deliver potential medicines to private companies for development (Dooren, Wall Street Journal, 5/21).
"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," NIH Acting Director Raynard S. Kington said in a press release. While using strategies "similar to approaches taken by pharmaceutical and biotechnology companies," TRND will work to "improve the drug development process itself, developing new approaches to accelerate it and make it less expensive," according to the press release.
"This is the first time NIH is providing support for specific, preclinical research and product development known to be major barriers preventing potential therapies from entering into clinical trials for rare or neglected disorders," said Office of Rare Diseases Research Director Stephen Groft said, adding, "While we do not underestimate the difficulty of developing treatments for people with these illnesses, this program provides new hope to many people world-wide." (NIH press release, 5/20).
The Wall Street Journal reports that one project being considered for funding by the new TRND is a potential drug that would be used to treat the neglected tropical disease schistosomiasis, a parasitic disease that afflicts about 200 million people worldwide (Wall Street Journal, 5/20), and kills roughly 280,000 people each year (Fox, Reuters, 5/20).
CQ HealthBeat reports that while "officials expressed optimism that they would have strong congressional support for the program through 2013," the program will get appropriations annually (Reichard, CQ HealthBeat, 5/20).This is part of the KHN Morning Briefing, a summary of health policy coverage from major news organizations. Sign up for an email subscription.