$24 million for rare and neglected diseases research

The National Institutes of Health is launching the first integrated, drug development pipeline to produce new treatments for rare and neglected diseases. The $24 million program jumpstarts a trans-NIH initiative called the Therapeutics for Rare and Neglected Diseases Program, or TRND.

A rare disease is one that affects fewer than 200,000 Americans. NIH estimates that more than 6,800 rare diseases afflict more than 25 million Americans. However, effective pharmacologic treatments exist for only about 200 of these illnesses.

Many neglected diseases also lack treatments. Unlike rare diseases, however, neglected diseases may be quite common in some parts of the world, especially in developing countries where people cannot afford expensive treatments.

The drug development process is complicated and expensive. Studies suggest that it currently takes more than a dozen years and hundreds of millions of dollars to take a potential drug from discovery to the marketplace. And the failure rate is high.

“This initiative is really good news for patients with rare or neglected diseases,” said Stephen C. Groft, Pharm.D., Director of the NIH Office of Rare Diseases Research (ORDR). “While Congress has previously taken important steps to help these patients, such as providing incentives for drug companies under the Orphan Drug Act, this is the first time it 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.”

“NIH traditionally invests in basic research, which has produced important discoveries across a wide range of illnesses,” said Alan E. Guttmacher, M.D., NHGRI Acting Director. “Biotechnology and pharmaceutical companies have enormous strength and experience in drug development, but to maximize return-on-investment work primarily on common illnesses. 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. NIH’s goal is to get new medications to people currently without treatment, and thus without hope.”

NIH already has many components of the drug development pipeline within its research programs. TRND will begin its work in collaboration with the NIH Chemical Genomics Center (NCGC), initially developed as part of the NIH Roadmap for Medical Research.

Molecules with potential therapeutic properties emerging from the NCGC screening process could be fed into the TRND drug development pipeline.

Numerous obstacles impede the development of new drugs for rare and neglected diseases. Moreover, for many rare diseases, the natural history of the disease is poorly understood and researchers lack clinical measures (such as blood pressure) that show whether a treatment is working.

To address these difficulties, TRND will seek a wide range of collaborations with academic researchers as well as partnerships with patient advocacy organizations, disease-oriented foundations and others interested in treatments for particular illnesses. TRND’s leaders hope that the collaborations will help lay the groundwork for clinical trials once that point in the drug development is reached.

NIH expects to use existing intellectual property policies to transfer licenses for TRND-discovered drugs to private companies or others for development, clinical testing and marketing.

Source: National Human Genome Research Institute, USA



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