The National Institutes of Health (NIH) is launching the first integrated drug development pipeline to produce new treatments for rare and neglected diseases. The $24 million initiative jumpstarts the trans-NIH Therapeutics for Rare and Neglected Diseases (TRND) program.
The program is unusual because TRND creates a drug development pipeline within the NIH and is specifically intended to stimulate research collaborations with academic scientists working on rare illnesses. The NIH Office of Rare Diseases Research (ORDR) will oversee the program, and TRND’s laboratory operations will be administered by the National Human Genome Research Institute. Other NIH components will also participate in the initiative.
A rare disease is one that affects fewer than 200,000 Americans. NIH estimates that, in total, 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. Private companies seldom pursue new therapies for these types of illnesses because of high costs and failure rates, and the low likelihood of recovering investments or making a profit.
"This initiative is really good news for patients with rare or neglected diseases," said ORDR Director Stephen C. Groft, PharmD. "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 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. While we do not underestimate the difficulty of developing treatments for people with these illnesses, this program provides new hope to many people worldwide."
TRND will work closely with disease-specific experts on selected projects, leveraging both the in-house scientific capabilities needed to carry out much of the preclinical development work, and contracting out other parts, as scientific opportunities dictate. Its strategies will be similar to approaches taken by pharmaceutical and biotechnology companies, but TRND will be working on diseases mostly ignored by the private companies. Importantly, TRND will also devote some of its efforts to improving the drug development process itself, creating new approaches to make it faster and less expensive.
If a compound survives the preclinical stage, TRND will work to find a company willing to test the therapy in patients. There are several stages to the clinical trials process that can take several years before the safety and efficacy of a new drug is determined. The Food and Drug Administration will only approve a drug for general use after it passes these trials. The clinical trials process is also expensive, but the failure rate is lower at this stage. TRND will seek to take advantage of several NIH resources that can help launch human studies, including the NIH Clinical Center, the NIH Rapid Access to Interventional Development, and the Clinical and Translational Science Awards program.
Numerous obstacles impede the development of new drugs for rare and neglected diseases. In addition to the reluctance of private companies to risk their capital on a potentially low return, relatively few basic researchers study rare diseases, so the underlying cause of the illness frequently remains unknown. Also, because rare diseases are rare, researchers often have difficulty recruiting enough people with the disorder to participate in a clinical trial once a candidate compound reaches the stage where it can be tested in people. Moreover, for many rare diseases, the natural history of the disease is poorly understood, so researchers lack the needed clinical measures (such as blood pressure) that can demonstrate 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 drug development is reached.
TRND is currently setting up an oversight process to help it decide which projects that address thousands of rare and neglected diseases will be pursued. Leadership currently envisions a small number of diseases being studied each year, with strict criteria used to determine which molecules will be studied for which diseases. 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: NIH News 5/20/09