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NIH to Help Bridge the “Valley of Death” for Rare and Neglected Diseases

BioMarin Pharmaceutical works on the drug Aldurazyme, used to treat arare disease.Yesterday, the National Institutes of Health announced the creation of its new Therapeutics for Rare and Neglected Diseases Program. TRND’s goal is to bolster drug development for rare diseases that affect less than 200,000 Americans as well as neglected diseases that lack treatments, despite being common in some regions of the world. Most neglected diseases are prevalent in developing countries that cannot afford expensive treatments. According to the NIH press release, there are more than 6,800 rare diseases that currently affect over 25 million Americans.

Congress will provide the TRND program with $24 million a year for five years to conduct preclinical research and work on product development for rare and neglected diseases. If a drug survives the preclinical stage, TRND will contract a company to test the treatment clinically. These funds come from the $10.4 billion the NIH received from the stimulus package, intended to be spent by September 2010.

The NIH is targeting these diseases because private companies avoid this small market with little profit appeal, leaving patients with no treatment options. Only about 200 rare diseases have effective treatments today, as there are high barriers of entry for the pharmaceutical industry. Studies estimate that a potential drug requires between 8 and 15 years and upwards of $800 million to develop a new drug and introduce it to the market. The preclinical process, a focus of the TRND program, is the most difficult hurdle, costing $10 million and between two to four years itself. Without a successful preclinical stage, a treatment only has a 10 to 20 percent chance of reaching clinical trials, hence the nickname for this period, the “Valley of Death.” The NIH plans to use its funding and in-house academic resources to ease this process. The program will also address the difficulties in working with rare diseases, such as recruiting enough clinical trial patients, by partnering researchers with patient advocacy organizations and disease-oriented foundations.

The National Organization for Rare Disorders keeps a current database of rare diseases and associated organizations.

Image: AP/Marcio Jose Sanchez

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