The current focus is on collaborating with disease foundations, industry, and academic investigators with disease-relevant assays to screen against the approved drug collection acquired by NCGC. Any new therapeutic use of an approved drug would require additional studies including clinical trials in that disease, approved by the U.S. Food and Drug Administration. Given the cost and limited quantities of the drugs in the collection, each partnership to screen the NPC will be evaluated based on the quality of each disease-related assay and its scientific merit.
Creating a new drug is expensive. Recouping the investment can be difficult for rare diseases, due to the small number of patients with the disease or, in the case of tropical neglected diseases, the limited ability of patients to pay for treatments. Today, therapies are available for less than 300 rare diseases.
Drugs that receive regulatory approval have been demonstrated to be reasonably safe and effective in the treatment of a specific disease or condition. When such drugs are used in large populations, new benefits or adverse effects can be discovered. Subsequently, the use of approved drugs can be expanded beyond what a drug was originally approved for to treat other health conditions.
Thalidomide is an example of repurposing a drug with serious adverse effects in one condition to treat another disease, according to the authors. In the 1950s, it was used as a sedative and as a treatment for morning sickness during pregnancy. It was later withdrawn because it was found to cause severe birth de
|Contact: Geoff Spencer|
NIH/National Human Genome Research Institute