With the cost of bringing a new drug to market now exceeding $1 billion dollars and taking up to 15 years to test and develop, global pharmaceutical companies are focused on return on investment or making a profit.
Of 7,000 rare diseases only 200 have effective pharmacologic treatments, according to NIH. Where does that leave 25 million Americans who suffer from 7,000 different rare diseases?  Millions of people are faced with the fact that new drugs will never reach them or their loved ones in their lifetime because global pharmaceutical companies control the entire drug development process.
To begin to address the global drug development crisis, The National Institutes of Health (NIH) this week announced the first integrated, drug development pipeline to produce new treatments for rare and neglected diseases.   The $24 million program jump starts a trans-NIH initiative called the Therapeutics for Rare and Neglected Diseases Program, or TRND.  The program, funded by Congress this spring, 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 these illnesses.
In essence, the government has realized it needs to take more control and become a pharmaceutical company focused on health not profits in order to try and help millions of people who currently have no hope.
The NIH Office of Rare Diseases Research will oversee the new cutting edge program, and TRND’s laboratory operations will be administered by the National Human Genome Research Institute (NHGRI), which also operates the NIH Chemical Genomics Center, a principal collaborator in TRND.
Thankfully our government has recognized the problem and is putting much needed dollars into programs to help find treatments for rare and neglected diseases.   As Dr. Francis Collins says, understanding rare diseases will lead to solving the most common diseases.
We now need many millions of dollars poured into rare disease research and into centers like the National Chemical Genomics Center who utilizes high through put screening techniques and robotic technology and can accelerate finding therapeutic targets for people suffering from rare diseases.
To learn more about TRND, go to the ORDR web site at https://rarediseases.info.nih.gov/TRND.