NIH Launches Therapeutics for Rare and Neglected Diseases (TRND) Program

MAY 10, 2010
by Terry Mac Dermaid, Assistant Director, PXE International

In 2009 the National Institutes of Health (NIH) dedicated $24 million to launch the first integrated, drug development pipeline to produce new treatments for rare and neglected diseases, a program called Therapeutics for Rare and Neglected Diseases (TRND). A rare, sometimes called orphan, disease is defined as one that affects fewer than 200,000 Americans. A neglected disease is one lacking substantial therapeutic development activity, ignored by drug developers, or by others instrumental in drug access, such as government officials, public health programs and the news media. Many genetic diseases are both rare and  neglected.

While more than 6000 rare and neglected diseases affect more than 25 million Americans, effective drug treatments exist for only about 200 of these conditions. TRND aims to encourage and speed the development of new drugs for rare and neglected diseases, and to bridge the wide gap in time and resources that often exists between basic research and human testing of new drugs. TRND will concentrate its efforts on the pre-clinical stage of drug development, striving to develop candidate drugs for rare and neglected diseases that will meet FDA requirements for an Investigational New Drug (IND) application, which is necessary for human testing. TRND expects to license most of its IND-worthy candidate drugs to biopharmaceutical companies for clinical developments.

A May 2009 statement issued by Genetic Alliance, an umbrella organization for more than 1,200 disease advocacy groups (PXE International is one of these), applauded the federal government for development of TRND, and “views meaningful collaborations with external partners as a key element in the program”. “Collaborations through open and transparent processes are essential for change that will benefit health outcomes for all stakeholders.” PXE International, along with all of the advocacy organizations, has great hope that once this program begins in earnest it will result in improved systems for all drug development, regardless of whether the diseases are part of the TRND program or not. Sharon Terry, executive director of PXE International and CEO of Genetic Alliance, said, “The most important aspect of this program is the systems change it will cause. New learning and negative results will be shared – this in itself is novel and will accelerate drug development.”

For more information on TRND, please visit the following links

NIH Announces New Program to Develop Therapeutics for Rare and Neglected Diseases, NIH News, May 20, 2009

TRND FAQs, National Human Genome Research Institute, March 5, 2010