The purpose of the new, Congressional mandated, NIH Therapeutics for Rare and Neglected Diseases (TRND) Program is to build on the chemical genomics efforts of the NIH Roadmap to develop new drugs for patients with neglected and rare diseases. The opportunity is commensurate with the need, since the Human Genome Project has revealed the genetic underpinnings of thousands of neglected and rare diseases. TRND will bridge the wide gap in time and resources that often exists between basic research and human testing of new drugs. The effort is grounded in, but aims to improve upon, existing processes for drug development in the pharmaceutical industry. TRND will focus not only on developing drugs for such neglected and rare diseases, but also on scientific paradigm and technology development to improve success rates in the preclinical stage of drug development generally. Specifically, TRND's neglected and rare disease-specific goal will be to generate clinical candidate compounds and data packages on them that are sufficient for Investigational New Drug (IND) Applications to the FDA. TRND is a collaborative drug discovery and development program with governance and oversight provided by the Office of Rare Diseases Research (ORDR). Program operations will be within the intramural research program adjacent to the NIH Chemical Genomics Center (NCGC) and will be administered by the National Human Genome Research Institute (NHGRI).
