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Research

Rare Disease Research and Therapeutics

a child with Proteus syndrome

NCATS supports research aimed at accelerating new treatments for rare and neglected diseases. There are more than 6,500 rare diseases that affect about 25 million Americans, yet there are fewer than 250 treatments currently available. In some cases, private industry and nonprofit groups are unable to focus on the development of therapeutics for rare diseases due to associated high costs and failure rates.

NCATS seeks to develop new collaborative models of rare disease research by facilitating interactions among government, industry, academia and patient constituency groups. Partners work together to advance the process of discovering new therapeutics through scientific and technological innovations that may improve success rates in the pre-clinical stage of the drug development pipeline.

New discoveries about the molecular basis of rare diseases, coupled with ongoing rare disease research, can offer unprecedented scientific opportunities as bottlenecks in the drug development pipeline are identified and overcome.

NCATS supports the following rare disease research programs.

Therapeutics for Rare and Neglected Diseases

The Therapeutics for Rare and Neglected Diseases (TRND) program, which is part of the NCATS Division of Pre-Clinical Innovation, is geared to encourage and speed the development of new drugs for rare and neglected diseases. This program specifically is intended to stimulate drug discovery and development research collaborations among NIH and academic scientists, nonprofit organizations, and pharmaceutical and biotechnology companies working on rare and neglected diseases.

Bridging Interventional Development Gaps 

The Bridging Interventional Development Gaps (BrIDGs) program, part of the NCATS Division of Pre-Clinical Innovation, provides successful applicants developing therapeutic agents with access to critical resources in support of pre-clinical studies. Investigators do not receive grant funds, but instead, gain access to services, which enable researchers to submit Investigational New Drug (IND) applications to the Food and Drug Administration.

Office of Rare Diseases Research

The Office of Rare Diseases Research (ORDR) in the NCATS Office of the Director supports and coordinates rare disease research, responds to research opportunities for rare diseases, and provides information on rare diseases. ORDR serves the needs of patients who have any one of the thousands of rare diseases known today. To accomplish this, ORDR coordinates and fosters relationships with a variety of stakeholders, from patient advocacy groups to academic institutions, as well as other NIH Institutes and Centers.

Rare Diseases Research Advocate Retires

Stephen C. Groft

Long-time rare diseases research champion Stephen C. Groft, Pharm.D., director of the NCATS Office of Rare Diseases Research, announced his retirement effective Feb. 8, 2014. Learn more about Groft’s legacy.

In a recent PhRMA Catalyst blog, Groft discusses collaborating to speed rare diseases research.