Kaufmann Appointed Head of NCATS’ Office of Rare Diseases Research

Will Retain Role as NCATS’ Division of Clinical Innovation Director
 

On Sept. 3, 2015, NCATS Director Christopher P. Austin, M.D., named Petra Kaufmann, M.D., M.Sc., as director of the Center’s Office of Rare Diseases Research (ORDR). Kaufmann’s new leadership role will include overseeing NCATS’ Rare Diseases Clinical Research Network (RDCRN), the Genetic and Rare Diseases Information Center, and the NIH/NCATS Global Rare Diseases Patient Registry Data Repository/GRDR® program. She will retain her current position, held since May 2014, as director of NCATS’ Division of Clinical Innovation (DCI), which includes the Clinical and Translational Science Awards (CTSA) Program.

“Research on rare diseases has advanced into the mainstream as an area of disproportionate medical need, a basis of insights into common diseases, an avenue to identify commonalities among diseases and a testing ground for new translational approaches — all of which are accelerating the translational science process,” Austin said. “Because of shared science and shared goals among NCATS’ ORDR and DCI programs, placing them both under one person’s leadership will enhance synergy, increase our Center’s ability to advance translational science and help bring more treatments to more patients more quickly.”

Kaufmann has had a longstanding interest in and commitment to rare diseases research. She is deeply involved in the rare diseases community and has a distinguished track record leading rare disease programs and complex collaborations in academia and government. Her training and experience span neurology, biostatistics, clinical research and clinical trials.

“I am delighted with this new opportunity to bring the rare diseases community and the broader clinical and translational community together,” Kaufmann said. “I am optimistic that we can make great progress in advancing research to respond to the needs of the millions of people living with rare diseases through collaboration among our grantees, staff, patient advocacy organizations, industry, NIH colleagues and other federal agencies.” 

Prior to joining NCATS, Kaufmann was at NIH’s National Institute of Neurological Disorders and Stroke (NINDS), where she directed the Office of Clinical Research and initiated NeuroNEXT, a network for neurological trials. Before coming to NIH in 2009, Kaufmann was a tenured associate professor of neurology at Columbia University in New York City, where she worked in the neuromuscular division, electromyography laboratories and pediatric neuromuscular clinic. Kaufmann also has worked on natural history studies, biomarker development and clinical trials in amyotrophic lateral sclerosis, muscular dystrophies, spinal muscular atrophy and mitochondrial disorders.

ORDR was established in 1993 within the NIH Office of the Director under the leadership of Stephen C. Groft, Pharm.D. In 2011, ORDR became part of the newly established NCATS. ORDR guides and coordinates NIH-wide activities involving research for a broad array of rare diseases, responds to research opportunities for rare diseases, and provides information on rare diseases. Groft served as director of ORDR until his retirement in February 2014.

 

Posted September 2015