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Push to Accelerate Drug Discovery for Rare Diseases Is Led by GRANDRx Initiative

New Rochelle, April 6, 2010—Leaders in the fields of health advocacy, industry, academia, and government gathered to explore the challenges preventing more rapid discovery and development of novel therapies for rare and neglected diseases and brainstormed on strategies such as the GRANDRx initiative to build collaborative gateways for applying technology to identify new drug treatments. The resulting roundtable discussion is published in ASSAY and Drug Development Technologies, a peer-reviewed journal published by Mary Ann Liebert, Inc. The paper is available free online

Sharon Terry, president and CEO of the Genetic Alliance, moderated the panel discussion entitled “Assay, Preclinical, and Clinical Brick Walls and Opportunities for System Change Through GRANDRx,” which brought together four distinguished scientists and advocates including: Christopher Austin, MD, Director of the National Institutes of Health Chemical Genomics Center (NCGC) and Senior Advisor to the Director for Translational Research at the National Human Genome Research Institute (NHGRI), James Inglese, PhD, Deputy Director of the NCGC, David Meeker, MD, Executive Vice President at Genzyme (Cambridge, MA), and Patrick Terry, Acting-CEO of the Grand Therapeutics Foundation (Washington, D.C.) and a business entrepreneur in the biotechnology and disease advocacy community.

The discussion focused on three main areas—assay development and high-throughput screening, preclinical testing, and clinical trials--in which there are so-called “brick walls” obstructing progress toward new drug development for rare diseases, which collectively affect millions of people worldwide. The participants outlined the relevant issues and presented their viewpoints on how the current system for drug discovery research, preclinical and clinical testing of experimental compounds, and regulatory approval requirements could be modified to accelerate the process of bringing new drugs to market.

One such collaborative endeavor intended to build gateways through these “brick walls” is the Gateway to Rare and Neglected Disorders Therapeutics (GRANDRx) initiative (www.grandrx.org). The mission of GRANDRx is to create a road map for the development of robust systems for translating basic scientific discoveries into safe and effective therapeutics.

“GRANDRx’s success depends on vastly improving the current system, and this paper will deliver this message broadly. It is the responsibility of all stakeholders to step up and develop this new system collaboratively,” says Sharon Terry.