Data sharing is a common request in the rare disease community. Unfortunately, the realities of our current research infrastructure make it problematic for researchers to share their data. The net result is a plethora of data silos that can dramatically delay our understanding of diseases, especially ultra-rare diseases, that can lead to treatments.
This problem of data silos was expressed by several key opinion leaders in a recent editorial Data Silos are Undermining Drug Development and Failing Rare Disease Patients published in the Orphanet Journal of Rare Diseases.
In this panel discussion, Peter Ciszewski, President of CheckRare talks with three authors of that editorial, Paul Howard, PhD, Director Of Public Policy at Amicus Therapeutics, Eric Marsh, MD, PhD, Associate Professor of Neurology at the Children’s Hospital of Philadelphia, and Andrew Mulberg, MD, FAAP, Senior Vice President at Neurogene, Inc.
As discussed by the panel, the problem of data silos is a systemic problem and requires all institutions, including the U.S. Food and Drug Administration (FDA), the National Institutes of Health (NIH), journal publishers, research institutes, and the pharmaceutical industry, to provide incentives to share data more willingly to help accelerate rare disease research.
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