The Office of Rare Diseases Research (ORDR), National Center for Advancing Translational Sciences (NCATS), NIH in collaboration with PatientCrossroads, Children Hospital of Philadelphia and WebMD, has launched a pilot program to establish a Global Rare Diseases Patient Registry and Data Repository (GRDR). The Fibrous Dysplasia is one of 15 organizations without a patient registry that has been chosen to participate.
The goal is to create a resource of aggregated de-identified patient information from rare disease patient registries to facilitate clinical trials, translational research, comparative effectiveness research, and analyses of data across many disorders and ultimately drug developments and therapeutics for the millions of rare disease patients.
The long term goals of the GRDR is develop a sustainable resource with governance structures and an organizational architecture guided by open-science principles; and provide a sustainable resource funded by private-public partnerships.
ORDR has selected 34 organizations to participate in the GRDR pilot program. 19 organizations have patient registries and 15 organizations do not have registries. Information provided by patient organizations, in response to an RFI, was evaluated according to selection criteria and scored by a review committee. Organizations were selected based their scores and in attempt to have representation across many rare diseases and organization size.
To find out more about the FD/MAS patient registry please visit: http://www.fibrousdysplasiaregistry.com/