The Friedreich’s Ataxia Research Alliance (FARA) and the Critical Path Institute‘s (C-Path) Data Collaboration Center (DCC) will work together to create a database of clinical data for Friedreich’s ataxia (FA).
Access to the large-scale database is expected to expedite research on the disease and clinical trials of therapies for FA.
The database will be used to promote partnerships among researchers to further the understanding of the natural course of the disease, as well as potential biomarkers — substances produced in the body that can be used as indicators of disease states and responses to treatment.
Also, it will help identify possible clinical endpoints for clinical trials of drug candidates in patients with FA. This will facilitate the design of clinical trial protocols that are more efficient and allow new therapies to be tested faster and more effectively.
“FA is a rare disease which is progressive, affects multiple organ systems, and is fatal. Treating the disease is an urgent unmet need,” Jennifer Farmer, executive director of FARA, said in a press release. “FA research has reached a critical juncture, where several therapies have undergone or are in clinical trials, and additional new therapies are expected to start clinical development in the next few years.
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“The purpose of this project is to leverage and share as much information as possible, to more fully understand progression of disease, how that progression can be captured in measurable endpoints, and the effect of placebo treatment,” Farmer added. “We want to ensure that we are using all the information available to design the most efficient and robust clinical trials, giving potential therapies the best chance of success.”
The project will create a database of clinical information for Friedreich’s ataxia that integrates knowledge from different sources. The database can then be shared and utilized by FA researchers.
The DCC will approach companies that have carried out clinical trials in FA to obtain their clinical data. The DCC will also share natural history data collected by FARA’s research network.
The project will include three datasets, with the first dataset expected to be shared in the next few weeks. Additional datasets to be shared are presently being discussed. Patient identifiers will be removed from the datasets before they are aggregated into a single database according to rigorous scientific procedures by C-Path’s DCC.
“With a long history of expertise in data standards development, curation, and oversight of multiple data sharing initiatives, C-Path has the unique opportunity to help coordinate the collaborative contributions from data owners and integrate that data into a single database for this rare disease,” said Richard Liwski, C-Path’s chief technology officer and director of the DCC.
“Our approach has been successfully implemented in multiple C-Path consortia, and it is our hope that this proven experience will translate well toward the ongoing success of this important project,” Liwski said.
The DCC was founded in 2014 to provide solutions to managing large-scale data for scientific research. It will play a key role in this collaborative project, which will benefit from the DCC’s experience with clinical data standards, data aggregation, and sharing.
FARA raises funds for research and public awareness promotion, bringing together research scientists, patients, healthcare professionals, government agencies, pharmaceutical companies, and other organizations whose mission is to cure FA and related conditions.