Effort will make Friedreich’s ataxia data more accessible for research

FARA teaming with C-Path to integrate datasets into RDCA-DAP initiative

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by Mary Chapman |

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The nonprofit Critical Path Institute (C-Path) and the Friedreich’s Ataxia Research Alliance (FARA) are partnering to bolster the library of data from people with Friedreich’s ataxia (FA) and make it more accessible for researchers to develop new treatments.

The effort will involve integrating additional FA datasets into C-Path’s Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP), an initiative funded by the U.S. Food and Drug Administration for characterizing rare diseases and speeding up the development of new therapies.

“FARA is excited to continue our collaboration with C-Path and foster data sharing from natural history studies and clinical trials to enhance research and analysis aimed at improving outcomes and developing treatments for Friedreich’s ataxia,” Jennifer Farmer, FARA’s chief executive officer, said in a press release from C-Path. “We believe in making patient contributions of data as meaningful as possible and one way to honor this is by ensuring that such data is available to the research community through a secure and robust platform.”

Among the new datasets are the natural history studies FA-CHILD (NCT03418740), which is complete, and an expanded version of the ongoing FA-COMS (NCT03090789). FA-CHILD, sponsored by the Children’s Hospital of Philadelphia, enrolled and monitored 108 pediatric FA patients over time to find ways to follow FA progression in this young patient population. The FARA-sponsored FA-COMS study, which is aiming to develop and validate clinical outcome measures in FA, now features more than two decades of data from 1,450 FA patients in the U.S., Canada, New Zealand, Australia, and India.

Integrating these findings into RDCA-DAP should help researchers gain a better understanding of FA’s natural course toward a better understanding about how experimental therapies may be helpful.

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Integrating FA datasets

The Friedreich’s Ataxia Integrated Clinical Database (FA-ICD), which was launched in 2019 by C-Path’s Data Collaboration Center and FARA to enable collaborative FA research and data sharing, will be discontinued to centralize and enhance FA data access in the RDCA-DAP, which provides analytical tools and advanced security.

The FA-ICD, which contains clinical trial and natural history study data, was managed by C-Path and integrating it into RDCA-DAP will let researchers access the FA-ICD dataset and new FA datasets on a single platform.

“The incorporation of FA-ICD into RDCA-DAP substantially expands our data resources and sharpens our analytical precision,” RDCA-DAP Executive Director Alexandre Bétourné, PhD, said. “With this enhanced scope and clarity, we’re better equipped to uncover insights that drive the development of effective therapies for Friedreich’s ataxia.”

By leveraging “the comprehensive data from FA-ICD, now fully integrated within RDCA-DAP, we are better equipped to accelerate the pace of discovery, fostering the development of novel therapeutic approaches and substantially improving patient outcomes,” Bétourné said.

Integrating the information into RDCA-DAP fortifies the platform’s capabilities and allows for a more effective use of data for research and development, according to C-Path. Previous contributions of FARA data increased the regulatory evidence that led to the approval of Skyclarys (omaveloxolone), the first disease-modifying treatment for FA.

Along with streamlining research efforts, the effort should enhance the global research community’s access to high-quality, actionable data, the aim being to reduce the time and costs associated with bringing new treatments to market.

“RDCA-DAP’s extensive resources can amplify the impact of the existing data and foster new discoveries in FA research,” Farmer said. “This collaboration will build upon our strong, sustained, and productive partnership with the C-Path team.”

More information about the collaboration is available by sending an email to [email protected].