Unlocking Rare Disease Research: How Advanced Data Sharing is Transforming Clinical Research

The development of treatments for rare diseases faces a significant challenge – a lack of comprehensive data. Of the thousands of rare diseases known, only a few hundred have any viable treatments. One of the major roadblocks is the scarcity of natural history study data, which tracks the progression of diseases over time. To overcome this, the Rare Diseases Clinical Research Network (RDCRN) was formed, supported by 23 research organizations. However, each organization had its own isolated data storage, making collaboration difficult. Quantum Insights partnered with the RDCRN to build a cloud-based solution hosted on AWS, migrating legacy data, and enabling seamless collaboration between organizations. This new platform addressed the challenges of data standardization, collaboration, and identity management. By creating standardized Case Report Form (CRF) templates, Quantum Insights ensured that data from all 23 organizations followed the same structure. Data cleansing tools were also implemented to ensure consistency and accuracy.

A Collaborative Data Ecosystem

The cloud solution allowed data to be securely stored in a HIPAA and IRB-compliant environment, with built-in scalability for various data types such as images, videos, and genomics. For the first time, researchers could access all relevant data from a centralized location, streamlining the data access process, and enabling faster identification of key information. To foster collaborative research, the platform was designed as a Virtual Research Organization (VRO). Researchers had access to reusable tools, statistical analysis resources, and shared datasets, encouraging cooperation across the network. The platform was later enhanced with Palantir Foundry, providing even more advanced data analytics and storage tools, allowing scientists to focus more on research and less on technical challenges.

Driving Efficiency and Transparency

By transitioning to this collaborative cloud system, the RDCRN and its partners gained more flexibility in how they analyze and share data, driving scientific research forward. Researchers could now work together to solve complex challenges in rare disease treatment, generating new insights more efficiently. Additionally, the U.S. Government could now engage with the RDCRN in a more transparent, data-driven manner, facilitating smoother collaboration with the consortia. This innovative approach to data sharing and collaboration is revolutionizing rare disease research, allowing for faster progress in developing life-saving therapies.