P248: Innovations in Therapy Development for Rare Diseases through the Rare Disease Cures Accelerator –Data and Analytics Platform
Poster Presenter
Heidi Grabenstatter
Scientific Director, RDCA-DAP
Critical Path Institute United States
Objectives
Progress toward the development of therapies for rare diseases is hampered by a limited biological understanding of most diseases and their progression and challenges in evaluating patients and designing adequate clinical trials.
Method
Data that could be used to characterize many diseases is often collected in multiples formats, however, each data source contains a limited number of subjects and different data elements, and is frequently kept proprietary in the hands of the study sponsor.
Results
The Rare Disease Cures Accelerator – Data and Analytics Platform (RDCA-DAP®) is an FDA-funded effort to overcome these persistent challenges. By aggregating data across all rare diseases and making that data available to the community to support understanding of rare diseases and inform drug development, RDCA-DAP aims to accelerate the regulatory approval of new therapies. RDCA-DAP curates, standardizes, and tags data across datasets to make it findable within the database, and contains a built-in analytics platform to help visualize, interpret, and use it to support drug development. RDCA-DAP also coordinates data and tool resources to serve a diverse array of rare disease stakeholders that includes academic researchers, drug developers, regulatory reviewers and patients and their caregivers.
The platform currently holds over 70 international datasets for 35 different rare diseases, with an ever-expanding and diversifying database. The platform users range from industry and academia to foundations who are actively leveraging the database to inform their research and drug development programs. In addition, the RDCA-DAP team is developing data-driven solutions for several rare diseases under the premises of the RDCA-DAP Task Forces. Each Task Force convenes patient advocacy groups, foundations, key opinion leaders and industry in a pre-competitive environment to drive single, regulatory-endorsed deliverables aimed at disentangling bottlenecks in drug development and solving unmet needs in rare diseases.
Conclusion
Our active Task Forces and collaborative groups include efforts for Friedreich’s Ataxia, Progressive Supranuclear Palsy, rare mitochondrial and inherited metabolic diseases and Limb-girdle muscular dystrophies. RDCA-DAP aims to develop a Task Force for each organ or bodily function impacted by rare diseases. Finally, we encourage rare diseases stakeholders to utilize the RDCA-DAP platform to develop solutions on their own and/or through collaboration with our program. Platform access: https://portal.rdca.c-path.org/. Connect with our team: RDCA-DAPadmin@c-path.org
