Data Collaboration Tool for Accelerating Rare Disease Research Celebrates Success in First Year

Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP)

The RDCA-DAP, funded by the FDA, was first launched in September of 2019. Its aim was to make data more accessible for researchers of the rare disease community, because without data, effective research can’t be done. As a part of this mission, the team hoped to encourage collaboration among researchers. Their goal is to eliminate bottlenecks in development of therapies by increasing researchers’ ability to access the information they need.

The platform specifically includes data on the characterization of diseases such as their natural history, symptoms, advantageous clinical trial designs and more. It was built by using the insight of the very individuals who would be benefitting from the platform. The concept was discussed by NORD, C-Path, and other stakeholders through webinars, public meetings, interviews, focus groups, and more. There were meetings with groups who had similar goals, to see how they could collaborate on both future ideas and previous progress to accelerate therapies.

This year, the event to discuss the platform’s progress was held virtually on October 19, 2020. The event included over 400 attendees ranging from industry partners, regulatory agencies, patients, and academics.

2020 Event

The 2020 virtual event discussed the accomplishments of this platform within the first inaugural year. Topics included-

  • Updates on the data infrastructure
  • New enhancements of the platform
    • Data search tools
    • Data sorting tools
    • Data analyzing tools
  • Sharing of prototypes

Researchers also shared examples of how the platform has already been beneficial. For example, patient data has helped a new biomarker for polycystic kidney disease receive accelerated approval. Additionally, prototypes have been built that can integrate data from many rare diseases in order to generate an advanced model of how diseases progress. This can ultimately lead to faster drug development for these conditions.

Benefits for Phenylketonuria

Phenylketonuria (PKU)  has a patient registry through NORD called IAMRARE. Researchers explain that by adding data like this on disease characterization and natural history to the RDCA-DAP platform, we can develop a better understanding of how this disease operates. Additionally, combining patient data helps us to determine what it is that matters most to patients. With this knowledge, we can design clinical trials that will be most beneficial for the rare community.

Get Involved

If you would like to help contribute to this data platform, would like to use the database, or want to assist with the development of this project, click here. Data is being accepted now and the user interface will be up and running by 2021.

Additionally, if you would like to listen to the full recording of the virtual workshop you can do so here.

You can read more about this platform here.

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