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NORD Launches Two New Patient Registries for Rare Diseases

The National Organization for Rare Disorders (NORD®) has initiated a call for applications to establish two new patient registries on its IAMRARE® platform. This important step is made possible through funding from the Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP®), a collaboration with the Critical Path Institute which receives support from the U.S. Food and Drug Administration (FDA).

Tracey Sikora, NORD's Vice President of Research and Clinical Programs, emphasized the significance of this initiative, stating, “This opportunity builds on NORD's work supporting rare disease nonprofits. Research studies led by rare disease communities are an important source of data, and insights from IAMRARE-hosted studies have contributed to the development of clinical outcome assessments, updates to treatment guidelines, and at least one FDA approval.”

Starting from April 2027, successful applicants will collaborate with NORD to create and launch a patient registry tailored to their rare disease community. NORD will oversee the development and hosting of the registry website, while also providing necessary training to the registry sponsors. This training will include establishing a registry advisory board, developing a comprehensive study protocol, customizing survey instruments, navigating Institutional Review Board (IRB) processes, and laying out data-sharing criteria with researchers.

Application Criteria for Interested Organizations

To qualify for this opportunity, applicants must fulfill the following requirements:
1. Be a 501(c)(3) organization representing a rare disease community.
2. Priority will be given to NORD Member Organizations.
3. Commitment to a reduced implementation fee of $5,000, payable upon contract signing.
4. Commitment and resources must be in place to commence registry work by April 2027, and a natural history study should be conducted for a minimum of five years.
5. At least two paid or volunteer staff members must contribute a combined total of at least 20 hours per week to the registry.
6. A U.S.-based Principal Investigator (PI) must be designated.
7. Sufficient financial resources should be allocated to cover annual maintenance costs, estimated at approximately $5,000 per year.
8. Commitment to submit data produced by the registry to RDCA-DAP.
9. Support from the disease community is crucial, including input from experts for project design and ensuring patient engagement and retention.

How to Apply

The application window is currently open and will remain so until January 10, 2027. Successful applicants will be notified by February 1, 2027, and the implementation of the registries is set to begin in April 2027. For further details and to apply, please visit the NORD IAMRARE website.

About the IAMRARE® Platform

NORD's IAMRARE platform is vital for advancing knowledge about rare diseases, currently hosting more than 55 active registries funded by patient advocacy organizations. These registries aim to collect critical patient-reported data, which in turn supports the broader goal of enhancing the understanding of rare diseases.

About RDCA-DAP

The Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP) is an innovative initiative by the FDA that establishes a standardized framework to facilitate the characterization of rare diseases. Its overarching aim is to accelerate the development of therapies that can change lives for those affected by these conditions.

About NORD

Established in 1983, the National Organization for Rare Disorders (NORD®) is a prominent nonprofit organization dedicated to improving the health and quality of life for over 30 million Americans impacted by rare diseases. With collaborations with more than 350 disease-specific organizations and 49 designated Rare Disease Centers of Excellence, NORD is committed to advancing research, education, and advocacy on behalf of these communities. To learn more, visit rarediseases.org.