Rare Disease Report

Be Part of the NORD Natural History Database Project

JANUARY 22, 2016
RDR Staff
Recently, we spoke with Jacqueline Kraska, Research Programs Manager at the National Organization for Rare Diseases (NORD) about the Natural History Study Database project, who can get involved, award deadlines (Jan 29th), and the important purpose behind the project. 

What is the NORD Natural History Study Database Project?

This is a new project to build and launch 20 disease-specific natural history studies for patient organizations interested in capturing data on the natural progression of their diseases over a period of time.
We are very excited about this.   The NORD Natural History Study Database Project is one component of the greater NORD Registry Project, which we built to address what is widely recognized as one of the greatest needs and inherent challenges of the rare disease community—having enough natural history data to help medical researchers better understand how diseases develop and progress over time.   
This project is made possible through a grant from the FDA and contributions by NORD.  Patient groups can apply to be one of the 20 groups selected.  By the end of the year, 20 more patient organizations will be driving research for their rare diseases with registries as part of NORD’s Natural History Study Database.

Why should an advocacy group want to be involved in the project?

Data from a patient registry can help to:
  • Unite the patient and research community;
  • Learn about a disease/condition;
  • Improve quality of care;
  • Develop treatments;
  • Develop research hypotheses; and
  • Improve the management of healthcare.
  • Funding to build and launch the registries is provided through the FDA grant. 

Why only 20 rare diseases?

Several patient groups are already using the platform and NORD will continue to develop additional studies in the future.  Resources through this particular program, however, are available for the development of 20 disease-specific studies.

How does an advocacy group apply? Are there any restrictions?

The application is online on the NORD website, where we have a dedicated page for the project.   You can access the information directly from our homepage, rarediseases.org.
The application and review scoring criteria for assessing applications are outlined on the webpage.  Patient organizations are encouraged to apply, even if they are not certain they meet all criteria.

When is the deadline and when will the awardees be announced?

The deadline to apply is January 29, 2016. Questions may be directed to Jacqueline Kraska, NORD Research Program Manager, at research@rarediseases.org or (203) 304 7205. Awardees will be announced on Rare Disease Day (February 29, 2016),

Who decides who the awardees will be?

A Program Selection Committee whose members are experienced in rare disease research-related fields will oversee the selection process. The selection criteria are posted on the NORD website.

What data will be collected?

Natural history data capturing longitudinal information about the disease will be collected. Natural history studies can yield vital information that is essential to clinical trial design such as biomarkers, demographics, genetic and environmental variables, disease progression, and patient perspectives on the challenges of living with the disease.

Who owns the data and how will the privacy of the patients be protected?

The identifiable and de-identifiable data are owned by the specific patient organizations. Each organization decides how and with whom to share the data. A subset of the de-identified data collected from registry participants across the platform is shared with NORD to support research on all rare diseases, including studies comparing diseases.  Studying one rare disease can often lead to findings about other diseases.

Is the data safe?

Yes.  The registry follows strict government guidelines to assure patient information is protected. The platform is also served over HTTPS, providing encryption of traffic to prevent eavesdropping and prevent man-in-the-middle attacks. Communication between the registry platform application server and the database are also encrypted.

Why can’t an advocacy group do their own natural history study. Why use the NORD-FDA program?

They can do their own natural history studies. However, NORD is providing a standard natural history study and the tool to collect the data, along with support and guidance to ensure the study is safe and effective.  Ours builds upon previous efforts to develop common data elements, questions, and ontologies for rare diseases to provide a generalized baseline for future efforts.  We developed it for designing, deploying, and maintaining these types of studies in a cost-effective and efficient way, which we know are important criteria for patient organizations. 


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