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NORD-FDA Natural History Study Project

NORD is pleased to announce a cooperative project with the FDA to fill the unmet need of a generalized, standard approach to natural history study development across rare diseases. Working with disease patient groups and the FDA, NORD has developed natural history studies for nineteen rare diseases.

Together, we are developing a registry toolkit containing best-practice tools and templates that will aid future organizations to initiate and conduct natural history studies that are designed to support drug development programs. Additionally, we will build upon previous efforts to develop common data elements, questions and ontologies for rare diseases to provide a generalized baseline for future efforts. Each study will be sponsored by the disease patient group, will include the common data elements created as part of this project, and will include disease-specific research.

We invite proposals from disease patient groups to join this project. From the applicants, we will conduct a selection process using the review criteria below to find the twenty proposals that best fulfill the goals of the project.

For more information, read the official announcement.

Application criteria

  • 501(c)(3) Organizational Member of NORD – more info here
  • Commitment and resources to administer a registry and collaborate with NORD for at least five years to properly evaluate outcomes
  • Support from disease community and experts who will be able to contribute to the design and success of the project, including selecting disease-specific questions and ensuring patient engagement and retention


Review scoring criteria

  • Unmet medical needs
  • No existing or limited natural history data
  • Research gaps that could potentially be addressed by a natural history study



NORD Natural History Study Project Application

Contact information

[email protected]
203-744-0100, ext. 222

Contact Information


For Questions:

Jacqueline Kraska
[email protected]


20 Rare Disease Patient Groups Selected for Natural History Studies Project

Twenty rare disease patient groups have been chosen to develop natural history studies with the assistance of the National Organization for Rare Disorders (NORD) supported in part by a cooperative agreement with the U.S. Food and Drug Administration (FDA). Read the full announcement here.

Hereditary Neuropathy Foundation

Organic Acidemia Association

XLH Network, Inc.

CCHS Family Network

Pitt Hopkins Research Foundation

The OMSLife Foundation

Platelet Disorder Support Association

Global Foundation for Peroxisomal Disorders

APS Type 1 Foundation

Scleroderma Research Foundation

Galactosemia Foundation

Desmoid Tumor Research Foundation

International Pemphigus & Pemphigoid

The Morgan Leary Vaughan Fund

Adult Polyglucosan Body Disease (APBD) Research

Bridge the Gap-SYNGAP Education and Research Foundation

United Leukodystrophy Foundation

Lipoprotein(a) Foundation

Worldwide Syringomyelia & Chiari Task Force.