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.
- 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
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
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
Worldwide Syringomyelia & Chiari Task Force.