A goal of the foundation is to better represent our small but growing community. Our newest project requires the support of each family that is impacted in creating a patient registry for the YWHAG Community.
A registry is a database that collects and stores information about a group of individuals, most of whom are living with the same disease. Registries are a powerful tool for focusing research on a specific disease. One challenge of advancing the scientific understanding of rare diseases is finding enough people who eligible to participate in research studies. Establishing a registry can help tackle that challenge.
Specific Objectives of Registries:
To connect affected patients, families, and clinicians.
To learn the natural history, evolution, risk, and outcomes of specific diseases.
To support research on genetic, molecular, and physiological basis of rare diseases.
To establish a patient base for evaluating drugs, medical devices, and orphan products.
References: https://www.ncbi.nlm.nih.gov/books/NBK208609/
Registries can be different things to different people and organizations. Families may see a registry as an educational tool to better understand symptoms making their child sick. Research institutes and universities may see registry data as a way to validate animal studies, or to establish once overlooked disease endpoints for further clinical research. Pharmaceutical or biotechnology companies might want to establish disease progression or burden, or supplement data of the necessarily small clinical trials. Regulators such as the FDA are now accepting registries as part of the marketing approval requirements.
Reference: https://globalgenes.org/wp-content/uploads/2019/01/GG_toolkit_rare-registries-web-hyperlinked.pdf
In the coming weeks we will be reaching out for your support in building a registry for our community to better serve our kids now and all future families.
Start the process now by checking out the latest section added to our site: https://www.ywhagfoundation.org/patient-registry