We need your help in taking a critical step toward learning more about our children and adults hopefully identifying effective treatments to help them experience life to the fullest. The online Sturge-Weber International Patient Registry has recently been launched to any individuals diagnosed with Sturge-Weber syndrome (SWS) or who have a port wine birthmark in the forehead and/or eye region.
We are optimistic about the future benefits of creating this important on-line library of information about SWS which has grown out of 25 years of mutual information sharing by patients and their families.
Brief explanations regarding registry composition and importance shared by Ronald J. Bartek, Co-Founder/Founding President, Friedreich's Alaxia Research Alliance (FARA)
Registries and natural history databases are proving to be invaluable tools for patient communities from the beginning to the end of the R&D continuum. From the basic/discovery research stage through clinical trials, approvals and the post-market environment, they are helping engage patients early, often and effectively; providing scientists with better understanding and characterization of diseases; attracting industry partners to well organized disease groups, and helping educate funding agencies and regulators.
Registries are databases of information on individuals affected by a certain disease or condition. The type of data included in registries will vary depending on the purpose of the registry. Contact registries, which are useful for organizing the disease community and clinical cohorts, include names, contact information, and perhaps some demographics. Registries that are intended to be used for research, often called natural history databases, will also contain substantial clinical information, such as age of onset, symptoms, medical images and examination/test results. The type of information included in a registry will vary for each disease.
Contact Registries primarily contain demographic information, most often entered by the patient/patient family, helpful in identifying who the patients are, where they are located and how they can be contacted. The organization holding the registry can use it to keep the registrants informed of research in which they might want to participate along with contact information for the sites where that research is to be conducted. Because contact registries can be invaluable tools for recruiting subjects for clinical research, they often include enough information on registrants (e.g., DOB, specific diagnosis, ambulation capability, travel restraints, associated conditions such as heart disease, diabetes, etc.). The organization can review such information to predict which registrants are most likely to meet the inclusion/exclusion criteria for each research project and provide these registrants with the contact information for the research site nearest them.
Natural History Registries/Databases contain more extensive clinical information, entered by patient/patient family and/or clinicians, that can be exceedingly helpful throughout the entire therapy development spectrum. Natural history data is essential, for example, in fully understanding and characterizing a disease, determining which symptoms will be most important to patients over time, identifying clinical endpoints effective in measuring changes in those symptoms in clinical trials, searching for biomarkers, selecting subjects best suited for each research project, determining optimal clinical procedures that will be well tolerated by the subjects and will instruct the clinical endpoints, etc.