DATA COLLECTION AND SUBMISSION
The purpose of the Registry is to collect uniform and meaningful clinical data on patients with DYT-1 Dystonia. Since, at the time of enrollment, patients will be at various stages in their disease course and medical care, the Registry is focused on collecting basic information but eventually information such as previous treatments, drug therapies etc. associated with the management of their disease. Both retrospective and prospective data collection are encouraged and a standard set of case report forms (CRFs) will be utilized by participating individuals and physicians (refer to Attachment 1 for sample Registry CRFs). Data collection will focus on contact information, familial DYT-1 history, medications, previous treatments and others.
Secure Internet technology and other electronic advancements will be utilized to enhance data collection, education, and reporting capabilities. Where secure Internet technology is not available, or if it is not the choice of the patient to use them, data are collected on paper CRFs. The data collected are maintained by Tyler’s Hope Foundation and it’s partners in an electronic database. Paper originals will be scanned and maintained electronically as source documents.
Since a number of these patients are children, a primary Registry contact person will be designated and will be responsible for completing and submitting the standard DIPR-1 Registry CRFs (Attachment 1) and Link.
Please refer to the Data Collection and Submission section of the DIPR-1 Registry Manual for guidance on the data collection and submission process.
QUALITY ASSURANCE OF DATA
Data collected through this Registry will first be entered via Tyler’s Hope website or one of the partnership websites and stored in a database. Staff of the Registry program will review the data for missing datapoints, incomplete information, and discrepancies with previously submitted data. If necessary, issues will be resolved with the individual electronically, by telephone, or FAX. Quarterly this data will be reviewed by a Data Monitoring Committee (DMC) and once deemed usable will be marked in the Registry with the date of DMC review. Only then, will this data be available as Registry data.
All data management and analysis will occur in a validated computing environment. However, Registry data are not verified against source documents, in contrast with clinical trials, where source verification is performed in accordance with Good Clinical Practice (GCP) guidelines.
STATISTICAL METHODS AND DATA REPORTING
Data will be analyzed and reported periodically and upon individual requests from participating physicians. In addition, annual reports summarizing de-identified data from this registry will be provided to appropriate regulatory authorities and all registry stakeholders. Analyses will be performed using the SAS statistical software system. Data are summarized using descriptive statistics for patients and family members enrolled in the Registry.
Refer to the Data Reports section of the DIPR-1 Registry Manual for a more detailed explanation of possible data reports and the process for requesting a report.
CONFIDENTIALITY
Patient Confidentiality
To maintain patient confidentiality, patient contact information is stored in a separate database than the patient’s medical information. These are linked by the Registry identification (ID) number and Registry Family identification number. Additionally, all patients are referenced by Registry ID and Registry Family ID. If supplemental laboratory or imaging reports are submitted to the Registry, the patient’s name should be deleted and the patient’s ID number and Family ID number should be added to each attached page.
Physician Confidentiality
All physician information is confidential. Furthermore, physician-specific patient data will remain confidential and will not be released to other physicians without the patient’s prior written approval, in accordance with applicable laws related to the confidentiality of patient information. No physician-to-physician data comparisons will be made.
PATIENT DISCONTINUATION
Patient participation is voluntary. A patient may decline to participate or withdraw consent at any time and without affecting their medical treatment.
RISKS AND BENEFITS
Benefits
Patients and their family members will benefit by participating in the DYT-1 Registry in many ways. Their primary benefit will be – support group, newsletter with up to date research – what’s happening, road to a cure
Risks
Since participation in this registry does not require any assessments or procedures, the only risk to the participant is disclosure of personal health data. Every effort has been taken to protect the individual’s identity and health information in accordance with the Code of Federal Regulations Part 46 – Protection of Human Subjects.