INTERNAL NOTES. Explaining the study flow, IRB, informed consent, and the role of advisory boards may ease participants’ worries. This article should address concerns about survey difficulty. The goal is to convert discomfort about the registry into excitement about it. The call-to-action block at the bottom will be determined by proximity of article publication date and launch.
That’s right. The patient registry is a research study. It aims to aid in finding answers to important questions about the natural history of MdDS. Using the IAMRARE® platform powered by the National Organization for Rare Disorders (NORD®), the MdDS Patient Registry will gather information through validated surveys and custom questionnaires.
This research study not clinical research. A research study aims to gain knowledge about a phenomenon, while clinical research aims to develop new treatments, diagnostics, or preventative measures by testing them in humans.
But the data collected through the patient registry can help researchers explore potential treatments or launch clinical trials for MdDS. To protect the privacy of individuals, the data they will have access to will always be de-identified, with all personal information removed. Participants will have the opportunity to be notified about MdDS clinical trials as well as view their data in comparison to others within the registry.
The feature works such that when an individual has submitted a survey, they are able to see graphs of aggregate data for the questions they have answered. An example using a race question is shown below. The graph shows data from all participants who answered the same question about race. This allows an individual to see how their answer compares to the whole, within the registry.
Similarly, de-identified patient data, in aggregate, will be analyzed to inform healthcare providers of information to improve standards of care.
Learn more about the IAMRARE® program.
Enroll in the MdDS Patient Registry.