Patients frequently use experimental agents outside a standard treatment or clinical trial, but outcome data is not captured. This presents missed opportunities to capture real world data in the context of a rare disease that could significantly accelerate development of new therapies and combinations and/or support better matching of therapies and prevent complications for other patients. I know there are concerns about the accuracy of patient reported data, but data could be annotated as patient reported so that it can be properly integrated with the rest of a patient's data.
Idea No. 950