The role of the patient is huge. I know of an example of a someone in the U.S. who went from having a child who was the first in the world diagnosed with a specific rare disease—literally the first—to creating a patient group of now around 50 patients worldwide, all within the span of five years, to actually becoming a professor of medicine within the span of the same five years. He was a professor of computer science—so my colleague—and he actually started to create therapies for his son's disease, all in five years, I believe.
Patients have great power to enact change. Yes, there is a balance between the two. Patients can't do everything. They need to work with pharma. They will need to work.... The key to me is to identify early as many of these individuals as we can, making sure that they don't fall through the cracks and that the diagnostic odyssey stops as early as possible so that patients can start working on the treatment side.