PRA recently performed a feasibility study to identify investigators who see patients with a rare neuromuscular autoimmune disease and who have the capability to perform a Phase II clinical trial. It was estimated that there may only be 1,000 to 3,000 patients in the US and Europe with the condition.
The major challenge that PRA faced was identifying the investigators and sites that see these patients. The protocol required 42 patients, and it was initially estimated that at least 35 sites would be needed to enroll these patients. The feasibility team selected 18 countries in North America, Latin America, Western Europe, Central and Eastern Europe, and Asia. Country selection was based on data from PRA’s investigator database, input from our local country experts, and Medical Informatics data searches that showed where the greatest numbers of physicians treat or study patients with the disease.