PRA recently performed a feasibility study to identify investigators that see patients with a rare neuromuscular autoimmune disease and 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 participants, and it was initially estimated that at least 35 sites would be needed.
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 its local country experts, and Medical Informatics data searches, which showed where high numbers of physicians treat or study patients with the disease.