A patient advocacy group supporting patients with a rare metabolic disorder in the western Pacific wanted to help provide access to effective treatments. There are currently no approved medicines available for this condition and, although there are promising treatments in development, there were no clinical trial sites set up in this region of the world, at the time. The advocacy group had heard of patients in the US experiencing marked improvement in their symptoms using a novel drug in phase 3 clinical trial. The group had also heard good things about several phase 2 investigational products being studied in the US and EU. With patient interests in mind, the advocacy group reached out to WEP Clinical to request support.


WEP Clinical first investigated all the companies, worldwide, developing some form of treatment for this disease. Of the companies we found, the majority were familiar to us, and we had had prior communication with many of them. Using our contacts at these companies, we reached out, on behalf of the advocacy group, to see if anything could be done to help these desperate patients. Some were not in a position to help, while others were more open to discussing potential solutions. WEP Clinical was able to discuss potential expanded access use of products with many of the companies. In the end, one of the companies agreed to provide its drug to several patients through an International Charitable Access Program (ICAP).