The sponsor is a global biotechnology company developing promising new medicines for patients with serious rare diseases. The company has a portfolio of approved medicines as well as a pipeline of transformative medicines currently being studied in clinical trials.
The company currently has a portfolio of US and European commercially approved, ground-breaking medications that are used to treat life-threatening hereditary conditions. While the company manages the commercial sales and strategy in these major markets, it wanted help making these products available on an unlicensed basis to patients in additional territories. The sponsor wanted to make the products available in as many countries as possible, so that no patient is left without treatment due to geography. To complicate the matter, these drugs are high priced treatments, so careful considerations had to be used when designing the program.