What are Key Considerations When Launching a Rare Disease Drug in a Specialty-Naive Market?

Gilbert: Key considerations for launching orphan therapy in a specialty-naïve market involves understanding the market, understanding the manufacturer demographic. Is this a new startup biotech firm? Is this a large manufacturer who’s been in this space for a while? Is the product the first product on the market available to these treatments? Or is this a market where other treatments have been available and so the marketplace is well known to specialty pharmacy?

Additionally, understanding where the physicians are, and their level of experience with specialty pharmacy and what their potential barriers might be. Do they understand that there may be capacity issues in the beginning where there’s a high demand of patients who have been waiting for treatments for a long time, and getting those patients on therapy? When you consider commercializing new orphan products, you have to look at all of these different dynamics in order to create a program, support systems, tools, and design to best support both the physician and the patient. That will lead to ultimately those patients having access to treatment.