Orphan drugs are medicinal products intended to treat diseases so rare that sponsors are reluctant to develop them under usual marketing conditions. Orphan drugs are a growing market, due in large part to tax incentives, longer periods of market exclusivity, and shorter, smaller clinical trials, as well as the premium pricing associated with many orphan drugs. Despite increased emphasis on orphan drug development, only five percent of rare diseases currently have available treatment options. As sponsors continue to invest in rare diseases, it is important for them to understand the strategic and practical considerations of the planning and development process in order to manage the delicate balance between budget and timelines and maximize the opportunity for successful entry into the market.
