Pharmacoeconomic analyses of treatments for rare disease

Although more than 7000 diseases have been classified as rare in nature, estimates place fewer than five percent of these diseases as being a current focus of substantial drug development. Several challenges exist concerning the basic understanding of these conditions overall, including a lack of understanding surrounding natural history, epidemiology, and effective treatment options in the real-world. The purpose of this paper is to discuss issues concerning unmet needs of rare disease and adoption or reimbursement decisions, including ethical and policy considerations associated with the treatment of rare diseases. Elements that are often inadequately addressed by a standard economic calculus to evaluate rare disease such as distributive justice are also addressed. Overall, the opportunity costs of supporting orphan drug use to treat rare disease must be carefully balanced by the necessities of treating patients with more common diseases wherein cost-effective treatments may more readily exist.