Pharmaceutical pricing, cost containment and new treatments for rare diseases in children.

Cost-containment in healthcare spending has become a central issue in public policy and healthcare reform, especially as the affordable care act adds millions of people to public and private insurance rolls. In this climate, longstanding criticism of pharmaceutical pricing has grown sharper, and many in both policy and medicine have characterized the costs of newly developed drugs as both exorbitant and wasteful of scarce healthcare resources. At the same time, pharmaceutical research and development pipeline costs are increasing exponentially.