Companies that develop therapies for rare diseases and conditions face unique challenges that stem from the small patient populations available for enrollment in clinical trials and the inherently small markets for these products. Starting with the enactment of the Orphan Drug Act, the federal government has recognized that policies need to be developed to provide incentives for companies to develop products for patients suffering from rare diseases. To continue to meet unmet medical needs, US policies must continue to provide incentives for research and development. As policies are developed, policy makers must take into account the different circumstances confronting biotechnology companies developing products for patients with rare diseases. This panel will provide details about orphan product development and make recommendations about the policies that are necessary.

• Highlight the challenges confronting companies seeking to develop products for rare diseases and the success of certain policies in fostering the development of products
• Discuss pending issues confronting policy makers and how solutions must account for the particular circumstances surrounding development of products for rare diseases
• Describe company efforts to develop products for rare diseases