Various stakeholders, mostly European, ponder what techniques and standards should be used to assess the efficacy and cost efficiency of medical technology for managing rare diseases. Among the parties are patients, physicians, assessment agencies, and the plasma product industry. The topics include key concepts in health economics for hæmophilia organizations, how to assess the cost-effectiveness of assessment, comparative effectiveness research in the US and primary immunodeficiency diseases, patient engagement, and orphan drugs in oncology. There is no subject index. Annotation ©2012 Book News, Inc., Portland, OR (booknews.com)