title: SMi'S 4th Annual Orphan Drugs and Rare Diseases Conference (October 19-20, 2015--London, UK).
abstract: The story of rare diseases is a story of people. Often very young, and usually with little hope of treatment, it is often a desperately sad and tragic story, but increasingly a story of hope. Statistics are inescapable when discussing the subject: small numbers of patients in huge populations, the massively disproportionate contribution to pediatric mortality, and the necessarily high prices for rare drugs. Almost every speaker at the SMi's Fourth Orphan Drugs and Rare Diseases conference provided figures that illustrated just how different this area is to the macro-world of the blockbuster drug.
authors.affiliation: Thomson Reuters, London, UK. scientific.prousjournals@thomsonreuters.com.
authors.forename: P
authors.initials: P
authors.lastname: Robins
mesh_terms: D004345:Drug Industry; D006801:Humans; D008131:London; D009965:Orphan Drug Production; D035583:Rare Diseases / Q000188:drug therapy*
keywords: Alkaptonuria; Orphan drugs; Patient groups; Rare diseases; Vimizim