RARE DISEASES, EXTRAORDINARY ASPIRATIONS. HIGHLIGHTS FROM THE SOCIETY FOR MEDICINES RESEARCH SYMPOSIUM, HELD OCTOBER 13, 2016-COVENT GARDEN, LONDON, UK

More than 7,000 rare diseases are known that collectively affect some 6-7% of the developed world.s population. Individually, any single rare disease may only affect a handful of people making them historically a relatively unattractive prospect for the biopharmaceutical industry to target. However, ground-breaking legislation starting with the Orphan Drug Act that was passed in the U.S. in 1983 now provides financial incentives for companies to develop orphan drugs, designated to treat rare or "orphan" diseases. Today, rare diseases and the drugs that treat them are sufficiently attractive that many companies and research institutes now have units dedicated to this area of research. A panel of international experts from academia, industry and disease advocacy organizations shared their views on how rare diseases can be effectively tackled, as exemplified using case studies and opinion pieces covering basic science, drug discovery and development, and the impact of regulatory factors. This Society for Medicines Research Symposium was held at The Grand Connaught Rooms, Covent Garden, London, with sponsorship provided by Charles River, The European Federation for Medicinal Chemistry, GlaxoSmithKline, Pharmidex and Vertex.