BACKGROUND: During the past two decades, orphan drug policies have had a substantial impact on developing new products for rare diseases. In the US nearly 1100 drugs and biological products have been designated as orphan products, and over 240 of these products have been approved for marketing. In Europe an incentive regulatory incentive system started about 15 years later and there have been nearly 160 orphan designations and a total number of approved products are 12 other countries in the world make significant progress as well. Rare diseases are conditions that occur in fewer than 200 000 patients (approximately 7.5 in 10 000 people) in the US or. than 5 in 10 000 people in Europe. The system is developing itself further in the direction of the implementation of new innovative areas, e.g., classification of medically plausible subsets based on genomics and/or proteomics, orphan drugs for patients refractory to existing standard therapy (e.g., non-response to MTX due to adverse effects).
OBJECTIVE: We aim to focus on the various epidemiological aspects of orphan diseases and therapies. But, also at orphan drugs as a broader model for drug development. History shows that a major part of universal medical knowledge we have gained over centuries, started with ‘rare disease research’.
METHODS: The proposed symposium will include speakers from regulatory bodies (EMEA/FDA), industry, patient groups and the medical community. In all phases of orphan drug development epidemiology adds: to answer the pivotal question whether the disease is rare, how variable the natural course of the disease is, to assess whether drug therapy is efficacious and safe, etc. The field of orphan drug development is dynamic and raises constantly new questions in need for sound epidemiology. For instance, MS was a rare disease in the past but today not anymore.Nowthe question is pertinent: is there a distinctable subset ofMSpatients qualified to have a ‘rare’ disease? What are the clinically relevant outcomes in these patients to evaluate therapies appropriately? And what about risk management in the context of a rare disease, a rare therapy and an even rarer unwanted effect.
