Orphan medicine legislation spurs on research, innovation and growth, claims report
EU legislation aimed at providing incentives for research, development and marketing of medicines to fight rare (orphan) diseases has far exceeded initial expectations, a newly published report by the European Commission has claimed. Since the introduction of an EU regulation on orphan medicinal products in 2000, more than 450 applications have been submitted for 'orphan drug' designation. Of these, more than 260 applications have been approved and are undergoing clinical tests, while 22 new orphan medicines have gone on to receive a marketing authorisation. And while the report surmises that the true impact of the incentives will only be revealed progressively as longer term experience is accumulated, it estimates that one million patients suffering from rare diseases may have already benefited from these new medicines. Orphan medicinal products are intended for the diagnosis, prevention or treatment of life-threatening or very serious conditions that are rare. While only a small number of patients suffer from one of the 5,000 to 7,000 orphan conditions that exist today, it is predicted that rare diseases affect some 30 million people in the 25 EU Member States. The regulation introduced the possibility of placing on the market an orphan medicinal product through an EU procedure that grants a single marketing authorisation, valid throughout the EU. This so-called centralised procedure - a Commission decision based on the scientific evaluation of the European Medicines Agency (EMEA) - offers the advantage of the widest possible EU market with one single authorisation. Since November 2005, the centralised procedure has become compulsory for orphan medicinal products. Among the incentives offered through this procedure are a 10-year market exclusivity, reduction of application fees, and advice from EMEA. Support is also given to research into rare disease and orphan medicinal products through the EU's multi-annual framework programme for research. Under the Fifth Framework Programme (FP5), a total of 47 projects were granted EUR 64 million in the thematic section 'Quality of life and management of living resources'. Research was conducted in fields ranging form genetics, neurology, and metabolic diseases to oncology, mycology and infectious diseases. The Sixth Framework Programme (FP6) saw the introduction of new types of projects designed to provide new knowledge through multidisciplinary approaches. In addition to providing much needed medicinal solutions, such incentives, the report notes, have also helped to stimulate industrial activity. Since the implementation of the regulation, a significant number of start-ups have been created, while many existing companies have begun research on rare disease. Jobs related to orphan medicinal products have also increased by 43 per cent, a pace which the report claims, is faster than general industry trends. Spending on research and development (R&D) on rare disease has also grown faster than general medicinal R&D investment, more than two-fold, for the period 2000-2005. Furthermore, the regulation has also spurred on innovation, according to a recent review by EMEA of the orphan applications received between 2000 and 2004. The review shows that a total of 53 per cent of applications have been for novel/innovative products.