The European Medicines Agency has reported 12 new medicines being made available over the past year for the treatment of rare diseases. This shows an improving picture in Europe with an increase from only four approvals in 2011.
Medicines for rare diseases can qualify for ‘orphan drug’ designation and so benefit from a range of incentives including reduced fees and up to 10 years of market exclusivity. They include treatments for unusual metabolic conditions such as Gaucher disease, therapies for particular cancers and for genetic disorders like cystic fibrosis, each of which can be devastating for the affected individuals and their families. Together they impact 30 million Europeans. But each disease affects only a small proportion of the population, so the economics of bringing them to market present additional challenges.
Because of the advantages offered by orphan drug status, treatments for rare diseases are increasingly seen as an alternative model to the traditional 'blockbuster' approach, with companies such as Alexion making them their focus. Alexion's Soliris, for example, has recently received orphan drug status for the prevention of delayed graft function after transplantation in both the EU and the US.
As well as offering its own suite of support measures, the EMA collaborates with the US and Japanese regulators to promote joint applications and to provide technical support. Half of the orphan drug applications the EMA received last year made use of this procedure, saving time and money in bringing these new medicines to an international market.