EU orphan drug approval is "too slow"

A study published in the British Journal of Clinical Pharmacology reveals that the development of orphan drugs in the European Union is progressing at an alarmingly slow pace. Of 255 drugs classified as having orphan drug status over a four year period, only 18 have gone on to reach final approval. The discrepancy between the propor-tion of approvals issued by the European Medicines Agency (EMEA) for orphan drug applications (7%) and other medicine filings (79%) is criticized by co-author Silvio Garattini, from the Mario Negri Institute for Pharma-cological Research in Milan, Italy. Prof Garattini said: "the lack of reliable methods for evaluating orphan drugs in a small number of people probably explains the poor quality of the applications." He added: "however, it is clear that less stringent criteria are acceptable for orphan drugs than for drugs for more common diseases, partic-ularly in view of the small number of patients."

The report's authors conclude that lack of government funding is to blame for the situation. Their solution is to call for the establishment of a special fund for the devel-opment of orphan drugs and for appropriate changes to the tax treatment of sponsors willing to produce orphan medicinal products.

Jeffrey Aronson, chairman of the BJCP's editorial board contrasted the performance of the USA with the EU's, saying: "there are more incentives - 1,100 drugs and biological products were designated orphan products [in the USA] between 1983 and 2002 and 231 were ap-proved." Dr Aronson added that the UK's National Institute for Health and Clinical Excellence (NICE ) does not generally approve drugs that cost more than L30,000 ($52,500) per Quality Adjusted Life Year, noting that, most orphan drugs cost more.