Larger companies planning to market orphan drugs for rare diseases in the EU will now have even more financial incentive after regulators announced greater fee reductions.
The European medicines Agency (EMA) said that the changes will take place from the beginning of 2014 and will offer reduced regulatory fees for companies that are not micro, small or medium-sized enterprises (SMEs).
From next year, non-SME companies submitting a marketing application for an orphan drug will be eligible for a 75 per cent fee reduction for non-paediatric-related initial and follow-up protocol assistance. Previously this reduction was only 40 per cent for larger firms.
The EMA will also introduce a 10 per cent fee reduction for initial marketing-authorisation applications, where currently there is no reduction. There will also be a 100 per cent reduction for pre-authorisation inspections, updating the current situation where no fee reduction is offered.
These incentives are intended to encourage more pharma companies to enter the growing rare disease market, which has tended to be off-putting for drug makers due to the limited customer base.
In the EU an orphan designation is given to drugs where the prevalence of the condition in the EU must not be more than 5 in 10,000 or it must be unlikely that marketing of the medicine would generate sufficient returns to justify the investment needed for its development.
However, the situation is slowly changing due in part to regulator incentives and the need to move beyond more common conditions.
In addition to the fee reductions for larger companies, the EMA also confirmed the current financial assistance for SMEs.
This includes free-of-charge services for all initial and follow-up protocol assistance, initial marketing-authorisation inspections and pre-authorisation inspections.
Other incentives for orphan drug developers in the EU include ten years of market exclusivity and the chance to make a single marketing authorisation application the EMA.
