Orphan drug approvals rise, but high costs pose challenges for patients

Although the pace of approvals for new orphan drugs - medicines that treat relatively rare conditions - have increased in the USA and Europe in recent years, patients are facing growing challenges accessing those drugs, a newly completed study by the Tufts Center for the Study of Drug Development at Tufts University has concluded.

During the 14-year period 2000-2013, 86 orphan drugs were approved in the USA, up from 65 during the prior 18-year period 1983-2000, while in Europe 96 orphan drugs were approved in 2000-2013, more than double the 44 approved in the earlier period, according to Tufts CSDD.

Orphan drugs are those developed for rare diseases and conditions that affect fewer than 200,000 people in the USA, or five per 10,000 or fewer people in the European Union.