Tufts CSDD Report Finds Orphan Drug Approvals Have Increased, But Challenges Exist

Orphan drugs are defined as those developed for rare diseases and conditions that affect fewer than 200,000 people in the U.S., or five per 10,000 or fewer people in the European Union. Although the pace of approvals for new orphan drugs have increased in the United States 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 U.S., 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. Among the challenges that limit patient access to orphan drugs in the U.S., relative to Europe, is higher cost-sharing by patients, which can lead to increased levels of non-compliance.

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