Orphan drug approvals of 2014: Europe and the United States

Abstract

Introduction: The development of orphan drugs for the treatment of rare diseases has attracted increasing interest from the pharmaceutical industry. The year 2014 saw an increased level of orphan drug approvals, possibly reflecting the increased attention given to rare diseases.

Areas covered: The review discusses all the orphan drug approvals of 2014. It first considers the 12 European approvals, which includes eight novel chemical entities (NCEs). It then considers the 44 US approvals, which includes 17 NCEs. The discrepancies in approval timings between these markets are then discussed.

Expert opinion: The increase in the number of orphan drug approvals appears attributable both to an increased awareness of rare diseases and to the FDA’s efforts to expedite the approval of drugs to treat rare diseases and serious conditions. Europe’s failure to follow suit appears to be one factor in the delays between US and European approvals of orphan drugs.

Keywords:

• European Medicines Agency
• FDA
• orphan drugs
• rare diseases

Declaration of interest

The author has no relevant affiliations or financial involvement with any organisation or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents, received or pending, or royalties.

Notes

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