Access and availability of orphan drugs in the United States: advances or cruel hoaxes?

Abstract

The Orphan Drug Act of 1983 in the United States collapsed the barrier between patients with rare diseases and promising drug treatments. Over the subsequent 30 years, > 400 ‘orphan drugs’ became available to them. However, with thousands of rare diseases still left with no treatments at all, many efforts are being put toward generating more investment for discovery, new clinical trial methods and more efficient approval processes. But, a new threat to access has emerged from the costs patients increasingly must bear. We call for a coordinated systems engineering approach that makes more treatments available to more people without unintended negative consequences on individual elements of the process.

Keywords::

• access
• economics
• orphan drugs
• rare diseases
• regulation
• research