Next-Generation Orphan Drug Delivery

According to the National Organization for Rare Disorders (NORD), a disease is rare if it affects fewer than 200,000 Americans. There have been around 6,800 rare diseases defined so far, affecting around 30 million people in the US alone. Big Pharma used to ignore this segment of the market, but now developing so-called orphan drugs to treat these rare diseases is  industry priority.

The global orphan drug market is lucrative and growing. According to BCC Research, this sector was worth around $82.6 billion in 2011, growing to almost $86 billion in 2012 . In a 2013 report from Evaluate, the global orphan drugs market was projected to be worth $127 billion by 2018. “This sector is predicted to grow at 7 to 8% a year–twice as fast as the rest of the market--to make up 15% of the [total drug] market by 2018,” says Andreas Weiler, global head of strategic marketing for SAFC, the custom manufacturing and services unit of St. Louis-based Sigma-Aldrich. “It’s not just a business of small companies. About half of the top 20 selling orphan drugs originated in Big Pharma.”