Addressing Rare Disease Drug Product Manufacturing Challenges

By Shawn Cain, Senior Vice President, Development and Manufacturing, PCI Pharma Services

Once largely considered to be too costly to develop, given the limited number of patients affected by a particular disease, legislation providing financial incentives and exclusivity — coupled with new technologies and breakthroughs in biopharmaceuticals — have changed the orphan drug landscape.

An orphan drug is one that’s developed specifically to treat a rare disease, which is defined as a condition that affects fewer than 200,000 people in the US or one in 2000 in Europe.¹ It’s estimated that there are more than 7000 rare diseases affecting 300 million people around the world.² With only 5% of rare diseases having an approved treatment, there is clearly a large unmet medical need. Recent developments, however, are enabling more orphan drugs to be approved than ever before, providing life-saving therapies to patients across the globe.

Even with economic and regulatory incentives, developing orphan drug formulations presents several challenges. Achieving clinical and commercial success requires biopharmaceutical companies to overcome a number of development and manufacturing challenges related to fast-tracked timelines, the high value and limited availability of active pharmaceutical ingredients (APIs), formulation and scale-up. Addressing these challenges requires experience, expertise and scalable technologies, along with a flexible and agile supply chain.