The US Food and Drug Administration defines a rare disease as a disease with fewer than 200K patients in the US. Despite this apparently low number of occurrences, over 7K rare diseases affect a staggering 30M patients in the US alone, thereby necessitating solutions to advance their development and ensure a flawless journey from vein-to-vein.

Breakthroughs in advanced therapy offer a beacon of hope for patients. Yet, navigating the journey from identifying mutations to delivering effective therapies poses significant challenges. The conventional healthcare model doesn’t fit, requiring alternative approaches and a robust clinical supply chain.

This featured article explores challenges faced in developing and delivering advanced therapy medicinal products, including dispersed patient populations, complex manufacturing processes and regulatory hurdles, and how Marken has adopted a hypercare model for all rare disease shipments.