The Challenge: 

A pharmaceutical client recently developed a gene therapy to treat a genetic disorder that impacts infants and young children, causing them to irreversibly decline, with few other satisfactory treatment options available. The client contractedUniphar to design and implement an Expanded Access Program (EAP) with the objective of providing worldwide access to their therapy for all qualifying patients as quickly as possible. This EAP presented several unique challenges from a regulatory, logistical and commercial perspective as this therapy was a world-first treatment. Most countries participating in the EAP did not have appropriate approval processes in place as they had never imported or approved such a treatment previously. The therapy was also very high in value, leading to the possibility of significant charges and customs duties for the client and patients. In addition, the therapy had to be stored at -80°c, could not be X-rayed in customs, and had to be used within 14 days of shipment. The EAP incorporated a number of different payment models, beginning with a paid programme and later expanding to include a free-of-charge model along with other innovative payment methods.

Our Solution:

Uniphar deployed our 50 years of experience of the distribution of medicines and the related regulatory frameworks, as well as our passionate team of high-calibre logistics and clinical specialists, to meet the EAP’s unique needs and ensure timely delivery of this potentially lifesaving therapy in all the countries in the programme. This was the first global EAP for a high-value gene therapy product in a critical disease area, carving a hopeful path for many more therapies to come. The most important success, however, was that the EAP provided a significant number of patients across more than 40 markets access to essential, timely treatment.