Case Study: Protocol-Based Access Programs
Designing and Delivering Expanded Access for Rare Disease Therapy
50 Patients. 20 Sites. 8 Countries.
The Challenge
A large pharmaceutical company had acquired a rare disease company and wanted to expand access to patients outside of a trial for one of the acquired assets. The acquiring company had limited experience in rare disease and expanded access programs, thus needed a partner with the expertise to design and deliver a program and help the company develop the internal procedures and governance for expanded access.
Our Approach
With a significant portion of patients in the US, Germany and Spain, Tanner worked with the company to draft the regulatory submissions for a US expanded access Treatment Protocol and a Compassionate Use Program in Germany and Spain. Tanner managed the submission of the protocols to BfArM and AEMPS, while the company managed the submission to the US FDA. On an annual basis, Tanner supported the company with the development of the annual reports for submission to each regulatory agency. Outside of the core countries, Tanner supported the treating HCPs and sites with all regulatory activities for named patient supply.
