There is a heightened sense of urgency by all stakeholders to accelerate development of therapies for rare disease. The FDA shares this urgency, but it also must protect the public safety and follow the well-defined laws and regulations governing the process.
The challenge (or opportunity) is to develop creative approaches, which accelerate access and stay within the regulatory boundaries – or find ways to shape the boundaries.
In this session, Dr. Tim Franson will outline how the use of Intermediate Clinical Endpoints (ICE) could be such a creative approach. ICE measures have shown promise when applied to biomarkers and more common diseases, such as cancer. It could be equally promising for use in rare disease – but it is not without obstacles. In this session, Dr. Tim will outline the promise of ICE, the obstacles to their adoption, and a path forward.
About Tim Franson, M.D.: Dr. Franson is the Chief Medical Officer for YourEncore and a leading regulatory expert on rare disease. He was a key architect of PDUFA 2 – 5, was the VP of Global Regulatory Affairs for Lilly, and co-authored the first patient advocacy-initiated guidance on rare disease submitted to the FDA.
About Joseph Lamendola, Ph.D.: Dr. Lamendola is the Senior Vice President of YourEncore’s biopharma practice. Prior to YourEncore, Dr. Lamendola was the VP of US Regulatory Affairs for Bristol-Myers Squibb and was responsible for 20+ approvals across 10+ therapeutic areas.