Orphan Insights, Part 3: Breakthrough Therapy Designation
Orphan Insights is a short series of Q&A assembled from the many discussions I have with companies developing medicines and therapies for rare diseases and who are seeking regulatory advice. These questions are related to when, why and how to engage with the regulators, and take advantage of the various designations applicable to their orphan programs. Each “Part” in this series will pose questions and offer strategic and thought-provoking responses. By no means are these straight forward answers, I hope however that you find the considerations helpful. I encourage you to reach out should you want to discuss anything further.
Part 1 outlined the three most common questions around Orphan Drug Designations, in Part 2, we discussed the PIP.
Part 3 will discuss the Breakthrough Therapy Designation (and everything that comes with it).