CLL Society writes to FDA in support of its Rare Disease Endpoint Advancement (RDEA) Pilot program. However, we also offer a number of recommendations to strengthen the effort, given that CLL in particular could benefit from FDA’s efforts in this realm. Our heterogeneity, indolence, and response to previous therapies can combine to make endpoints difficult to identify and study. Our recommendations include FDA (a) take a broader approach to identifying needed expertise for the pilot, (b) ask drug sponsors to clearly identify the rare disease a novel endpoint might be applicable to when requesting it for more common conditions, and (c) give preference to promising novel endpoints in life-threatening rare diseases for which existing treatments fail to impact outcomes in later stages.
Read the letter to FDA and all our recommendations:
