Breakthrough Therapy Designation And Accelerated Approval With Inadequate CMC Program Development: Hurry Up And Wait

By Scott Burian, Ph.D., Kevin Barber, Ph.D., David Shoemaker, Ph.D.

This article examines the development and marketing application review processes for the chemistry, manufacturing, and controls (CMC) aspects of products targeting serious conditions in the context of the FDA’s expedited accelerated approval (AA) and breakthrough therapy designation (BTD) programs. Inasmuch as these expedited programs were primarily developed to accelerate clinical development with little to no regard for accompanying manufacturing requirements, companies often find themselves having completed their clinical programs far in advance of the development of an adequate CMC program to support a marketing application. This article presents strategies for accelerating your CMC program in an attempt to complete your clinical and CMC development simultaneously in order to maximize the benefit of these expedited programs.