iSpeak Blog

ISPE Members Leading the Discussion on CMC Issues in Breakthrough Therapy Designation


This post is the first in series of reports on the many regulatory-related activities your fellow ISPE Members are undertaking, and how you can add your voice to these impactful initiatives. The Advancing Breakthrough Therapies for Patients Act was incorporated into the US FDA Safety and Innovation Act (FDASIA) to expedite the development of new drugs or treatments that show dramatic potential in early phase studies.

CMC Challenges for Industry Of concern to industry are the CMC/GMP issues that might lag the accelerated clinical development program, and inspection readiness issues brought on by these expedited timelines.  Parts of the development process will have to be greatly compressed, or possibly even deferred until post-approval, to keep pace with the expedited clinical timelines.

ISPE Members Rise to the Challenge An ISPE Breakthrough Therapies working group has been formed within the North American Regional Focus Group of ISPE’s Regulatory and Compliance Committee (RCC). The working group was convened to implement ideas generated by the participants of a workshop held at the ISPE/FDA cGMP Conference in June. The working group, which is being led by Eric Thostesen of Janssen and which also has the support of ISPE’s PQLI group, will facilitate dialog between industry and the FDA and help address implementation challenges. In August, the Breakthrough Therapies working group led the development of ISPE’s comments on the FDA draft Guidance for Expedited Programs for Serious Conditions (which included Breakthrough designation as one of four expedited pathways), recommending that the FDA take a risk-based approach to CMC review and inspection and asking the FDA to clarify the aspects of manufacturing development that could be negotiated for completion either during review of the marketing application or as part of a post-approval commitment.  See those comments here.

The Discussion Continues The next Breakthrough Therapies workshop will be held on 6 November at the ISPE Annual Meeting in Washington, D.C., and will include a panel discussion with invited FDA regulators.  The ideas generated at this workshop will be used to develop papers and case studies intended to help both industry and the FDA with a smooth implementation of the program in recognition of its importance in meeting medical needs. Can’t make it to Washington, D.C.?  Leave your comments here.