Cell and gene therapy (CGT) sponsors are wrestling hard with how to prepare for having to make CMC changes throughout the development, submission, and post-approval stages, while facing accelerated timeframes and significant knowledge limitations.
The nexus of issues that the change component presents is a prominent concern in the intense discussions now taking place at public forums on how to go about: ● creating viable strategies for assuring CGT product quality in an accelerated development context, and ● conveying that assurance to regulators.
At a session on accelerated development at the June 10-12 CASSS CGT Products Symposium held in Bethesda, Maryland, Celgene Global Regulatory CMC Director Yoko Momonoi and Voicin Consulting Life Sciences (LCLS) CMC Director Alexandra Beumer Sassi presented industry views on how to manage the inevitable changes that come with having to create viable CGT processes on compressed timelines.
Momonoi and Sassi both stressed the need for upfront planning and early and proactive agency interactions to identify: ● likely process changes that could impact product quality and comparability across the development and post-approval phases, and ● a viable strategy for instituting and assessing them.
Presenting a regulator perspective on addressing the CMC pressures created by the accelerated development and review timelines that CGTs often face at the CASSS session was CBER Office of Tissues and Advanced Therapies (OTAT) CGT Division Branch Chief Stephen Oh.
Oh reviewed the landscape of advanced therapy approvals and IND submissions, and how the expedited programs are coming into play – offering insights on working with CBER to chart pathways and avoid pitfalls.
[Editor’s Note: For a review of Oh’s comments at the session, see IPQ September 28, 2019. For additional regulator and industry perspectives on the CMC challenges that industry faces on route to realizing the promise of advanced therapies and insights on how collaborations between government, industry, and academia can help address them, see IPQ September 17, 2019.]