Regulators facing the diversity and complexity of advanced therapies are being challenged with achieving the right balance between providing the regulatory flexibility needed to foster their development and assuring that CMC processes are in place to maximize product safety and avoid the mishaps that could significantly derail their progress.
Agencies like the US FDA are recognizing that striking this balance demands a lot of careful thought and dialogue with sponsors and a high level of expertise among the reviewers involved.
Compounding the challenges are the expedited timelines under which many of these new therapies may be reviewed – where the approval pathway has to be defined and the unique learnings acquired, even as the process is in a fast-forward mode.
Among those who are well-positioned to give voice to the stresses on the regulatory process that this new wave of advanced therapies is creating are the Director of the Center for Biologics Research and Development (CBER), Peter Marks, and the Director of NIH’s National Institute of Biomedical Imaging and Bioengineering Tissue Engineering/Regenerative Medicine Program, Rosemarie Hunziker.
At the opening session of the 2017 PDA/FDA conference, held in September in Washington, D.C., the two offered their perspectives on the product development and regulatory challenges of advanced therapies and what FDA and NIH are doing to help address them and support medical product innovation.
[Editor’s Note: For a more detailed review of the CMC challenges sponsors are facing in developing cell/gene therapies (CGTs) and how the regulatory expectations are taking shape, see IPQ’s December 2017 Monthly Update.]