Huygens (Consultant): Another common error can be based on the use of new technologies. In CAT, the applications of different technologies are expanding rapidly. Companies must help regulators to understand how these technologies work, what advantages and risks they present, and how a development program will consistently mitigate any risks. If you are adapting technologies used in biopharma, you need to clearly outline how they will fit in your CAT process.
Tente (Humacyte): When the regulators make a recommendation, companies are wise to take it seriously. In my experience, many regulatory problems occur because companies ignored specific input or advice provided by regulators. Whether it is based on a gap in science or a personality conflict, this is almost never a wise decision and is often unnecessary.
Boerner (Argos): Another mistake I see is that innovators don’t take advantage of the established forums focused on CAT issues, such as the Association for Regenerative Medicine (ARM). There are now several groups working with regulators to develop more efficient and streamlined pathways to regulatory review. We are still in a period when only a few products have advanced to late stage development and commercialization, so these alliances can be a major resource.
Tente (Humacyte): In addition to what you present to regulators, you also want to focus on how you appear in these meetings. You want your positions to be credible and backed up by data wherever possible. And you don’t want your innovations and solutions to look like you are cutting corners. This is one area where consultants who have IND and BLA experience can really help.
Huygens (Consultant): For companies outside the EU working with regulators in the EU, many are new to the role of the Qualified Person (QP). EU regulations require that each batch of product is certified by the QP prior to release for use in clinical trials or the commercial market. This is a professional who can facilitate developing the Investigational Medicinal Product Dossier (IMPD) and help in managing product development changes (e.g., change in release specs) while maintaining compliance to the product specification file. The CAT company and the QP should work closely together during clinical development and provide the QP with the necessary data to make an educated assessment. This is a fully independent decision and the QP takes personal responsibility for making a product available in clinical trials or to the market.
Tente (Humacyte): Another big miss is when companies don’t maintain a detailed summary report for a development program. This should be updated in real time and will be needed to support regulatory submissions including the IND. Companies should refer to guidance and standards for summary reports from ASTM, NIST and other industry organizations.