Q&A: Key Regulatory Considerations for Moving Your Cell Therapy Research to the Clinic
Meet the Speakers
Lynn Csontos
Lynn Csontos, Vice President of Regulatory Affairs at STEMCELL Technologies, has worked in the biotechnology industry for over 25 years. Her background started with small molecule API and drug products, and she uses that knowledge to navigate the regulatory landscape currently being defined for cellular, gene, and tissue engineered therapies.
Mike Jones
Mike Jones, Director of Clinical Services and Alliances at STEMCELL Technologies, has worked in the biotechnology industry for over 12 years, notably in Product Management and Regulatory Affairs. In the last 7 years, he has focused on supporting customers with the use of STEMCELL products as ancillary/raw materials for cell therapy manufacturing.
The cell and gene therapy market continues to advance with a significant impact on healthcare. As a cell therapy developer, qualifying raw materials used in your manufacturing process is critical to success in your translational journey. As a supplier, STEMCELL can help you reduce time and costs for translational projects. Our Services for Cell Therapy team can walk you through key regulatory considerations that will support the success of your clinical candidates. On June 11, 2024, STEMCELL hosted a half-day, virtual event, “Translational T Cell Talks: Scaling for the Future.” At this event, Lynn Csontos and Mike Jones answered participants' questions about ancillary material qualification and the trends in regulatory requirements for clinical trials. Below is a transcript of the Q&A session and a link to the full presentation.
Webinar: Key Regulatory Considerations for Moving Your Cell Therapy Research to the Clinic
Watch the talk led to this Q&A. This talk was originally presented at our Translational T Cell Talks half-day virtual event which featured 6 other talks with T Cell experts. Learn about crucial regulatory strategies for enhancing clinical candidate success, covering the latest developments and best practices in materials qualification and supplier partnerships.
Q&A Report
How much more relaxed is the ancillary material guidance for early-phase clinical trials vs. commercial products? Is it okay to not have full-GMP-level rigor for some materials?
Lynn Csontos
I would say there’s more relaxation around ancillary materials from the regulators for early phase clinical trials. For example, the process validation or the method validation of a given reagent hasn't been completed at the time of customer use in early phase clinical trials. This reflects the guidance from the FDA for GMP for Phase I trials. We’ve found that taking a similar approach has been successful thus far. One area where we have found very little wiggle room is the viral safety profile. Regulators are being very sticky about that, which I highlighted in the presentation. As such, that is something you will need to work with your supplier on to ensure you have the proper documentation on your Certificate of Origin to demonstrate your ancillary materials have an acceptable viral safety profile.
In the face of ever-changing technological solutions in cell product manufacturing, how do the manufacturers and the regulators meet each other’s needs, from application submissions through approvals?
Lynn Csontos
Well, that is a fantastic question, and I'm not sure I have an answer. I'm going to give you my opinion, and that is that they are working very closely together. There are many forums, some mentioned in my presentation, such as ISPE, ISCT, ARM, and Biophorum. All these organizations are working with regulators to develop standards, guidelines, and requirements for the manufacture of cell and gene therapies all the way down to ancillary materials that STEMCELL manufactures. It is a learning curve for both the market and the regulators. If you are a sponsor of a cell and gene therapy, you should open that communication channel with your local regulator to ensure you're staying on track with the requirements.
What are some challenges that STEMCELL has helped previous sponsors/clients/customers overcome?
Mike Jones
From our perspective, and this is thankfully becoming less common, we've seen a number of clinical trial applicants submitting their Investigational New Drug (IND) or Clinical Trial Application (CTA) without having first gone through the ancillary material qualification process. In those cases, the regulators will typically come back to the applicant with many questions about their material controls that require the sponsor to go back to their suppliers and gather more information, and sometimes even put additional materials controls in place. That whole process can take a lot of time, and the IND remains on hold the whole time. Over the years, we've seen it becoming more and more standard for sponsors to carry out a robust risk assessment beforehand and make good use of pre-IND meetings with the FDA or the equivalent in other jurisdictions.
In your experience, what is the most common issue that you see slows a project down?
Mike Jones
STEMCELL has worked with customers to support the use of our products in nearly 90 clinical trials so far, and most have included the use of non-GMP products. So, it's most common in those cases for customers to have additional requirements in terms of how the product is tested, as well as in the manufacturing controls that are in place and the grade of raw materials used. We've been successful in working with the sponsors to put customized solutions in place to address those gaps. We have a dedicated team at STEMCELL called the Services for Cell Therapy program that works one-on-one with clinical trial sponsors for those types of solutions.
If you could give one piece of advice to someone who’s considering spinning out a biotech from academia, what would it be?
Lynn Csontos
Start with the end in mind, which means, design quality and safety ‘into’ your product. A part of that is ensuring you effectively qualify the ‘right’ reagents/raw materials for your process to build quality and safety into your final therapeutic product. And one more thing—partner with your supplier early!
Webinar: Qualification of Ancillary/Raw Materials for Clinical Use
Watch this presentation which gives a step-by-step guide to qualify ancillary materials for clinical use. It will focus on the path to qualification, region-specific regulations and guidelines, common industry issues and case studies.
