By the bioMérieux Connection Editors
Despite the COVID-19 pandemic, the ATMP market has surged over the past couple years. Many cell and gene therapies are among the first of their type developed—as with any pioneer in the pharmaceutical space, growing pains are to be expected.
Three ATMP experts recently discussed how the COVID-19 pandemic has impacted pharmaceutical quality control processes and the main takeaways for new ATMP developers.
Meet The Experts
Kanti Thirummorthy is the VP of Technical Operations at Neogene Therapeutics and has a background in PD, MS&T, QC, and QS. She joined the ATMP space in 2014 and values the ability to work on innovative solutions for patients.
Michael Mestaz is a Healthcare Account Manager at bioMérieux with extensive experience in laboratory settings. Michael brings years of QA/QC knowledge related to consumer, patient, and product safety.
Karen Mullen is a Regional Sales Manager at bioMérieux with a demonstrated history in the biotechnology industry. She is passionate about new technologies, particularly in Pharmaceutics, Medical Devices, Biotechnology, Animal Health Management, and Product Development.
Accelerated Timelines Require Nimble Quality Processes
Michael: Vaccines and therapeutics for COVID-19 have come to market in record time. How did this affect the mindset around quality in the industry?
Kanti: Quality processes have had to be nimble. Development, qualification, and verification of methods were needed in record time. I think the rest of the industry can learn from the ATMP area, especially the autologous area. They had to reinvent how quality systems and risk management could work when the product had to be released within a short duration. This includes deviations and non-conformities that need to be closed out quickly, and that doesn’t mean that when we do this we are not “crossing the T’s or dotting the I’s,” it’s just how you do it in a systematic manner.
Even in this vaccine scenario you can see that some people succeeded and some people didn’t. I think this is a lesson we should learn and continue to go forward with. I’ve been on both sides of the aisle where quality works with the organization and then there are places where quality wields the hammer and then that’s it. It must be a mutual education of how we get out of this—it’s a joint conversation to be had between the laboratory, production, quality control, and risk management.
Karen: It has forced people to think outside the box and work differently. The ATMP market has had to be much more innovative in making sure they have a quality product, just due to the nature of the product, and the rest of the industry can learn a lot from that.
Kanti: It’s not the quality and regulation functions themselves, it’s how we implement the processes. Quality of the product is much more than compliance of the product, and when we start understanding that piece then we find the right solutions in between. And the companies that do that, succeed.
Karen: I like your comments regarding how Traditional Pharma can learn how the ATMP industry has adapted not only during the pandemic, but as an industry itself. The nature of on autologous product leads to out of the box thinking when it comes to product and patient safety. ATMPs are pioneering and setting the example of how success can be achieved through collaboration and through process development. Just because you are compliant doesn’t necessarily mean the product is automatically safe. Deviations have to be closed out quickly as they don’t have the luxury of releasing the product to storage. Autologous products are released directly to patient. This is literally life and death.
Looking To The Future: The Need For Faster, Regulated Technology
Michael: What emerging diagnostic technologies do you think will have the biggest impact moving forward?
Kanti: Shorter timeframe of testing with technologies fully comparable with the gold standards will have a huge impact. Many of the safety tests fall in this category. If these faster technologies can become compendial so it is standardized, it will help the industry.
Michael: Considering that some of these things can take a while to become compendial, how do companies like yourself address those methods?
Kanti: Four or five years ago the PCR methods available were not fully comprehensive. ATMP companies ended up building in-house PCR methods and we did a huge comparability study. Just validating the PCR method is great, but it’s not the end of the story. So having the full package from the vendor is essential.
Michael: Gene editing is gaining more attention, but what are the safety implications?
Kanti: Gene editing is happening in the industry in multiple ways, in vivo and ex vivo, with very different requirements for each. In vivo would have much more safety questions. Vendors need to make sure their products, including validation packages, are comprehensive, which means a comparison between compendial methods and their technology. It saves the customer considerable time and energy if they don’t have to do compendial comparability.
Karen: It’s like a brand new frontier of science that you’re starting to journey into but you don’t know what the boundaries are yet.
Kanti: Correct.
Michael: How has your interaction with the FDA or other regulatory agencies changed during this time? Do you think these changes are here to stay?
Kanti: Agencies and companies have created ways to have virtual interactions quite fruitfully. Agencies conducted a few virtual audits, and the logistics worked well. Some of this is bound to stay. I think it will become a hybrid of virtual and in person audits. The preparation for the audit didn’t change at all. There was a WAR room and interaction room, just in the electronic world. You need the support of your IT team, but it’s entirely doable.
Michael: Do you believe any part of the industry needs to be better regulated?
Kanti: Some standardization may help. For example–vector copy number standardization.
Michael: What would be the benefits of standardization?
Kanti: Vector copy number stands out as it has been a hot topic of discussion since 2018, at many conferences and amongst customers. For all transfused therapies, the vector number shall be no more than five. The agency gave a target, but depending on your technology, the result was very different. We ended up with a consortium of companies coming together and engaging with the agency to come to terms on how to get the final result they were requesting.
Karen: There’s a delicate balance.
Kanti: It doesn’t have to be a USP chapter, but to give opinions would help customers decide where the standardization and limit boundaries are.
Karen: COVID has been a pain on many levels, but it forces a different collaboration, especially among regulatory bodies and it’s nice to see that interaction between manufacturers themselves.
Be sure to read part one of the conversation between Kanti, Michael, and Karen, in which major quality control challenges presented by the pandemic were discussed.
Opinions expressed in this article are not necessarily those of bioMérieux, Inc.
