By the bioMérieux Connection Editors
In 2020, the Food and Drug Administration (FDA) received over 230 applications from cell and gene therapy developers to begin clinical trials. In a year in which the world was disrupted by the COVID-19 pandemic, the ATMP industry remained on pace with their 2019 output.
Three experts recently gathered to discuss how ATMP developers weathered the pandemic and the potential implications on quality control.
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.
Supply & Demand: A Major Challenge
Michael: In your opinion, has the pandemic been a catalyst for changing the landscape of the ATMP market in respect to quality control and manufacturing?
Kanti: In some sense, yes. The RNA space has exploded. There are a number of RM vendors now which was not the case a few years ago.
Michael: What have been the biggest quality and manufacturing challenges for the ATMP market recently?
Kanti: Simple things like syringes and plastics have become a real issue. Bigger companies—those that are more established—anticipated that, and they locked up quite a bit, including ourselves. We locked up some supplies with our vendors, but if it is something that a company has not anticipated, it’s an issue. Usually at some point that will trigger back in different ways. We can only stock so much and the pandemic doesn’t show any signs of going away per se, so who knows how long this period is going to be.
Michael: Are you seeing this issue get better as the pandemic evolves? Are providers and vendors able to manufacture more and address the problem?
Kanti: I don’t know yet. I haven’t seen huge changes and differences. Right now, one of the things we’re looking at is storage. We are looking at -80 and -150 (freezer storage) and we know the back log and long lead times are there. You would think that every pharmacy that wants to store Pfizer and Moderna vaccines have already bought the -80 that they want, but still the industry has not yet caught up. We are still seeing huge lead times for -80s, which never used to be the case.
Michael: What are you or others in the industry doing to try and compensate for these large lead times? I’m sure it has an impact on timelines—what can be done?
Kanti: It depends on the pace of the company. Commercial companies don’t have much recourse. They cannot just bring new vendors on. But earlier stage companies are looking at other vendors. It’s not that visible, but people are constantly conscious that there could be shortages.
Michael: What can vendors do when working with early-stage companies like yourself that would set them apart?
Kanti: It may be a wish list on my part—I don’t know how much vendors would do this. They are going to look at their portfolio and see who the bigger customer is, but the smaller customers are probably suffering more because there’s only so much bandwidth. But if the vendor companies would allocate some [supplies] for these smaller companies because pipeline is always important, it would help. Even if salespeople are addressing these issues and being transparent saying, “We are anticipating long lead times, can you plan your requirements for the next six months and let me know?” – that kind of proactive conversation would help.
Karen: I think back to everyday life early in the pandemic … some of the shortages we went through at the start of the pandemic, even basic personal supplies couldn’t be had. We try to be very cognizant of the fact that all customers need enough to get them through, but it’s definitely a balancing act.
Keys To Success In The Wake Of The Pandemic
Michael: Bringing a drug to market can be an arduous task in the best of circumstances. What does a pharmaceutical company need to do to be successful in the face of a situation like the pandemic?
Kanti: Be nimble. Be willing to try new technologies. For this, vendors have to be forthcoming in sharing more information and helping early-stage companies to adopt these technologies with assistance and partnership to address regulatory aspects. There are many, many small companies doing really good research and price becomes a barrier to many things. I understand everyone has to make money but some aspects of any kind of help in that manner is appreciated for companies to get a handle on things. I do understand things are very expensive, but I also see companies billing people left right and center for things that are pretty common.
Karen: You raise an interesting perspective on that.
Michael: How has COVID changed the approach to clinical trials, which is a critical step in bringing a product to market?
Kanti: There were a few months when patient enrollment was difficult. Clinical trials had to stop at one point if they were not deemed essential. Asking patients to come back at intervals to get samples was not possible, but those patients were among the first to get the vaccine and we are back to normal now, fortunately.
Michael: What advice would you give to a new startup company in the ATMP market regarding quality strategy and micro testing?
Kanti: The ATMP space is quite large and diverse now. The strategy will have to be specific to the particular needs. For example, the needs of an autologous therapy would be different from an allogeneic therapy. Quality strategy can be phase appropriate, but it will be best to plan fully and execute in phases. In cases where the patient needs a very quick turnaround of tests, we need rapid tests that are comparable to compendial methods. In many autologous therapies sample quantities are also a constraint. The thought process has to be rigorous in thinking about the number of patients at risk. Autologous is 1 patient vs. an allogenic product that could treat 100s and the risk is very different.
Be sure to read part two of the conversation between Kanti, Michael, and Karen.
Opinions expressed in this article are not necessarily those of bioMérieux, Inc.
