Navigating Cell & Gene Therapy Regulations: How Does Your CDMO Match up?

Whether you are a large or new and emerging biotech company, many companies find themselves lacking the internal resources and/or expertise to properly support regulatory submissions. Both large and small operations alike must find the balance between getting life-changing medicines to patients in a timely, continuous fashion, all while adhering to the crucial regulatory guidelines that make these medicines safe, pure, and potent.

A Rapidly Growing Market and An Evolving Regulatory Landscape

When you combine the need to quickly get life-saving products to market and navigating regulatory guidelines with a rapidly growing industry and evolving regulations, any biotech company can find themselves in quite the conundrum. A perfect example of this scenario is the evolving Cell & Gene Therapy (C&GT) industry.

The market for C&GT products, viral vectors, and viral vaccines, is truly unique and in many ways, much different from other biopharmaceutical products. C&GT products are potential “one-and-done” treatments. This is a paradigm shift from traditional biologics—e.g., monoclonal antibodies—where the C&GT market is looking to make cures for patients rather than long-term treatments. This changes the supply chain paradigm and has many existing biologics innovators switching to CG&T. C&GT products can have a dramatic impact on patient lives and while there is a heavy sense of urgency to get them to market, it is still unchartered territory for many biotech innovators. Aspects like high drug pricing, uncertainty as it pertains to healthcare reimbursements, and scale-up costs, are all contributing to both large and small biotech companies’ abilities to get their viral vectors to market in a timely fashion.

The uniqueness of the industry and the means of manufacturing viral vectors, also calls for specialized facilities and expertise:

• Existing recombinant protein/mAb facilities are not designed for viral vector manufacturing.
• Specific HVAC requirements increase the already high operating costs.
• A scalable Quality Management System is needed to enable speed while maintaining robustness.

Despite the novel uniqueness of C&GT products, the market demand for them continues to rise. According to a 2020 report, the C&GT market is expected to be worth $14 billion by 2025, with a CAGR growth rate of over 30 percent. Furthermore, the US Food & Drug Administration (FDA) expects that the C&GT industry will continue to grow at a rapid pace:

“We anticipate that by 2020 we will be receiving more than 200 INDs per year… And by 2025, we predict that the FDA will be approving 10 to 20 cell and gene therapy products a year based on an assessment of the current pipeline and the clinical success rates of these products.”

Scott Gottlieb, MD, Commissioner of the FDA

To help alleviate any extraneous delays of C&GT approvals, regulators such as the FDA and the European Medicines Agency (EMA) have published several formal and informal guidelines to help viral vector companies successfully and safely produce their life-changing products. Unfortunately, due to the evolving nature of the C&GT industry, regulations surrounding these products need to be quickly updated to keep up with the rapidly growing field and its development.

Despite changing regulations, there are CDMOs who are true C&GT experts who possess not only the scientific experience in developing C&GT products, but who can nimbly navigate evolving regulations. No matter the size of your biotech company, navigating regulatory guidelines can be a daunting task—making a CDMO’s experience with regulators a top priority.

While the sense of urgency to get your C&GT product to market is high, the average turnaround time to get a BLA takes just under four years. Long turnaround times and evolving regulations is creating an industry-wide CMC bottleneck—elongating the process to get C&GT products to patients. Neither your company nor your patients can afford to get your viral vectors stuck in a regulatory approval spin-cycle.

CDMO leaders in the C&GT industry can lessen the blow of this CMC bottleneck while still driving development acceleration, and the experienced CDMO will understand the nuances and novelties of the C&GT industry. This example is just one of the many indicators of what makes a CDMO an expert in viral vector services.

Base Level Qualities of an Expert C&GT CDMO

Whether you have a CDMO or are thinking about engaging a CDMO for viral vector services, it’s important to inquire about their base level expertise in the industry. For example:

• Do they expect significant and continuous change to happen to C&GT products in decades to come? The C&GT market is growing and changing at a rapid pace, and the right CDMO should always be able to show how they’re able to pivot with market changes.
• Do they focus on early clinical trials to expedite existing approval pathways to speed up development for quick market launch? Though C&GT approvals can take time, there are existing pathways for approval and the right CDMO will have streamlined processes and procedures to reduce the time it takes to get from development to market.
• Do they engage in future alignment to regulatory frameworks for C&GT products? While no CDMO can predict the future, the savvy CDMO will be able to calculate when regulatory changes may happen based upon their past experiences. This is key for speeding up product approvals.
• Do they collaborate closely and have a good relationship with regulators? Whether it’s the FDA or the EMA, it’s important to know that the CDMO is in good standing with regulators. Not only will the right CDMO have a good relationship with regulators, but they will also collaborate closely with them via formal and informal communications.

Though these questions just scratch the surface of what makes a CDMO an expert in viral vector services, it’s important to get them answered. These questions open the crucial, bigger conversations necessary to fully understand a CDMO’s commitment to your viral vector aspirations, as well as their commitment to continuously manufacturing the viral vectors patients need.

And as part of the CDMO’s commitment to helping you get products to market faster, they also need to be able to maintain business continuity to be able to meet regulatory health authority expectations and commitments. This is even more apparent now as COVID-19 has been a challenging hurdle for any company in the biotech space to overcome. Whether you’re a small or large operation, having to juggle maintaining a safe production space while still meeting regulatory requirements such as inspections, have separated the experts from the novices. The right CDMO should already have in place the proper business continuity processes and procedures to maintain all things related to production—but what about regulatory inspections? How do CDMO’s accommodate inspections while keeping their production facilities safe?

Virtual Inspections: How Adaptive is Your CDMO?

With travel complications still being a concern, many historically in-person meetings have gone virtual—including inspections from regulators like the FDA and EMA. If a CDMO doesn’t have means to support this new virtual relationship with regulators, they will find it difficult to keep your C&GT program running smoothly and without delays. The right CDMO will develop robust means of showing their facilities and procedures with regulators, virtually.

These types of virtual inspections are more than just a WebEx meeting—the CDMO must be able to fully immerse regulators in their sites, in order to be able to hit all the necessary marks of a regulatory inspection. The right CDMO will create a virtual environment that:

• Is in line with regulatory authority remote assessment guidelines—currently only the EMA has formal guidance regarding virtual inspections.
• Uses reliable software such as Microsoft Teams and SharePoint to not only show the site to regulators, but share the necessary and required documentation as well.
• Utilizes real-time, high-quality video streaming of GMP facilities with the capabilities of providing a full, 360-degree view.

When a CDMO engages in this type of mixed reality, it communicates two things. The first is that they are committed to business continuity and can adapt to whatever challenges our global industry may face. It also shows that they won’t sacrifice their regulatory responsibilities—it shows that they value communication with regulators and for the C&GT industry, that collaboration is monumental to the success of any C&GT product.

Thermo Fisher Scientific: Your Collaborative, C&GT Partner

To us, it is apparent—C&GT innovators of all sizes need a CDMO partner that can help them successfully reach the next clinical milestone as quickly as possible, all while mitigating regulatory risk and providing the lowest overall cost to the business. We offer a unique combination of end-to-end solutions for cell and gene therapy development, manufacturing, and distribution.

We view ourselves as not just your C&GT CDMO, but as your partner of viral vector services, driven by your success and getting life-saving products to patients.