Current & Future Landscapes of Cell and Gene Therapy: Analytics & Regulations
What Is the State of Cell and Gene Therapy in the Current Market??
The outlook for this young industry is very promising indeed. From a growth perspective, it is predicted that the global size of the cell and gene therapy market will reach approximately $42.56 billion by 2030 and grow at a CAGR of 39.42% between 2022 to 2030.1 Many new companies are surfacing, and exciting technologies that hold tremendous promise are emerging, as we will discuss.??
The relative youth of the cell and gene therapy space within the larger pharma industry does present challenges, though. For regulators and quality workers particularly, it can be difficult to determine how to ensure that new products that are being created with new processes are safe and capable of treating specific indications. For sponsors, working with a CMDO that has an expert quality team is crucial to the outcome of your project.??
Our experts, Barry Conner, MBA, VP of Quality & Regulatory, and Michael Paeltz, M.D., director of Quality Control, discussed the role of quality teams and regulatory bodies in the advancement of the cell and gene therapy space in a recent LinkedIn Live discussion, briefly summarized here.??
What Does It Take for Drug Companies to Successfully Navigate Quality and Regulatory Requirements??
For drug makers, meeting these requirements means putting in the time and effort to thoroughly understand your product, and support in that area is something that you should expect from your CDMO partner. With the rapid expansion of this inherently advanced industry, there is constant flow of new technologies, new guidance and regulations, and new contract organizations offering new or updated services. In short, there is a lot to take into consideration for anyone seeking a path to market. To optimize your drug’s potential, you must understand what has been done before and how you can do it better. Most critically, you must be able to fully characterize your product and produce something of quality, and that requires choosing the right CDMO partner.??
From a Quality Perspective, What Benefits Should Companies Expect From Working With a CDMO??
Drug companies can benefit strongly from working with a partner who understands the fundamental phases of GMP. Experienced people know that specific items must be understood for Phase I vs. Phase II and onward along the full pathway to market.??
Good CDMOs have those people.??
The right partner will be able to not only help determine the critical pathways that must be developed for drug products, but they will also then communicate that to their clients in a clear manner. Having this information will be crucial when it comes time to approach regulatory authorities.?
Everyone involved must do their due diligence to ensure quality, and while an expert partner will certainly operate in a streamlined manner, quality can never be sacrificed for the sake of speed. The GMP requirements for each phase must be followed to the letter so that when your project is completed, you will be able to show regulators precisely how your product will benefit patients. When you demonstrate this to the EMA or FDA, for example, you should be able to walk them through your process and show how it aligns with regulations and their current thinking. Without strict adherence to quality standards, you could experience delays in your program, and you might have suboptimal results when you get to clinic and start dosing patients.??
Keep in mind that regulators are focused on constantly learning in this new space, and they are becoming more advanced in their thinking. This challenges drug makers to take every step necessary to deliver safe and efficacious drug products, and your CDMO’s quality and regulatory expertise are the keys to meeting their requirements and experiencing the best possible outcome for your product.??
What Do You Need to Know When It’s Time to Approach Authorities With an NDA??
First and foremost, as a drug developer, you must have a clear understanding of your program from concept to completion. You will have to walk regulators through each step and demonstrate your controls, and if you cannot do this in a crystal-clear manner, it can lead to delays and the need for additional documentation. Possibly, additional studies could be required to prove you have control over your process and product.??
In essence, you need to tell a clear story from beginning to end:?
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On that last point, remember that not every process goes perfectly; however, this provides opportunities to improve it, and you will be required to provide evidence for your corrective actions. Being able to tell a complete story will help regulators understand your product and process, and ultimately make them comfortable that your product is safe and efficacious. The purpose of this industry is to improve the lives of patients, after all, so demonstrating that your drug can do that is paramount.??
It Pays to Be Proactive?
Our experience includes helping clients prepare for Phase III and the filing of a biologics license application (BLA), and there is a lot of work and documentation involved in that process. We know that taking the right steps on the front end, however, including managing paperwork and maintaining a cohesive, detailed, transparent story of your drug’s fully compliant journey, will make standing in front of regulators at this point much easier. That is the kind of support we give, and that is what you should expect from your partner.?
How Can an Experienced Quality Team Help Develop Better Drug Products??
One of the most important considerations for a quality team is the implementation of advanced technologies. We are experiencing a boom in the development of exciting new technologies, including improved computer systems, instruments that can characterize cells using light, next-generation sequencing, and nano-pore systems. All these advancements open the door to new possibilities for the rapid and safe advancement of biologics.??
To drive this industry forward and ensure that high-quality drug products make it to patients in a timely manner, we must be prepared to go beyond the US Pharmacopeia Convention’s (USP) current thinking and integrate the latest technological advancements into our processes. It is easy to get stuck in the rut of doing things a certain way — the way you have always done them — but that is not the best way to accelerate the creation of new, more effective medicines.??
Certainly, there is a fine line to be walked between old and new technologies, and comparability studies are essential. For people in the quality domain, new technologies can be embraced, just in a quality-minded fashion. That means engaging the appropriate regulatory bodies regarding validation and comparability activities and ensuring our approach is scientifically sound. This takes effort and expertise, but when those steps are properly taken, we have the opportunity to improve patient outcome through better accuracy and specificity in our analytics.??
An Innovative Analytics Example?
Focusing on next-generation sequencing, we can now do single-cell, single-plasmid, and single-vector sequencing with high accuracy, which means we can demonstrate that everything in a product is what you say it is. This is a giant leap forward from using polymerase chain reactions (PCRs). This technology also enables advanced troubleshooting, so that whether you are using lipid nanoparticles (LNPs), viral vector or MRNA, you can quantify and verify the accuracy of the DNA. This used to take weeks, but that time has been drastically reduced and continues to shrink. This is the kind of advantage that staying on the forefront of technology provides, and if such technologies are integrated into a CDMO’s services properly — with quality and regulatory compliance at top of mind — the benefits to clients and patients can be massive.??
Navigating the Path Forward??
The cell and gene therapy industry has the technology to improve the safety, efficacy and potency of drug products, which amounts to better medicines — faster — for the patients who need them. To ensure we implement them properly, though, we must take a measured approach that engages regulatory authorities and ensures the quality of drug products and processes. That is our approach at Matica Bio, and that is a sure way to advance the capabilities of the cell and gene therapy industry.??
Reference?
1 Biospace.com. Cell and Gene Therapy Market Size, Growth, Trends, Forecast Report 2022-2030. Published Aug. 11 2022.?