To Deliver Cell and Gene Therapies to More Patients, Innovation in Diagnostic Testing is Essential

The best part of working in the life sciences field is having a daily view of the new technologies and solutions that improve the quality of life for people across the world. One of the most innovative and exciting areas is the advancement of individualized medicine through the successful development and implementation of cell and gene therapies (CAGT).

Individualized treatments like CAGT are poised to scale rapidly over the next decade, both in terms of the number of unique therapeutics and in the overall number of patients being treated. Since 2000, there have been a total of 23 of these therapies approved by the FDA. By 2025, it is estimated that the FDA will be approving 10 to 20 new CAGT offerings each year.

This inflection point is due to the enhanced understanding from both the pharmaceutical industry and regulatory bodies through the successful implementation of the first generation of CAR T-cell therapies. As more data and knowledge is gathered, pharmaceutical companies are strategizing the best way to design their future offerings to encourage adoption at scale. Key to this adoption is investment in the evolution of quality testing processes to minimize risk and maximize output while manufacturing these complex therapeutics.

In the case of CAGTs, because we are dealing with literal life and death considerations, the stakes for microbiology quality control are much higher and require a significant amount of quality analysis including sterility and endotoxin and mycoplasma testing to ensure viability and safety. By integrating diagnostic partners upstream in the process, as early as the preclinical step, pharmaceutical companies can optimize the approval stage processes for developed therapies and move into deployment as rapidly as possible.

Given the delicate and complex nature of these therapies, innovation is required to meet testing needs for new therapies; there often isn’t an existing off-the-shelf solution that adequately addresses diagnostic testing needs. Regulatory bodies are aware of this and require a robust diagnostic testing plan, which specifies partner involvement as early as the preclinical and design stages to account for quality control variables in emerging innovations.

Evolving the Role of Diagnostic Testing Within Pharma

Over the past 2 decades, diagnostic companies have worked alongside their pharmaceutical partners to provide innovative solutions that evolve beyond the existing standard testing to create new tools and techniques to ensure that the industry is meeting regulatory requirements in a more streamlined manner. These experienced testing partners are collaborating with CAGT developers to satisfy the constantly evolving regulatory requirements in the space, while also raising confidence that these new tools, approaches, and methods are fit for the purpose of ensuring the safety and efficacy of these innovative new therapies.

The standard methods for diagnostic testing, often referred to as the “compendial” methods, were designed in the context of traditional sterile pharmaceutical products. As a result, these methods poorly match the needs of the world of CAGT. For example, the compendial methods often assume a long shelf-life product that can accommodate lengthy testing protocols requiring weeks of time.

When it comes to CAGTs, we’re dealing with advanced diseases in which time is of the essence and the therapeutic itself has an incredibly short shelf life, sometimes measured only in a few weeks. As such, diagnostic tests must provide effective, objective results while using a smaller sample size, with simplified data analysis, all on a truncated timeline.

How to Identify the Right Diagnostic Partner

Of course, innovation in diagnostic testing for cell and gene therapies is only one consideration when looking for a partner. To leverage industry best practices, CAGT should seek out diagnostic partners that 1) maintain a deep rooted knowledge of the complex history and the current/emerging landscape of the pharmaceutical industry, 2) have a proven track record of successfully developing and deploying trustworthy testing solutions against the backdrop of a myriad of regulations in pharma labs, and 3) possess both the vision and infrastructure in place to solve problems in real time to bring new, quality solutions to production.

As an emerging field, CAGT is something that’s rapidly evolving; we learn new things literally every day. There’s a constant influx of information and webinars for scientists and decision makers. To synthesize these new developments, the right diagnostic testing partner must have a strong ability to not only understand, but communicate, a customer’s testing story effectively. In turn, they must also be able to maintain a consistent presence in the conversations that are driving the industry forward. If a partner excels in both of these areas, they can do more than just advise on the design of a testing solution. Through collaboration, a trusted partner can develop grounded methodology based on best practices, identify measurement considerations, and also consider constantly changing regulations.

It’s truly an exciting time in the cell and gene therapy space, and diagnostic partners must come alongside the major players to guide best practices and ensure that quality control processes are appropriate and robust. While we can focus on applying technologies to invest in new innovation for testing, it’s equally important that we are able to translate these innovations into stories that resonate with the appropriate audiences to encourage wider adoption among the companies that are inventing new ways to save patients’ lives.

About the Author

Miguel Villa brings more than 2 decades of experience leading complex Life Sciences teams to his role of Vice President, Industrial Applications for the Americas at bioMérieux. In his position, Villa is responsible for managing multinational, multidisciplinary teams across divisions including scientific affairs, sales, marketing, application support, and technical assistance across the US, Canada, and Latin America. Villa also works to continue the development of bioMérieux's global vision of producing innovative solutions that protect public health. For more information, visit www.biomerieux.com.