Navigating nuanced CGT clinical trial logistics

It is no secret that cell and gene therapy clinical trials are already among the most complicated therapeutic areas in research. However, given the transformative benefits for patients who are typically limited in viable treatment options, the industry is experiencing record investments in CGT development, with 406 clinical trials initiated in 2023. In 2024 alone, seven cell and gene therapies were approved by the U.S. Food and Drug Administration for various therapeutic needs.

Offering hope to these patient populations is critical, but as those in the life sciences industry know, trial and patient journey complexities can pose significant barriers to the success and sustainability of these individualized treatments. Managing the vein-to-vein therapy process can be as intricate and individualized as the patients it aims to treat. Within the larger CGT trial framework, the complex logistical nuances for how treatments requiring sensitive and precise handling are tracked and progressed from apheresis to infusion needs special attention.

At the 16th annual SCOPE Summit in February, research and development industry stakeholders came together for open conversations regarding key ways to elevate patient-centered clinical trial innovation. There was a particularly insightful discussion on the necessity of mitigating risk and complexity in CGT trials. It highlighted how advances in interactive response technology (IRT) platforms for drug supply can be highly beneficial when thoughtfully designed to address the unique challenges of this complex therapeutic area. This is especially true when these platforms are integrated into a comprehensive consultative approach led by specialized expertise.

Setting the stage: Trial logistics

Logistical considerations for CGT studies are quite different from other therapeutic areas, with a critical focus being effective end-to-end sample management.

Current manufacturing of cell therapies is a laborious, complex and costly process, especially when considering limited availability of production materials, logistics, supply chain, cold storage and stringent regulatory guidelines. This process can represent one of the most challenging aspects of a CGT trial. Unlike traditional investigational products, these therapies demand individualized biological production processes that encompass cell isolation and genetic modification, expansion and preservation.

Additionally, tracking the entire process from cell collection to infusion is intricate and extensive. This includes tracking multiple numbers associated with the investigational product (IP), such as a sample identifier, a manufacturing number, a lot number and a release number, all of which are unique to a patient. Along with tracking and documenting progress and managing potential risks with real-time adjustments, maintaining precise temperature-controlled storage and transport at every step of movement of the supply chain is vital.

Trial sponsors, study teams and sites have to account for the variabilities and multiple ‘what if’ scenarios throughout the manufacturing process, which can impact patient dosing, create delays in access or cause risk of contaminated, lost or inconsistent cell batches. Each patient’s treatment requires precise orchestration between collection sites, manufacturing facilities and treatment locations, creating a complex web of interdependent operations.

Effective collection to infusion

For all types of clinical trials, integration of a purpose-built interactive response technology platform provides end-to-end visibility of IP movement for accurate tracking of shipments. Each relevant stakeholder can easily see their responsibilities, including supply chain managers requiring a bird’s-eye view of IP monitoring to address issues quickly.

In planning for all the steps within a CGT trial and what may require keen monitoring and adjusting, a clinical technology provider partner can help identify nuanced capabilities needed within a tailored IRT to help uphold good clinical practice, regulatory compliance and patient safety standards. For CGT trials, IRT platforms with flexibility, a high degree of configurability and a comprehensive library of existing cell and gene specific components enable sponsors and study teams to apply varying expertise where needed, building precisely per the protocol requirements.

This is why early in trial planning, it is key to include technology providers to work with sponsors, study teams and therapeutic experts to map out every step needed from IP development through administration, answering questions, such as:

• What is the chain of custody for the step-by-step process that will need to be accounted for in IRT platform design for this specific trial?
• What is every possible logistical step needed to make sure treatment administration occurs as planned?
• Are we complying with regulatory requirements from end-to-end?

Protocol-specific design

Every aspect of a trial can impact protocol design and create unnecessary amendments, trial delays and risk of not meeting regulatory requirements. As such, it is important that IRT platforms are designed with the protocol in mind, especially for complex CGT trials. CGTspecific IRT configurability should aim to:

• Account for safe and compliant processes, in accordance with good clinical practice (GCP) parameters, regulatory compliance and patient safety standards.
• Support data validity, preventing errors that occur with tedious or repetitive manual data entry and tracking processes.
• Ensure data is reconciled, with key integrations and automated workflows for quality source data and to eliminate the need for continuous data reconciliation steps.
• Provide flexibility in study design and service support, which can be tailored to meet the complexity and specific needs of each protocol and allow for more efficient collaboration with supply chain partners.

Why flexible IRT matters for CGT trials

As noted previously, CGT trials are unique and complex in many ways, especially as the personalized nature of advanced treatments means each batch follows its own unique timeline with specific handling requirements. This makes it critical for sponsors, study teams and technology providers to anticipate and address unusual scenarios that could affect protocol adherence and potentially derail the trial all together.

A few noteworthy examples of where highly flexible and agile IRT platforms can help reduce risk within CGT trials, include:

• Patient recruitment and enrollment for CGT trials, especially regarding rare disease patient populations, can be challenging. This can make adequate supply for sites difficult. Advanced IRT platforms can provide real-time visibility into IP stock and offer supply optimization suggestions. It is also possible for IRT platforms to help support patient-centered approaches, including decentralized solutions (e.g., home visit coordination, virtual provider visits, electronic consent, etc.), aiming to improve the patient experience and related retention.
• When a CGT trial participant receives a specific therapy developed just for them, the IRT platform needs to adapt and allow for the unique processes or actions for each cell therapy treatment, such as temperature excursion and management (e.g., monitoring cryogenic storage requirements).
• In some cases, a sponsor may request a secondary product depot location where transportation to site for administration requires treatment to be stored in liquid nitrogen for thawing and preparing at the site. IRT programming needs to be able to designate and label a ‘ready to use’ status when the secondary depot team has thawed and prepared the treatment, and then, systemically know to create an expiry date for each kit/ treatment in the platform.

Despite trial complexity, advanced IRT solutions must be designed to enable the relevant stakeholders to control their points of process without disrupting the trial activities outside of the system.

Consultative guidance from experts

As with any trial-specific technology, it takes more than an advanced solution to help plan for trial success. It requires a collaborative mindset where sponsors, study teams, therapeutic experts, trial design experts and technology providers come together as early as possible in trial planning phases.

Working together, it is beneficial to emphasize potential areas of concern and best practices for trial efficiency from each perspective, all in alignment to uphold GCP, patient safety and data quality standards and comply with regulatory requirements. How an IRT is designed specifically to meet the needs of that trial and related participants depends on this cross-stakeholder planning and dialogue.

For example, sponsors with limited experience in CGT trials may require guidance from therapeutic experts, study teams and seasoned clinical IRT platform and service providers. This support can help them effectively navigate the unique complexities of these trials and create efficiencies in operational processes. In such scenarios, a consultative approach is essential to ensure a high degree of sensitivity and oversight throughout the therapy development and administration process.

Staying ahead of complexities to come

Monitoring the success of CGT R&D in providing patients with benefits compared to currently available options, if any, will be critical to better understanding how related trials will need to be fine-tuned to improve outcomes. As we look ahead to future CGT development, trial sponsors will need to consider the evolving landscape and expanding complexities. For example, will multi-country R&D activity grow beyond the U.S. and China? And, if so, how does that impact trial harmonization efforts and related oversight needs from clinical technology providers and tailored IRT platforms?

Having the ability to use one’s own cells to create therapies to meet their individual needs is at the heart of personalized health care. How R&D stakeholders improve upon this advanced approach will continue to evolve. Just as CGT R&D changes, experienced clinical technology providers must continue designing and updating tech-enabled solutions with innovation, providing more flexibility in capabilities per individual CGT trial needs while always maintaining consistency in high security and safety standards as well. 

References

IQVIA Institute. (Mar 2024). Strengthening Pathways for Cell and Gene Therapies: Current State and Future Scenarios.

Macpherson, J. (2025). From the Editors: Cell & Gene Therapy Approvals in 2024. ISCT, Telgraft Hub.

16th Annual SCOPE Summit [Conference]. (2025, Feb 3-6). Orlando, FL.