The clinical trial infrastructure for cell and gene therapies is overloaded. How can we strengthen it?

A limited number of investigative sites are qualified to conduct cell and gene therapy (CAGT) trials, and the number of studies has soared since 2016 with more than 2,500 currently in progress, according to the FDA¹. Most are at top-tier academic research centers, and data show staff at those institutions are burning out². Kathy Scott, Associate Director with Parexel’s Site Alliance Americas Team, works on the front lines of this swelling crisis. She shares her vision of what sponsors, sites, and clinical research organizations (CROs) can do to expand CAGT research to community-based sites and build a more sustainable system. 

Restructuring demands collaboration and raises tough questions

Using the same academic research centers for CAGT trials leads to site fatigue, perpetuates the lack of patient diversity, and impedes access to new treatments. Moving trials from overburdened institutions to community-based sites could solve these problems, but it will require collaboration and capital investment by key stakeholders. 

In March of 2023, I participated in a panel discussion on this topic at the Society for Clinical Research Sites Oncology Site Solutions Summit. There was broad consensus among site, sponsor, and CRO attendees that moving CAGT trials into communities is essential to ensure enough sites are available to develop future products. Toward the end of an hour-long, intense discussion, one site representative asked: “Can we please start having these discussions with all of us in the same room? We keep having these discussions in silos.” That plea resonated with me. 

In my work at Parexel, I have found that sites aspiring to CAGT clinical research are frustrated. They cannot find a comprehensive resource detailing the operational requirements of conducting these complex trials. As a result, inexperienced sites find it difficult to navigate the standards established by the Foundation for the Accreditation of Cell Therapy (FACT) and other international certification organizations. The CAGT field needs introductory-level education and training materials for sites. Producing new, easily accessible resources would require seasoned players to share their knowledge and experience. Can this be done?

Currently, most CAGT trials occur in institutions where every study-related procedure, assessment, and medical intervention is conducted within their walls. But expanding CAGT research to community-based sites will require them to manage external providers for some procedures. For example, medical practice investigators may need to work with accredited blood banks for leukapheresis. Or they may need to partner with hospitals to handle the serious adverse events (SAEs) associated with administering CAR T-cell therapies, such as cytokine release syndrome and neurotoxicity. Will hospital research sites already conducting CAGT trials be willing to provide in-patient care services to medical practice research sites in their community (for non-competing trials)? Will accredited treatment centers (ATCs) of commercial cell therapies be willing to provide in-patient care to research participants? These and other tough questions need answers if community-based sites are to make progress in CAGT research.

I propose that sponsors, sites, and CROs collaborate to discuss new operating models and begin addressing these questions. Even without an official collaboration to promote the greater good, all players can be part of the solution.

Here are critical actions each of the three main stakeholder groups can take now: