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

By Kathy Scott, Associate Site Alliance Director

4 min

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:

Three main stakeholder groups


Streamline protocols and move beyond comfort zones

The scope of CAGT clinical trial protocols is crippling workflow and productivity at sites. Studies replete with exploratory endpoints, assessments, and genetic testing strain resources³. Some of these elements are required; others could be re-assessed. Large academic centers now have so many competing studies (whether CAGT or not) and so few available resources that they must strictly limit the number of trials they undertake. At Parexel, we see institutional disease and resourcing review groups increasingly reject resource-intensive trials. For sponsors, streamlining CAGT protocols is the most effective way to lessen the burden on sites. 

Sponsors are understandably risk averse. They feel comfortable with sites that have a CAGT track record, well-published PIs, and a single contract covering all components of care, from screening visits to apheresis to managing SAEs. However, we advise clients to comprehensively assess why they hesitate to consider community sites and to quantify the risks and tradeoffs.  

We believe most sponsors will benefit by moving from what is familiar and expanding the base of CAGT research sites. First, we find that many community-based hospitals did not suffer as much loss of staff as larger institutions during the pandemic-related ‘Great Resignation’ of healthcare workers. Community sites are eager to conduct CAGT trials because they want their patients to access these potentially life-altering and life-saving treatments. Seeding CAGT capabilities into more regions will support the uptake of approved CAGTs. Finally, community practices and PIs could potentially recruit more racially and ethnically diverse and other historically underserved patient populations.⁴

Sponsors must invest in site development before initiating a trial, requiring vision and discipline alongside resources and capital. But companies can begin with small steps. For example, would sponsors consider reimbursing sites to attend FACT workshops to learn the standards and capabilities required for certification? Would sponsors be willing to approach their ATCs to encourage them to support in-patient services for research patients from medical practice sites? It will be critical to devise trial budgets to make these services more financially attractive for hospitals, blood collection centers, and other providers. Sponsors might even contract with flagship regional hospital systems and cell collection centers to support multiple community research centers.


Examine resourcing and begin addressing gaps

At Parexel, when we talk with community-based practices that want to participate in CAGT trials, the biggest hurdle for them is the in-patient care requirement and the procedural complexities of cell collection. Frequently, these sites must conduct a thorough gap assessment and then explore the options available to outsource the services they will need.

They need to ask themselves:

  • Do we have existing partnerships with a hospital for in-patient management?
  • Do we have a blood collection center for leukapheresis?
  • Do we have an infusion center to administer preparative treatment and investigational CAGT products?
  • Do we have or can we obtain Institutional Biosafety Committee certification?
  • Is it feasible to seek FACT accreditation?

Sites can leverage their existing sponsor and CRO relationships to request formal assessments of their CAGT readiness using standard pre-qualification checklists. Evaluations should include detailed reports identifying which operational aspects require further development. 


Support site, build relationships, and achieve cost efficiencies

At Parexel, we believe CROs that establish relationships with community-based research centers will serve sponsors and clinical trial participants more effectively. We work to identify non-academic research institutions already conducting CAGT trials, especially those with greater capacity for new trial opportunities, and profile their capabilities and experience. With that knowledge, we can provide targeted support and advocate for them with sponsors during the site selection process.  

To ensure the continued growth of CAGT-capable sites, CROs must locate community-based researchers who want to run these trials and determine how to help them fill operational and expertise gaps. This level of support for sites represents a significant resource and capital investment. Establishing relationships with community- and regional-based hospital systems may allow the placement of studies at multiple regional locations with institutional oversight, standardization, and cost efficiencies.

At Parexel, we facilitate advisory groups for direct communications between sponsors and community sites. We aim to foster a transparent dialogue about what support is required and which operational models are feasible for both. 

  1. Brennan, Z. (2023) Thousands of gene and cell therapies are inundating FDA reviewers as the agency tries to keep up, Endpoints News. Available at: (Accessed: April 4, 2023).
  2. Rotenstein, L.S. et al. (2023) “The association of work overload with Burnout and intent to leave the job across the healthcare workforce during COVID-19,” Journal of General Internal Medicine [Preprint]. Available at:
  3. “Rising Protocol Design Complexity Is Driving Rapid Growth in Clinical Trial Data Volume,” (2021) [Preprint]. Tufts Center for the Study of Drug Development. Available at: (Accessed: April 11, 2023).
  4. "Woodcock, J. et al. (2021) “Integrating research into community practice — toward increased diversity in clinical trials,” New England Journal of Medicine, 385(15), pp. 1351–1353. Available at: