In short
- Patients with chronic, progressive diseases face a multitude of health and QoL challenges. Their treatment options are limited, particularly when standard therapies fail to address underlying disease mechanisms
- Emerging advanced therapies (for example, gene therapies, cell therapies, antibody-drug conjugates) offer the potential to modify disease progression, but introduce significant operational and logistical complexity
- Careful site selection, patient identification, and infrastructure readiness are critical to ensuring successful execution of late-phase trials in the chronic disease area
The challenge
A biopharma sponsor initiated a late-phase, registrational study of an innovative advanced, personalized therapy in a large heterogenous patient population with a chronic condition associated with progressive disease burden.
Despite strong scientific rationale, regulatory alignment, and positive press coverage, the study encountered critical issues:
- Significantly slower-than-expected enrollment, with only a fraction of planned patients recruited over an extended period
- Initial site selection misalignment, where several sites lacked access to the appropriate patient population, or didn’t have the capabilities required for study execution
- High operational complexity, including specialized handling and coordination requirements
- Inconsistent site performance, leading to variability in execution, and delays across regions
As timelines slipped, and risks to the overall program increased, the sponsor initiated a trial rescue and transitioned to PSI to stabilize and recover the study.
The approach
We developed and implemented a comprehensive rescue strategy. Based on our prior rescue experience with complex trials and advanced therapies, we focused on rapid assessment and targeted interventions.
Detailed portfolio-wide study assessment
Existing sites, processes, and performance metrics were reviewed to identify the root causes of underperformance.
Strategic site optimization
Underperforming sites were deprioritized or replaced, while high-potential sites with better patient access and specialized infrastructure for the advanced therapy were activated.
Focused enrollment acceleration
Patient identification strategies were refined, including improving referral pathways, and increasing collaboration with KOLs and PIs (including targeting new KOLs and PIs, those within our network that we knew would be perfect fits for this highly-technical trial).
Enhanced training and support
Sites received tailored guidance to manage the advanced therapy’s technical and logistical requirements more effectively.
Operational streamlining
Communication pathways and study processes were optimized to reduce delays and improve coordination across stakeholders.
Renewed regulatory engagement
Ongoing dialogue with regulatory authorities ensured continued alignment, and supported the study’s expedited development pathway.
The outcome
By tackling operational risks, enrollment accelerated significantly, reversing the earlier delays and site performance stabilized, with greater consistency across regions. The study was successfully brought back on track, with a clearer path toward completion and regulatory submission.
PSI take time to understand the challenges and formulate strategies to deliver the work. The way they brief, train, and support the sites is excellent. I can rely on their energy and commitment.
Key lessons
Early warning signs should trigger rapid intervention. Delays in enrollment, and inconsistent site performance can quickly compound and seriously jeopardize the success of your trial if not timely addressed. Initiating a rescue is a decisive, but necessary, action.
Advanced therapies demand the right sites, not just more sites. Capability and patient access must be prioritized relatively equally, and you should not be afraid to pivot to sites better-placed to deliver the trial on time, rather than those where a historical relationship may exist. Activating the right KOLs and PIs is crucial for trials – and also underpins successful rescues.
Even well-designed, late-phase trials can encounter significant operational challenges, especially when evaluating complex therapies in chronic conditions. A structured, proactive rescue strategy can reverse underperformance, restore momentum, and ensure that promising treatments remain on the path to patients. Sponsors return time and again to PSI for our on-time enrollment and delivery of the most important (and difficult) clinical trials across the industry.
Contact PSI to discuss your cell therapy trial needs
Abbreviations
CRO, Contract Research Organization;
QoL, Quality of Life;
, Key Opinion Leaders;
Principal Investigators.
