May, 2026
Why execution is now the defining challenge in cell and gene therapy trials
A patient undergoes cardiac surgery on Monday. By Tuesday, they develop a serious complication and are enrolled in a clinical trial.
Blood is collected and shipped to a manufacturing site in another country, where it’s transformed into a personalized therapy before being sent back to the hospital.
From the moment that therapy is ready, there’s only a narrow window — just a few hours — to deliver and administer it.
Every step must align. Every delay carries risk.
This is the reality of cell and gene therapy clinical development. And it’s reshaping how clinical trials are designed and delivered in practice.
Innovation has moved ahead. Execution is catching up
Cell and gene therapies (CGTs) represent one of the most significant advances in modern medicine. Many are designed for patients with limited or no alternative treatment options, often targeting rare or complex conditions.
At the same time, the pace of innovation is accelerating. Advances in analytics, manufacturing, and scientific understanding are enabling faster identification and development of new therapies.
According to insights shared at the IQVIA summit, a leading industry forum for clinical development and innovation, emerging biopharma companies now drive the majority of innovation, while trial volume and competition continue to increase.
As innovation accelerates, the pressure on clinical development systems is increasing.
In response, the way clinical trials are executed is evolving — raising questions about whether traditional approaches are still fit for purpose.
Cristina Salado Manzano, Director of Advanced Therapies, Innovation and Patient Support Programs at Alcura, says: “We still rely on traditional clinical development models — they’re the foundation for how trials are structured, regulated, and delivered. What’s changed is the level of precision required to operate them. The model itself isn’t wrong. But you need to be more prepared and operate in a different way.”
Why traditional assumptions no longer hold
Traditional clinical development models are built around stability. Products can be manufactured in advance, stored, and distributed when needed. Timelines can flex. Supply chains are designed to absorb delay. Cell and gene therapies introduce a different operating environment.
“In some cases, regulatory processes are designed around timelines that simply don’t align with the reality of CGT delivery,” says Cristina. “You may have weeks to approve something — but only hours to deliver a therapy.”
Many therapies are:
- Patient-specific
- Time-sensitive
- Highly dependent on temperature control
- Subject to complex regulatory pathways across multiple jurisdictions.
Cristina says: “In some cases, the patient triggers the supply chain. Collection, manufacturing, analytics and delivery all depend on that individual pathway. This removes flexibility. If a shipment is delayed, the impact isn’t operational inconvenience. It can affect product viability, patient safety, and trial outcomes. In this context, logistics doesn’t simply support the clinical trial — it can determine whether the trial can proceed.”
What this looks like in practice
The complexity of CGT trials isn’t theoretical. It’s operational. In autologous therapies, where a patient’s own cells are used, each treatment pathway is unique.
A single trial may involve:
- Collection of patient material in one country
- Manufacturing in another
- Cross-border regulatory approvals
- Return of a viable therapy within a defined time window at a controlled temperature, usually below -150ºC.
Even within the same program, variability can be significant. Manufacturing timelines can differ from patient to patient. Supply routes may need to change based on geography, infrastructure, regulatory requirements or wider geopolitical factors.
“I remember one case where a clinical trial design had to be adjusted because the therapy couldn’t physically reach certain regions within its viable time window,” says Cristina.
Where complexity is underestimated
Many CGT programs originate in academic environments or emerging biopharma organizations. The initial focus is rightly on scientific development and funding. Operational complexity is often considered later.
This can lead to challenges in:
- Regulatory readiness
- Supply chain design
- Infrastructure availability across regions
- Coordination between stakeholders.
Cristina highlights that these challenges often emerge late in the process: “We’ve seen situations where a program was ready to begin — but couldn’t proceed because it hadn’t met required manufacturing compliance standards. Everything else was in place, but without the right regulatory and quality foundations, the trial simply couldn’t start.”
From planning to real-time coordination
The shift in CGT clinical development is not about replacing the model. It’s about operating it differently.
Planning must begin earlier and involve more functions. Clinical, manufacturers, regulatory, supply chain, and operational teams must work together from the outset to design a trial that is both scientifically and operationally viable while compliant with regulatory standards.
Risk assessment becomes crucial rather than optional. Teams must anticipate:
- Delays in transport
- Temperature deviations
- Regulatory timelines
- Variability in manufacturing.
“This requires a higher level of coordination across the entire ecosystem,” says Cristina. “You’re not just running a clinical protocol. You’re coordinating a complex system across multiple stakeholders.”
Why partnerships are critical
No single organization can deliver every element required for CGT clinical development.
Successful trials depend on coordination between:
- Developers
- Manufacturers
- Logistics providers
- Regulatory authorities
- Hospitals and clinical sites
- Patients and patient advocates.
Each plays a distinct role, and each must align in real time. Incorporating the patient voice helps ensure trial design and delivery are shaped around real-world needs, experiences, and outcomes.
“Specialized partners bring expertise in areas such as regulatory management, temperature-controlled logistics, and supply chain design,” says Cristina. “Their role isn’t to replace internal capability, but to extend it. This shift reflects a broader trend across the industry: moving from linear execution to integrated delivery models.”
What this means for clinical development leaders
For leaders across R&D, procurement, clinical operations, and supply chain, the implications are clear.
Scientific innovation alone is no longer enough. Execution must be designed with the same level of rigour as the therapy itself.
This includes:
- Engaging operational and supply chain expertise early
- Designing trials around real-world constraints
- Building partnerships that support end-to-end delivery
- Investing in coordination and communication across teams
As CGT pipelines continue to grow, these considerations will become increasingly important.
Looking ahead
“The clinical development model isn’t being replaced", says Cristina. “It’s evolving. New approaches, including in vivo therapies and more integrated supply models, and digital orchestration platforms powered by AI, may reduce some of today’s constraints over time. But for now, success depends on how effectively organizations can operate within a complex, fast-moving environment. This isn’t a temporary shift. It reflects the direction of travel for clinical development as a whole.”
She adds: “In cell and gene therapy, innovation doesn’t end in the lab. It must be delivered — precisely, reliably, and at speed. Because in these trials, execution isn’t a supporting function. It’s the difference between a therapy existing — and a patient receiving it.”
