Why are therapies involving modified cells and tissues difficult to evaluate for safety and effectiveness?

In the field of translational medicine, therapies involving modified cells and tissues are difficult to evaluate for safety and effectiveness primarily due to their increased complexity compared to traditional pharmaceutical agents.1

These advanced therapeutic products often operate through distinct, highly specific mechanisms that may not be fully understood by researchers. Because these mechanisms are not always clear, it becomes challenging to establish standardized validation strategies to ensure the treatments are both safe and effective for patients. Furthermore, as biotechnology continues to refine these strategies, the resulting increase in the sophistication of these agents complicates the regulatory and clinical testing processes, which are already burdened by high costs and the need to protect public safety.1