Pharmacovigilance and Regulatory Affairs

Pharmacovigilance in Gene and Cell Therapies

Gene and cell therapies represent a revolutionary class of treatments with curative potential, yet they come with complex and evolving safety profiles. These products often involve novel vectors, long-term biological activity, and individualized treatment regimens, making their risk assessment fundamentally different from conventional pharmaceuticals. Adverse events may be delayed, unpredictable, and patient-specific, requiring lifelong follow-up and innovative pharmacovigilance strategies. Pharmacovigilance systems must accommodate unique product attributes, such as persistence of genetic material, immune system activation, and off-target effects. Specialized registries, digital tracking tools, and long-term cohort studies are essential to monitor patient outcomes over extended periods. Regulatory expectations include comprehensive Risk Management Plans (RMPs) and proactive signal detection mechanisms, tailored to address the inherent uncertainties in these therapies. The integration of clinical insights, genomics, and patient-reported data enhances the ability to identify emerging risks. Collaboration among manufacturers, clinicians, and regulatory bodies ensures that the safety of gene and cell therapies is closely monitored while supporting innovation. As these therapies become more common, their pharmacovigilance frameworks must remain adaptive and scientifically rigorous.