Designing And Delivering Cell And Gene Therapy Clinical Trials: A Strategic Approach To CAGT Study Conduct And Operations

By Erin Finot, MS, MBA, Vice President, Immuno-Oncology and CAGT; and Diego Correa, MD, PhD, Vice President and Global Head, Cell and Gene Therapy Center of Excellence

Cell and gene therapies (CAGTs) represent a groundbreaking approach in medicine, offering new treatments for genetic and rare disorders. As over 2,000 active CAGT trials progress globally, these therapies underscore the need for adaptive regulatory frameworks and robust operational strategies. Regulators, including the FDA and international bodies like the International Council for Harmonization (ICH), are developing guidelines that address unique CAGT requirements, such as extended safety monitoring and environmental impact considerations. Initiatives like the FDA’s Diversity Action Plan (DAP) further encourage diverse patient representation in trials to improve treatment inclusivity.

CAGT trials face specific operational complexities due to their innovative nature, requiring specialized trial designs and strategic patient and site identification. Adaptive and decentralized models help retain patients and provide broader access across diverse populations, while early logistical planning ensures trials remain scalable and compliant with regulatory demands.

IQVIA Biotech supports CAGT trial execution by offering integrated regulatory, operational, and data-driven solutions. These include specialized lab services, real-time data insights, and cross-functional coordination from preclinical through to commercial stages. Their expertise in managing long-term studies, patient retention, and complex trial execution is crucial for overcoming the unique challenges of CAGT.

By embracing tailored regulatory pathways, streamlined patient engagement strategies, and strategic operational planning, CAGT trials can reach the patients who need them most, advancing treatment options and shaping the future of healthcare innovation.