Clinical And Regulatory Considerations For Gene Therapy Products

By Ash Kaur, MD, RAC, Director of Regulatory Affairs and Product Development, Cardinal Health Regulatory Sciences

After decades of slow and steady progress, gene therapies have experienced important recent breakthroughs, demonstrating the potential to slow disease progression, improve outcomes, and, in some cases, potentially cure an array of illnesses. More than 25 cell and gene therapy products have been approved by the FDA so far, marking exciting progress for sponsors and patients alike. However, developers of these cutting-edge therapies continue to encounter existing and new challenges as they navigate complex clinical and regulatory hurdles.

While most traditional products follow now tried-and-true processes for critical steps in the development process, advanced therapies do not have standard approaches due largely to each product’s unique, patient-specific nature and the potentially limited patient populations for each therapy. Regulatory standards are also continuously changing as the evidence necessary to prove the safety and efficacy of each of these new therapies changes to reflect the evolving science behind these treatments.

Ash Kaur, MD, RAC, Director of Regulatory Affairs and Product Development for Cardinal Health Regulatory Sciences, shares clinical and regulatory considerations to best navigate the expedited programs made available by the FDA.