Analysis of cellular and gene therapy product reviews in the United States

Abstract

The emergence of cellular and gene therapy (CGT) products has profoundly transformed healthcare by addressing previously unmet medical needs. However, developing these innovative therapies presents complex regulatory challenges that require thorough examination. This study aimed to identify strategies to mitigate potential delays or rejections in the CGT approval process. By analyzing review documents from the United States Food and Drug Administration, we found that quality concerns were the primary focus of Complete Response letters and postmarketing commitments, while safety concerns predominantly shaped postmarketing requirements, reflecting persistent uncertainties around CGTs. The unique characteristics of CGTs were also evident in their individualized clinical trial designs. Although the regulatory landscape is intricate, the increasing diversity of CGTs and accumulated experience have clarified key product-specific challenges. To facilitate approvals, it is crucial for applicants to address these deficiencies early, while we recommend that regulatory authorities re-evaluate the scope of utilizing postmarketing requirements. Enhanced collaboration among academia, industry, and regulatory authorities is essential to identify balanced, effective strategies, while continuous information gathering and monitoring are vital to ensure the safe, long-term administration of approved CGTs.