“Analysis of cellular and gene therapy product reviews in the United States” by Cheng-Fang Weng, Jhe-Yuan Dong, Shiuan-Fei Lin, Ai-Lei Jiang, Yu-Li Cheng and Lin-Chau Chang, was published in the journal Regulatory Toxicology and Pharmacology on June 16, 2025.
The article 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 between April 2010 and June 2023, the study 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.
We gratefully acknowledge Project LEAP (Lead, Excel, Aspire, Pharmacy)NTU for its support of the Platform for Research and Inspiration in Regulatory Science.
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感謝「臺大藥學發展永續基金計畫」對於國際藥政法規科學研究平台之支持，翁晟芳、董哲源、林炫妃、姜愛蕾、鄭鈺立、張琳巧，以 “Analysis of cellular and gene therapy product reviews in the United States” 為題之文章，已於2025年6月16日發表於 Regulatory Toxicology and Pharmacology 期刊。
本研究回顧美國食品藥物管理局 (FDA) 生物製劑評估與研究中心 (CBER) 自2010年4月至2023年6月間核准之細胞與基因治療 (cellular and gene therapy, CGT) 產品審查資料，探討如何減少CGT產品在審查過程中延遲或駁回的風險。研究結果顯示，品質疑慮為Complete Response letters顯示之首輪審查後的缺失與上市後承諾（postmarketing commitments）的主要議題；而安全性疑慮則是上市後要求（postmarketing requirements）關注的焦點，反映CGT產品仍存在不確定性。此外，CGT產品亦展現出高度個別化的臨床試驗設計。雖然法規環境仍具挑戰性，CGT產品類型多元性與審查經驗的累積已逐步釐清特定產品面臨的挑戰。研究結果建議，申請商應及早處理相關缺失，而法規機關亦應重新檢視上市後要求之範圍，並促進學術界、產業界與主管機關間之合作，以尋求兼顧風險與效益之平衡策略。同時，持續性的資訊蒐集與監測亦至關重要，以確保CGT產品在核准後之安全與長期使用。
請參考原始文獻。
