人類細胞治療製劑臨床試驗GTP訪查現況調查   全文下載

The Status of GTP Inspections on Clinical Trials of Human Cell Therapy Products

為確保人類細胞治療製劑臨床試驗之安全與效用，並保障受試者之權益，食品藥物管理署對人類細胞治療製劑臨床試驗計畫，依人體細胞組織優良操作規範（Good Tissue Practice, GTP），事前訪查研究用細胞治療製劑製造相關場所，確保捐贈者之細胞組織經過適當的傳染病病原篩檢，在製造過程中未受污染，且不致因製造不當而影響研究用細胞治療製劑效用與完整性。本研究調查109年度辦理之人類細胞治療製劑臨床試驗案GTP訪查現況，並與歷年平均值比較。結果顯示臺灣之人類細胞治療製劑臨床試驗，已逐步由醫療機構主導之學術研究，朝向由藥商主導以查驗登記為目的之方向發展，且臨床試驗之II期與III期案件數成長，亦顯示細胞治療製劑上市具樂觀前景。本研究並分析GTP訪查觀察事項所載之缺失態樣，其中「作業程序」相關缺失最常見，而「收受與配送」、「品質計畫之建立與維持」及「紀錄」相關缺失比例，較歷年平均增加，「物料與試劑」、「設備」及「環境管制與監控」相關缺失比例則有下降的趨勢，顯示作業程序之完善，還有收受、配送及紀錄與品質計畫維持等實務運作的良好管控，是持續確保GTP符合性的關鍵。

To ensure the safety and efficacy of the clinical trial on human cell therapy products, and to protect the rights of the subjects in the clinical trial, Taiwan Food Drug Administration (TFDA) has conducted Good Tissue Practice (GTP) inspections on facilities which manufacture the human cell therapy products for clinical trials. The inspection on manufacturing facilities is carried out, in advance of the execution of clinical trial, to make sure the donor's cell tissue will be screened for communicable diseases and the manufacturing is without contamination risk, thus, to preserve the quality and integrity of the product. This study surveyed the GTP inspections on manufacturing facilities of human cell therapy products for clinical trial conduced in 2020, and compared to previous years. The results have shown that the clinical trial of human cell therapy products have shifted from medical institution initiated scientific research to drug maker led application for registration purpose. In addition, the application number of phase II and III clinical trial has increased, and this reflects the optimistic future of the human cell therapy products. Deficiencies analysis through GTP inspection indicated that ''procedures'' related issues are the most commonly noticed deficiencies, whereas the related deficiency ratios of ''receipt and distribution,'' ''establishment and maintenance of a quality program,'' and ''records'' have increased as compared with data from previous years. On the other hand, the related deficiencies on ''supplies and reagents,'' ''equipment,'' and ''environmental control and monitoring'' have declined. These results indicate that the refined procedures, as well as the well-controlled operating practices such as receipt, distribution, records and maintenance of quality program, are the keys to keep facilities on the right tract of GTP compliance.