Identification of the risks in CAR T-cell therapy clinical trials in China: a Delphi study

Weijia Wu; Yan Huo; Xueying Ding; Yuhong Zhou; Shengying Gu; Yuan Gao

doi:10.1177/1758835920966574

Identification of the risks in CAR T-cell therapy clinical trials in China: a Delphi study

Ther Adv Med Oncol. 2020 Oct 17:12:1758835920966574. doi: 10.1177/1758835920966574. eCollection 2020.

Authors

Weijia Wu¹, Yan Huo², Xueying Ding³, Yuhong Zhou⁴, Shengying Gu⁵, Yuan Gao⁶

Affiliations

¹ Department of Clinical Pharmacy and Pharmaceutical Management, School of Pharmacy, Fudan University, Shanghai, China.
² National Institution of Food and Drug Control, National Medical Products Administration, Beijing, China.
³ Engineering Technology Research Center of Cell Therapy and Clinical Translation, Shanghai Science and Technology Committee.
⁴ Department of Medical Oncology, Zhongshan Hospital, Fudan University, Shanghai, China.
⁵ Clinical Research Center, Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, China.
⁶ Department of Clinical Pharmacy and Pharmaceutical Management, School of Pharmacy, Fudan University, Pudong District, Shanghai, 200433, China.

Abstract

Aims: Within the past few years, there has been tremendous growth in clinical trials of chimeric antigen receptor (CAR) T-cell therapies. Unlike those of many small-molecule pharmaceuticals, CAR T-cell therapy clinical trials are fraught with risks due to the use of live cell products. The aim of this study is to reach a consensus with experts on the most relevant set of risks that practically occur in CAR T-cell therapy clinical trials.

Methods: A Delphi method of consensus development was used to identify the risks in CAR T-cell therapy clinical trials, comprising three survey rounds. The expert panel consisted of principal investigators, clinical research physicians, members of institutional ethics committees, and Good Clinical Practice managers.

Results: Of the 24 experts invited to participate in this Delphi study, 20 participants completed Round 1, Round 2, and Round 3. Finally, consensus (defined as >80% agreement) was achieved for 54 risks relating to CAR T-cell clinical trials. Effective interventions related to these risks are needed to ensure the proper protection of subject health and safety.

Conclusion: The Delphi method was successful in gaining a consensus on risks relevant to CAR T-cell clinical trials in a geographically diverse expert association. It is hoped that this work can benefit future risk-based quality management in clinical trials and can potentially promote the better development of CAR T-cell therapy products.

Keywords: CAR T-cell therapy; Delphi study; clinical trial; risk identification; subject protection.