Table1_Benefit assessment of extended dosing in cancer patients after their withdrawal from clinical trials.docx

Background: Clinical trials have been widely recognized as an effective treatment approach by physicians and cancer patients alike. Physicians’ evaluations suggest that many patients are likely to continue experiencing benefits from extended dosing of investigational new drugs even after withdrawing from clinical trials.
Objective: Given the uncertainty surrounding the efficacy and safety of investigational new drugs, it is essential to continually assess the benefits of extended dosing for patients.
Methods: The trial group for this study comprised patients who requested extended dosing after withdrawing from clinical trials at Hunan Cancer Hospital between 2016 and 2020. The control group consisted of patients who received conventional treatment and were enrolled in a 1:1 ratio. Follow-up assessments were conducted every 3 months for both groups, and included monitoring of patients’ health status, survival time, disease control or remission, treatment modalities received, and medical costs.
Results: A total of twenty-three patient pairs were successfully matched for this study. The Ethics Committee approved extended dosing for all patients in the trial group, with an average gap period of 16.48 days between their withdrawal from clinical trials and continuous access to the investigational drugs. The median overall survival for patients after withdrawal from clinical trials was 17.3 months in the extended dosing group and 12.9 months in the control group, with no significant difference observed between the two groups (p > 0.250). The median total cost of treatment after the previous clinical trial was 38,006.76 RMB, of which the median cost of therapeutic drugs for conventional treatment was 15,720 RMB, while extended dosing was provided free of charge.
Conclusion: Extended dosing can indeed provide benefits, including survival benefits and economic benefits, to cancer patients after their withdrawal from clinical trials and will clinically present an additional treatment option for patients.

背景：临床试验已被临床医师与癌症患者广泛认可为有效的治疗手段。临床医师的评估显示，即便在退出临床试验后，诸多患者仍可从研究性新药（investigational new drugs）的延长给药方案中持续获益。
研究目的：鉴于研究性新药的疗效与安全性尚存不确定性，持续评估延长给药方案对患者的获益情况至关重要。
研究方法：本研究的试验组纳入2016至2020年间于湖南省肿瘤医院退出临床试验后申请延长给药的患者；对照组纳入接受常规治疗的患者，两组按1:1比例匹配。两组均每3个月进行一次随访评估，评估内容包括患者健康状况、生存时间、疾病控制或缓解情况、接受的治疗方案以及医疗费用。
研究结果：本研究共成功匹配23对患者。试验组所有患者的延长给药方案均经伦理委员会批准，患者从退出临床试验到开始持续使用研究性新药的平均间隔时长为16.48天。退出临床试验后，延长给药组患者的中位总生存期为17.3个月，对照组为12.9个月，两组间差异无统计学意义（p > 0.250）。既往临床试验结束后，患者的治疗总费用中位数为38006.76元人民币，其中常规治疗的治疗性药物费用中位数为15720元人民币，而延长给药方案则免费提供。
研究结论：延长给药方案确实可为退出临床试验后的癌症患者带来包括生存获益与经济获益在内的多重益处，同时可为临床患者提供额外的治疗选择。