Kumar Prajna P, Dalal Jeroze, Jadhav Manoj P
Shire, 300 Shire Way, Lexington, MA 02421, USA.
Novartis Healthcare Private Limited, Hyderabad, 500081, India.
Contemp Clin Trials Commun. 2016 Sep 14;4:155-160. doi: 10.1016/j.conctc.2016.09.002. eCollection 2016 Dec 15.
Several approaches to clinical trial monitoring, including the Risk Based Monitoring (RBM) are aimed at the protection of the human subjects (safety), improved data quality, and ultimately, reducing the cost of drug development and operations. There exists minimal evidence globally about the perceptions and the level of confidence among the clinical staff on the merits of RBM. The present study assessed the perception among clinical research staff globally (developed and emerging countries) on the applicability and adaptability of RBM.
An electronic questionnaire survey consisting of twelve items was developed, validated, and then circulated globally via email to three thousand clinical research staff members at various investigational sites. This survey collected information on the use of RBM and factors that relate to clinical trial cost, data quality, subject safety, and the readiness to adopt RBM practices. The survey responses were summarized and analyzed by using the information e.g. responder's age, sex, clinical research role, global location, and experience in clinical research trials.
Responses were received from ten countries, six emerging and four developed. Of the 3000 surveys sent to emerging (1,000) and developed (2,000) countries, a total response of 595 (261 vs 334) participants was received, respectively. The emerging versus developed group had 100 vs 137 participants with complete responses (CR); 34 vs 35 participants with partial responses (PR); and 127 vs 162 participants were disqualified with no exposure (NE) responses. About 67% of the overall responders were investigators, followed by 23%, 10% coordinator and other staff respectively. There was not significant difference in feedback between the researchers in developing versus emerging countries ( = 0.20) with regards to their perception of RBM reducing the overall cost of conducting a clinical research. Responders from emerging countries had a more favorable response than in the developed countries. Similarly, when asked if RBM will be more effective in addressing data quality ( = 0.006), patient safety ( = 0.05) and findings fraud/fabrication ( = 0.01), researchers from emerging countries indicated more confidence than researchers from developed countries. There was also a significant difference in the readiness to adopt RBM between responders of emerging versus developed markets ( < 0.0001).
This unique study performed across ten emerging and developed countries strongly supported the need for systematic global training, education, and implementation of RBM regulatory guidance, with an aim for better safety of subjects and improved quality of clinical trial data. Furthermore, studies with larger sample sizes are recommended to provide an evidence-based approach.
几种临床试验监测方法，包括基于风险的监测（RBM），旨在保护受试者（安全）、提高数据质量，并最终降低药物开发和运营成本。全球范围内，关于临床工作人员对RBM优点的看法和信心水平的证据极少。本研究评估了全球（发达国家和新兴国家）临床研究人员对RBM适用性和适应性的看法。
设计了一份包含12个项目的电子问卷调查表，进行了验证，然后通过电子邮件在全球范围内分发给各个研究站点的3000名临床研究人员。该调查收集了有关RBM使用情况以及与临床试验成本、数据质量、受试者安全和采用RBM实践意愿相关的因素的信息。通过使用诸如应答者的年龄、性别、临床研究角色、全球位置以及临床研究试验经验等信息，对调查回复进行了总结和分析。
收到了来自10个国家的回复，其中6个是新兴国家，4个是发达国家。在发送给新兴国家（1000份）和发达国家（2000份）的3000份调查问卷中，分别收到了595名参与者的回复（新兴国家261名，发达国家334名）。新兴国家组与发达国家组分别有100名和137名参与者给出了完整回复（CR）；34名和35名参与者给出了部分回复（PR）；127名和162名参与者因无有效回复（NE）而被取消资格。总体回复者中约67%是研究者，其次分别是23%的协调员和10%的其他工作人员。在发展中国家与新兴国家的研究人员对RBM降低临床研究总体成本的看法方面，反馈没有显著差异（P = 0.20）。新兴国家的回复者比发达国家的回复者反应更积极。同样，当被问及RBM在解决数据质量（P = 0.006）、患者安全（P = 0.05）和发现欺诈/伪造行为（P = 0.01）方面是否会更有效时，新兴国家的研究人员比发达国家的研究人员表现出更多信心。新兴市场与发达市场的回复者在采用RBM的意愿方面也存在显著差异（P < 0.0001）。
这项在10个新兴国家和发达国家开展的独特研究有力地支持了对RBM监管指南进行系统的全球培训、教育和实施的必要性，目的是提高受试者安全性并改善临床试验数据质量。此外，建议进行更大样本量的研究以提供基于证据的方法。
