Bang V, Mallad A, Kannan S, Bavdekar S B, Gogtay N J, Thatte U M
Department of Clinical Pharmacology, Seth GS Medical College & KEM Hospital, Parel, Mumbai, India.
Department of Pediatrics, TNMC and BYL Nair Hospital, Mumbai, India.
Int J Risk Saf Med. 2014;26(2):61-70. doi: 10.3233/JRS-140613.
Off-label use of drugs is widely prevalent in children mainly due to a limited data generated in children during drug development process. Parents play a critical role in giving consent for their child to participate in clinical trials. Very few studies have assessed the opinion of the parents regarding such use and permitting their child to participate in clinical trials.
In view of lack of information about the awareness among parents regarding both off-label drug use in children as well as about allowing their child to participate in clinical research especially from a developing country, this study was conducted.
Adults accompanying patients in a tertiary care hospital were administered a validated, structured questionnaire following written informed consent. The questionnaire consisted of 18 items broadly divided into 5 themes - parental views on safety and labelled use of drugs in children, awareness of off-label drug use in children, communication from healthcare worker about it, parental views on off-label drug use in children and willingness to allow their child to participate in a clinical trial. Chi-square or Fisher's exact probability test and McNemar's test were used for analysis.
Initially, a majority of the participants felt that the drugs used in children in hospital (89.5%) and prescribed by a family physician (80.3%) were either safe or extremely safe while after the concept of off-label drug use is explained, a significant reduction in the proportion (59.3% in hospital and 59.8% by family physician) of parents felt the same. Only 30% parents were aware of off-label drug use in children. Ninety-three percent of the parents wanted to be informed whenever a doctor prescribes a drug in an off-label manner and a similar percentage felt the off-label drug use would increase the side-effects. Seventy three percent parents felt the off-label drug use is illegal and 57% would ask for change to a labelled drug in case of such prescription in their children. A majority of the parents would allow their child to participate in case of a life-threatening condition (59.8%) or in case of a chronic illness (51.3%) but significantly less when their child is healthy.
The present study has found a low level of awareness regarding the concept of off-label drug use in children amongst the public. Our study also shows that parents expect that the doctor explains the fact to them, although they appear to vest a large amount of trust in the doctor's judgement in doing the best for their sick child. Parents were more willing to allow their child's participation in clinical research if their child was seriously ill than if healthy, indicating the need to educate the society about the need for clinical research so that they could take more informed decisions.
药物的非标签使用在儿童中广泛存在，主要原因是在药物研发过程中针对儿童产生的数据有限。父母在同意其孩子参与临床试验方面起着关键作用。很少有研究评估父母对于此类使用以及允许其孩子参与临床试验的看法。
鉴于缺乏关于父母对儿童非标签药物使用以及允许其孩子参与临床研究的认识的信息，尤其是来自发展中国家的信息，开展了本研究。
在一家三级护理医院，在获得书面知情同意后，对陪同患者的成年人发放一份经过验证的结构化问卷。问卷由18个项目组成，大致分为5个主题——父母对儿童药物安全性和标签使用的看法、对儿童非标签药物使用的认识、医护人员关于此的沟通、父母对儿童非标签药物使用的看法以及允许其孩子参与临床试验的意愿。采用卡方检验或Fisher精确概率检验以及McNemar检验进行分析。
最初，大多数参与者认为医院用于儿童的药物（89.5%）以及家庭医生开的药（80.3%）要么安全要么极其安全，而在解释了非标签药物使用的概念后，认为同样情况的父母比例显著降低（医院为59.3%，家庭医生处为59.8%）。只有30%的父母知晓儿童非标签药物使用情况。93%的父母希望医生以非标签方式开药时能告知他们，并且类似比例的父母认为非标签药物使用会增加副作用。73%的父母认为非标签药物使用是非法的，57%的父母表示如果孩子遇到这种处方情况会要求换成有标签的药物。大多数父母会在孩子面临危及生命的情况（59.8%）或患有慢性病时（51.3%）允许其孩子参与，但孩子健康时允许参与的比例显著降低。
本研究发现公众对儿童非标签药物使用概念认识水平较低。我们的研究还表明，父母期望医生向他们解释这一情况，尽管他们似乎非常信任医生为其患病孩子做出最佳判断的能力。与孩子健康时相比，如果孩子身患重病，父母更愿意允许其参与临床研究，这表明有必要向社会宣传临床研究的必要性，以便他们能够做出更明智的决定。
