What to consider when considering 'off-label' drug prescriptions for children
2014年2月，美国儿科学会（AAP）发布a statement on the off-label use of drugs in pediatric patients. As the AAP pointed out, changes in the federal regulations and incentives for pediatric drug research over the last decade have led the Food and Drug Administration (FDA) and pharmaceutical companies to add pediatric guidance to the prescribing labels for more than 500 medications.
然而,, the topic of off-label use remains highly important in pediatrics and public health, because many medications prescribed for children in the U.S. today have never been studied in children.
"While the rate can vary greatly depending on the specialty and environment, in general, less than 50 percent of medication labels have any guidance for providers on a drug's use in children," saysFlorence Bourgeois, MD, MPH, an emergency physician at Boston Children's Hospital who studies pediatric drug development practices. "At an academic medical center, that rate might even be as high as 60 to 80 percent for the medications used among hospitalized patients."
However, lack of child-specific prescribing guidance does not mean that the FDA prohibits a given drug's use in children. Nor does it mean that the drug is experimental or requires special consent from the patient's family or guardian.
Rather, it means that there are no data or"substantial evidence" from "adequate and well-controlled investigations"upon which the FDA can make a determination either way regarding that drug's safety and effectiveness in children.
"'Off-label' use has negative connotations, but a lack of pediatric guidance is not the same as if a drug were to have a pediatric contraindication," Bourgeois explains. "Rather, it points to a paucity in the evidence about how a given drug should be used."
超越标签以寻求处方指南
在决定是否开出没有小儿标签的药物时，AAP和资产阶级都同意提供者必须依靠其最佳判断力和最佳证据。正如双方指出的那样，有几个来源提供者可以求助于给定药物的儿科使用和安全，例如*：：
- peer-reviewed literature
- consensus statements
- AAP政策和实践指南
- information from organizations such as the Cochrane Collaboration and UpToDate
- the FDA's MedWatch program
"Providers should also rely on their own and their colleagues' expertise," Bourgeois adds, "as well as on any expert opinions and trial data they can access.
她继续说：“缺乏正式证据并不意味着没有证据可以指导处方。”“但是，发现，编译和评估证据可能具有挑战性，并为提供者而采取额外的努力。”
提供者可以在帮助给定药物的小儿证据基础方面发挥重要作用，尤其是对于新生儿，或对于几乎没有可用数据的罕见疾病的儿童。Bourgeois说：“ AAP的声明鼓励提供者成为该过程的一部分。”“如果您经常开出标签外药物，请考虑发布或展示您的经验。”
Cautions and concerns
She notes that providers are sometimes reluctant to prescribe medications to children off-label for fear of causing harm. And while that could prevent children from receiving a potentially beneficial treatment, some hesitation is understandable. For instance, there can be greater medical and legal risks when writing an off-label prescription. Also, some insurance carriers will not cover the cost of drugs prescribed off-label or will discourage patients from filling the prescription.
她说：“最终，每个提供商都对自己的决定负责，如果您开了毒品并且有损害，则可能会增加法律责任的风险。”
She adds that providers should consider providing more information to patients and families than they normally would with an approved drug and engage them more closely in the decision.
"Inform the family about the evidence behind your recommendation to prescribe the drug, and be sure to document that evidence before writing the prescription," she advises. "And above all, use your best judgment. Only prescribe a drug off-label if you think there is sufficient evidence that the treatment will benefit the child."
*This list is provided for informational purposes only, and should not be construed as an endorsement by Boston Children's Hospital of any of the included sources.
