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Off-Label Drug Use in Pediatrics

Presently, there is extensive use of off-label drugs as well non-approved or even extemporaneously prepared drugs in both outpatient and inpatient care settings.  Reports indicate that nearly all pediatric patients are at risk of having at least one of these drugs administered to them. It is important to take extra caution when using off-label drugs as the clinical consequences can be very grave. This paper discusses the use of these drugs and describes strategies for increasing safety in the same regard.

The use of off-label drugs has generated debate and different opinions are fronted on the issue. Indeed, the standards for application as well as the circumstances under which the use of this drugs occurs vary from one jurisdiction to another. Regulation of these standards is done by relevant medical bodies as well as legislation, although the practice is illegal in some jurisdictions. Prescribing off-label drugs in pediatrics, as in other care settings, is justifiable under some circumstances but convincing clinical proof that such use will yield the desired patient outcome must be present (Golocorbin-Kon et al., 2015).  As such, circumstances for prescribing these drugs include when the doctor is convinced beyond reasonable doubt that, as per professional judgment, such prescription is “the safest and most effective therapeutic option for the patient” (Golocorbin-Kon et al., 2015, p.36). For example, there could be a drug undergoing the process of approval and legal registration and as such is yet to be officially released to the market (but it has undergone sufficient clinical trials and the results are already known by the scientific community); a doctor can prescribe such a drug if he thinks it is the best and most effective therapeutic option. Another example in the current conversation is that of a doctor preparing a suspension from, say tablets or capsules, and administering it to the patient; that suspension can be considered an off-label drug. Such a situation can be considered especially when the patient is unable to take the drug in its other form.

Considering the increased use of off-label drugs, some strategies are merited that would make the practice safer. One such strategy is for prescribing authorities to ensure they follow and strictly adhere to professional guidelines and standards. In other ones, prescribing must be informed by scientific evidence, perhaps except in emergency cases where there is no other alternative. Second, prescriptions must guarantee respect of the rights and needs for the client. Thus, this strategy would involve obtaining an informed consent prior to prescription. Such informed consent would, of course, follow a discussion with the patient (in this case the patient’s parents or guardians) whereby they would be informed that the drug to be prescribed has not been registered for the use it is going to be used for, or perhaps the dose being prescribed is not approved for the patient weight or age (Kimland & Odlink, 2012). Such a discussion would also entail explaining to the parent/guardian the off-label drug’s benefits, risks, and any adverse reactions associated with it. Discussing available alternatives would also be appropriate.

Another strategy for ensuring safer use of off-label drugs is the more obvious one of conducting more clinical trials in children to increase knowledge on contextual pharmacology. This would also be accompanied by increased post-marketing surveillance to generate data and as such remain more knowledgeable about drug safety in the pediatric setting. Bavdekar and Gogtay (2005) suggest the provision of updated information about off-label drugs, for instance through a newsletter to the medical fraternity to ensure they are kept abreast of various aspects of off-label indications.

To conclude, it is imperative to mention a few off-label drugs that require extra care and attention.  The need for such care and attention is elevated by the complexity and uncertainty surrounding age-related dosing guidelines as well as the confusion surrounding adverse effect profiles as far as age variation is concerned. One example of an off-label drug that calls for extra attention and care is paracetamol that is given to premature infants; complications are likely to arise because of the infants weight and age.  Adrenaline administered via inhalation as opposed to intravenously also calls for extra care, just like codeine and morphine, drugs whose pediatric information is missing.