Examining Off-label Medicine Use in Paediatric Healthcare

Our life science experts Dr Juli Mansnérus, Charlotta Bonsdorff and Petro Vornanen seized the opportunity to contribute to the intricate field of medical law as they provided their expertise to the Office of the Ombudsman for Children in the form of an article regarding off-label medicine use in children.

A summary of the article is provided below. For the whole publication in Finnish, please refer here.

Off-label use of medicines refers to the practice of prescribing medication to a patient outside the scope of the medicine’s approved indications. As an intrinsic part of their profession, medical professionals are subject to certain ethical requirements. These ethical requirements may obligate them to obtain informed consent from a patient and to base their treatment endeavours upon generally accepted good practices and experience. In light of this, off-label use may be an ethics-stretching resort in the realm of healthcare. The need for it may be driven by the scarcity or non-existence of other treatment options. On one hand, it can be a lifeline for people with severe or rare conditions for which no other treatment exists. On the other hand, the lack of standardized dosing and safety information raises perplexing questions about the risks and benefits of off-label drug use.

Nearly half of the medications administered to paediatric patients deviate from the information provided in the official prescribing information, including indications, dosage, administration method, and/or target group. The younger the child, the less researched and authorised information is available regarding the efficacy and safety of the drug. Simultaneously, a significantly lower number of clinical trials are conducted on children compared to adults, as there is a desire to avoid exposing children to potential risks associated with research.

When prescribing medical care off-label, medical professionals are faced with the multifaceted question of fulfilling the requirements of their professional ethics while still providing the most effective medical care for their patients. Fulfilment of the ethical requirements imposed upon medical professionals requires an intricate analysis of the patient, their level of development and their ability to comprehend their circumstances and the treatment options available to them.

The ethical requirements and off-label medicine prescriptions for children:

Categorically speaking, children include a wide range of people with significantly different levels of maturity. Particularly young children in their early stages of development must rely on their parents while other, more mature children may be able to persist in the modern world wholly on their own. Both extremes are, however, considered as children. This mismatch between children is approached through weighing in their levels of development.

It is implicit that children perceive their surroundings and information in different manners, depending on their age and maturity. A near-adulthood child may have perception abilities equivalent to an adult, whereas a child approaching their teenage years may require simpler terminology, expression and forms of communication. Thus, it is essential for a paediatrician to commit to a comprehensive analysis of the child’s level of development, and to adjust their approach to informing the child patient accordingly. In the conduct of care for children in the early stages of their development, the bar is raised for meeting the ethical requirements regarding the provision of information in a comprehensible manner. Also, the Finnish Parliament’s ombudsman has outlined that the attending physician is obligated to inform the paediatric patient and/or their guardian about the risks and uncertainties associated with off-label drug use. This is particularly significant for recognising and responding to adverse drug effects, as careful monitoring of the benefits and drawbacks of medication is crucial. If the desired response is not achieved or if the child suffers significant adverse effects, discontinuation of the medication should be promptly considered.

Balancing the ethical requirement of providing information to the patient in a comprehensible manner with the capability of the child patient to understand the provided information is further complicated when the medicine is prescribed off-label. The physician must formulate their reasoning and information so that the patient comprehends why the medicine is prescribed off-label. They must also meet their ethical requirements of only providing care based on generally good practice and their experience and in accordance with their training. This subjects the provision of information to even more scrutiny as justifying the use of medicine for something it has not been approved for requires further informing the patient of such use. Children are in a special position also in this case, as the paediatrician must adjust their level of communication according to the development of the child so that the child patient acquires enough information to provide an informed consent to the off-label use of medicine.

All in all, the physician is required to communicate the therapeutic reasons that led to the decision to use the drug off-label. Simultaneously, it is essential to evaluate the child’s right to access treatment using off-label medications. The patient’s right to self-determination does not automatically guarantee the right to receive off-label drug treatment. The physician, guided by their expertise, may limit the patient’s access to off-label treatment in situations where standard treatment practices have proven ineffective and where such treatment would not be in the child’s best interest. However, the paediatric patient has the right to receive high-quality healthcare, and in situations where conventional treatments are insufficient, the possibility of receiving off-label drug treatment can be considered a right of the patient.



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