Prescribing Unlicensed Medication to Children
What are unlicensed medications?
The GMC’s guidance on good practice in prescribing and managing medicines and devices at paragraph 103(1) states that ‘the term ‘unlicensed medicine’ is used to describe medicines, which are used outside the terms of their UK licence or that have no licence for use in the UK. Unlicensed medicines are commonly used in some areas of medicine, such as paediatrics, psychiatry, and palliative care’.
In the UK, most medications have a licence, known as their ‘Marketing Authorisation’, which is provided by the Medicines and Healthcare products Regulatory Agency (‘MHRA’), that is the UK governmental Licencing Authority. This licence will confirm which conditions the medicine can be used for; including the age range of patients in which it can be used, the appropriate dose, and how the medicine should be administered (for example, orally, or intravenously).
In order for the Licensing Authority to approve an application for a particular medicine, the drug company must have conducted clinical trials to demonstrate that the medicine is effective and safe. All clinical trials are regulated in the UK by the MHRA and must be approved by an independent research ethics committee. However, if children have not been included in the trial, the licence may not include use for children. For similar reasons, it may also not include an indication for use in a particular condition.
This situation is not uncommon for drugs used in paediatric practice as sufficient trials in this population may not have been conducted and some conditions may be rare in children. In 2007, UK and European Law introduced a requirement for pharmaceutical companies to undertake studies in children as part of the development plan for new medicines, and so it is anticipated that the number of medicines licensed for use in children will begin to increase significantly over time.
When can unlicenced medications be considered for use?
The GMC’s above-mentioned guidance confirms that unlicensed medicines may be prescribed in certain circumstances if, on the basis of an assessment of the individual patient, it is determined for medical reasons that it is necessary to do so to meet their specific needs.
The GMC says that prescribing unlicensed medication may be necessary in the following situations –
- If there is no suitably licensed medication that would meet the patient’s needs. For example; where there
is no alternative licensed medicine that would meet the needs of a child, but a medicine, licensed for the same condition in adults, would do so; the dosage specified for a licensed medication would not meet the
- patient’s need; the patient needs a medicine in a formulation that is not specified in an applicable license.
- If a suitably licensed medicine would meet the patient’s needs but is not available at the time.
- If prescribing forms part of a properly approved research project.
- If there is a serious risk to public health, allowing the MHRA to temporarily authorise the sale or supply of
an unlicensed medicine (e.g. often used as a vaccine).
- If a prescription only medicine that is unlicensed in Northern Ireland has been supported under the
Northern Ireland MHRA Authorised Route (NIMAR).
The GMC also says that when a doctor prescribes an unlicensed medicine, they must –
- Be satisfied that there is sufficient evidence or experience of using the medicine to demonstrate it is safe
and effective, in accordance with principles of evidence-based medical practice;
- Take responsibility for prescribing the medicine and for overseeing the patient’s care, monitoring and any
follow-up treatment or ensure there are arrangements in place for another suitable doctor to do so;
- Make a clear, accurate and legible record of all medicines prescribed and where the doctor is not following
common practice, their reasons for prescribing an unlicensed medicine.
Unlicensed Medication in children – considerations for practice
Guidance from the Royal College of Paediatrics and Child Health (‘RCPCH’)(2) confirms that those who prescribe for a child should choose the medicine which offers the best prospect for that child, as well as being aware that such prescribing may be constrained by the availability of resources. Children should be able to receive medicines which are considered safe, effective, appropriate for their condition, palatable and available with minimal clinical risk.
The informed use of some unlicensed medicines is necessary in paediatric practice, but the RCPCH advises that ‘where available, an appropriate licensed preparation should be prescribed and supplied in preference to an unlicensed preparation’. They go on to say that all health professionals should have access to reliable up-to-date information on any medicine they prescribe, dispense, or administer, and on its availability.
Medicines for Children(3) is a partnership of three organisations who work towards improving child health in the UK and around the world. They comment that most medicines prescribed for children have a licence for use in adults, which means clinical trials have demonstrated efficacy and safety in adults. If a doctor believes that a specific medication is safe and effective to use in children, they are able to prescribe it for the child. As discussed above, doctors need to be acting in the child’s best interest, using the most appropriate medications to help the child. Doctors are required to read all information from medical publications about the relevant medications use in children before prescribing, and could be advised to seek further advice from other experts before deciding to prescribe an unlicensed medication.
Essentially, as per the RCPCH’s guidance, any use of an unlicensed medication should be ‘supported by a respectable, responsibility body of professional opinion’.
Overall, please be reminded that you are legally accountable for any prescription decisions that you make.
Consideration of local policies
The RCPCH’s guidance also notes that some NHS Provider Organisations have sought to introduce systems and procedures to monitor the prescribing of unlicensed medications by staff members due to the current emphasis in healthcare culture on evidence-based medicine and risk management.
Considering the above, the RCPCH suggests that NHS Provider Organisations should support therapeutic practices that are advocated by a respectable, responsible body of professional opinion and if this can be demonstrated, then the courts would not hold this as a breach of the duty of care.
Thus, the ideal situation would be that any unlicensed use of medicines was agreed as part of a national, regional or hospital approved guideline, and so we would advise healthcare practitioners to check if their Trust or Employing Body has any policies or leaflets about this matter.
The first port of call for prescribing should be the British National Formulary for Children (BNFC)(4). The BNFC does contain information on unlicenced medications and clearly states this below the relevant entries. As described on the NICE website: ‘Information in BNF for Children has been validated against emerging evidence, best-practice guidelines, and advice from a network of clinical experts. BNF for Children includes a great deal of advice that goes beyond marketing authorisations’; ‘because licensed indications frequently do not cover the clinical needs of children’; ‘Careful consideration has been given to establishing the clinical need for unlicensed interventions with respect to the evidence and experience of their safety and efficacy’.
This may provide reassurance; however, it is always important to remember that the GMC states that the doctor must ‘be satisfied that there is sufficient evidence or experience of using the medicine to demonstrate safety and efficiency’.
We would advise prescribers to be extremely cautious and seek advice from their employing organisation or professional body if the medication, it’s indication or it’s formulation is not included in the BNFC.
Regarding consent, the GMC guidance confirms ‘you must give patients, or their parents or carers, sufficient information about the medicines you propose to prescribe, to allow them to make an informed decision’.
The GMC does note that some medicines are routinely used outside the terms of their license (for example, in treating children in emergencies or where there is no realistic alternative treatment). In such cases, the GMC has advised that if the information is likely to cause distress to patients, or their parents or carers, then it may not be practical or necessary to draw attention to the licencing of the relevant medicine if this could adversely affect the child’s treatment. On the other hand, in cases where prescribing unlicensed medicines is supported by authoritative clinical guidance, it may be sufficient to describe in general terms why the medicine is not licensed for the proposed use for the patient.
Notwithstanding the above and in any event, the GMC’s position is supportive of the RCPCH’s position that when an unlicensed medicine is used in children it is paramount to always ensure doctors answer questions from patients, or their parents or carers, about such medicines fully and honestly. Thus, general rules apply in that health professionals should respect children and their parents to participate in decisions on the health and wellbeing of the child and are required to ensure that those decisions are appropriately informed. However, they also say that, in general, it is not necessary to take additional steps beyond those taken when prescribing licensed medicines, to obtain the consent to prescribe or administer unlicensed medicines and licensed medicines for unlicensed applications as it is important to remember that the license status of a drug should not necessarily be perceived as reflecting what is or is not best for the child patient.
Informed consent and consideration of side-effects
Related to informed consent, Medicines for Children have confirmed that the doctor has a duty to advise if there are any common side effects that the child may experience after receiving the medication. There could be some side-effects which are unknown, which is also the case with a licensed medicine, so the patient (or parents or carer of the patient) should be advised to contact the doctor or nurse if they are concerned about anything unusual. There is an option to report any suspected side-effects to a UK Safety Scheme.
However, there are circumstances where a health professional may decide to provide additional information when prescribing that is usually judged necessary – which may be when a medicine is new or experimental, or when the balance of risk versus benefit is less clear or when the concerns of some patients, or their parents and carers, suggest a more detailed discussion is needed.
Any information shared must be given in a way that the relevant person can understand and needs to be accurate and consistent. This is especially important in cases where the patient and responsibility for the administration of medicine is being handed over to someone else (as with parents, carers and teachers).
If the above guidance does not answer your question, please do not hesitate to contact us on 0300 30 32 442 or CaseManager@mdsuk.org and we will be more than happy to assist you further.