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Case for standardising practice

The number of children with complex health needs is increasing.1 Caring for these children requires healthcare professionals to consider and make decisions on a wider range of treatment options, considering the relative evidence for each pathway and their anticipated benefit for the child.

It is important that children and their families are involved in discussions and decisions relating to all aspects of their care as they are the experts on the child's experience of health and quality of life. Understandably, families will often want all treatment options to be considered for their child. It is the role of the healthcare professional to use their expertise to support families in exploring options, but also to manage expectations and advise in situations where treatments may not be in the best interests of the child.

For us as parents the most important thing is that when our child does pass away we know we’ve done absolutely everything to give them the best quality of life and for as long as possible.

Family, 2021

Social media and access to information on the internet have changed the way that children and families seek out advice and access health provision.2  This has led to an increased awareness of novel and unproven interventions. In some circumstances it has resulted in conflict between clinicians and families, and very rarely has led to families taking their children out of the UK for treatment.3 4

It is important to recognise that paediatricians routinely work with parents to develop constructive relationships for the benefit of their child. But occasionally a breakdown in communication5 and loss of trust6 7 can occur, particularly when a family has felt that their own expertise and knowledge of their child has been ignored by the healthcare team.8  On rare occasions such disagreements have played out in high profile court cases, resulting in great distress to families and healthcare professionals alike. 

Doctors should see the parent as the expert on that child.

Family, 2021 

When done well, ESOs reaffirm the principles of shared decision making and give assurance to the family that everything possible has been considered. However, in recent years, the process by which ESOs have been sought and provided by individual clinicians has come under scrutiny. 

Perceived problems of the ESO process can include: 

• concerns that those giving second opinions may not have the requisite skills or expertise to do so
• examples where the doctor giving the second opinion has not reviewed the child’s notes, met with the family, or examined the child
• a perception of bias on behalf of families that the chosen professional may not be truly independent
• concern by the healthcare team that the ESO sought by the family is not based on the medical evidence provided in the child’s health record
• variable practice across the UK in terms of both when a second opinion is sought and the way this happens
• that the ESO process is not recognised by most hospital Trusts/Health Boards and service planners, leading to concerns that, in some paediatric specialties, there is a national shortage of experts prepared to undertake this work..

Aims and scope

This guidance has four overarching aims: 

• to standardise practice and improve transparency of the ESO process
• to develop a helpful resource for healthcare professionals, service planners and families to draw upon when requesting, providing, and receiving an external second opinion
• to prevent disagreements in healthcare
• to make recommendations to service planners, professional bodies and governments on the actions needed to adopt and support this guidance locally.

The themes, processes and principles of this work will be useful in all ESO scenarios, whether gained by individuals, external MDT or national advisory panels.

Whilst the guidance is primarily aimed at paediatricians and families under the care of paediatric services, it is recognised that there are increasingly occasions where the families of older adolescents under 18 years of age are involved in challenging discussions relating to transition to adult services. In these circumstances, it may be appropriate for adult clinicians to refer to this guidance in case they are requested to provide the ESO.

Many aspects of healthcare provision for children are outside the scope of this guidance, including:

• diagnosing the child’s underlying condition
• treatment decisions for the unborn child
• second opinions on unproven interventions.
• seeking best practice advice for the management of children with perplexing presentations and fabricated or induced illness (see )
• best practice advice or recommendations on managing conflict (see  and ).

This work draws on legal and medical professional frameworks adopted by the UK. The overarching principle that the child’s best interests is paramount, is drawn from a UK context that is described throughout this document. 

While the information and recommendations presented here seek to provide transparency around ESOs, they may also help to support and inform the court system by encouraging paediatricians to undertake expert witness work.

Most importantly, this document and its accompanying guide for families (which can be downloaded on the landing page), aims to support families to understand how they should be involved in the decision-making process. The views of parents are included throughout this document, and are accompanied with examples of good practice that illustrate the importance of shared decision making between the healthcare team and the family. 

The clinical context - how decisions are currently made in practice

Medical decision making can be complex and at times uncertain. Whilst many decisions are informed by a well-established evidence base or mandated NICE guidance, some are inevitably judgements that consider the risks and benefits of a particular course of action. The emphasis on shared decision making and patient centred care has motivated the medical profession to embrace a broad culture change where the views of patients and families are regularly considered and often at the centre of discussing treatment options.

Shared decision making in the best interests of the child guides practice in paediatrics. All families should be allocated a named consultant who maintains overall clinical responsibility for the child’s care. It is important to recognise that every paediatrician also operates within a team; the term ‘team around the child’ describes key members of the multidisciplinary team who work together to provide holistic co-ordinated care for that patient. As the number of children with complex needs increases, team-based decisions are now regarded as standardised good practice in many paediatric sub-specialities.

In all circumstances the family should be regarded as a key partner: informed, participating, and consenting 

Parent, 2021

For those children where decision making around their treatment is complex, a referral may be made for an external second opinion (ESO) from a multidisciplinary team (MDT) in a separate hospital or alternatively from a national advisory panel. Paediatric cardiac surgery and oncology are examples of this. Occasionally the child’s named consultant may seek an ESO from an individual consultant at another hospital. In all cases the child and family should be involved in decision making in every stage of the process.

For most children getting a second opinion is not about going to court, it is about diagnosis, treatment and exploring different thinking.

Healthcare professional, 2021 

While ESOs in all their formats are just one element in the decision-making process, they have become increasingly important in ensuring every avenue is explored to ensure consensus is achieved. Throughout this guidance we aim to describe the ‘usual’ - for example, a routine consultation between two hospital MDT meetings - as well as the ‘less usual’ - namely a more formal ESO from an individual clinician. We suggest that the same principles underpin both processes.

Legal framework

While families have some legal and ethical rights to make decisions for their child, they do not have a specific legal right to request a second opinion.9 10 A healthcare professional will rarely refuse a reasonable request by a parent for an ESO if it is in the best interests of the child. While it may fall within their professional duty, there is no legal requirement that a healthcare professional must request or obtain a second opinion.

Healthcare professionals must legally be able to demonstrate they have provided appropriate care for their patients. This includes being able to show they have weighed the risks against the benefits and made reasonable and logical decisions about what should be done. These decisions must be in line with ‘a responsible body of medical opinion’.11 12 A healthcare professional who cannot show this may be considered negligent.

Courts will take account of professional and clinical guidance that it is good practice to request an ESO in determining what constitutes a responsible body of opinionl13  however, the courts appreciate that it is not always feasible to obtain a second (or expert) opinion even if it would be desirable, particularly in situations where a decision must be made quickly.14  

Healthcare professionals cannot be legally required to give care or treatment that they do not consider is in their patient’s best interests.11 The law accepts that two views on appropriate treatment can co-exist as long as each has a logical basis and represents a responsible body of professional opinion.12  Therefore, if an MDT disagrees with an ESO, their refusal to carry it out will not necessarily be considered negligent or against the child’s best interests.  Even if the court decides it is in the child’s interests to have a particular treatment, the first MDT/healthcare professional would not be compelled to provide it.

Regulatory professional framework

The General Medical Council (GMC) is the organisation that protects patient safety and sets standards for medical education and training in the UK. Where necessary, the to prevent a doctor from putting the safety of patients, or the public’s confidence in doctors, at risk. Good medical practice describes what it means to be a good doctor and the GMC asks doctors to demonstrate trust and respect for human life in their practice.

Whilst parents do not have a legal right to request an ESO, specifically requires doctors to “respect the patient’s right to seek a second opinion”. This document supports a patient’s right to make free, informed health care choices in order to clarify clinical facts and define treatment options. Responding to a patient’s request for an ESO fulfils a doctor’s obligation to respect patients’ rights and to provide the highest standard of care.  

Commissioning framework

There is currently no formal commissioning arrangement in place that recognises or facilitates the ESO, resulting in variable practice across the UK. Upon consulting with paediatricians and specialist healthcare professionals, it is clear that this work is viewed as extremely onerous, frequently done ’out of hours’ and, in the main, unremunerated. Whilst informal arrangements are made between medical directors at hospital Trust/Health Board level, the lack of a national commissioning framework frustrates formal governance arrangements, job planning and the proper resource to support an optimal process.   

The way that specialised services are commissioned in England is changing, as new legislation is introduced to put integrated care systems on a statutory footing by July 2022. Integrated care boards will be financially responsible for some specialised services from 2023/2024 and will work with regions to ensure services and standards of care are maintained. There are opportunities for local systems to work with hospital Trusts/Health Boards and service planners on understanding the best model in which to embed and support this guidance.   

Unlicensed or experimental interventions

The makes it clear that doctors are expected to reason carefully when prescribing medications or other interventions. That responsibility also applies when a doctor is providing an ESO. An ESO must represent reasoning based on sound theoretical knowledge and understanding (usually resulting from appropriate training qualifications), clinical experience, and available evidence.

Unlicensed and ‘off label’ medicines

Medicines for Children has written an . 

Whether or not a product is ‘licensed’ for the indication for which it is proposed is an important part of that reasoning. A licensed medicine has been assessed for efficacy, safety, and quality by a regulatory authority (in the UK, by the Medicines and Healthcare Regulatory Agency, MHRA) and has received a ‘marketing authorisation’. The ‘marketing authorisation’ or ‘license’ restricts the claims manufacturers can make for that medicine and is based on evidence provided by clinical trials.15 16   

and such as stem cell and gene therapies are subject to similar UK regulation. 

‘Off-label’ refers to the use of medicines that have a marketing authorisation, but whose licence does not include the indication in question. That might be because it is licensed for a different condition, or in a different age group. ‘Unlicensed’ medicines are those whose manufacturers have no authority to market at all in the UK, often because they are still undergoing clinical trials. Such medicines can sometimes be made available to prescribers by the pharmaceutical company through or schemes.   

The  acknowledges that effective clinical practice sometimes requires doctors to prescribe medicines for indications that are ‘off-label’, or less commonly, unlicensed. Under those circumstances, the GMC points out that there is greater responsibility on the prescriber to set out the reasoning carefully and clearly and must: 

• be satisfied that there is sufficient evidence or experience of using the medicine to demonstrate its safety and efficacy
• take responsibility for prescribing the medicine and for overseeing the patient’s care, monitoring, and any follow up treatment, or ensure that arrangements are made for another suitable doctor to do so
• make a clear, accurate and legible record of all medicines prescribed and, where you are not following common practice, your reasons for prescribing an unlicensed medicine.

This practice is fairly common in paediatrics as historically clinical trials often didn’t include children which meant the manufacturer didn’t have enough evidence to market the product in this age group, irrespective of its actual effectiveness and safety.16 17  

However, the presence or absence of a licence should certainly inform prescribing decisions. A licence indicates that the product has been assessed for efficacy in that indication as well as for safety and quality; the presence or absence of those assessments must be relevant when it comes to well-reasoned judgments and prescribing recommendations on the part of doctors.

ESO and unlicensed or experimental interventions

The ability to request, provide or receive an ESO is dependent on there being an expert who is able to offer such an opinion. A clinician who wishes to provide an informed ESO about an intervention for a particular clinical indication must show that he or she has the necessary and specific knowledge and training (see Providing an external second opinion). In the ESO, he or she must then provide evidence (research or, less persuasively, from personal experience) to support a recommendation. 

This can, but does necessarily always include, treatments offered on compassionate grounds. For these treatments there may be a good evidence base but cost is prohibitive and/or licensing is incomplete. Other treatments offered on compassionate grounds are for unproven therapies and should be seen in the same light as novel or experimental treatments.

Use of an intervention within its product licence means that a respectable and responsible body of professional opinion and experience endorses its use for that indication. Such endorsement is usually lacking where there is no licence. Recommendation for off-label use in children can often be made by showing that the medication is safe in children, albeit for a different indication, and/or that it is effective for this indication, albeit in adults.  

That is much more problematic in experimental interventions. There is a danger that clinicians and families confuse the term ‘novel’ or ‘experimental interventions’ with something that is ‘improved’ when in fact the converse may apply. By definition there is little clinical experience or evidence to support the effectiveness or safety of experimental or novel treatments.

It is unlikely, therefore, that there will be a ‘respectable and responsible body of professional opinion and experience’ in respect of novel or experimental interventions. While it is not impossible that introduction of such an intervention might nevertheless be in a child’s interests, an ESO that recommended it would need to offer extremely persuasive theoretical reasoning in order to offset the lack of evidence and experience available to support it.

Ultimately, any recommendation from an ESO relating to unlicensed or experimental interventions should be both reasonable and in the best interests of the child.

Key terms and definitions

List of key terms and definitions

Child or children	Babies, children and young people under the age of 18 across the UK.
Critical friend	A trusted professional, usually within the same organisation, who is requested to offer a critique of a colleague’s management of a challenging situation; for example, either to advise on treatment decisions or to help navigate circumstances where the child’s family and clinical team disagree on the correct course of action.
Clinical Ethics Advisory Committee (CEAC)	A group consisting of health and other relevant professionals who provide advice and support to clinicians and families on ethical issues that have arisen during clinical practice.
Expert witness	A healthcare professional, generally appointed by a solicitor, whose skills and experience qualify them to testify on a particular medical area in a court or tribunal setting.
External second opinion (ESO)	An external medical opinion on the patient’s proposed course of treatment from a second independent health professional/MDT within an appropriate specialty. The initial suggestion to get an ESO may come from a patient/family, or from the child’s health care team. This is for children facing significant life changing decisions, and the second opinion should come from a separate Trust or Health Board.
Family	Parent, carer or guardian identified as the person with parental responsibility of the child. This could include the local authority.
Family advocate	A named point of contact who can provide information to the family on the ESO process, and who can signpost to sources of support. See this section for more information.
Health organisations	NHS Trusts and NHS Health Boards across the UK.
Healthcare professional	A clinically qualified person who is working within the scope of practice as determined by their relevant professional body and who is registered with that body as competent to practice.
Mediation	An impartial service designed to support patients, families and NHS staff work together towards resolution of disagreements.
Multidisciplinary team (MDT)	A group of professionals from one or more clinical disciplines from hospital and community settings who together make decisions regarding recommended treatment of individual patients.
Named consultant	A named consultant taking on overall clinical responsibility for the child.
National Advisory Panel	A multi-disciplinary team of experts organised at national level to support decision making around treatment options in complex and rare diseases. Such panels may enhance consistency and transparency around care pathways and aim to ultimately improve survival outcomes.
Parallel planning	Parallel planning offers an opportunity for children with life limiting conditions to live their lives to the full while plans are made for managing their ongoing care and end of life.
Referral for second opinion	The administration and paperwork that is required to support the process of requesting an external second opinion.
Service planner	Organisations responsible for planning, commissioning, or providing health services.
Team around the child	The professionals that contribute to the wider health and wellbeing of the child. This would include the child’s named consultant, the MDT, GP and any professional in an advocate role.

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• 9Gillick v West Norfolk and Wisbech Area Health Authority [1986] AC 112, 184 per Lord Scarman.
• 10These are not ‘rights’ as such, but are better understood as responsibilities and are contingent on their being exercised in the child’s best interests: Re A (Children) [2001] 1 Fam 147 (HL) per Ward LJ.
• 11 a b R (Burke) v General Medical Council & Ors [2005] EWCA Civ 1003
• 12 a b Muller v King’s College Hospital NHS Foundation Trust per Kerr J.
• 13See further: C v North Cumbria University Hospitals NHS Trust [2014] EWHC 61 (QB) and also AUTHOR Margaret Brazier and Jose Miola, ‘Bye-Bye Bolam: A Medical Litigation Revolution?’ (2000) 8 Medical Law Review 85–114.
• 14Mulholland v Medway NHS Foundation Trust [2015] EWHC 268 (QB) per Green J.
• 15 Off-label or unlicensed use of medicines: prescribers’ responsibilities.
• 16 a b Royal College of Paediatrics and Child Health/Neonatal and Paediatrics Pharmacy Group. The use of unlicensed medicines or licensed medicines for unlicensed applications in paediatric practice. 2013 /resources/use-unlicensed-medicines-or-licensed-medicines-unlicensed-applications-paediatric
• 17 In 2007, European (including UK) law introduced a requirement for pharmaceutical companies to undertake studies in children as part of the development plan for most new medicines. Over time, it is anticipated that the number of medicines licensed for use in children will increase. When a licensed medicine becomes available it should, therefore, be prescribed and supplied in preference to an unlicensed preparation.