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Compassionate and innovative treatments in children: a proposal for an ethical framework
  1. Joe Brierley1,2,
  2. Vic Larcher2
  1. 1
    Paediatric and Neonatal Intensive Care Unit, Great Ormond Street Hospital for Children, London, UK
  2. 2
    Clinical Ethics Service, Great Ormond Street Hospital for Children, London, UK
  1. Correspondence to Dr Joe Brierley, Paediatric and Neonatal Intensive Care Unit, Great Ormond Street Hospital for Children, London WC1N 3JH, UK; brierj{at}

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Optimism is a good characteristic, but if carried to an excess, it becomes foolishness. Theodore Roosevelt1

Ethics and innovative therapies in children’s medicine

We would define an innovative therapy as any newly introduced treatment, or a new modification to an existing therapy with unproven efficacy and side effect profile, which is being used in the best interests of a patient, often on an experimental and/or compassionate basis.

Innovation in treating those who are suffering has been a driving force in the advancement of medical knowledge and treatment for many centuries. Many commonly accepted techniques, for example vaccination or transplant surgery, have developed from the use of innovation in response to human need. In critical care medicine, the need for innovation is driven by the obligation to rescue, often in circumstances that do not permit prolonged deliberation. In contrast there exists an obligation to protect the weak and vulnerable from interventions that are unlikely to achieve their intended benefits, but which may be accepted because they provide hope in the face of an otherwise bleak outcome.

When accepted and proven approaches are failing a critically ill or suffering child, and some promising experimental therapy exists, it may be argued that there is a moral obligation to strive to obtain that therapy for the patient. Whilst those using innovative therapies should remain mindful of that ubiquitous maxim associated with Hippocrates, primum non nocere (first, do no harm),2 perhaps an alternative ethos might be equally pertinent. If, as Plutarch suggested, “the omission of good is no less reprehensible than the commission of evil,”3 it might plausibly be argued that failure to use a therapy that has some chance of benefit, in this context, can be interpreted as failing to avoid harm.

However, Silverman specifically expressed the contrary view in considering the “advances” in neonatal intensive care towards …

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