Although many therapies in child-health are not supported by evidence from research, this is far more likely to be so in rare diseases for which innovative treatment is often considered.
The ethical considerations surrounding such therapy in children are complex. The balancing of the potential benefits and harms of rescue therapies, together with maintenance of respect for the rights and welfare of such a vulnerable population’s is a challenging task.
Innovation, whilst appropriate in such cases, is only truly ethical if it is accompanied by mechanisms that evaluate and report its effects, protect the child from avoidable potential harm, and ensure that any conflicts of interest are addressed. Furthermore, appropriately informed consent from those with parental responsibility regarding the innovative and experimental nature of the therapy is mandatory, together with assent/consent if possible from the child.
Crucially the implication of scarce, and usually expensive, resources used for increasingly speculative treatments must be balanced with their probability of success, and the continuing burdens of therapy to the child should be considered.
We suggest an ethical framework in which such innovations might be undertaken with this especially vulnerable population.