LAW/THUR/01 AN ETHICAL FRAMEWORK FOR THE REGULATION OF THE COMPASSIONATE USE OF INNOVATIVE TREATMENTS: EXPERIENCE OF A TERTIARY PAEDIATRIC HOSPITAL

R Mulvaney, V Larcher. Great Ormond Street Hospital, Centre for Paediatric Ethics, London, UK

Aims: To develop a framework for ethical decision-making for the compassionate use of innovative treatments that are not the subjects of research protocols. It should be open and transparent and sufficiently protective of patient safety without sacrificing possible benefits to patients. This framework will incorporate a system in which outcomes are measured, reported and analysed.

Methods: A retrospective ethical analysis of 10 cases of the proposed use of compassionate or innovative treatments and discussion with the clinicians involved.

Results: Of the 10 cases, seven concerned children with oncological/immunological problems for which off-trial compassionate innovative/experimental treatment was requested and three involved metabolic conditions requiring enzyme replacement therapy and intensive care (see table). Six oncological/immunological problem cases were initially referred to the chairman of the Research Ethics Committee, but were immediately referred to the chairman of the Clinical Ethics Committee (CEC) for prospective consultation. Four of these were retrospectively reviewed at the CEC. One was referred to the Gene Therapy Advisory Council prospectively and the CEC retrospectively. In one metabolic condition case a prospective clinical ethics consultation was requested initially with retrospective CEC deliberation.. Two metabolic condition cases involved a prospective multidisciplinary team meeting including clinical ethics, the medical director and representation from relevant clinical units.

Conclusions: From consideration of these cases it was possible to develop an ethical framework for decision making. The essential elements would appear to be: defined clinical need; clear scientific basis; a determination of how the patient is likely to benefit; in-team consensus regarding the patient’s best interests; absence of other reasonable alternatives; fully informed consent from patient/family bearing in mind the experimental nature and risks; absence of coercion; a consideration of resource allocation and a commitment to outcome reporting to provide benefit …