Abstract

A balance needs to be struck between facilitating compassionate access to innovative treatments for those in desperate need, and the duty to protect such vulnerable individuals from the harms of untested/unlicensed treatments. We introduced a principle-based framework to evaluate such requests and describe its application in the context of recently evolved UK, US and European regulatory processes. 24 referrals were received by our quaternary children's hospital Clinical Ethics Committee over the 5-year period. The CEC-rapid response group evaluated individual cases within 48-hours; the main referrers being haematology/oncology, immunology or transplant services. Most requests were for drug/vaccine/pre-trial access or biological/cellular therapies. The majority of individual requests were approved ; neutral or negative opinions were given in 5, including 3 group requests. Recently evolved regulatory processes share common criteria and conditions to our framework including: demonstration of clinical need; sound scientific basis with lack of viable alternative; risks-benefit/best interests evaluation; arrangements for fully informed consent; no compromise of arrangements to test treatment for licensing purposes; consideration of resource implications. There are differences between individual processes and with our framework, with respect to procedures, scope, application format, costs and obligation to make available all outcome data. Our experience has emphasized the need for an independent, principled, consistent, fair and transparent response to the increasing demand for innovative treatment on a compassionate basis. We believe that there is a need for harmonization of the recent proliferation of regulation and legislation in this area.