A Framework for Ethical and Clinical Governance Evaluation of Innovative Medicines in Children: A Mixed Methods Study

AIM: The aim of this project was to develop and implement a working definition and ethical framework for use of innovative medicine in contemporary Australian paediatric practice. METHODS: A mixed methods research methodology was undertaken using a Delphi process to establish a definition of innovative medicines in paediatric patients. This definition was used to quantitate innovative medicines use in a retrospective quantitative review of non-formulary medications in a tertiary paediatric hospital. An existing ethical framework from the United Kingdom was adapted to supplement standard clinical governance processes in contemporary Australian paediatric practice. RESULTS: A definition of innovative medicines and an ethical framework for their use in paediatric patients was developed after four rounds of feedback from 18 Australian paediatric experts. A total of 2906 individual patient medication approval applications were reviewed, 1447 of these were included for analysis, 120 of which were for medications not on the Australian Register of Therapeutic Goods and six applications were considered innovative using the newly developed definition. CONCLUSIONS: Evaluation of the use of innovative medicines in paediatric patients is essential to ensure patient safety and critical clinical governance of medication use. An adapted ethical framework for Australian practice is a valuable tool to assist clinical governance processes.