N/A Anne Preisz1, Dr Helen Young
1Sydney Children’s Hospital Network, 2Sydney Health Ethics
Rare diseases in children may be devastating and life-limiting, with few treatment options. This context is emotive and high stakes as therapy often relies on access to an early phase clinical trial which have strict criterion, unproven efficacy and a lower safety profile.
Parents and clinicians are faced with multiple ‘competing sorrows’ as there are often more eligible children than trial places, and siblings with the same genetic disease may be excluded.
In this presentation we outline salient ethical considerations for rare disease clinical trials for children, acknowledging the importance of paediatric research and the complex nexus of a rapidly evolving biotechnology sector, novel gene therapies and the need to offer vulnerable children with few options, access to clinical trials. This necessitates developing an ethically defensible plan underpinned by principles of procedural and distributive justice. This ‘futureproofing should highlight ethical principles of proportionality, precaution and protection to reflect on and account for issues such as ‘dual agency’ for clinicians as investigators, therapeutic misestimation and the psychological wellbeing of patients and families recruited to trials, as well as the clinicians who care for them. This requires robust transdisciplinary research and interconnected and expert communities of practice.
Biography:
Bio to come