WATCH: Advanced Therapeutics Webinar 7: Ethical considerations in rare disease clinical trials

30 May 2022
Webinar series

In the 7th instalment of our Advanced Therapeutics Webinar series we focused on ethical considerations in rare disease clinical trials. As new treatments become available, and in populations without prior access, concerns around equity and suitability arise. Physicians, patients and their families can be faced with a situation where there are more patients than clinical trial spots. So, who gets the spot?

Hosted by Acting Director of Research, Dr Paula Bray, we heard from Clinical Ethicist Anne Preisz and Paediatric Neurologist Helen Young for their expertise on the ethical framework and the practical processes and decisions, while Kris Elvidge from the Childhood Dementia Initiative provided the patient and family perspective on the topic.



Anne Preisz:


Anne is the Sydney Children’s Hospitals Network (SCHN) Clinical Ethicist, leading the Clinical Ethics Support Service. She is a member of the Advanced Therapeutics and Medications and Therapeutics Committees. Anne has a clinical background as a specialist physiotherapist in neuromuscular and neonatal care. She has a particular interest in clinical research and has a Postgraduate Master’s degree in Bioethics, including human and animal research ethics, from the University of Sydney and then the RCH Children’s Bioethics Centre.

Anne is a trained Mediator and an Honorary Associate at Sydney Health Ethics University of Sydney and Senior Lecturer at the University of Notre Dame Medical School Bioethics department. She has published widely on paediatric ethics topics including publication ethics, paediatric intensive care unit (PICU) and family conflict issues, cognitive bias in clinical decision making and the ethics of paediatric reported outcome measures.

Helen Young:


Clinical Associate Professor Helen Young is a Paediatric Neurologist who has worked as a consultant Neurologist in the Neuromuscular service at The Children's Hospital at Westmead (CHW) and Royal North Shore Hospital since 2007. Helen completed her undergraduate training at The University of Edinburgh in 1993 and subsequently completed advanced training in both Sydney and the UK, including a sabbatical at The Dubowitz Neuromuscular Centre at Great Ormond Street Hospital in London.

Helen was awarded a Master’s Degree by research in Neuromuscular disease in 2007 and is currently enrolled in a Masters of Bioethics at the University of Sydney, with a particular interest in research ethics. She is the paediatric representative on both the Clinical Ethics Committee and the Human Research Ethics Committee at Royal North Shore Hospital. Helen also works as a neurologist in the CHW refugee health clinic and has advocated strongly for compassionate and appropriate treatment of children seeking asylum.

Kris Elvidge:


Kris has been working with rare disease patient organisations since 2008, enabling them to invest in the best science to develop effective therapies and communicating this research to all stakeholders. She is currently Head of Research at the Childhood Dementia Initiative, an organisation founded in 2020 to drive research and advocacy globally to disrupt the impact of childhood dementia on children and their families.

She was previously Head of Research at Sanfilippo Children’s Foundation which funds research into Sanfilippo syndrome, one of the more than 70 rare diseases that cause childhood dementia and has also worked with several muscular dystrophy patient organisations, both in Australia and the UK. Kristina completed a PhD in molecular biology at University of Western Australia and a postdoctoral at the University of Oxford.




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