Experimental treatments are given in Australian hospitals every year, but little is learned from them. Researchers at the University of Sydney are exploring how leading hospitals could better capture this knowledge, turning one-off cases into evidence that helps make the next patient safer.
In Australia, personalised human cell and tissue products, also known as biologicals, reach patients in several ways. Approved products are rare, so most arrive as experimental treatments, under a legal exclusion or through a regulated route such as the Special Access Scheme. In both cases the treatment is delivered, but little is learned from it beyond that one encounter.
Dr Christopher Rudge, Deputy Director of Sydney Health Law at the University of Sydney, has spent years on the gap between what the law permits clinicians and hospitals to do and how that work should be governed. His work calls for legal and ethical thinking to be embedded in experimental science from the outset, rather than applied retrospectively.
“The idea is to share responsibility across a team that crosses the usual disciplines,” Dr Rudge says, “so that each experimental procedure can become an opportunity for learning.”
Dr Rudge works alongside the scientists developing these therapies, reading each product against the law to see what it allows and what reform it might need. One collaborator, Professor Wojciech Chrzanowski, Professor of Nanomedicine at Sydney Pharmacy School, is developing treatments for Parkinson’s disease from extracellular vesicles, which the body uses to carry material between cells.
Parkinson’s disease affects more than 10 million people worldwide and its most promising new drugs cannot cross the blood–brain barrier on their own, so today they must be injected straight into the brain or spinal fluid. Carried inside a vesicle, the same drug could one day be given intranasally.
Where a treatment like this is given experimentally under a legal exclusion, Dr Rudge argues that hospitals should not only deliver care but also learn from each case.
Dr Chris Rudge and Professor Wojciech Chrzanowski collaborate on new medicines together
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LinkOur most advanced institutions have a real opportunity not just to advance medicine but to learn from well-governed innovation.
Deputy Director, Sydney Health Law
Much of the debate about clinical translation focuses on whether Australians can access experimental treatments. In practice, some already do. As the technology advances, more will seek them. The pressing concern, Dr Rudge argues, is that these one-off treatments should contribute to a broader evidence base.
“Some patients already receive these treatments through exclusions designed to support innovation,” Dr Rudge says, “Yet what happens is rarely reported, much less shared where others can learn from it. As molecular profiling advances, the right governance could allow each case to add to what we know.”
What’s missing is what health policy calls a learning health system, where producing knowledge is part of ordinary care.
The reason some of these treatments reach patients at all is that they sit outside the national scheme entirely: the cell and tissue products a hospital makes for its own patients. When the regulator steps back like this, responsibility does not disappear; it passes to the hospital, which becomes, in effect, the regulator of its own work.
Getting this wrong has already caused harm. Prior to 2018, some private clinics used regulatory exclusions to promote and administer unproven “stem cell” treatments, resulting in patient harm and at least one reported death. Before 2018 the exclusion followed the practitioner; the reforms confined it to the hospital, making hospital governance the condition on which the carve-out now rests.
“The exclusion rests on the old idea that treating a patient with their own tissue is medicine — not manufacturing — and medicine is the hospital’s domain rather than that of the goods regulator,” Dr Rudge says. “Some argue that government oversight should still apply, which is fair enough. Yet until structural reform is a real prospect, hospitals carry both the burden and the opportunity of governance. Where the science and standards are strong enough, a hospital can do advanced medicine in self-controlled conditions. But everything turns on getting the governance right.”
One part of good governance is consent and data. Research-ethics rules permit reuse where a patient has agreed, but in one-off treatments that agreement is often too thin to support later research. Consent designed well from the outset, where patients agree, can generate clinical insights, and specialist teams could do this routinely.
Most hospitals manage this responsibility case-by-case. Dr Rudge proposes that leading hospitals take a more systematic approach: an in-house regulatory sandbox, a supervised environment embedded within existing ethics and clinical governance, where every experimental case is documented consistently.
Not all hospitals would be equipped for this. Without the right expertise, such an approach could repeat the harm those reforms were meant to prevent. Done well, the model is not additional regulation so much as improved institutional accountability.
For therapies using extracellular vesicles, that means capturing how each preparation is made and measured, building the shared methods the field still lacks, and feeding them into a clinical commons.
It’s the child and the family who really know what is at stake.
Research assistant at Sydney Health Law and the Faculty of Medicine and Health
Parkinson’s is one context. The need for systematic learning is even more acute in rare diseases, particularly in paediatric care.
Families of children with extremely rare conditions often have limited options: experimental treatments overseas, or novel therapies accessed locally. Supervised experimentation may be the only realistic path to evidence.
Sara Wardak, a science graduate and law student, has thought about children’s rights in the clinic more than most. In a 2023 article in The Lancet Child & Adolescent Health, she argued that children belong in research as partners, not subjects. She began as a youth adviser herself, at sixteen, and is now a research assistant at both Sydney Health Law and the Faculty of Medicine and Health.
“Where children and their families decide to embark on a novel treatment,” she says, “it’s worth asking whether they would like their experience to inform those who follow.”
“For rare diseases, every case has to count,” Dr Rudge says. “Personalised medicine will require personalised ethics, thought through at both bench and bedside. Where the law has stepped back, the institutions closest to the work must set high standards in advance.”
As personalised medicines and experimental therapies become more common, ensuring they are delivered safely, ethically and in ways that generate valuable evidence is an increasingly important challenge for health systems worldwide.
At the University of Sydney, researchers in Sydney Health Law are working alongside scientists, clinicians and hospitals to develop governance frameworks that help turn individual experimental treatments into opportunities for learning, improving patient outcomes while supporting responsible innovation in healthcare.
We are actively seeking partnerships with hospitals, health services, policymakers and industry leaders interested in advancing the future of personalised medicine. To find out more about partnering with us, submit an enquiry here.
