Ms Sara Attinger1
1Macquarie University, Macquarie Park, Australia
Biography:
Sara is a researcher at Macquarie University and Sydney Health Ethics, University of Sydney. Their research interests lie at the intersections of law, medicine, and commerce, with recent focus on assisted reproduction, and cellular therapies. Sara has worked in the private and public sectors in legal, policy and commercial roles.
Abstract:
Background/Aims:
Drug regulators are under increasing pressure to facilitate access to innovative cell and gene therapies – a mandate often in tension with their core role of monitoring safety, efficacy and quality. While the difficulty of balancing these tensions is well-recognised, the precise role of regulators in access at different points in the translation life-cycle is not always clear, making it challenging to assess how well they might be fulfilling these mandates. This is important when, alongside demands for more access and funding, the authority of some regulatory bodies has been opened to legal challenge.
Methods:
We draw insights from qualitative analysis of in-depth interviews with 93 professionals and persons with lived experience (patients and carers) about patient access to stem cell-based interventions (SCBIs), focusing on views relating to regulation and governance.
Results:
Stakeholders’ values and assumptions relating to regulation and access could be both competing and complementary, including choice and ‘trying’, along with caution. While domestic availability was equated with legitimacy, there was frustration about lacking domestic options and information, and desire for research and trial access.
Conclusion:
With most cell and gene therapies in the earlier stages of clinical development in Australia, and much of that occurring outside the market approval pathway, we consider the ‘facilitator’ role of regulators as part of a broader constellation approach to governance for managing access. We suggest gaps and opportunities for regulators to better address the needs and expectations of these access communities, including contributing to information and utilising trusted governance infrastructure.