Gene therapy and you

What Australians told us

AMANDA RUSH AND OTHERS*

Gene therapies are creating new hope for people with serious conditions like haemophilia. These treatments can be life-changing, but they also raise important questions: how should we pay for them, who should have access, and what happens when the long-term effects are still uncertain?

Between June and September 2024, researchers in a study led by the University of Sydney spoke to 87 Australians – including people living with blood disorders, their carers, advocates, and members of the public. Using eight in-depth interviews and 15 focus groups, participants shared their views after watching an educational video on gene therapy and blood diseases. They were asked to reflect on four areas: what influences their decisions, the potential benefits and harms, barriers to access, and what still feels uncertain.

WHAT PEOPLE SAID

Five key themes emerged:

1. The patient context matters: Participants stressed that decisions should account for disease severity, life stage, prior treatments, and the lack of other effective options.
2. Access and fairness are critical: People highlighted barriers such as awareness, the role of clinicians in sharing information, travel distance, wait times, and the importance of person-centred care.
3. Hope and risks are both real: Many saw gene therapy as a chance to reduce symptoms or even cure disease, but they also worried about the unknown nature of side effects.
4. Costs spark debate: Views ranged from concern about the fairness of allocating scarce resources to recognition that gene therapy could save the health system money in the long term.
5. Uncertainty weighs heavily: The lack of long-term data on safety and effectiveness left many uneasy, with some worrying that better options might appear in future.

WHY PATIENT PERSPECTIVES ARE ESSENTIAL?

In Australia, bodies such as the Pharmaceutical Benefits Advisory Committee (PBAC) and the Medical Services Advisory Committee (MSAC) assess whether new treatments should be subsidised. Traditionally, this process looks at clinical safety, effectiveness, and cost-effectiveness.

This research, led by Professor Kirsten Howard and funded by the Medical Research Future Fund, is part of a national project called Development of a generalisable evaluation framework for high upfront-cost gene therapies. The project aims to build a framework that helps decision-makers weigh not just clinical and financial evidence, but also ethical, legal, and cultural dimensions—alongside patient and family perspectives.

WHY IT MATTERS

Gene therapies are pushing health care into new territory. They bring hope, but also uncertainty and complexity. This study shows that Australians value not only medical results and costs, but also fairness, access, and the realities of living with disease.

By combining structured Health Technology Assessment (HTA) frameworks with consumer voices, Australia can make better, more accountable decisions that balance innovation with equity.

READ MORE

HFA is a partner in this University of Sydney research project and is very grateful to the people and parents of children with haemophilia who participated in the focus groups and interviews and the survey, which followed later.

Results from this research project were also presented as a poster at the Australian Bleeding Disorders Conference 2025.

Rush A, et al. Patient, consumer and societal perceptions on high-cost gene therapies for haematological disorders.

You can view the poster on the HFA website here

*Authors:
Amanda Rush, Maria Gomez, Kirsten Howard, Pippy Walker, Garry Lynch (The University of Sydney); Kristine Pierce (University of NSW); Richard De Abreu Lourenco, Rosalie Viney (University of Technology Sydney); Kylie Mason (Peter MacCallum Cancer Centre, The Royal Melbourne Hospital); Jo Watson (Pharmaceutical Benefits Advisory Committee).