One study, five themes: what patients, families and the public raised about paying for a high-cost gene therapy
People in Australia were asked what might influence funding decisions on high-cost gene therapies.
The authors grouped their answers into five themes, and cost is only one of them.
The paper, published in Gene Therapy on September 17, 2026, reports qualitative interviews and focus groups with 87 stakeholders recruited in Australia. The discussions covered high-cost gene therapies and health technology assessment (HTA), the process governments use to decide what to fund. The original study is best read as a map of stakeholder perceptions, not as a clinical or economic outcomes study.
The question behind the paper
Australian HTA processes are principally informed by clinical and economic evidence, and are also intended to be consultative and reflective of Australian community values. The researchers asked how people affected by disease and members of the general population think about value when a gene therapy carries a high upfront price and remaining uncertainty. That makes the paper input to deliberation, not a replacement for the evidence.
The sample comprised 55 people with lived experience, including patients, family members, caregivers and advocates, plus 32 members of the general population. Discussions covered haemophilia B, sickle cell disease and beta-thalassaemia. The participants were not a representative global population, and their views do not measure whether a therapy works or whether a health system should fund it.
The study, in short
- What it is: qualitative interviews and focus groups on high-cost gene therapies and public funding decisions.
- Where it was published: Gene Therapy, September 17, 2026.
- Who took part: 87 stakeholders recruited in Australia: 55 people with lived experience and 32 members of the general population.
- Conditions discussed: haemophilia B, sickle cell disease and beta-thalassaemia.
- When fieldwork ran: June to September 2024.
- What it measures: perceptions and preferences, not clinical effectiveness or cost-effectiveness.
87 stakeholders raised five themes; cost was one of them.
Five themes in how stakeholders see value
1. Patient and disease factors
Participants raised disease prevalence and impacts, and discussed disease severity and patient life stage as influences on individual decision making. One inference: the same clinical result may weigh differently depending on the patient’s life stage.
2. Access is part of the intervention
Geography appeared in both groups’ discussions, and consumers also raised travel, accommodation and time costs. Consumers valued person-centred care, both groups raised trust in clinicians, and general-population participants raised wait time as a barrier. A therapy that is theoretically available but practically hard to reach may be valued differently from one that fits the patient’s real care pathway.
3. Benefits and harms include quality of life
Consumers recognised the potential to minimise symptoms or cure disease. Both groups saw quality-of-life and societal benefits, and participants also raised possible adverse effects. One inference: these perceptions can inform the questions a developer or investor asks.
4. Cost is not only the list price
Both groups were aware of the high cost of gene therapy. Consumers also raised out-of-pocket costs and potential savings from avoiding future care, while general-population participants raised the health-system costs and opportunity costs for government decision makers. For a funding case, the price therefore sits inside a larger story about who pays and what delivery requires.
5. Uncertainty remains a value question
Both groups raised effectiveness uncertainty and the possibility that better alternatives may arrive later. They also wanted to hear from people who had already received the therapy, and evidence of prior success influenced both groups. One inference: for a programme team, durability and follow-up data are part of the value case, because they are how a funder hears from treated patients.
What this means for a CGT program
Two of the five themes change what a programme team does before an HTA meeting: budget and follow-up design.
GTC analysis: on budget, consumers raised out-of-pocket costs, while the general population raised health-system costs and opportunity costs. A market-access budget that models only the health-system view would leave the patient-side costs unpriced.
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GTC analysis: on regulatory strategy, both groups wanted to hear from people already treated, and the authors note that long-term data in specific disease cohorts is not available. One inference: a follow-up protocol that captures treated patients’ own experience could serve the regulator’s durability question and the funder’s evidence question with one dataset. The paper does not test that.
GTC analysis: for investors and developers, the useful pre-HTA question is which dossier assumptions are measured, which are modelled, and which rest on stakeholder perception alone. The question set travels; the Australian answers may not.
Five themes give a team five questions to put to its value dossier before an HTA meeting.
Evidence boundary
This was a qualitative study of 87 stakeholders recruited in Australia. They took part in interviews and focus groups between June and September 2024. The results describe perceptions and preferences.
They do not measure clinical effectiveness, cost-effectiveness, payer behaviour or realised quality-of-life outcomes.
The authors flag several limitations. Volunteer consumers may be more motivated to seek new treatments than others with lived experience, and general-population participants may have been more interested and knowledgeable. Facilitators may also have subconsciously introduced their own experience.
The authors say their structured question prompts likely limited that last risk, and the methods record that one team member did the NVivo coding of the interviews.
Frequently asked questions
What did the study in Australia examine?
It explored how patients, families, caregivers, advocates and members of the general population think about value in funding decisions for high-cost gene therapies.
How large was the study?
The qualitative study included 87 stakeholders recruited in Australia: 55 people with lived experience and 32 members of the general population.
What limitations does the study report?
The authors flag these limitations. Volunteer consumers may be more motivated to seek new treatments. General-population participants may have been more interested and knowledgeable about gene therapy. Facilitators may have subconsciously introduced their own experience. GTC note: the findings are perceptions from a qualitative sample recruited in Australia, not measurements of clinical or economic outcomes.
Sources
- Patient, consumer and societal values, perceptions and preferences on high-cost gene therapies. Gene Therapy, published September 17, 2026.
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