Case study

Supporting families through complex gene therapy journeys

21/10/2024
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Key Results

49 rare disease families

participated through workshops, interviews, events & surveys

89% of caregivers

found the support content useful for them and their child

50+ content type mock-ups designed

to support across five key stages of the gene therapy journey

Background

Trial Flow was configured through extensive co-design with rare disease families, advocacy groups and experts to support children with Duchenne Muscular Dystrophy and their caregivers through a complex gene therapy research journey. 

Challenge

High-stakes enrolment decisions

Gene therapy research for a life-limiting rare disease can place significant emotional and cognitive burden on families.

Families may face complex screening and enrolment decisions, including “all or nothing” choices about participation.

Prolonged engagement and retention

Long-term monitoring, which can extend over many years in some studies, also requires sustained motivation and engagement.

The sponsor needed clear, sensitive support that could help families understand the journey, prepare for key milestones and feel supported through both expected and unexpected outcomes.

Our solution

Patient-centred support across the gene therapy journey

Trial Trial Flow was configured through a deeply patient-centred approach, informed by rare disease families, advocacy groups and experts.

Support was structured across five key stages:

Eligibility
Planning and preparation
Gene therapy infusion
Short-term monitoring
Long-term monitoring

The solution included age-specific content for children and caregivers, with a focus on both informational and emotional support. It also considered unexpected “unhappy paths,” ensuring families could be supported even when their journey did not follow their hoped for route.

Child interacting with Trial Flow on an iPad with his mother

Outcomes

Improved confidence & understanding

89% of caregivers found content useful

Strong qualitative feedback

From families and the sponsor team

speech marks

Your power wheel is this psychosocial aspect that is helping kids, and the parents as well. You are speaking to the gaps I cannot speak to.

Patient Lead

Pharmaceutical Client

speech marks

Thank you for working to improve lives!

Parent & co-design workshop participant

Laptop, tablet and phone screens showing mock-ups of the information and support flows for this project.

Example early design mock-ups

[Overview of the outcomes. Rearrange the cards below as needed!]

Supporting families through complex journeys?

Talk to us about how Trial Flow can be configured around the needs of specific patient populations, supporting engagement, adherence and retention throughout demanding study journeys.

Trial Flow Phone (Left)

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