The (clinical) trials and tribulations of gene therapy – why meaningful patient engagement strategies matter
May 9, 2025 | clinical trials, gene therapy, patient engagement, patient perspectives
By Sarah Mohamad and Lauren Coleman-Bennett
Gene therapy has the potential to change lives, slowing or stopping disease progression or even correcting symptoms entirely, but deciding whether to take part in a gene therapy clinical trial isn’t simple. What makes this decision so difficult, and is it more difficult than it needs to be?
Meet David – a look inside one patient’s gene therapy trial experience*
David is a 27-year-old graphic designer who is newly married. His doctor has just told him that in only a few years, he can expect to notice the first symptoms of Huntington’s disease, the same disease that took his father’s life. David remembers watching him lose his ability to walk, talk and care for himself. His personality shifted, his memory faltered, and eventually, David barely recognized his father before he passed away. Now, David is facing the same future.
Unlike his father, David has options. After hours of Googling, he reads about a gene therapy clinical trial that might slow the disease. However, there’s a catch. The therapy has never been tested in humans before; it’s experimental and, like many gene therapies, irreversible. If he decides to participate, he may be ineligible for future, potentially more advanced gene therapies. The trial requires years of follow-up appointments to monitor long-term effects, invasive procedures, and regular hospital visits far from home. There’s no guarantee it will work, and as a first-in-human trial, many of the risks remain unknown.
David’s mind is flooded with questions. He asks some of them out loud: “What are the risks of gene therapy? Will I be cured?” His doctor seems unsure, rifling through websites and pausing for long stretches while searching for answers that never really come. Over the next few months, most of those questions remain unanswered, but with no other treatment options available, David reluctantly agrees to have his eligibility assessed for the gene therapy trial, hoping that things will become clearer in the sign-up process.
Instead, David encounters:
- A 50-page consent form with dense, technical language that feels more like a legal contract than a supportive guide
“Is this my best chance at a future or an unnecessary risk? Could this therapy cause complications decades from now? I wasn’t going to have children just in case they inherit this Huntington’s. If I take part in this trial, could it stop me from passing the bad gene on?”
- Doctors using unfamiliar terms such as immunosuppression, vectors and biomarker effects
“Do I need a science degree to keep up with all this?”
- Unclear responses when he asks about long-term risks and the pros and cons of participation
“What does Phase I even mean? Why do they keep bringing up mouse data?”
- Invasive treatment options that feel extreme – some trials involve brain surgery, while others inject viruses into the spine
“Have these procedures ever been done successfully before? Don’t viruses make you sick?”
- Logistical concerns about costs, time off work, the need to relocate, insurance implications and caregiving support
“Will I have to move near the hospital site?
“Why do some of these trials require 10 or even 15 years of follow-up?
“What does follow-up really mean?
“Will I be looked after when the trial ends, or will I be left without support?
“What does this mean for my health insurance?
“How will this affect my family, my job, my finances?”
David walks away afraid, uncertain, disheartened. He tells himself that he’ll wait for a proven treatment or for more certainty, but time isn’t on his side.
What difference can a meaningful patient-engagement strategy make to a clinical trial?
It helps people feel informed, empowered and in control. Meaningful patient-engagement strategies start by listening to real patient experiences. They’re designed to empower people to make confident, informed decisions about their own health. David’s story shows that when communication isn’t clear, tailored and developed in partnership with patients, they can feel lost, uncertain and unable to take control of their choices, and if that’s the case, can we really say that their consent is truly informed?
A meaningful patient-engagement strategy makes trials more accessible and more representative, which can strengthen trial outcomes, health outcomes and asset success. Patient-centred design and engagement help to remove barriers to participation (eg logistical, cultural and communication challenges). By making it easier for a wide range of people to take part, trials become more inclusive, the data more reflective of real-world populations, and the findings more relevant to real-world settings, and the opportunity to address health inequalities grows.1
Early engagement can speed up participant enrolment, reducing costs and increasing the likelihood of trial success. Nearly 80% of clinical trials fail to meet enrolment timelines. These delays mean that Phase II–IV trials can take up to twice as long as originally planned, with running costs at about $37,000 per day and delays in market entry costing $600,000 to $8 million per day.2 However, when people with the condition have been meaningfully involved early on, including through insight gathering or co-creation of trial materials, trials have enrolled up to three times as many participants than those without their involvement.3
Sustained engagement with patients throughout a trial and beyond can improve retention, preserve data quality and increase the chances of trial completion. Dropout rates can exceed 20%, risking the validity of trial data.4 Up to 80% of registered trials are never completed, often because of poor recruitment and retention.1 How, then, can you address this? Effective engagement strategies are built on understanding the root causes of dropout. They address patient needs, barriers and facilitators that can affect decision making, such as the burden of participation, unmet expectations and inadequate understanding from the very start of a trial.1
Post-trial engagement supports positive trial experiences, keeping the doors open to future trial participation.1 Engagement shouldn’t end when the trial does. A meaningful engagement strategy should include plans for clear, timely post-trial communication to improve participant experience and build lasting trust. Making sure that participants have positive trial experiences and study updates from the trial team, including the final results, helps participants feel valued and more likely to engage in future research.1
What does a good patient-engagement strategy look/feel like?
Now, imagine if David’s experience had been different:
- During his visit, David’s doctor proactively brings up the possibility of participating in a gene therapy trial, something she learnt about at a recent webinar. This time, the doctor speaks with confidence, providing a clear overview of the trial’s requirements. She even has an FAQ document made for HCPs, helping her answer David’s initial questions with ease
- David is intrigued. His doctor encourages him to take some time to think and hands him a few resources – a pamphlet, an infographic and a short video – all in language that he understands. These explain what gene therapy is and what he can expect from a clinical trial
- A few weeks later, he decides that he wants to participate. After some tests and having his eligibility confirmed, he receives a dense, 50-page informed consent form, but this time, it comes with an illustrated booklet that breaks it all down, explaining the science in everyday words, not technical jargon. It answers his questions as though it can read his mind: the risks, potential benefits, and what he can expect the trial experience to be like from one day to another
- While weighing his options, David connects with a patient advocacy group listed on the back of the same booklet, where he hears from other patients who have been through similar experiences. Their stories help him feel supported, less alone and more prepared
- When he finally decides to take part in the trial, he’s given a beautifully designed guidebook, something that feels almost like a companion. Inside, it takes him through each step of the journey, explaining what to expect. There’s a built-in planner to help him keep track of appointments, questions to consider asking at his appointments, and even thoughtful tips on how to prepare for procedures and recover comfortably
- After David receives the gene therapy infusion, HCPs and clinical trial site staff check in with him regularly – they have the tools and training to share regular updates, reminders and emotional support, so he never feels lost or uncertain. His guidebook holds his hand, so David knows what to expect – how many visits, when they’ll happen, and how to fit them around his work and family life
- He knows exactly how he’ll stay connected for the long-term follow-up and when and how he’ll get updates on the results of the trial
With the right clinical trial engagement strategy, David doesn’t feel like just a participant. He feels like an informed decision maker. Ensuring that patients like David have access to clear, supportive and transparent communication is necessary for truly informed consent and essential for trial success!
If you want to explore how to build a patient-centred engagement strategy for your clinical trial, contact Sarah.Mohamad@amiculum.biz. For more insights, visit the AMICULUM News and Insights page.
*This fictional patient story is based on published research into real patient experiences, including barriers, concerns and decision-making factors influencing clinical trial participation and engagement.
References
- Dobra R, Wilson G, Matthews J et al. A systematic review to identify and collate factors influencing patient journeys through clinical trials. JRSM Open 2023;14:20542704231166621. doi: 10.1177/20542704231166621
- Johnson O. An evidence-based approach to conducting clinical trial feasibility assessments. Clin Investig (Lond) 2015;5:491–9. doi: 10.4155/CLI.14.139
- Crocker JC, Ricci-Cabello I, Parker A et al. Impact of patient and public involvement on enrolment and retention in clinical trials: systematic review and meta-analysis. BMJ 2018;363:k4738. doi: 10.1136/bmj.k4738
- Bell ML, Kenward MG, Fairclough DL, Horton NJ. Differential dropout and bias in randomised controlled trials: when it matters and when it may not. BMJ 2013;346:e8668. doi: 10.1136/bmj.e8668
This content was provided by Amiculum
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