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Unravelling barriers to gene therapy access: Patient perspectives in a novel treatment landscape



For those of us watching the emerging treatment landscape, the past two years have seen a particular and exciting boom in the approval of innovative cell and gene therapies.

Across multiple conditions, 2022 saw the US and EU grant 11 new regulatory approvals, with 2023 following closely behind with 7 Food and Drug Administration (FDA) approvals in the space.

Promising recent news has also seen the FDA lift a 2-month partial clinical hold on Iovance Biotherapeutics’ Phase II trial of LN-145 – a tumour-infiltrating lymphocyte therapy designed to target non-small cell lung cancer.

Core to this progression was joint working between the organisation and both the FDA and an independent data monitoring committee as a core component in ensuring additional trial monitoring and safety measures were in place, highlighting the critical value of collaborative approaches when advancing the development pipeline.

Already, therapies for conditions such as Duchenne muscular dystrophy (Elevidys), relapsed or refractory multiple myeloma (CARVYKTI), and Haemophilia B (HEMGENIX) have received regulatory approvals, with multiple trials ongoing and additional therapies on the way at research and IND-enabling stages.

With an ever-growing pipeline, such therapies are beginning to revolutionise our approach to supporting many rare diseases with severe unmet needs.

Despite this, key barriers to ensuring that the patients who could truly benefit from these therapies are able to access them remain.

So how do we pinpoint the core hurdles and leverage innovations in technology to tackle these challenges?

Patient Perspectives in the Gene Editing Landscape

The sickle cell disease (SCD) space, historically hindered by underfunding and limited care options, is one that appears to be particularly thriving within the realm of cell and gene editing.

From CRISPR/Vertex’s Casgevy, to bluebird bio’s Lyfgenia, 2023 truly was rounded out by some spectacular advancements for a patient community that has lived with a dearth of innovation for decades.

While some are notably enthusiastic about the potential opportunities this may confer in revolutionising their future care, there remain significant challenges for the wider SCD population regarding education around, and access to, these therapies.

In light of this, our recent work sought to explore patient perspectives around these challenges in the real-world.

Soon to be presented at this year’s British Society for Haematology (BSH) Meeting, this focused on a survey of almost 100 individuals with SCD that captured key insights around their awareness of current gene therapies, their main sources of information, their concerns around these therapies, and what they felt were the biggest barriers to access.

While the majority of patients (49 per cent) reported either ‘limited’ or ‘no’ awareness of gene therapies, with only 26 per cent aware of gene therapy clinical trials, our data highlighted that more than half of patients still felt that access to treatments like gene editing was ‘very important’.

Critically, 56 per cent of patients felt inadequately informed about the potential risks and benefits of this treatment, with its newness and uncertainty around safety, side effects, and complications forming key areas of concern during patient decision-making.

Despite almost 70 per cent of patients reporting that they predominantly relied on healthcare professionals for information about new therapies, and almost 50 per cent utilising online resources or social media, it was in fact firsthand experiences and testimonials from other patients who had already received treatment that formed the most vital piece of information for patients when considering treatment.

This was closely followed by general statistics or scientific evidence demonstrating long-term safety, efficacy, and outcomes following treatment – patients most commonly hoping to see a reduction in pain crises.

At a barrier to access level, more than half of patients feared that the cost of the procedure, and whether funding could be secured to support its availability to all patients, would be a core challenge as the use of these therapies becomes more ubiquitous.

Although some identified barriers were ones without a clear solution, such as the need to offer treatment to the most severely affected patients or those with a specific genotype, most of the highlighted challenges were those linked to a need for better evidence and education that targets multiple key stakeholders.

From better public awareness and patient advocacy, to evidence-generation around the real-world impacts and potential cost benefits that in turn drive government or commercial support, it is clear that creating powerful data and insights will be central to future advancements in the gene editing landscape.

Tackling Patient Concerns Through Technology & Innovation

As clinical trials and post-marketing authorisation work in gene editing continue to progress, the ability to demonstrate the firsthand and eventual long-term impacts of treatment will be critical to patient confidence and adoption.

Approaches that track and demonstrate this longitudinally over the coming years, particularly as new therapies continue to emerge across different disease areas, will be crucial.

Indeed, ensuring that patients, clinical teams, and regulatory authorities have access to in-depth and real-world physiological, quality of life, and safety data is vital to supporting future advancements within this space – a challenge that can be tackled with the growing use of technology and innovative approaches to data capture and evidence generation.

While many of us have seen the promising success stories that have begun to surface with each successful trial and regulatory approval, the ultimate goal is to ensure that these therapies can safely reach more patients at pace.

With the success of the current pipeline of therapies set to determine and potentially pave the way for future cell and gene therapies in other disease areas, such work is more critical than ever.

For those who want to learn more about our research in this space, we welcome you to connect with us at the upcoming BSH Meeting in Liverpool, or reach out at [email protected].

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