From miracle medicine to missed patients: Closing the CGT belief gap

Let’s work together to deliver on the promise of CGTs

Cell and gene therapies (CGTs) are one of the most meaningful advances in modern medicine, offering hope to patients and families facing conditions that were previously considered untreatable. They have the potential to reduce treatment burden, improve quality of life, and in some cases even lead to long-term remission or cure.

Medical communicators can ensure that those advancements make it to patients. At the moment, CGT adoption is stymied by reputational challenges, complex safety narratives, and gaps in knowledge, but it doesn’t have to be that way. The CGT belief gap is the space between scientific possibility and stakeholder confidence: the point at which approved therapies exist, but HCPs, patients, caregivers, and systems are not yet ready to understand, trust, refer, or adopt them.

An integrated strategic approach that aligns early communications with market access strategies can close this gap and to ensure that CGTs reach every patient possible.

Regulatory approval doesn’t guarantee patient uptake

CGTs are advancing rapidly, with the potential to transform or even cure serious and rare diseases. Yet even when therapies are approved and payers recognize the potential of one-time, high-value treatments—some with multi-million-dollar price tags—this does not automatically translate into widespread clinical adoption.

The commercial success of a given CGT hinges on (1) product-specific characteristics (e.g., efficacy, safety, cost, or reimbursement), (2) broader ecosystem factors (such as operational infrastructure, referral pathways, and the size of eligible populations), and (3) stakeholder behavior impacted by their belief in long-term results. These first two variables are very difficult to modify, but industry leaders can make a difference in the third one through education and communication.

Healthcare providers’ (HCP) and patients’ reluctance and lack of trust in CGTs are not set in stone. In fact, medical communicators are well-positioned to drive outreach efforts to ensure that key stakeholders are ready to understand, trust, and deliver these therapies at scale.

Confidence and trust are core issues

It is imperative to understand the entrenched beliefs and value drivers behind HCP, patient, and caregivers’ CGT decision making. Across these stakeholders, a consistent theme emerges: lack of understanding or comfort with CGTs. Even when therapies are approved and reimbursed, they are not always seen as a safe, practical, or a known option within treatment pathways.

Some CGTs have demonstrated meaningful and sustained benefit for appropriate patients, but stakeholder concerns around safety, durability, logistics, and long-term follow-up can still limit adoption.

Across products, CGTs do not face identical barriers to commercialization, although they do share several common challenges. Both cell therapies and gene therapies require significant stakeholder engagement, complex reimbursement discussions, and the development of new clinical and operational pathways within healthcare systems. For cell therapies, commercial difficulties have been largely driven by operational and manufacturing constraints such as scalability, logistics and costs.

Gene therapies share some of these challenges, such as the need for specialist training and complex delivery. However, the additional barriers that they face related to deeply rooted perceptions of safety and long-term risk can be addressed through education.

Unfortunately, several high-profile gene therapy programs have experienced rare but highly publicized safety failures, some of which resulted in severe immune reactions or deaths. While these events are statistically uncommon, their visibility and tragedy has had an outsized impact on public perception. The result is a lingering caution that extends beyond individual products and onto gene therapy as a whole.

Compounding this is the still-evolving nature of durability data. Many gene therapies are relatively new, and while the intent is to provide one-time, long-lasting benefit, the reality is that long-term outcomes are still being observed. This uncertainty contributes to the perception that a “one-shot cure” remains out of reach.

Further, HCPs may be more comfortable with older, established treatment approaches and lack training in gene therapy approaches that would make them receptive to these technologies. As a result, they may not fully understand how these products are developed, when they are appropriate to use, or what patients’ variable responses will be. This creates a knowledge gap that directly affects confidence, and ultimately adoption.

Communicating the value, durability, and long-term implications of gene therapies requires a far greater educational effort, and the burden of closing that gap is correspondingly more significant.

 

Thoughtful provider communication as a catalyst for CGT adoption

Medical communicators are well-positioned to educate healthcare providers on CGTs to increase confidence and uptake. This is especially true in areas like rare disease, which may have limited treatment options.

Medical education should not begin at launch: Shifting perceptions and building confidence will take time and persistence, and this work must begin while their products are in development. Tactically, communicators can develop unbranded disease state education alongside strategic publications. Together, these can outline unmet needs and carve out space in the scientific community for changing behaviors and increasing knowledge.

Many HCPs will not have extensive prior exposure to gene therapy, which means that education needs to be accessible, structured, and focused on decision-relevant understanding. Training can focus on topics such as how therapies differ, the current safety profile of specific therapies, long-term outcomes, and how to interpret variability in patient responses.

Additionally, communicators must ensure that they are reaching HCPs where they already are, which requires understanding where they are seeking information and ensuring that the data is getting through to them. For example, if information is not accessible in digital tools or AI-supported decision platforms, it risks becoming effectively invisible. Healthcare organizations can actively shape how scientific content is structured, accessed and interpreted in AI‑mediated environments to ensure that their information makes it into the hands of those who need it most.

Improving the patient and caregiver experience

Patients and caregivers come to CGT with different disease states, and at different moments in their lives. Whether it is a 65-year-old patient with a disease in an advanced stage or a newly diagnosed 5-year-old sitting next to their parent, everyone deserves clarity and reassurance. CGTs are, by definition, highly specialized, and patients and caregivers are often asked to absorb new, detailed information during a stressful period. The information that is being given to them needs to be clear and practical as they work to make an informed decision.

Digestible, patient- and caregiver-centric communication around CGTs should:

• Translate complex science into a clear clinical narrative
• Explain the safety profile in a nuanced manner
• Frame uncertainty in a way that supports informed decision-making

Bridging the CGT belief gap

At Avalere Health, we are committed to reaching EVERY PATIENT POSSIBLE. For CGTs, an important part of that is supporting HCPs, patients, and caregivers’ ability to trust in these innovative technologies. Medical communicators play a vital role conveying these messages and turning breakthrough therapies into real-world outcomes.