In part 5 of this series, we made the case that evidence generation in cell and gene therapy (CGT) is a multi-year, post-approval commitment. That evidence agenda exists for a reason: it directly supports the people on the other end of these therapies, who are making some of the most consequential decisions of their lives based on incomplete information. Here we turn to those decisions, and to why elevated patient engagement is a critical driver of commercial success.

The CGT patient journey is emotionally and logistically demanding in ways few other treatment experiences match. Patients are typically navigating serious, often life-threatening conditions while facing travel to an authorized treatment center (ATC), apheresis, conditioning, weeks of disrupted routines, and a recovery that extends well beyond the infusion itself. Delays, inconsistent terminology across providers, online misinformation, and fragmented communication between sites compound fear and confusion at exactly the moments when clarity matters most.

Engagement Starts with the Full Ecosystem, Not Just the Patient

Effective patient engagement in CGT requires a meaningful shift in framing. The patient is not the only stakeholder in the experience and, in many cases, they’re not even the primary decision-maker. Caregivers, who may be partners, parents, or adult children, often carry the practical and emotional load of navigating treatment logistics.

Patient advocacy groups (PAGs) can often shape how patients first hear about a therapy and frequently serve as a trusted intermediary throughout the journey. Peer networks influence expectations in many ways. Providers, both at referring centers and at the treating ATC, set the tone for what patients should expect at each stage. Payers, while less visible to patients, ultimately influence what is possible.

Manufacturers must take the time to map this full ecosystem, including the relationships, information flows, and friction points across it. Doing so positions them to design engagement strategies that actually reach patients and influence the experience at the moments that matter. Otherwise, they could miss the most influential touchpoints entirely.

Clarity, Credibility, and Countering Misinformation

CGTs are scientifically complex, and the public narrative around them has become increasingly noisy. Patients and caregivers encounter a confusing mix of accurate clinical information, dated media coverage, anecdotal accounts on social media, and in some cases active misinformation. The result is patients and caregivers often making high-stakes decisions while sorting through conflicting signals about what a therapy can and can’t do.

One misperception is especially sticky: 66% of patients still see CGT as experimental and risky (InspiroGene 2025 CGT Annual Report) rather than an approved, commercial treatment, even though the first CAR-T was approved back in 2017. This gap is more about messaging and perception, not science, and it is precisely where PAG-led education and awareness can move patients from fear to informed consideration.

Manufacturers can play a constructive role here, but only if they approach it with credibility. That means providing clear, balanced information about risks and benefits, acknowledging the limits of what is known (particularly around durability and long-term outcomes, as discussed in Part 5), and proactively countering misinformation with accurate, accessible content. It also means investing in materials that patients can easily digest, both in terms of health literacy and emotional readiness. A glossy brochure full of technical language does not help a newly diagnosed patient process a treatment decision; a clear, step-by-step explanation of what the journey actually looks like does.

Partnerships with PAGs are often the most credible vehicle for this kind of content. Patients trust advocacy organizations in ways they don’t trust manufacturers, and well-designed partnerships allow accurate information to reach patients through channels they’re most comfortable with, as highlighted by a CMO in InspiroGene’s 2025 CGT Annual Report: “In my experience as a treating clinician and patient advocate, one of the most important steps to increasing access to CGTs is empowering and educating patients with the resources that will enable them to have informed conversations with their physician about their disease and their treatment options.”

Streamlining the Journey from Diagnosis to Long-Term Follow-Up

A patient considering a CGT typically interacts with multiple care sites such as

  • A community oncologist or specialist who identifies them as a candidate
  • A treating ATC that delivers the therapy
  • Ongoing follow-up providers who manage their care for years afterward

Each handoff is a potential point of friction, and inconsistencies in language or expectations can erode confidence in the treatment decision.

Streamlining these touchpoints, and harmonizing the language and expectations across them, is one of the most impactful things a manufacturer can do to improve continuity. When the referring physician, the ATC coordinator, and the manufacturer’s patient support team are all using the same terminology, describing the same timeline, and setting the same expectations, patients experience the journey as coherent. This reduces the likelihood that patients will view the process and “chaotic” and keeps attrition rates lower.

This is particularly important during the period between initial referral and infusion, when patients are most vulnerable to hesitation, misinformation, or competing recommendations. It’s also important in long-term follow-up, where patients can feel disconnected from the treatment ecosystem once the acute phase ends. Sustained, consistent engagement across the full journey reinforces confidence in the decisions patients have made and supports the long-term outcomes data that is so foundational to the commercial story.

Encouragingly, in mid-2025, the FDA removed the CAR-T REMS and cut the required post-infusion proximity period from four weeks to two, shrinking the time patients and caregivers must stay near a center before getting back to their routines at home. Delivery is shifting to the more patient friendly outpatient setting; per ARM, 45% of CAR-T administration was in the outpatient setting in 2025 vs. 16% in 2022. And a growing line of OIG advisory opinions now allows manufacturers to cover travel, lodging, and meals for qualifying patients and caregivers, reframing CGT from a therapy for those who can afford to relocate into one within reach of far more people.

The Bottom Line

The patient experience in CGT is a core commercial lever that influences referral momentum, treatment completion, long-term adherence to follow-up, and the credibility of the therapy in the broader market. Manufacturers that map the full stakeholder ecosystem, invest in credible and accessible information, partner meaningfully with PAGs, and work to harmonize the journey across sites of care are the ones whose patients reach treatment and stay engaged through the long tail of follow-up. Treating patient engagement as a checkbox can be a pathway to failure, with patients abandoning treatment at exactly the moments when better engagement would have kept them on the path.

Next in the series: Success Factor 7 – White Glove Patient Support Services.