As part of our advanced therapies access series, we asked George Eastwood of the Emily Whitehead Foundation to share his perspective on one of the field’s most urgent challenges: ensuring that scientific progress translates into real patient access.
There is a lot to be encouraged by in advanced therapies right now. CAR-T cell therapy, one of the most revolutionary breakthroughs in modern medicine, has changed outcomes for patients who otherwise have very few treatment options, or no treatment options, left to reverse their disease. That is real progress.
But I spend a lot of time thinking about the distance between what advanced therapies can do and how many people who can benefit from them get the chance to receive them.
That is where I think the conversation in the advanced therapies community needs to go.
We tend to talk about access as if it is a later-stage problem. First comes the science. Then the approval. Then, eventually, the system catches up.
This may have made sense earlier in the development of these life-saving treatments. I do not think it makes sense anymore.
We are far enough along now to know these therapies have extraordinary impact. So when patients who qualify are not still able to access treatment, that is not a side issue. It is a central issue.
In my presentation at ASH 2025, I talked about some of the barriers to access that still stand in the way. Awareness is one. Referral timing is another. Logistics, treatment-center availability, and the financial burden are barriers too. None of this is especially surprising to people who work in our field. What concerns me, though, is how often these barriers are acknowledged without being confronted (managedhealthcareexecutive.com).
Since 2017, almost 50,000 patients have received CAR-T cell therapy. Through CAR T Vision, an international coalition aimed at ensuring all eligible patients have the opportunity to receive life-saving, potentially curative CAR T-cell therapy, we have set a goal of helping move that number to 100,000 by 2030. The Emily Whitehead Foundation supports this goal because it gives the field something concrete to organize around. But I also think it points to something larger. Even as we make progress, we are still treating only a portion of the patients who may be eligible.
Public reporting from Europe showed 4,888 CAR-T recipients in 2023. Separate modeling work puts the annual global patient population that could plausibly meet current approved-style CAR-T criteria in the low hundreds of thousands, with a base estimate around 216,600 per year. Those are modeled figures, not a perfect count, and they should be treated that way. Still, the overall picture is clear. The number of patients who might benefit is far larger than the number who are being treated.
That gap exists for a lot of reasons, and I think the field sometimes oversimplifies them.
Cost matters, of course. But access is not only a cost problem. It is also a recognition problem. It is a referral problem. It is a timing problem. It is a center-capacity problem. It is an infrastructure problem. It is a coordination problem. By the time those things pile up around a patient, the distinction between them is not very meaningful.
What matters is whether the patient gets treatment in time.
That is why I keep coming back to awareness.
I do not mean awareness in a vague promotional sense. I mean whether the right people know enough, early enough, to act. Does the treating physician know when referral should happen? Is the patient made aware of a therapy’s existence before the window starts closing? Does the local care team know how to get the patient onto the right treatment path without losing weeks in the process?
In a field like this, awareness has very vital consequences. It changes what happens next.
The same is true of capacity. When people say there is a capacity issue, that can sound abstract. It is not abstract to a family trying to figure out where treatment can happen, how far they have to travel, whether a center can take them, how long it will take, and what happens if the disease progresses while all of that is being sorted out.
This is why the patient perspective matters. It changes the scale of the problem. It takes language that can sound administrative and puts it in human terms.
The patient perspective cannot be left at the margins.
Another thing I think about is that we sometimes mistake progress in the field for progress in access. They are not always the same thing. More approvals do not automatically mean broad availability. New indications do not automatically mean smoother pathways. A therapy can be groundbreaking and still remain out of reach for too many people.
That is why I believe the next phase of work in advanced therapies has to be more grounded in delivery. Not delivery as an afterthought. Not delivery as something to be solved once the exciting work is done.
If a therapy is difficult to access, difficult to navigate, and limited to a narrow set of institutions, then patient impact will remain narrower than it should be, even if the science keeps improving.
I am optimistic about where the advanced therapies field is headed. And there are many people across industry, clinical care, advocacy, and policy who understand the urgency. But I also think we need to be direct with ourselves about proving that these life-saving therapies can be accessed more broadly and more fairly.
That means better coordination across the whole ecosystem. It means listening more closely to patients and families. It means paying attention to what happens before treatment, not just after it. And it means being honest that access is not a supporting issue in advanced therapies. It is a fundamental issue.
For me, the real test ahead is not proving the power of these therapies. We know they work.
The test is whether we can build a system that allows more patients to receive them.