Promoting CAR T Access Focuses on Awareness, Capacity, and Economics

Although some chimeric antigen receptor (CAR) T-cell therapies have been in use since 2017, it is estimated that only a fraction of patients with treatable hematologic malignancies in the US and European Union (EU) are receiving these treatments. The push to address this gap has taken on a greater significance as long-term data from the earliest CAR T-cell studies are now confirming the curative potential of cellular therapies.

The CAR T Vision report published in June 2025 was produced by a consortium of stakeholders including patient advocacy groups, medical societies, academic and community treatment centers, and other subject matter experts. They issued recommendations in 3 key areas with a goal of doubling the number of patients receiving CAR T-cell therapy by 2030.¹

“The statistics are unacceptable,” said Miguel-Angel Perales, MD, who is a cochair of the CAR T Vision steering committee. “One in 5 patients who is eligible, both in the US and the EU, actually gets to CAR T therapy. I think we’re at the point where we have to make a difference. It’s unethical to have a potential curative therapy that that we cannot bring to patients.”

Perales, chief of the Adult Bone Marrow Transplant Service at Memorial Sloan Kettering Cancer Center (MSK) in New York, spoke with Targeted Oncology^® about the problems the group has identified and the key short-term and long-term steps being taken to expand the accessibility of CAR T-cell therapy.

Targeted Oncology: What are the most common reasons that patients are who are CAR T-eligible are not able to receive it?

Miguel-Angel Perales, MD: The fact that many patients are eligible for CAR T are not getting the treatment is a major challenge that we face, and this is true both in the US and in Europe. It’s estimated that only 20% of eligible patients are getting treated. I think it’s important to put this in the context of the fact that these treatments are potentially curative. We now have long-term follow-up data, certainly in patients with lymphoma and with [acute lymphoblastic leukemia], that support the fact that there is a cure rate in these patients.^2-4 I would estimate in patients treated with lymphoma—we have 3 FDA-approved products, and 2 of them approved in the second line—that there’s probably a cure rate of around 40%. So, I think when you have a therapy that is effective [and] potentially curative for patients, and only 20% of patients are getting to that treatment, it’s a major challenge.

There are multiple reasons for this. We’ve had these therapies approved for almost for a decade in the US, and I think part of it is still awareness, which sounds a bit strange 10 years later…both on the part of the patients, but also the treating physicians in the community. Many of them are aware of the treatment but may not necessarily know who is eligible for CAR T cells.

That’s one of the challenges we face, that physicians in the community are also treating multiple other diseases. They’re not experts in cell therapy, and they may not even be experts in large cell lymphoma. When they trained, they were exposed to [hematopoietic cell] transplant, and so they may have in their mindset, if this patient is not eligible for transplant, they shouldn’t be eligible for CAR T cells, which is incorrect. Furthermore, the advances that we’ve seen in transplant are such that we’re transplanting patients today who we wouldn’t transplant 10 to 15 years ago. So, I think there’s a there’s a big awareness and understanding gap in terms of eligibility.

Then the logistical aspects—the footprint of CAR T cells in the US is still limited geographically. There are patients [who] are 3 hours or more away [from a CAR T center]. We had a publication this year looking at ZIP codes in relation to the ZIP code of the CAR T-cell center.⁵ Clearly, the further you are away from a CAR T center, the less likely you are to be seen or treated, so there are geographic limitations. There are states in the US that don’t have a CAR T-cell center available in the whole state. There are other barriers in terms of insurance reimbursement and delays in referral, such that when the patient eventually does get seen, it may be too late.

Those are all the challenges we face. Many of these we are aware of, and we’re now collecting data to support and to identify these challenges, but now we have to pivot and try and make a difference.

What are the biggest successes of CAR T Vision and its stakeholders in promoting access to CAR T-cell therapy?

CAR T Vision was started earlier this year. We had our launch meeting early in the year. The first report was issued in June. The importance of this effort is that it is a multi-stakeholder effort. We bring in physicians’ representatives, patient advocacy, [and] different organizations in this ecosystem, and it’s a global initiative. It’s currently focused primarily on the US and the EU. Interestingly, some of the barriers we face are similar, and some of them are very specific to different regions or even different countries within the EU. But it’s thinking globally of the challenges and the barriers and how can we work together.

The success this year has been to launch the vision, to bring awareness to the vision, and bring all these people into the same room to discuss this, because ultimately, we approach this from different angles. My main focus is as a physician is taking care of patients and also doing research in this space, but it’s important to bring in the…patient advocacy [and] regulatory aspects of this—for example, FACT [Foundation for the Accreditation of Cellular Therapy] is involved—everybody around the table, so that we work together to try and solve these issues.

In order to make a difference, you can’t just sit around the table and talk about problems. The first step is to bring the stakeholders together, identify and define the problem, and then we have to pivot to start to take action. That’s where we are now moving into 2026. What can we do to actually make a difference? As part of that effort, we have formed different working groups to split up the steering committee team, and we brought additional outside experts to get more voices at the table.

The 3 areas that we’re focusing on now in these 3 working groups are awareness and understanding around the CAR T space, looking at resources and capacity, and…looking at the financial aspects, how do we develop this sustainable environment to finance both the development…and delivery of CAR T cells? By narrowing on these different areas, we can identify, “What are the gaps? Can we focus on 1 or 2 points that we can try and make a difference?” and start developing actions that can really make a difference in this space.

What are the top priorities going forward for improving patient access?

The priorities in terms of improving access to CAR T cells are going to come out of the working groups. The 3 areas [are] awareness, resources, and financing. We have to hit all 3. I don’t think you can, in isolation, say, “Well, if everybody knew about CAR T cells, everybody would get CAR T cells.” We know that’s not going to happen. We have to look at this in a multilayered way. For each working group, each priority we have to define, what are the things we can immediately start working on in 2026? What are the things that are going to take longer and may…be 2027 [or] 2028 goals?

What we want to do is double the number of patients who are have access to CAR T cells by 2030. We spent a lot of time discussing about what’s an ambitious goal and what’s a realistic goal. But I think if we’re going to make a difference, we have to start taking action. We can’t just talk about what the problems are. We need to identify areas where we can take action. The REMS [Risk Evaluation and Mitigation Strategy] program, for example, in many ways, was perceived as a potential barrier. I think those changes in the US, at least hopefully, will lead to less barriers, or at least one less barrier to access.

What effect has the removal of the REMS program had?

A major aspect of the REMS was restrictions around the patient. The patients were required to be close to the center for the first 28 days. They couldn’t drive for 8 weeks. Those were major barriers for [patients]. I don’t think people realized that, but if you live 3 hours away from [the tertiary care center] and suddenly you have to relocate them for a month, and you need a caregiver, some people don’t have a caregiver who can relocate. They may have a spouse [or] young kids at home, or [the caregiver] doesn’t get paid if they don’t get work. That’s a major barrier. The fact now that we’ve reduced that to 14 days and it’s focused on clinical safety is a major win.

We have a paper that just came out for one of the CAR T products, looking at the main toxicity of CAR T cells, the cytokine release syndrome and the neurotoxicity, most of that is resolved by day 14.⁶ It’s very unlikely for patients who haven’t had it, where it’s gone away for to develop it after day 14. That intense monitoring that they need at the CAR T Center can be confined to those first 14 days.

I think the change in the REMS program reflects the clinical reality. Driving is a major issue. People, especially if they don’t live in a major urban area, need to be able to drive. Those things which may appear as very small things have a big impact, because I’ve had patients who’ve told me, “I understand what you’re telling me about the CAR T-cell [therapy], and it can cure my lymphoma, and I have no other option, but I don’t have the ability to come to New York for a month.” I’ve had patients tell me that. I had a patient in Pennsylvania, in the Lehigh Valley, and I said, “What about Philadelphia? They have a great CAR T program. I’m happy to make a phone call for you.” The patient said, “No, that’s also too far. I don’t want to impose on my family.”

We’re already seeing…our ability to have patients going home earlier than before. They’re still under our care [and] being watched closely, but they no longer have to be within 1 [or] 2 hours of the center, and we can monitor them in our regional network just as safely.

Does streamlining CAR T administration and monitoring have potential to improve access?

I think that’s very center-specific. These are therapies that can be given outpatient; that has many advantages. Many hospitals these days are at capacity, so any ability to take care of a patient outpatient is an improvement in terms of saving beds. But I think patients actually do better when they’re treated outpatient. There’s a lot of evidence…they tend to be more mobile, which is important for their physical recovery, and not confined in that hospital room. We’ve done a lot of work on the microbiome and the fact that the microbiome is very much disrupted during treatment for cancer, but there are studies showing…that when you treat patients who are transplanted in an outpatient setting, there is less disruption to the microbiome.⁷ We have data, both in transplant and in CAR T cells, that patients whose microbiomes are less disrupted have better outcomes. There’s some interaction between the immune system and the microbiome that’s such that patients whose microbiome…diversity is preserved do better.

Obviously, that requires infrastructure. At MSK, for example, our infusion suite is a 7-days-a-week operation. We have 12-hour shifts during the day so patients can be fully managed. In the outpatient [setting], you have to have the system in place overnight for emergencies. Some centers…have done a lot of work in this space with wearables, so they can monitor the patients [as] outpatients. They’re monitoring their temperature and vitals, and alarms go off...if something is off. There are different strategies that use technology to do this. It does require some infrastructure and capacity, but I think it’s also an area that we need to move into.

What do you see as the key aspect of CAR T Vision going forward?

The important thing is that this is a major international effort that we have all recognized for a long time…that there were barriers to delivering these treatments, and we’ve done a lot of work trying to resolve some of these barriers, but at the end of the day, the statistics are unacceptable. One in 5 patients who is eligible, both in the US and the EU, actually gets to CAR T therapy. We’re at the point where we have to make a difference. It’s unethical to have a potential curative therapy that that we cannot bring to patients. The first step is to bring people together from multiple stakeholders, internationally, [and] identify other problems. But now we have to pivot in 2026 into action mode, and if we’re going to make a difference, we have to identify problems that we can start working on. That’s going to require partnerships with the regulators. It’s going to require partnership with policymakers. Some of these things are going to require talking to payers about different reimbursement models but also talking to policymakers in different countries about ensuring that patients have access to curative therapy.

REFERENCES

1. CAR T Vision Report. CAR T Vision. Published June 2025. Accessed December 16, 2025. https://cartvision.com/executive-summary/

2. Abramson JS, Palomba ML, Gordon LI, et al. Five-year survival of patients (pts) from TRANSCEND NHL 001 (TRANSCEND) supports curative potential of lisocabtagene maraleucel (liso-cel) in relapsed or refractory (R/R) large B-cell lymphoma (LBCL). Blood. 2024;144(suppl 1):3125. doi:10.1182/blood-2024-200204

3. Neelapu SS, Jacobson CA, Ghobadi A, et al. Five-year follow-up of ZUMA-1 supports the curative potential of axicabtagene ciloleucel in refractory large B-cell lymphoma. Blood. 2023;141(19):2307-2315. doi:10.1182/blood.2022018893

4. Rives S, Maude S, Hiramatsu H, et al. S112: Tisagenlecleucel in pediatric and young adult patients (PTS) with relapsed/refractory (R/R) b-cell acute lymphoblastic leukemia (B-ALL): Final analysis from the ELIANA study. HemaSphere. 2022.6:13-14. doi:10.1097/01.HS9.0000843344.19780.98

5. Chung AP, Shafrin JT, Vadgama S, et al. Inequalities in CAR T-cell therapy access for US patients with relapsed/refractory DLBCL: a SEER-Medicare data analysis. Blood Adv. 2025;9(18):4727-4735. doi:10.1182/bloodadvances.2024015634

6. Hunter BD, Lunning M, Shadman M, et al. CRS or ICANS are rare beyond 2 weeks after lisocabtagene maraleucel infusion: data from clinical trials and the real-world setting. Transplant Cell Ther. Published online October 24, 2025. doi:10.1016/j.jtct.2025.10.024

7. Sung AD, Giri VK, Tang H, et al. Home-based hematopoietic cell transplantation in the United States. Transplant Cell Ther. 2022;28(4):207.e1-207.e8. doi:10.1016/j.jtct.2022.01.015