Where CLL Care Falls Short of the Science: Kirsten Axelsen, MS

Targeted therapies have transformed care for chronic lymphocytic leukemia (CLL), raising 5-year survival rates to nearly 90%, yet many patients still do not receive the testing and treatment that guidelines recommend. A September 2026 report from Charles River Associates (CRA), commissioned by BeOne Medicines USA and developed with input from blood cancer advocacy groups, details gaps in biomarker testing, utilization management (UM) policies such as step therapy and prior authorization, and nonmedical barriers such as transportation, along with 5 recommendations to close them.¹

In part 1 of this interview with The American Journal of Managed Care^® (AJMC^®), lead author Kirsten Axelsen, MS, a consultant at CRA, explains where the gap between science and practice is widest, why biomarker testing remains inconsistent, what patient groups shared that the literature misses, and how step therapy and Medicare Part D changes shape access. AJMC previously covered the report’s key findings.²

This interview has been lightly edited for clarity and length.

AJMC: The report says innovation is outpacing access. In plain terms, where is the gap widest right now between what the science supports for CLL and what patients receive?

Axelsen: First-line guideline-based treatment for CLL has really shifted from chemotherapy, which was untargeted across the board, to more targeted treatments. With that, we’ve seen increases in survival rates and a reduction in adverse effects typically found with these targeted therapies. But what hasn’t happened is adherence to guidelines and the biomarker testing that would identify patients who are eligible for these more targeted therapies. We’ve seen estimates as low as 15% of patients getting tested for all 3 biomarkers. CLL is a disease where the average age at diagnosis is around 72 years, and typically the approach is to watch and wait until symptoms appear and then move to therapy. That creates a bit of an issue in this patient population, which often has comorbid conditions, such as cardiovascular issues. We’re finding that between the watch and wait and the treatment, we’re not seeing the biomarker testing that would really improve care and reduce, as you say, this gap between the availability of a novel treatment that could help a person live longer and live better and what people are often getting, which is often just chemotherapy, which is not the recommended standard of care.

AJMC: According to a study cited in the report, fewer than 15% of Medicare patients with CLL were tested for all 3 of the most common biomarkers. Why is testing still so inconsistent, and what does that mean for treatment decisions?

Axelsen: We interviewed patient groups and provider groups from around the country in different states. The 4 states included in our reports were Florida, Texas, California, and New Jersey. We heard different things from different providers and patients in different parts of the country. In some cases, the provider may not know biomarker testing is available, or a patient does not have access to a specialist who would know. The provider may make assumptions, fairly or unfairly, that the patient wouldn’t have the ability to cover the cost, but in fact, some of these tests are covered by insurance. We also heard about transportation barriers. It was a combination of a lack of awareness and education and maybe some perceptions that this patient wouldn’t be able to get access to the test or to the targeted drug. While barriers still do exist in insurance, there are ways to get this treatment—especially in the Medicare program—these drugs are covered, although most drug plans still require somewhat sizable cost sharing.

AJMC: You spoke with groups like the CLL Society and the Lymphoma Research Foundation. What did you hear from them that the published research wasn't capturing?

Axelsen: We heard in our focus groups, and from them, that research wasn’t capturing a lot of the costs that are nonmedical. Transportation was continuously raised as a real barrier to care, and we talked about not only transportation costs but also transportation support. For example, in one discussion, we talked about how when you call your typical ride-sharing app, that person won’t help you get from your house to the car. These are people who are often both elderly and experiencing symptomatic disease. Just sending an automobile for them is not enough.

Another cancer survivor mentioned that in New York, while we have some of the best facilities available, taking the subway to a chemotherapy appointment is just not feasible, because the immune system is suppressed and a person would be exposed to a lot of different germs and viruses on the subway. So just having access to public transportation isn’t necessarily the same as having access to the right supports. That’s really what we came up with. I think a lot of the medical literature focuses on costs, on tests and whether those are getting done, and on adverse effects—all important things, but what we really heard about were some of the things outside of the medical costs that are real, sizable barriers to care for this patient population.

AJMC: From a payer's perspective, step therapy is a cost-control tool. Where's the line between reasonable management and a barrier that harms patients?

Axelsen: Utilization management should be based in clinical evidence. But there are reports of up to a quarter of new prescriptions for blood cancer therapies getting rejected. So the real question is, are those prescriptions getting rejected for justifiable clinical reasons or because a bureaucratic hurdle hasn’t been met? I think the general guideline is: Is that step edit or that rejection really justified by the evidence, by the guidelines from cancer providers, by the labels on the drugs, or by real-world evidence? When it’s not, then it’s just an unnecessary barrier to access that can result in the person not getting any treatment.

AJMC: How has the Inflation Reduction Act's Part D redesign changed how plans manage oral cancer drugs, and what should patients and prescribers expect?

Axelsen: Cancer medicines are one of the protected classes in Medicare, so they are all covered. What we are seeing, though, is an increase in utilization management that goes beyond coverage: things like step edits and prior authorizations for a lot of the higher-cost treatments, including cancer treatments, which are typically higher cost. The main thing that patients have to be concerned about is the hurdles. There’s also been a real shift in patients moving from standalone drug plans and fee-for-service into more of a Medicare Advantage environment, and those Medicare Advantage plans, which are more like managed care plans, can have more restrictive physician networks. That’s not directly related to the drug, but if a patient is not able to get access to a specialist—or a specialist they can reach geographically—through their health plan, that can be another barrier to care if they’re switched into, or they switch themselves into, a plan that has less access to the specialists who might know to do the biomarker testing or provide the other services they need to support their condition.

AJMC: What spurred the creation of this report and the collaboration behind it, and why was it needed now?

Axelsen: BeOne Medicines reached out to me, and I’m a consultant at Charles River Associates, to develop an objective assessment in partnership with patient groups of the barriers to care. The goal was to look across the different issues that patients may face from a whole-patient perspective: not just thinking about insurance coverage, not just thinking about access to specialists, but what is it that patients are facing? We discussed how we would do this, and one of the first principles was that it needed to involve the patient groups. We were fortunate to get partnership and input from the American Cancer Society Cancer Action Network, Blood Cancer United, the CLL Society, the Lymphoma Research Foundation, and Fred Hutchinson Cancer Center. Representatives from all those organizations informed the design of the research and also worked with us on input.

In addition, we convened patient groups and providers from different regions around the country to talk about rural issues and urban issues. That was really part of the philosophy of this report that BeOne, my coworker Laura Jenkins at CRA, and I decided was going to be primary: It needed to include rigorous evidence from the literature about these drugs, about how they work, and about what the right treatment protocols were based on what physician societies and clinical data said. But it also needed to have insight from patients about what they’re experiencing that we’re not necessarily seeing in the literature so that we could surface some of those barriers. We also agreed with BeOne and with the patient groups that we would look into state-level policy as well as federal-level policy, because those 2 really work hand in hand. The states regulate different aspects of insurance coverage and can also do a lot around awareness, whereas the federal government is more in the domain of Medicare coverage, certainly, but also of bodies advising on protocols for biomarker testing and other preventive steps. So it really came from BeOne’s desire to find a partner to pull together these different parts of the patient and provider community and have a very rigorous foundation for what we put together for this report.

References

1. Axelsen K, Jenkins L, Garvey L, Pan M. When Innovation Outpaces Access: Closing the Gap in CLL Care. Charles River Associates; September 2026. Accessed November 4, 2026. https://beonemedicines.com/patients/our-commitment-to-patients/

2. McNulty R. CLL treatment access lags behind innovation amid testing, step therapy gaps. AJMC. October 8, 2026. Accessed November 4, 2026. https://www.ajmc.com/view/cll-treatment-access-lags-behind-innovation-amid-testing-step-therapy-gaps