CLL Treatments Have Never Been More Promising. Why Aren’t More People Benefitting From Them?

Treatments for the most common form of adult leukemia, chronic lymphocytic leukemia (CLL), have changed dramatically over the past decade. Today, patients have the possibility to be treated with targeted therapies that can improve quality of life and push five-year survival rates to nearly 90%.

Yet for too many people living with CLL, innovation in medicine alone is not enough. Lack of access to or delays in biomarker testing, inconsistent use of clinical guidelines, and onerous insurance barriers can prevent patients from getting treated with the best possible care.

As the maker of foundational medicines for CLL and other blood cancers, BeOne Medicines knows more is possible. But scientific progress can only reach its full potential when patients can access the right care at the right time. That’s why BeOne partnered with Charles River Associates and blood cancer experts including CLL Society, Blood Cancer United, American Cancer Society Cancer Action Network, Lymphoma Research Foundation, and others, on a first-of-its-kind national assessment of the CLL care ecosystem to pinpoint barriers to timely, innovative care.

Matt Shaulis, General Manager of North America, BeOne Leadership.

“Innovation in CLL has transformed what’s possible for patients, yet too many still face barriers to benefiting from that progress. This report pinpoints where gaps persist, and where the blood cancer community must work together to close them.” – Matt Shaulis, General Manager, North America, BeOne Medicines

Understanding CLL

CLL mainly affects older adults, is often diagnosed around age 72, and is commonly discovered through routine bloodwork. Because CLL generally progresses slowly, many patients begin with a “watch-and-wait” approach before starting treatment. But every patient’s disease is different.

Age, comorbidities like cardiovascular disorders, patient preferences, and unique genetic risk factors should all shape treatment decisions.

Treatment innovations

For decades, systemic and untargeted therapies such as chemotherapy and early immunotherapy were the standard of care for CLL, but both had serious side effects and limited effectiveness. That changed with FDA approval of targeted therapies like the first Bruton’s tyrosine kinase inhibitor (BTKi) in 2014 and the first B-cell lymphoma 2 inhibitor (BCL2i) in 2016. These therapies have transformed CLL care by significantly improving long-term outcomes.¹

Continuous BTKi therapy has demonstrated durable benefit, with second-generation BTKis showing exceptional long-term survival. Emerging fixed duration treatments could further change the treatment paradigm if they can deliver more durable responses, keep the disease under control for longer, and offer greater convenience.

Five Ways to Close the CLL Care Gap: Getting the Right Treatment to the Right Patient

So, while innovative treatments have continued to advance, the challenge now is ensuring more patients can benefit from these novel advances. This will require action on five fronts.

1. Expand Access to Comprehensive Biomarker Testing

Biomarker testing is a critical starting point in CLL because it can reveal genetic risk factors that help guide treatment decisions. Yet access remains inconsistent. One study cited in the report found that fewer than 15% of Medicare patients with CLL received all three of the most common biomarker tests.

Nathan, diagnosed with CLL at 45, was initially advised to begin chemotherapy without testing. After asking for biomarker testing, a second opinion led to a non-chemotherapy treatment. Today, he is in remission.

Patients should have timely access to clinically appropriate biomarker testing, regardless of insurance or location, and administrative barriers that delay testing should be reduced.

A family of three stands together outdoors, exuding unity and support, possibly sharing their cancer care journey.

2. Increase HCP and Patient Education

Clinical guidelines must continually evolve to reflect the latest data and best clinical practice, yet they are not always updated or consistently followed, with some patients still initiated on chemotherapy instead of targeted oral therapies. Healthcare providers need current guidance that reflects the latest clinical evidence, and patients need accessible, scientifically accurate information that helps them understand their choices. Health systems should keep care pathways aligned with national treatment guidelines and make it easier for patients to take an informed role in treatment decisions.

3. Improve Care Coordination for Timely Access to Specialists

Patients in rural or underserved communities may have less access to CLL specialists and may need to travel farther for care. Telehealth, stronger care coordination and continued investment in rural cancer infrastructure can help community oncology providers connect more patients with blood cancer expertise, particularly at key decision points in their care.

4. Reduce Insurance Barriers that Delay or Deny Access to Care

Even when a physician recommends a cancer treatment, insurance companies and pharmacy benefit managers (PBMs) often put burdensome barriers in the way. Step therapy and prior authorizations can delay and deny care and, in some cases, require patients to “step through” and fail on older therapies before gaining access to the treatment their physician believes is most appropriate. These policies can also create significant administrative burden for oncology practices.

In one survey, 91% of community oncologists said insurer or PBM policies do not align with clinical recommendations, while 97% said those policies interfere with access to treatments recommended by physicians. Policymakers should require clear and timely step-therapy exception processes, greater transparency around where these restrictions apply and stronger oversight to ensure coverage policies reflect current clinical guidelines and evidence.

5. Increase Transparency and Promote Responsible AI in Cancer Care

Patients are increasingly using AI to understand their diagnosis, treatment options and care journey, while insurers are also using AI in coverage decisions. AI can be useful, but patients need trusted, evidence-based information and clear safeguards around how these tools are used.

Insurers should disclose when AI plays a role in a coverage decision, and a qualified clinician should review any decision to deny or delay care. AI should not be the sole basis for those decisions.

“Thanks to advances in science and medicine, people living with blood cancers have more options for hope in their treatment approaches than ever before,” said Kirsten Axelsen, Policy Advisor, Charles River Associates. “But realizing that progress means closing these gaps in their ability to get access to modern medicines. By spotlighting these challenges, the blood cancer community, policymakers and healthcare leaders can turn these advances in CLL medicine into better outcomes for every patient.”

Read the full report, When Innovation Outpaces Access: Closing the Gap in CLL Care, at www.beonemedicines.us/CLLReport.

REFERENCES

Karr, M., & Roeker, L. (2023). A History of Targeted Therapy Development and Progress in Novel-Novel Combinations for Chronic Lymphocytic Leukemia (CLL). Cancers, 15(4), 1018. https://doi.org/10.3390/cancers15041018