CLL Treatment Access Lags Behind Innovation Amid Testing, Step Therapy Gaps

Gaps in biomarker testing, inconsistent adherence to clinical guidelines, and insurer utilization management policies continue to limit treatment access for many patients with chronic lymphocytic leukemia (CLL), according to a September 2026 report from Charles River Associates commissioned by BeOne Medicines USA.¹

“Science has transformed what is possible for people living with CLL, offering more personalized, effective, and tolerable treatment options than ever before,” the authors wrote. “Yet too many patients remain unable to fully benefit from these advances because of inconsistent access to comprehensive biomarker testing, barriers to guideline-concordant treatment, [utilization management] policies that delay treatment, and lack of affordable coverage.”

The report synthesizes academic and policy literature with interviews from the blood cancer community and closes with 5 recommendations for policymakers, health plans, and health systems.

Why CLL Treatment Access Has Not Kept Pace With Innovation

CLL primarily occurs in older adults, with a median age at diagnosis of 70 to 72 years, and comorbidities can complicate treatment selection, the authors wrote.¹ Following FDA approval of the first Bruton tyrosine kinase (BTK) inhibitor in 2013 and the first B-cell lymphoma 2 inhibitor in 2016, targeted therapies have increased 5-year survival rates to nearly 90%, according to the report. On October 2, 2026, the FDA also approved the noncovalent BTK inhibitor pirtobrutinib (Jaypirca; Eli Lilly) for adults with previously untreated CLL or small lymphocytic lymphoma without known 17p deletion.²

Biomarker Testing, Step Therapy Among Key CLL Access Gaps

Even when physicians prescribe guideline-preferred therapies, step therapy and prior authorization can delay or restrict access. In a survey from the Community Oncology Alliance cited in the report, 91% said insurer or pharmacy benefit manager policies do not align with clinical recommendations, and 97% said insurance policies interfere with patient access to recommended treatments. At an Institute for Value-Based Medicine^® event in the Detroit, Michigan, area, an oncology pharmacist similarly said step therapy that pushes ibrutinib ahead of newer options remains a recurring holdup.³

As of July 2026, 19 states require biomarker testing coverage for all state-regulated plans, and as of 2026, 26 states ban or limit copay accumulator programs.¹ But coverage remains fragmented, the authors noted, because many state laws apply only to state-regulated plans.

“Only when the impact of access barriers is understood and reduced can patients fully benefit from innovative medicines,” the authors wrote.

5 Policy Recommendations for CLL Treatment Access

The authors recommend that all states require state-regulated plans to cover medically necessary biomarker tests for CLL and that Congress extend similar requirements to self-funded employer plans. They also support provisions of the Safe Step Act that would require these plans to offer a step therapy exception process and respond to exception requests within 72 hours.

Other recommendations include guideline-concordant CLL care pathways, continued Medicare telehealth flexibilities, investment in the Rural Health Transformation Program, and responsible use of artificial intelligence (AI). The report calls for Medicare Advantage and employer-sponsored plans to disclose when AI is used in care decisions and to show that a qualified clinician evaluated a decision before issuing a denial, which the authors wrote could help prevent AI from serving as the sole basis for denials.

“Ensuring that treatment decisions are guided by clinical evidence, and not administrative barriers or insurance design, is essential to improving outcomes for people living with CLL,” the authors concluded. “By acting on these recommendations, payors, healthcare providers, patient advocates, and policymakers can help strengthen a healthcare ecosystem that keeps pace with innovation and ensures every patient has timely access to the right treatment at the right time, regardless of where they live or the type of insurance they have.”

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 October 7, 2026. https://beonemedicines.com/patients/our-commitment-to-patients/

2. McNulty R. FDA approves pirtobrutinib for untreated CLL/SLL without 17p deletion. AJMC®. October 2, 2026. Accessed October 7, 2026. https://www.ajmc.com/view/fda-approves-pirtobrutinib-for-untreated-cll-sll-without-17p-deletion

3. Joszt L. Oncologists grapple with sequencing, access as treatments multiply. AJMC^®. September 30, 2026. Accessed October 7, 2026. https://www.ajmc.com/view/oncologists-grapple-with-sequencing-access-as-treatments-multiply