Real-World Evidence Fills Gaps in Blood Cancer Research

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Most of what we know about treatments, adverse events, and the overall survival of patients with chronic lymphocytic leukemia (CLL) and mantle cell lymphoma (MCL) comes from clinical trials. Confirmatory real-world evidence, especially in the Medicare population, is limited. In existing studies, many address only a few types of outcomes. The introduction of newer targeted therapies has led to gaps in evidence, evidence that is needed to understand more fully the burden of these blood cancers.

To provide detailed information about different treatment types for CLL and MCL in routine clinical settings, researchers from AstraZeneca and RTI Health Solutions conducted observational studies in Medicare populations. These studies reported real-world evidence on how treatment types can affect patients’ health outcomes (such as adverse events and survival) and financial outcomes (direct medical costs to both patients and the healthcare system).

Adverse events from cancer treatments often require medical intervention and may disrupt a patient’s treatment schedule, which in turn can lead to suboptimal health results and economic consequences. The annual economic burden of adverse events and other disease-related medical costs of CLL alone in the United States is expected to climb from $740 million in 2011 to over $5 billion in 2025. Ravi Goyal, the lead author of these studies, observed, “Up-to-date understanding of health outcomes and the financial burden of treatments for blood cancers like CLL and MCL in nonclinical-trial settings is critical for keeping patients, physicians, and policymakers informed and for accelerating evidence-based decision making. This research helps provide such evidence to the stakeholders.”

Study measures and endpoints were as follows:

  • Baseline patient characteristics
  • Treatment characteristics
  • Overall survival
  • Adverse events
  • Healthcare resource use and costs

Study results highlighted the following findings:

  • There is considerable patient susceptibility to adverse events
  • There is a substantial economic burden to Medicare
  • There is a significant increase in financial burden as the number of adverse events increase

Read these studies  

Goyal RK, Nagar SP, Kabadi SM, Le H, Davis KL, Kaye JA. Overall survival, adverse events, and economic burden in patients with chronic lymphocytic leukemia receiving systemic therapy: real-world evidence from the Medicare population. Poster presented at the 61st Annual Meeting of the American Society of Hematology; December 2019. Orlando, FL. [abstract] Cancer Med. 2021 Mar 18;10:2690-702. doi: 10.1002/cam4.3855. https://www.rtihs.org/publications/overall-survival-adverse-events-and-e...

Goyal RK, Jain P, Nagar SP, Le H, Kabadi SM, Davis K, Kaye JA, Du XL, Wang M. Real-world evidence on survival, adverse events, and health care burden in Medicare patients with mantle cell lymphoma. Leuk Lymphoma. 2021 Jun;62(6):1325-34. doi: 10.1080/10428194.2021.1919662. https://pubmed.ncbi.nlm.nih.gov/33966583/

Additional research involving CLL and MCL

Goyal RK, Nagar SP, Kabadi SM, Davis KL, Le H, Kaye JA. Overall survival, adverse events, and economic burden in Medicare patients with chronic lymphocytic leukemia receiving cancer-directed therapy. Presented at the 61st American Society of Hematology (ASH) Annual Meeting and Exposition; December 9, 2019. Orlando, FL. [abstract] Blood. 2019 Nov 13;134(suppl 1):796. doi: 10.1182/blood-2019-122842. https://ashpublications.org/blood/article/134/Supplement_1/796/427022/Ov...

Kabadi SM, Goyal RK, Nagar SP, Davis KL, Le H, Du XL, Jain P, Wang ML, Romaguera JE, Kaye JA. Overall survival, adverse events, and economic burden in Medicare-insured patients with mantle cell lymphoma receiving cancer-directed therapy. Presented at the 61st American Society of Hematology (ASH) Annual Meeting and Exposition; December 7, 2019. Orlando, FL. [abstract] Blood. 2019 Nov 13;134(suppl_1):63. doi: 10.1182/blood-2019-122841. https://ashpublications.org/blood/article/134/Supplement_1/63/424464/Ove...

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