Second-Level Appeals of Denied Anticancer Medication Claims in Medicare Part D

Importance: Despite the goal of utilization management (UM) to facilitate high-value cancer care, concerns about care denials persist. Evaluating justifications for denied claims can clarify the quality of UM decisions. Objective: To examine second-level appeals for denied anticancer medications in Medicare Part D to assess the quality of UM decisions. Design, Setting, and Participants: This cross-sectional study used a large language model (ChatGPT 5 Mini, OpenAI) to analyze texts of second-level appeals decisions on denied Part D medications, rendered by a Medicare-contracted reviewer. Data were analyzed from March 13, 2025, to May 12, 2026. Main Outcomes and Measures: From decision summaries, the reasons for first-level denials (as cited by Part D plans) and the outcomes of second-level appeals (favorable vs unfavorable) were examined. The reasons for unfavorable reviews (clerical vs nonclerical) were also examined. Results: We included 4952 second-level appeals from 2020 to 2024 by beneficiaries with hematological (n = 1155 [23.3%]), prostate (n = 876 [17.7%]), digestive (n = 650 [13.1%]), breast (n = 613 [12.%]), and lung, bronchus, and respiratory (n = 430 [8.7%]) cancers. Most appeals were for targeted therapy (n = 2889 [58.9%]) or hormonal therapy (n = 959 [19.4%]). The most common reasons for initial denial or appeals were "non-medically acceptable" indications (ie, off-label use; 3410 [68.9%]), formulary exceptions (545 [11.0%]), and failure to meet preapproval coverage criteria (mostly on-label prescribing; 463 [9.3%]). Overall, 19.5% (95% CI, 18.4%-20.6%) received favorable reviews, and 55.2% (95% CI, 53.8%-56.6%) and 25.3% (95% CI, 24.1%-26.5%) received unfavorable reviews due to clerical and nonclerical issues, respectively. Favorable reviews were more prevalent among appeals for drugs initially failing to meet preapproval coverage criteria (61.1% [95% CI, 56.7%-65.6%]), but less so among appeals for off-label therapies (16.5% [95% CI, 15.2%-17.8%]). More than 87.9% (95% CI, 86.1%-89.6%) of successfully appealed off-label therapies were for medically acceptable indications supported by Medicare-approved clinical compendia and/or peer-reviewed literature. Conclusions and Relevance: In this cross-sectional study of second-level appeals for denied anticancer medications in Medicare Part D, a considerable number of successfully appealed claims (especially for therapies prescribed on label) were found, which suggests potentially inappropriate UM decisions by Part D plans. However, the appeal outcomes varied substantially by the nature of the denials. Streamlining the preapproval process for on-label therapies and establishing accessible, transparent criteria for off-label prescribing may optimize UM processes for anticancer medications.