Nearly 1 in 5 second-level appeals of Medicare Part D cancer drug denials were overturned by an independent Medicare-contracted reviewer, a cross-sectional study found. 

The roughly 20% second-level overturn rate “may seem low,” but if plans conducted accurate initial reviews and appeals, initial denials would likely be accurate, which means the second-level overturn rate should be close to zero, first author Youngmin Kwon, PhD, of the Department of Health Policy at Vanderbilt University Medical Center, Nashville, Tennessee, told Medscape Medical News.

The “most worrisome finding,” however, was that denials involving drugs prescribed for FDA-approved indications and rejected for failure to meet coverage criteria were overturned far more often than denials involving off-label use — about 60% vs 17% — which suggests that many clinically appropriate therapies may have been denied initially, Kwon said. 

The findings were published online on July 23 in JAMA Network Open. 

Utilization management measures, including prior authorization and formulary restrictions, are intended to encourage high-value care and control spending. However, these measures have been criticized for delaying treatment, increasing administrative burden, and contributing to care discontinuation and financial toxicity in patients with cancer.

Kwon and colleagues examined 4952 second-level Medicare Part D appeals involving cancer drugs submitted from 2020 through 2024. These appeals had already undergone an internal first-level review by the patient’s Part D plan before being sent to a Medicare-contracted independent reviewer.

Most appeals were for targeted therapies (58%), followed by hormonal therapies (19%), immunotherapies (10%), conventional chemotherapy (8%), and ancillary therapies (5%). Patients had a range of cancer types, including hematologic, prostate, digestive, breast, and lung.

Overall, 19.5% of second-level appeals were successful; among unsuccessful appeals, 55.2% were denied for clerical reasons, which may include missing documentation or supporting citations, and 25.3% for nonclerical reasons.

Most second-level denials (69%) involved off-label drugs, which Kwon said was unsurprising given potential questions about their safety and effectiveness.

Appeals involving failure to meet preapproval coverage criteria had the highest success rate at 61%, while only 16.5% of second-level appeals for off-label therapies were approved. Appeals requesting coverage of drugs excluded from Part D and tiering exceptions almost universally failed. Additionally, about 20% of formulary exception requests were approved, whereas appeals involving Part D exclusions or tiering exceptions rarely succeeded.

Among unsuccessful appeals for off-label use, 72% were rejected for clerical reasons.

Nearly 88% of successful appeals for off-label use involved indications supported by Medicare-approved drug compendia or peer-reviewed evidence.

Appeal criteria are “not always clear-cut, which may be frustrating for oncologists and their staff,” Kwon said. That means “providing supportive citations included in a Medicare clinical compendium or high-quality peer-reviewed evidence is key.” The evidence should apply to the patient’s specific cancer and comes from mature, peer-reviewed research rather than conference abstracts or early-stage studies, he added.

Despite efforts to reform utilization management, Kwon said “more could be done to standardize and streamline coverage criteria across Part D plans for clearly clinically appropriate medications, especially drugs prescribed on-label.”

Adam C. Powell, PhD, president of Payer+Provider Syndicate, a healthcare strategy and operations advisory firm, noted that an overturned denial does not necessarily indicate that the utilization management process failed. Appeals for non-medically accepted indications were often unsuccessful and “may in some cases be protecting patients from receiving inappropriate care,” said Powell, who wasn’t involved in the study.

Still, the findings suggest there are “a considerable number of potentially inappropriate denials by Part D plans,” the authors wrote, supporting efforts to streamline utilization management, particularly for drugs prescribed for FDA-approved indications.

Funding for the study was provided in part by grants from the Agency for Healthcare Research and Quality and the National Cancer Institute. Disclosure information for authors is available with the original study publication. Powell reported no relevant disclosures for this article.