Medicare Part D Anticancer Drug Appeals Overturned 1 in 5 Times
09/28/2026
Key Takeaways
- In second-level Medicare Part D appeals for denied anticancer medications, non–medically accepted indications or off-label use were the most common denial category at 68.9%.
- Only about one-fifth of second-level anticancer medication appeals received a favorable review, and unfavorable outcomes were more often clerical than nonclerical.
- Appeal success differed sharply by denial rationale, with the highest reversal rate in denials tied to failure to meet preapproval coverage criteria and lower success in appeals involving non–medically accepted indications.
- Successful off-label appeals were usually supported by Medicare-approved compendia or peer-reviewed literature, while rejected outside citations in unfavorable appeals were commonly judged nonrepresentative, investigational, or limited to abstracts or nonapproved journals.
A cross-sectional analysis of 4,952 publicly available second-level Medicare Part D appeals for denied anticancer medications from 2020 through 2024 examined decisions issued by a Medicare-approved Qualified Independent Contractor (QIC). Using natural language processing (NLP) with ChatGPT 5 mini, investigators classified denial reasons and appeal outcomes from decision summaries, with main outcomes defined as favorable review vs clerical and nonclerical unfavorable review. Secondary analyses examined how medically accepted indications were adjudicated and why external citations were rejected in unfavorable off-label appeals, and the sample was drawn from oncology conditions and medications for cancer and treatment-related symptoms identified through the Cancer Medications Enquiry Database, with adjusted and sensitivity analyses also performed.
Overall, 19.5% of second-level reviews were favorable (95% CI, 18.4%-20.6%), while 55.2% were unfavorable for clerical reasons (95% CI, 53.8%-56.6%) and 25.3% were unfavorable for nonclerical reasons (95% CI, 24.1%-26.5%). Many disputes at this stage were therefore resolved on administrative rather than direct clinical grounds.
Outcomes varied sharply by the initial denial rationale, with 61.1% of appeals denied for failure to meet PA coverage criteria receiving a favorable review (95% CI, 56.7%-65.6%) and 16.5% of non–medically accepted indication appeals doing so (95% CI, 15.2%-17.8%). Among successful off-label appeals, 87.9% were supported by Medicare-approved compendia and/or peer-reviewed literature (95% CI, 86.1%-89.6%). Formulary-exception appeals were favorable in 20.2% of cases, while appeals involving excluded drugs and tiering exceptions rarely succeeded, and rejected outside citations in unfavorable off-label appeals were commonly described as nonrepresentative, investigational, or limited to abstracts or nonapproved journals.
The authors noted that second-level appeals represent a selected subset of denied Part D claims rather than all initial denials, so the findings describe adjudication patterns within a filtered appeals population. Because the analysis was cross-sectional, it characterized review outcomes rather than patient outcomes or causal effects, and overturned denials may suggest potentially inappropriate denials without establishing plan-level error in every case. The study also relied on NLP classification, although a manually recoded 5% sample showed high agreement for denial reasons and appeal outcomes (κ=0.81 and κ=0.84), and the main patterns were similar when 2020 appeals were excluded.
The authors concluded that roughly one-fifth of appealed denials for anticancer medications were overturned at the second review stage, that outcomes varied sharply by the reason for the original denial, and that many unfavorable reviews turned on clerical rather than clinical grounds.
Clinician Questions
What were the most common denial categories in second-level Medicare Part D appeals for anticancer medications?
In 4,952 second-level Medicare Part D appeals for denied anticancer medications, non–medically accepted indications or off-label use were most common at 3,410 appeals (68.9%), followed by formulary exceptions at 545 (11%), failure to meet prior authorization coverage criteria at 463 (9.3%), drugs excluded from the Part D benefit at 300 (6.1%), and tiering exceptions at 234 (4.7%).
Which Medicare Part D anticancer drug appeals were most likely to receive a favorable second-level review?
Appeals for denied anticancer medications that were initially rejected for failure to meet prior authorization coverage criteria had the highest favorable second-level review rate at 61.1% (95% CI, 56.7%-65.6%). By contrast, appeals involving non–medically accepted indications had a 16.5% favorable rate (95% CI, 15.2%-17.8%), while excluded-drug and tiering-exception appeals were rarely successful.
What evidence supported successful off-label anticancer medication appeals in Medicare Part D?
Among favorable off-label anticancer medication appeals in Medicare Part D, 87.9% (95% CI, 86.1%-89.6%) were supported by Medicare-approved compendia and/or peer-reviewed literature. In the same favorable off-label group, 15.2% (95% CI, 13.3%-17%) included an acceptable external citation and 8.4% (95% CI, 6.7%-10%) were adjudicated using both compendium and external citations.
Why were external citations rejected in unfavorable off-label Medicare Part D appeals for anticancer medications?
Among 94 rejected external citations in unfavorable Medicare Part D appeals for denied anticancer medications seeking redetermination of medically accepted indication, the most common reasons were that the evidence was not representative of the requested drug use or cancer condition in 33 of 94 cases (35.1%), was investigational in 21 of 94 (22.3%), was available only as abstracts in 19 of 94 (20.2%), or was abstract material not from an approved journal in 18 of 94 (19.1%).
Recommended Reading
- For more on prior authorization reform: Prior Authorization & the AMA's Push for Reform