Real-World Analysis of Insurer Rejection Rates for Specialty Oral Anticancer Prescriptions in a Nationwide Sample of Patients with Blood Cancer
Authors: Doshi Jalpa A. , Li Pengxiang , Geng Zhi , Lei Xiudong , Pettit Amy , Armstrong Katrina A. , Huntington Scott F , Lin John K 
Published: December 2024

Background: Remarkable increases in the available number of innovative, specialty oral anticancer medications (SOAMs) have yielded improvements in tolerability and survival for leukemia, lymphoma, and myeloma. However, SOAMs' high prices and longer treatment durations make blood cancers expensive to treat, leading U.S. insurers to restrict formulary coverage for SOAMs and implement more stringent prior authorization (PA) requirements and step therapy (ST) policies that could delay treatment initiation or require treatment plan changes. However, little real-world evidence exists on insurer rejection of SOAM prescriptions and most assessments of PA/ST burden are limited to prescriber survey data. This study aimed to estimate rates of insurer rejection and reasons for rejection of new SOAM prescriptions for patients with blood cancers, overall and by insurance type.
Methods: This retrospective analysis used prescription and medical claims from the 2021-2023 Symphony Health Solutions Integrated Dataverse database, which uniquely captures whether a prescription submitted to the pharmacy is approved or rejected by the patient's insurer and the reason for rejection. All patients with evidence of ≥1 new SOAM prescription for any blood cancer between 01/01/2022 and 12/31/2022 were included in the sample (index date = date of first SOAM prescription). Key outcomes included SOAM prescription rejection and the reason for rejection. All analyses were conducted separately for patients who received a prescription for (a) a brand-name only SOAM agent (i.e., with no generic available) and (b) an SOAM agent with a generic alternative available (i.e., Gleevec® or imatinib, Afinitor® or everolimus, and Revlimid® or lenalidomide). Rates were calculated overall and by insurance type (Medicare, commercial, or Medicaid). Logistic regressions assessed the association between insurance type and SOAM rejection while controlling for patient age, sex, race/ethnicity, education, income, region, NCI comorbidity index, number of total prescriptions in the 6 months pre-index, SOAM agent, and index month.
Results: The final sample included 15,842 patients (mean age 65.4 [SD: 15.8] years; 54.7% male; 54.8% white), of whom 58.2%, 36.1%, and 5.8% were enrolled in Medicare, commercial insurance, and Medicaid, respectively.
Among the 9,272 patients prescribed brand-name only SOAM prescriptions, the initial insurer rejection rate was 25.7% and was substantially lower for patients in Medicare (16.0%) compared to commercial insurance (39.0%) or Medicaid (29.9%). Common reported reasons for rejected brand-name only SOAM prescriptions included PA/ST requirements (29.8%) and lack of formulary coverage (12.5%). Medicare patients were more likely to have PA/ST as the reason for rejection and commercial patients were more likely to have lack of formulary coverage as a reason for rejection. Even after controlling for patient sociodemographic and clinical characteristics, the commercially insured had 3 times the odds (OR: 3.07, 95% CI: 2.70-3.49) and those with Medicaid had 2 times the odds (OR: 2.06, 95% CI: 1.63-2.63) of rejection compared to those with Medicare. Very few (&lt;10%) of the rejected prescriptions were eventually approved within 90 days, across all insurance types.
Among the 6,570 patients prescribed an SOAM with a generic available, the initial rejection rate was higher at 34.2% and 35.3% for the brand-name and generic counterparts, respectively. Substantial variation was observed in rejection rates and reasons by insurance type and SOAM agent. Multivariate analyses showed that the odds of rejection were several-fold higher among commercial and Medicaid compared to Medicare patients. Less than 26% of initially rejected prescriptions were approved within 90 days, across all insurance types.
Conclusions: This nationwide, real-world study found that about one-quarter to one-third of new prescriptions for an SOAM agent received by blood cancer patients were rejected by their insurer in 2022. Patients with commercial insurance and Medicaid were more likely to have their SOAM prescription rejected compared to Medicare patients. This study highlights the critical need for further investigation and urgent consideration of legislative efforts to facilitate appropriate, timely access to life-extending therapies, particularly among commercial and Medicaid patients.