Prior Authorization, Costs Limit Access to Oral Blood Cancer Drugs

Most new prescriptions for specialty oral anticancer medications (SOAMs) for blood cancers were rejected by insurers when first submitted. Even after insurers approved the prescriptions, many patients never filled them, particularly when out-of-pocket (OOP) costs exceeded $175. These findings come from a nationwide claims analysis published in the Journal of Clinical Oncology.

At first submission, insurers rejected 64.9% of prescriptions for Medicare beneficiaries and 84.0% for commercially insured patients. Within 90 days, 54.5% of Medicare patients and 45.5% of commercially insured patients had both received approval and filled the prescription.

"Getting an insurer to approve a cancer drug is only the first hurdle," said senior author Jalpa A. Doshi, PhD, Leon Hess Professor of Internal Medicine and senior fellow at the Leonard Davis Institute of Health Economics at the University of Pennsylvania, in a news release. "We saw a second drop-off at the pharmacy counter when patients faced high out-of-pocket costs."

How was the study designed?

The retrospective study used prescription and medical claims from July 1, 2021, to March 31, 2023, from the Symphony Health Solutions Integrated Dataverse. Unlike most claims data sets, which capture only filled prescriptions, this database tracks whether each prescription was rejected by the insurer, approved but not filled, or approved and filled.

Investigators identified 12,134 patients with at least 1 new prescription in 2022 for a U.S. Food and Drug Administration–approved SOAM for leukemia, lymphoma, or myeloma. Of these, 61.0% had Medicare Part D coverage and 39.0% had commercial insurance. The most common index drug classes were:

• Immunomodulators (40.8% of Medicare patients; 33.6% of commercial patients)
• Bruton tyrosine kinase (BTK) inhibitors (17.7%; 15.0%)
• B-cell lymphoma 2 (BCL-2) inhibitors (13.0%; 14.8%)

Prescription status was assessed at initial submission and again at 90 days. Investigators also examined reasons for rejection and fill rates across 5 OOP cost levels. Patients with Medicaid were not included.

Why were prescriptions for oral blood cancer drugs rejected?

Prior authorization (PA) and formulary restrictions were the most common reasons for initial rejection. PA accounted for initial rejections in 32.1% of Medicare patients and 27.8% of commercially insured patients. By 90 days, PA-related rejections had fallen to 4.6% and 6.9%, respectively.

Formulary exclusion was a larger barrier in commercial plans, accounting for 17.1% of initial rejections and 8.0% of final rejections. Fewer than half (48.5%) of commercial prescriptions rejected as off formulary were eventually approved. By comparison, 71.3% of commercial prescriptions rejected because of PA were eventually approved.

In adjusted analyses, commercially insured patients had higher odds of rejection than Medicare patients:

• Rejection for any reason at initial status: odds ratio (OR), 3.27 (95% CI, 2.31-4.63; P < .001)
• Rejection due to formulary exclusion at initial status: OR, 8.75 (95% CI, 5.70-13.42; P < .001)
• Rejection due to formulary exclusion at final status: OR, 8.89 (95% CI, 6.11-12.92; P < .001)

Commercially insured patients also had statistically significantly higher odds of rejection due to PA and quantity limits at both time points. The study authors attributed the gap partly to Medicare Part D's protected-class status for oncology drugs, which generally requires plans to cover all or substantially all drugs in the class.

How did out-of-pocket costs affect prescription fills?

By 90 days, 30.5% of Medicare patients and 17.4% of commercially insured patients had an approved prescription they did not fill. The mean OOP cost for the first approved prescription was $933 for Medicare patients and $775 for commercially insured patients.

Fill rates fell sharply once OOP costs rose above $175:

• Medicare: 84.9% at $15 or less; 76.7% at $15.01-$175; 38.0% at $175.01-$500; 29.2% at $500.01-$2,000; 31.2% above $2,000
• Commercial: 80.0% at $15 or less; 71.3% at $15.01-$175; 53.8% at $175.01-$500; 30.9% at $500.01-$2,000; 21.6% above $2,000

The odds of not filling an approved prescription did not differ statistically significantly between the 2 insurance groups. Among patients who did fill, the time to fill exceeded 2 weeks for 17.1% of Medicare patients and 21.1% of commercially insured patients.

"These blood cancer medications can be life-prolonging, but our findings show that insurance coverage requirements and high out-of-pocket costs can delay or limit access," Doshi said.

What policy changes could improve access?

The authors noted that because most PA rejections were eventually overturned, PA may add administrative delays without changing many final decisions. They pointed to electronic PA tools and gold-carding policies, which exempt high-performing prescribers from PA, as ways to reduce this burden.

For Medicare beneficiaries, the Inflation Reduction Act set an annual Part D OOP maximum ($2,100 in 2026). It also created the Medicare Prescription Payment Plan (MPPP), which lets beneficiaries spread OOP costs into monthly payments; for example, $2,100 over 12 months is $175 per month. The authors noted that fill rates were much higher at OOP costs of $175 or less and that MPPP awareness and enrollment remained low in the program's first year.1

The study had several limitations. Investigators could not determine whether individual rejections were clinically appropriate, and they could not account for disease status, line of therapy, or drugs obtained through channels outside the claims data, such as manufacturer free drug programs. The study was supported by Blood Cancer United.

How are oncology teams managing financial toxicity with oral agents?

Prior Oncology Nursing News coverage has described team-based workflows for these barriers. In one practice, pharmacists handle PAs and copay assistance for commercially insured patients, and triage nurses help Medicare beneficiaries apply to manufacturer-sponsored patient assistance programs.

What are the key takeaways for oncology nurses?

• Expect an initial denial: Most new SOAM prescriptions were rejected at first submission, but most rejections due to PA were overturned within 90 days. Setting expectations early may reduce patient distress during the approval process.
• Ask about cost before the first fill: Fill rates dropped sharply above $175 OOP. Asking patients about their expected copay can identify those at risk of not filling the prescription.
• Confirm the fill: About 1 in 3 Medicare patients and 1 in 6 commercially insured patients had an approved prescription they never filled. Following up with the patient or specialty pharmacy after approval can catch prescriptions that were never picked up.
• Use the MPPP for Medicare patients: Medicare beneficiaries cannot use manufacturer copay coupons. The MPPP can spread Part D costs into monthly payments, and foundations and manufacturer free drug programs may also help.
• Plan for formulary exclusions in commercial plans: Off-formulary rejections were overturned less than half the time, so early discussion of formulary alternatives or the appeals process may prevent treatment delays.

References

1. Doshi JA, Li P, Geng Z, et al. Insurance and cost-related access barriers to specialty oral anticancer medications for blood cancers in a nationwide sample of Medicare and commercially insured patients. J Clin Oncol. Published online September 30, 2026. doi:10.1200/JCO-26-00247
2. Insurance barriers limit access to blood cancer drugs. News release. Penn Medicine. September 30, 2026. Accessed October 2, 2026. https://www.pennmedicine.org/news/insurance-barriers-limit-access-to-blood-cancer-drugs
3. Sandy B. Addressing financial toxicity: team-based workflows in oncology care. Oncology Nursing News. September 3, 2026. Accessed October 2, 2026. https://www.oncnursingnews.com/view/addressing-financial-toxicity-team-based-workflows-in-oncology-care