Why the First Denial of a Cancer Drug Isn't the Final Answer

Among 4,952 second-level Medicare Part D appeals involving cancer drugs submitted from 2020 through 2024, 19.5% ended in a favorable decision, according to a study published in JAMA Network Open. A second-level appeal is a review by an independent entity contracted by Medicare, which takes place after the insurance plan has upheld its own denial. Appeals for drugs initially denied because they did not meet prior authorization criteria had the highest success rate, at 61%.

Joanna Fawzy Doran, Esq., CEO and co-founder of Triage Cancer, spoke with CURE about why patients with cancer should not treat a first denial as the final word, how patients and their care teams share the work of an appeal and what options exist when treatment cannot wait. Lead author Youngmin Kwon, PhD, discussed the findings in a separate CURE interview.

What insurance problems do patients with cancer face most often?

Triage Cancer is a national nonprofit organization that provides free education on the legal and practical issues that come up as a result of a diagnosis. Insurance is the number one topic we get contacted about, whether it's understanding health insurance options, understanding how to use your coverage effectively or knowing what to do when insurance says no, we're not going to cover that care.

Why do insurance companies deny coverage for cancer drugs?

It's hard to say the reasons why medications are being denied, but we do know what insurance companies tell us the reasons are. The medication might be new, so it's labeled as experimental or investigational. Or maybe it's being used in combination with another medication, and somebody might say, "That's off-label use. It's not being used for the reason that it was initially approved, so we're not going to provide coverage."

That can happen even though there might be new scientific information that shows using it in combination with another treatment ends up being standard of care. Sometimes the science moves a lot faster than the law does, so insurance companies are relying on older information or older rules about particular medications and whether they will cover them.

Can patients with cancer appeal an insurance denial for a cancer drug?

Patients should never view the first denial as the final answer. Whether it's Medicare, Medicaid, an employer-sponsored plan or an individual health insurance plan, there is the opportunity to appeal that denial.

Lots of people will say, "I know I can appeal. I don't have to take no for an answer." Very few people do. When you look at the data across different types of plans, most people are in fact taking no for an answer. The harder part is understanding what the steps are and then having the time, the energy and the capacity to take them. When somebody does use the process, they are much more likely to get access to the care that they need.

What is the first step in appealing a denied cancer drug?

The first step is knowing what type of insurance you have and then understanding what the appeals process looks like for your type of insurance specifically. At Triage Cancer, we have a state-specific appeals toolkit that helps people understand the steps based on where they live and the type of insurance they have.

Your health care team prescribes your care for a particular reason. They don't typically prescribe care for no reason. That's why it's so important to work with your health care team to make the argument for why it's medically necessary for you to get that care, and that's the foundation of your appeal.

What happens when prior authorization for a cancer drug is denied?

Patients and health care teams are a partnership in lots of ways. A lot of times, providers are taking care of the business side of accessing care without a patient even knowing it's happening. If the insurance company says no to a prior authorization, providers are oftentimes filing the appeal right away. They're the ones going back to the insurance company and saying, "This is why this patient needs this care."

If it gets denied again, the patient might be notified by their insurance or by the provider. Denials impede access to care, and they create financial hardship. If people don't use the appeals process, they're much more likely to be trying to figure out how to pay for that treatment out of pocket.

[Patients should also] communicate with their health care team and say, "Are you going to file the appeal, or do I need to? Can you help me with the statement of medical necessity? Can you give me any additional information, like research studies or other literature, that I can use to make my argument?" It's a team effort, and we want both sides communicating effectively so things aren't falling through the cracks.

What does off-label use mean, and why do insurers deny off-label cancer drugs?

Insurance companies typically use medical guidelines when deciding whether to cover care, and off-label use is common in cancer care. The original study for a drug might have been in one type of cancer, and then there's research to see if it's useful in another type of cancer or even another medical condition. When a drug is used for reasons other than what it was originally approved for by the Food and Drug Administration, that creates the off-label situation.

It still falls back to the health care team to explain why that's the best course of treatment for that particular patient and why it's medically necessary, and to back up that decision with any information available in the medical literature.

What is an external appeal when private insurance denies cancer treatment?

With private insurance, so individual or employer-sponsored plans, you can appeal back to the insurance company to ask it to change its decision. If that's unsuccessful, you can go outside the insurance company to an independent entity, which is called an external appeal. That independent entity is going to look at whether it's medically necessary for a patient to access that care.

It's an important consumer right that advocacy groups fought for, but almost no one knows it exists. The data show across states that very few people are using it, and when I say very few, I mean like 100 people in the whole state.

Can an insurance appeal for cancer treatment be decided in 72 hours?

The length of time depends on the type of insurance you have. One place where the external appeals process is useful is the prior authorization stage. If the insurance company says no to that prior authorization, and it's time sensitive for that patient to start treatment, you can make the argument that it's urgent. You can file the internal and the external appeal at the same time and get a response back within 72 hours.

Many providers don't realize the external appeals process exists at all, or the value of filing the internal and external appeal together.

What should patients and caregivers do when insurance denies a cancer medication?

The number one thing we want patients and caregivers to know is that you do not have to take no for an answer. The next step should be appealing.

Help is available. Triage Cancer is one of those resources. You can always come to our website at triagecancer.org and learn about health insurance appeals and what your options are for next steps.

Reference

1. Kwon Y, et al. "Second-Level Appeals of Denied Anticancer Medication Claims in Medicare Part D." JAMA Network Open. Published July 23, 2026.

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