Immunotherapies are radically changing outcomes, but while helping patients, they are creating complexities surrounding their cost. At the 2019 Annual Conference of the National Comprehensive Cancer Network^® (NCCN^®), a roundtable of experts, including clinicians and payers, discussed how chimeric antigen receptor (CAR) T-cell therapy is disrupting conventions.

Stefanie Joho

Clifford Goodman, PhD

Consultant and advocate, Stefanie Joho, presented her experience as a former patient with metastatic disease who, having failed to respond to all standard-of-care options, had a complete response after treatment in a clinical trial with an anti–programmed cell death protein 1 inhibitor.

Roundtable moderator Clifford Goodman, PhD, kicked off the discussion: “We have this whole panoply of innovative therapies coming along with entirely different mechanisms of action, new delivery models, and sometimes a different site of care. But they are also prompting changes in how we think about paying for treatments. Innovation is that pebble in the pond that has very broad implications.”

One Ripple in the Pond: Payment Complexity

The positive implication of that “ripple in the pond” is the long-term survival, in some cases cures, associated with these immunotherapies. However, another far-reaching ripple that is not so positive is the astronomical cost of these treatments.

John W. Sweetenham, MD, FASCO

Frederick L. Locke, MD

Physicians on the panel estimated the cost of CAR T-cell therapy to be between $500,000 and $1.5 million, depending on specifics. “If it’s a complex clinical course with admission to the intensive care unit, it could be significantly higher,” added John W. Sweetenham, MD, FASCO, of Huntsman Cancer Institute at the University of Utah.

However, as pointed out by Frederick L. Locke, MD, of Moffitt Cancer Center, the inappropriate use of aggressive treatments at the end of life is also expensive, and immunotherapies often have a payoff. “I think $1 million is a lot, but if we are curing, let’s say, 40% of patients, then maybe a high cost is reasonable,” he suggested.

New Payment Models Are Needed

The “ripple effect” is also a good metaphor for how the expense of immunotherapy can impact the payer, according to Jennifer Malin, MD, PhD, Senior Medical Director of Oncology and Genetics at UnitedHealth Group. She pointed out that the actual payout by the insurer is several times the cost of the actual treatment, since reimbursement is still based on the conventional cost-plus payment model. This model is outdated in the immunotherapy era, she noted.

The margin approach may make sense when we’re talking about doxorubicin, but we are talking about innovative new therapies that are very expensive….

— Jennifer Malin, MD, PhD

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“We reimburse the hospital and the practice typically at some margin above the cost of the product,” Dr. Malin said. “The margin approach may make sense when we’re talking about doxorubicin, but we are talking about innovative new therapies that are very expensive…. So, an elephant in the room is, what is a reasonable profit margin on administering a drug?”

Dr. Locke commented that reimbursing several times the half-million-dollar cost of the drug is not reasonable, but some additional funding is justified. “An infrastructure is required for CAR T-cell therapy—ancillary staff are necessary to deliver the care, and these costs are difficult to quantify,” he said.

Dr. Goodman weighed in: “This is certainly a disruptive technology. It’s disrupting the current reimbursement system, which doesn’t work for that purpose.”

Treatment Centers Taking Risks

Even with reimbursements, under the current system, hospitals are left holding the bag on a large proportion of the CAR T-cell therapy costs. And treatment centers are assuming too much of the financial risk upfront, according to the physicians.

As a community-based practice, we don’t have additional sources of revenue that some academic medical centers have.

— Lalan S. Wilfong, MD

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Lalan S. Wilfong, MD, Executive Vice President for Quality Programs and Value-Based Care for Texas Oncology and Physician Liaison for McKesson Specialty Health said Texas Oncology, is rarely performing CAR T-cell therapy for Medicare patients, since adequate payment, at least for this population, is not assured. “As a community-based practice, we don’t have additional sources of revenue that some academic medical centers have,” he said.

“Our center is rarely doing CAR T-cell therapies for Medicare patients, and I think we’re at a huge risk,” Dr. Locke said. “If this doesn’t get figured out soon, we will be able to continue giving these treatments to Medicare patients.”

Toward a National Coverage Decision

Further muddling the financial issues is the lack of national consistency in the reimbursement of emerging therapies. Today, with any new therapy, the Centers for Medicare & Medicaid Services (CMS) lacks a national coverage determination, leaving coverage decisions to local Medicare administrative contractors. “I don’t believe that even today, any of the local carriers have made official determinations for local coverage, although they are unofficially covering CAR T-cell therapy,” Dr. Malin noted.

[CAR T-cell therapy] is going to remain mostly within the balance of the established stem cell transplant programs, since these programs already have the infrastructure to collect the necessary data.

— John W. Sweetenham, MD, FASCO

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UnitedHealthcare recently submitted a request to CMS that the organization make a national coverage decision, with the chief aim being less about cost and more about assuring consistent access for patients and “equity of acccess,” Dr. Malin reported.

Currently, CAR T-cell therapy is being administered according to its U.S. Food and Drug Administration label, she said, but insurers expect future data to show its efficacy outside of these indications. “We want to have a national process in place to be able to handle that,” she explained.

“How do we ensure that [coverage decisions] are consistently applied across the Medicare population?” she asked. Anything short of that “makes it challenging for us as a national health plan, because we don’t want to see inequities in how our members have access to treatments based on where they live,” Dr. Malin said.

CMS Wants Patient Registries

In February 2019, the CMS proposed to cover CAR T-cell therapy under a Coverage with Evidence Development (CED) agreement.¹ CED is a policy tool designed to help determine whether an innovation is necessary and beneficial in routine clinical settings. Under a CED, insurers agree to cover promising new technologies and treatments if patients enroll in studies that will generate additional safety and effectiveness information. In this case, treatment would be covered if it is delivered in a clinical study or CMS-approved registry in which patients are monitored for 2 years after treatment.

Evidence from the registries and studies would help CMS identify the types of patients who benefit from CAR T-cell therapy. This would inform a future decision by the agency regarding the types of cases in which Medicare would cover the treatment with no registry or trial requirement, according to a CMS press release.¹

“We believe that the CED is important, both to ensure ongoing research and to learn how CAR T-cell therapy performs in the real world, but especially in the Medicare population,” Dr. Malin said. “There have been very few published data on this age group.”

Until patients are seen as co-investigators, we’re limited in the scope and depths of our investigations. We need to treat the patient as a partner in research.

— Stefanie Joho

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Dr. Sweetenham believes the CED and patient registry mandate is a “good arrangement” at this time. “Because of the evidence requirements, at least for now, this treatment is going to remain mostly within the balance of the established stem cell transplant programs, since these programs already have the infrastructure to collect the necessary data. I think the knowledge and expertise will be gained at those centers that are best equipped.”

Dr. Wilfong agreed. “Continuing to collect evidence is very important in this population, because we don’t yet know for sure if they will have the same benefit as patients in the clinical trials, who are not the typical patients we treat.”

Stefanie Joho said having registries—and especially more patient-reported outcomes—is an important goal. “I think until patients are seen as co-investigators, we’re limited in the scope and depths of our investigations. The patient is the one with the disease. We need to treat the patient as a partner in research.”

There May Always Be Financial Risks—to Someone

Dr. Locke maintained that, even with a national coverage decision, he is not ready to trust that institutions will be made whole financially. “What I believe is missing is the actual mechanism to pay for this. It’s one thing to say, ‘Medicare, cover this.’ It’s another thing to actually pay for it,” he pointed out. For example, payments based on diagnosis-related group codes for hospital billing are often not adequate, he said. “So, it’s one thing to say yes, everybody can get CAR T-cell therapy, but how?”

It’s one thing to say, ‘Medicare, cover this.’ It’s another thing to actually pay for it.

— Frederick L. Locke, MD

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Dr. Wilfong weighed in from a community practice perspective. “We spend a lot of time making sure we get the right authorizations in place, that we understand coverage decisions from our different payer partners. We do this to make sure we get those payments in and don’t put ourselves at risk. If CAR T-cell therapy comes into that world, all of a sudden, I’ll be having to spend $400,000 for a vial of drug,” he said. “It’s going to be interesting to see what happens when a drug that expensive comes into the options.”

Dr. Wilfong also reminded everyone that even with national reimbursement, many patients are hard hit. “Every January is a challenge in our practices because that’s when most patients’ deductibles reset. The high-deductible plans are $5,000, $7,000 out of pocket, and patients need to pay this the first month of treatment…. If a patient doesn’t pay his or her deductible, guess who has to cover that cost? It’s me.” ■

DISCLOSURE: Ms. Joho and Drs. Goodman and Sweetenham reported no conflicts of interest. Dr. Wilfong receives a salary from McKesson Specialty Health. Dr. Locke has consulted for the Cellular Biomedicine Group and is a scientific advisor to Kite Pharma and Novartis. Dr. Malin is an employee of UnitedHealth Group.

REFERENCE

1. Centers for Medicare & Medicaid Services (CMS): CMS proposes Coverage with Evidence Development for chimeric antigen receptor (CAR) T-cell therapy. Available at https://www.cms.gov/newsroom/press-releases/cms-proposes-coverage-evidence-development-chimeric-antigen-receptor-car-t-cell-therapy. Accessed April 26, 2019.