February 03, 2021

To date, several immunotherapy agents (monoclonal antibodies and checkpoint inhibitors, oncolytic virus therapy, T-cell therapy and cancer vaccines) have been approved by the Food and Drug Administration (FDA)(Table 1) and are making their way to market at a rapid pace. 

Agent	Mechanism of Action	Type of Cancer (to date)	Product Examples
Checkpoint Inhibitors3	Molecule acts as a brake on immune cells (i.e., “the checkpoint”), allowing them time to recognize, and mobilize against, the tumor cells.	Melanoma, lung cancer	Yervoy®, Keytruda®
Oncolytic Virus Therapy4	Uses a virus as a vehicle to invade and destroy tumor cells.	Melanoma	T-VEC (ImlygicTM)
T-cell Therapy (or CAR T-cell Therapy)1,5	Involves removing a number of T cells from the patient’s tumor, genetically modifying them in a lab setting to enhance their cancer-fighting ability and then implanting them back into the patient.	Childhood acute lymphoblastic leukemia, adult B-cell non-Hodgkin’s lymphoma	KymriahTM, YescartaTM
Cancer Treatment Vaccine6	Given to patients already diagnosed with cancer to boost their immune system to destroy any cancer cells still in the body after other treatments have ended, stop a tumor from growing or spreading or prevent the cancer from coming back.	Metastatic prostate cancer	Provenge® (Sipuleucel-T)

These therapies are undergoing continued exploration in clinical trials to determine their impact on other types of cancers as well. As such, the above list is expected to grow. In situations where there has previously been an unmet clinical need, the FDA will often agree to an expedited review process and accelerated drug approval based on surrogate endpoints, or signs used in place of stronger indicators (e.g., a shrinking tumor or lower biomarker levels vs. longer survival or improved quality of life) to determine whether the immunotherapy treatment provides clinical benefit.⁷ The approval, however, is “conditional,” meaning that the drug manufacturer is still required to confirm clinical benefit of the drug through large phase 3 trials and more definitive endpoints, such as overall survival or progression-free survival.²

Employer Considerations

Though the concept of immunotherapy has been around for more than a century, only in the last 10 years have we seen such dramatic advances in the field.¹ Industry experts warn that these are still the early days of immunotherapy research, and while it is tempting to speculate as to the real value that these treatments may provide, it is still too soon to tell just how it will all play out.²

Who’s Eligible?

Immunotherapy is typically administered to patients with an advanced diagnosis or cancer that has recurred and spread after primary treatment.⁸ In many cases, an individual’s genes and tumor composition will dictate eligibility.⁹ Of note, less than half of patients undergoing cancer treatment will actually benefit at all from immunotherapy. In some cases, genetic tests are useful (and clinically indicated) for identifying those patients who are, indeed, good candidates for treatment. Ongoing research will continue to explore why some patients respond better to immunotherapy than others do.¹⁰

Explore the Business Group's Preicision Medicine Guide for more about genetic testing and gene therapies.

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Ultimately, it is up to the patient and their physician to decide on a suitable course of treatment. Providers are at liberty to use their discretion and can prescribe an immunotherapy drug off-label (i.e., when a drug is intended and FDA-approved for one purpose but used for another). Studies indicate that approximately 30%-50% of all U.S. oncology prescriptions have been off-label. In many cases, trial and error has proven treatment success, and these drugs go on to become standard-of-care treatment.¹¹ It is important to note, however, that off-label use can often be accompanied by financial hurdles. For instance, coverage for an off-label treatment may be denied by the insurance carrier. In appropriate situations and when coverage is denied for this reason, the patient should discuss with their physician the availability of clinical trial enrollment. A health plan or pharmacy benefit manager (PBM) cannot enroll a patient in a trial at the time of coverage denial, nor can the pharmacist, although a pharmacist may inform the patient of the availability of a clinical trial opportunity.

Patient Experience

Immunotherapy has been shown to produce fewer side effects and is generally better tolerated than traditional chemotherapy. Discontinuation rates across trials with these agents is routinely low.² One downside captured in clinical trials, however, is the possibility of damage to healthy tissue and organs (i.e., introduction of autoimmunity).^1,12 Health care providers must be trained on how to detect and treat immune mediated adverse events.²

How Do Current Immunotherapy Offerings Compare?

Testing of immunotherapy agents is being conducted in multiple clinical trials across multiple tumor types. While some agents are already on the market today, it is still relatively early to draw definitive conclusions on how these immunotherapy drugs stack up against one another.² That said, when comparisons are drawn against standard of care, immunotherapy results have shown tremendous promise. For example, a UCLA study found that an immunotherapy agent was able to help more than 15% of patients with advanced non-small cell lung cancer live for at least 5 years. An even greater percentage of patients (25%), with tumors marked by a specific protein, demonstrated a 5-year survival rate; when the study began, the average 5-year survival rate was 5.5%.¹³

How Much Does Immunotherapy Cost?

The high price tags of early market pioneers are telling. Merck’s checkpoint inhibitor, Keytruda®, for example, costs about $150,000 per patient, per year. CAR T-cell therapies have far exceeded this price range, with Kymriah^TM costing $475,000 to treat acute lymphoblastic leukemia in children and young adults. To put these numbers into perspective, the median cost of a single course of radiation therapy for breast, lung and prostate cancer is $8,600, $9,000 and $18,000, respectively.¹⁴ The average monthly cost of chemotherapy ranges from $1,000 to $12,000. If a patient, for example, requires four chemotherapy sessions a year, they would be looking at a total cost of $48,000.¹⁵ While immunotherapy agents come with a lofty price tag, the hope is that they also come with potential cost offsets like higher efficacy and fewer complications.

Employer Solutions

There are a few steps employers can take to ensure proper management of immunotherapy treatment costs:

• Work with your health plan, PBM and other partners to build a comprehensive cancer management program that provides navigation, second-opinion and decision-support services. Be sure to include educational material as well as information on alternative treatments and clinical trial opportunities.
• Talk to your health plan and PBM partners about how they are adjudicating immunotherapies and set expectations for how you will collaboratively determine future treatment adjudication (e.g., prior authorization and step therapy protocol). Ask for reports with potential cost and utilization impact based on your demographics.
• Review current policies with your partners and discuss specific guidelines regarding off-label use of immunotherapy treatments not approved by the FDA.
• Determine, with your partners, how best to educate and encourage employee patients to seek possible enrollment in a relevant immunotherapy clinical trial, particularly where there is an unmet clinical need or where off-label prescription coverage is denied by the health plan.
• Consider exploring and partnering with a cancer Center of Excellence (COE) that is well-equipped, from a staffing and infrastructure perspective, to administer and monitor proper immunotherapy treatment.
• Actively monitor the drug pipeline and new treatment developments. Coordinate with your health plan and PBM to update policies in response to the release of outcomes data from ongoing clinical trials for drugs that had previously received accelerated approval.

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