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Barriers to CAR T-Cell Therapy: Cost and Clinical Effectiveness

Barriers to CAR T-Cell Therapy: Cost and Clinical Effectiveness
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Published on July 12, 2021

What Is the Cost and Clinical Effectiveness of CAR T-Cell Therapy?

Use of cellular immunotherapies such as chimeric antigen receptor (CAR) T-cell therapy remains limited, partly due to barriers in cost and clinical effectiveness, said Jeffrey A. Tice, MD, of the University of California, San Francisco (UCSF), in a virtual presentation at the 2021 American Society of Clinical Oncology (ASCO) Annual Meeting.

CAR T-cell therapy involves the genetic manipulation of a patient’s T cells in a laboratory so the cells will bind with and kill cancer cells. The manipulated cells are restored to the patient through an intravenous infusion.

Four CAR T-cell therapies, axicabtagene ciloleucel (Yescarta), tisagenlecleucel (Kymriah), lisocabtagene maraleucel (Breyanzi), and brexucabtagene autoleucel (Tecartus), are approved by the U.S. Food and Drug Administration (FDA) to treat several types of lymphoma and leukemia. A fifth CAR T-cell therapy, idecabtagene vicleucel (Abecma), was approved in March 2021 for patients with treatment-resistant multiple myeloma.

To illustrate the cost-effectiveness challenges of CAR T-cell therapy, Dr. Tice shared a literature review published in the February 2021 issue of Drug Discovery Today looking at 95 studies on barriers to access and the use of gene therapies — a category of treatment that involves altering the genetic material in cells. This includes CAR T-cell therapies, which are produced by genetically modifying a person’s T cells. The review highlighted three challenges:

  • Health economics: CAR T-cell therapies have high short-term costs and come with uncertainties about cost-effectiveness.
  • Clinical evidence: Doctors are uncertain about the magnitude of benefits of CAR T-cell therapies, as well as their long-term benefits and harms.
  • Clinical trial design: CAR T clinical trials in some cases lack randomization and a control population. They also may have small numbers of patients, short follow-up periods, and inappropriate clinical endpoints.

Dr. Tice then shared research from the Institute for Clinical and Economic Review (ICER), an independent nonprofit group that provides reports on healthcare technology, including medical tests and treatments, using publicly available data.

The goal of the ICER is to provide “sustainable access to high-value care for all patients,” Dr. Tice said. The ICER considers both short-term affordability, including cost to the patient, and long-term value for money, which incorporates data on comparative clinical effectiveness and incremental cost-effectiveness, he said.

However, “people’s assessments are often influenced by other benefits and disadvantages of the therapy, and contextual considerations,” Dr. Tice said. For instance, people may favor a therapy that is the first available for a previously untreatable condition, or a therapy that may reduce disparities, said Dr. Tice.

As an example of using a cost-effectiveness ratio to compare costs and outcomes, Dr. Tice cited a report published by ICER in April 2021 following the FDA approval of idecabtagene vicleucel for multiple myeloma. The report discussed a phase 2 study of patients with multiple myeloma that had not responded to prior treatment. In the study, people treated with idecabtagene vicleucel showed a median progression-free survival of nearly nine months — meaning they went nearly nine months without having their cancer progress — and a median overall survival of 19 months. Based on the current list price of $419,500 for a one-time infusion, the cost-effectiveness was $319,000 per quality-adjusted life year, which is not considered cost-effective, Dr. Tice said.

“Overall, a cost of between $50,000 and $150,000 per quality-adjusted life year is considered cost-effective in the United States,” said Dr. Tice. A quality-adjusted life year is defined as equal to one year of perfect health, he explained. Data show that in the United States, most people are willing to pay approximately twice their annual salary for one quality-adjusted life year, he said, although actual patient costs are not the list price of the drug.

Other barriers to the use of CAR T-cell therapy include oncologists’ limited familiarity with the benefits and toxicities of the treatment, Dr. Tice said. Toxicities may be a deterrent for some patients, he added, as may be the logistics of treatment administration and limited availability of CAR T therapy, which is only provided at some cancer centers. Insurance barriers and inadequate reimbursement to treatment centers remain challenges as well. Any of these barriers, and others, “are likely to exacerbate disparities in care,” he concluded.

One critical missing area of information are the results from randomized trials of CAR T-cell therapies, which will eventually yield more information about the true net benefit of these therapies, Dr. Tice said during a discussion following the presentation. “Once the FDA approves something, ICER has to come up with a pathway for coverage,” so the organization tries to anticipate approvals and issue reports to coincide, he said. The challenge is making decisions without long-term data, which means that the clinicians issuing the reports have limited information with which to evaluate cost-effectiveness, he explained.

Dr. Tice disclosed that his institution, USCF, has a contract with ICER. He had no other disclosures.

~Heidi Splete

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