CAR T-cell therapy is making headlines, mainly for being a groundbreaking and life-saving approach to treat particular types of cancer and rare diseases, including childhood leukemia, but also for its very high price tag.
CAR T-cell therapy (chimeric antigen receptor) extracts a patient’s immune cells, genetically engineers them to fight cancer cells, and then reintroduces them into the body. The patient can have an extreme reaction to the rejuvenated immune cells and this can lead to extended hospitalization. So, in addition to the price of this gene therapy treatment, patients are likely to incur considerable costs related to surrounding requirements of care. Nonetheless, the therapy is expected to make a significant impact, curing conditions that were previously fatal, and there are many trials currently underway.
Only two of these CAR T-cell therapies have been approved by the FDA for oncology indications [at the time of writing]: Kymriah from Novartis for pediatric B-cell acute lymphoblastic leukemia (approved August 2017), and Yescarta from Gilead for the treatment of adult diffuse large B-cell lymphoma (DLBCL), which was approved in October 2017. The FDA also granted Priority Review for Kymriah for adults with relapsed or refractory DLBCL in January 2018. But already the price tag for both therapies (which are intended to be administered only once) has become a frequent topic of conversation and media coverage. The cost of Kymriah is US$475,000 and Yescarta is set at US$373,000. However, in an Evidence Report (February 2018), the US Institute for Clinical and Economic Review (ICER) found that both were cost-effective, stating, “both therapies provided improvements in response rates and survival for patients who have exhausted most other treatment options, and that the drugs are priced in alignment with their clinical value.”
There is, however, another challenge for physicians keen to use these treatments. With CAR T-cell therapy being such an innovative treatment, there are no established payment codes for US insurers, such as Medicare, to work with. These codes are necessary to ensure reimbursement. Physicians have many patients for these new therapies on their waiting lists but are forced to wait for confirmation that the costs will be covered. Bloomberg reported that after two months following Yescarta’s approval, only five patients had received the treatment. There are signs that insurers are working to address the issue, but in the meantime, some hospitals are choosing not to admit patients until the coverage situation is clarified, while other hospitals are admitting patients regardless of the insurance situation, as this is possibly their last chance at receiving life-changing treatment.
These therapies are likely to be the first of many new gene therapy treatments. For a therapy that offers such hope to cancer patients who may have exhausted conventional and even newer targeted therapies, it is desperately frustrating that they could miss out on life-saving treatment due to reimbursement challenges and missing codes.
Read a more detailed discussion of the situation in our Expert Insight, ‘Challenges of the new gene therapy era: a focus on CAR T-cell treatment.’