The Centers for Medicare & Medicaid Services (CMS) have finalized nationwide coverage of chimeric antigen receptor (CAR) T-cell therapy, replacing the previous policy under which the decision of treatment coverage was left to Medicare's regional administrators.
"As the first type of FDA-approved gene therapy, CAR T-cell therapies are an important scientific advancement in this promising new area of medicine and provide treatment options for some patients who had nowhere else to turn," commented Seema Verma, MPH, Administrator of CMS. "[This] coverage decision provides consistent and predictable patient access nationwide. CMS will work closely with our sister agencies to monitor outcomes for Medicare patients receiving this innovative therapy going forward."
A treatment that involves genetically modifying a patient's own T cells so that they are better geared to attack cancer cells, CAR T-cell therapy has provided a substantial breakthrough in the treatment of B-cell hematologic malignancies. Two CAR T-cell products are currently approved by the FDA: axicabtagene ciloleucel (Yescarta®, Kite Pharma), indicated for aggressive B-cell non-Hodgkin lymphoma (B-NHL), and tisagenlecleucel (KymriahTM, Novartis), indicated for aggressive B-NHL and for B-cell acute lymphoblastic leukemia (B-ALL) in children and young adults. The next generation of CAR T cell products for B-cell malignancies are already being developed.
"Before CD19-directed CAR T-cell therapy, the response rate of available and experimental agents in multiply relapsed and refractory pediatric B-ALL and aggressive B-NHL was approximately 20% to 40%, and response duration was short," wrote Caron Jacobson, MD, Medical Director of the Immune Effector Cell Therapy Program at Dana-Farber Cancer Institute, and colleagues in a perspective piece published last month in JAMA on the issue of Medicare and paying for CAR T-cell therapy. "Tisagenlecleucel definitively improved this outcome in pediatric B-ALL, with minimal residual disease–negative complete responses occurring in [81% of] patients… In adults with aggressive B-NHL, axicabtagene ciloleucel led to responses in [83% of] patients, with 58% of patients having a complete response."
The issue with Medicare and CAR T-cell therapy, say Dr. Jacobson and colleagues, is the price, at a one-time cost of $475,000 for B-ALL and of $373,000 for B-NHL. In addition, "these therapies carry risk related to the activation and expansion of immune effector cells on reinfusion, and 20% to 30% of patients require admission to the intensive care unit. The cost of care, then, can be even greater than the price of the products," note the authors. "Some hospitals report setting limits or declining to offer CAR T-cell therapy at all because of reimbursement concerns."
Prior to the new nationwide approval of CAR T-cell therapy coverage under Medicare, CMS had recently proposed a national coverage policy that would cover CAR T-cell therapy only at the prescription of a certified hospital or cancer center and exclusively for treatments of relapsed or refractory malignancies that used the patient's own cells. This proposal was considered by many experts to be overly restrictive, comment Dr. Jacobson and colleagues, and it was likely to become quickly outdated.
The new decision by CMS includes broader coverage. Medicare will now cover CAR T-cell therapies for FDA-approved indications when they are administered under an approved FDA Risk Evaluation and Mitigation Strategy (REMS). Medicare will also cover CAR T-cell therapies for off-label uses recommended by CMS-approved compendia.
"We remain committed to supporting the efficient development of safe and effective CAR T-cell therapies," commented Ned Sharpless, MD, Acting Commissioner of the FDA. "We will continue working with our partners at CMS and the National Institutes of Health's National Cancer Institute (NCI) to help advance the development and availability of these therapies to patients in need."
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Image credit: Alex Ritter, Jennifer Lippincott Schwartz, and Gillian Griffiths. Courtesy of the National Institutes of Health