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CAR T-cell therapies may be cost-effective for patients with lymphoma subtype
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The chimeric antigen receptor T-cell therapies axicabtagene ciloleucel and tisagenlecleucel may meet a cost-effectiveness threshold of less than $150,000 per quality-adjusted life-year gained for adults with diffuse large B-cell lymphoma, according to results of a study published in Journal of Clinical Oncology.
The cost-effectiveness of the anti-CD19 CAR T-cell therapies — each of which costs $373,000 — depends on long-term outcomes compared with chemoimmunotherapy and stem cell transplants, researchers noted.
First-line chemoimmunotherapy induces long-term remission in about two-thirds of patients with DLBCL. Salvage chemoimmunotherapy or stem cell transplants can sustain remission for patients with relapsed or refractory disease; however, patients with multiple relapses generally have poor outcomes.
Axicabtagene ciloleucel (Yescarta, Kite Pharma) and tisagenlecleucel (Kymriah, Novartis) received FDA approval for treatment of relapsed or refractory DLBCL after two or more lines of therapy, or refractory DLBCL less than a year after stem cell transplantation.
“CAR T cells have changed the treatment paradigm for multiply relapsed/refractory DLBCL,” John K. Lin, MD, VA health services research and development fellow at Center for Health Policy and Center for Primary Care and Outcomes Research at Stanford University, and colleagues wrote. “Our results suggest that [axicabtagene ciloleucel] and tisagenlecleucel provide meaningful survival gains for adults [with this disease]. If highly effective in producing long-term remissions, they may also meet a cost-effectiveness threshold of less than $150,000 [per quality-adjusted life-year gained].”
Lin and colleagues studied the cost-effectiveness and budget impact of these therapies by using a decision analytic Markov model to determine 5-year PFS at 40% and 30% for axicabtagene ciloleucel, and at 35% and 25% for tisagenlecleucel.
They independently compared the therapies with salvage chemoimmunotherapy and stem cell transplantation for each scenario among a hypothetical group of adults (mean age, 58 years) with relapsed or refractory DLBCL.
Undiscounted life years, discounted lifetime costs, discounted quality-adjusted life-years (QALYs), and incremental cost-effectiveness ratio with a 3% annual discount rate served as the study’s primary outcomes.
The model showed that axicabtagene ciloleucel increased life expectancy by 8.2 years at $129,000 per QALY gained (95% uncertainty interval [UI], 90,000-219,000) when assuming 5-year PFS of 40%, and by 6.4 years at $159,000 per QALY gained (95% UI, 105,000-284,000) when assuming 5-year PFS of 30%.
Tisagenlecleucel increased life expectancy by 4.6 years at $168,000 per QALY gained (95% UI, 105,000-414,000) when assuming 5-year PFS of 35% and by 3.4 years at $223,000/QALY gained (95% UI, 123,000-1,170,000) when assuming 5-year PFS of 25%.
Health care costs in the United States would increase by about $10 billion over 5 years if all indicated patients received CAR T-cell therapy, according to researchers. However, reducing the price of therapy to $250,000 for axicabtagene ciloleucel and to $200,000 for tisagenlecleucel would lower the cost to less than $150,000 per QALY, even with a 5-year PFS of 25%.
Researchers acknowledged study limitations, including the fact current trials designed to evaluate CAR T-cell therapies for patients with DLBCL are single arm. This limits comparability of treatments, researchers said.
“The challenge with paying for CAR T raises broader issues with prioritization — whether pharmacologic research and development is optimally incentivized,” Lin and colleagues wrote. “There are multiple cancer therapies, the values of which seem to be inferior to that of CAR T. Still other therapies were approved by the [FDA] without demonstrating overall survival benefit. We should ask whether it is reasonable to continue paying a premium for therapies that provide incremental benefit while budget constraints limit access to more transformative therapies.”
A CAR T-cell therapy coverage proposal drafted by CMS could pave the way for patients to join a nationally audited registry that would include clinical and patient-recorded outcomes, Jonathan D. Campbell, PhD, and Melanie D. Whittington, PhD, both of the department of clinical pharmacy at University of Colorado, wrote in an accompanying editorial. This registry could inform future CAR T reimbursement and coverage policies, they wrote.
“In this brave world of innovative, individualized and potentially curative therapies, we must be good stewards of limited health system resources, while working together toward characterizing and achieving fair value,” Campbell and Whittington wrote. “Government-funded and actionable research into value-assessment frameworks and fair value payments is missing and clearly needed to align CAR T payments with limited health system resources.” – by John DeRosier
Disclosures: Lin reports no relevant financial disclosures. Please see the study for all other authors’ relevant financial disclosures. Campbell reports a consultant/advisory role with Kite Pharma, Novartis and PhRMA. Whittington reports no relevant financial disclosures.
Perspective
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Nirav Shah, MD
CAR T-cell therapy for DLBCL is a potentially life-saving treatment for those with relapsed, refractory disease. The excitement around CAR T-cell therapy is limited by the realistic concerns about widespread delivery of these products given the astronomical costs associated treatment. Both approved products for DLBCL — axicabtagene ciloleucel and tisagenlecleucel — cost $373,000 per treatment, which does not include the costs to deliver care in qualified medical centers. In this publication, cost-effectiveness of CAR-T cell therapies in aggressive B-cell lymphomas was evaluated utilizing a decision analytic Markov model.
When applying the most optimistic 5-year predictions, both treatments were felt to be cost-effective at a threshold of less than $150,000 per QALY; however, should CAR T fail to meet these optimistic predictions, it quickly would become a cost-ineffective therapy.
Even with these predictions, the additional costs to the health care system would increase by $2 billion annually per year if all eligible patients received CAR T cells, which is astonishing given that — prior to CAR T — the total annual cost of care to all patients with non-Hodgkin lymphoma was only $2.9 billion in 2013. The authors proposed several alternative pricing models, including payment only for patients who achieve complete response, or a decreased price structure for CAR T therapies to make them more cost-effective. However, without regulatory pressure or competitive pricing of future CAR T therapies, it is unlikely for industry to take such action on its own, as has been seen in the case with other life-saving therapies.
Although advances in medical science have altered the natural history of many malignancies, unsustainable pricing of drugs continue to be a limiting factor. New technologies to reduce costs associated with CAR T cell therapy are indicated.
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